Policy and Medicine

Institute of Medicine Report on Essential Benefits

The Patient Protection and Affordable Care Act of 2010 (ACA) was enacted to help uninsured Americans obtain health insurance.  As part of this effort, private health insurance plans will be offered to low- and moderate-income individuals and small employers through state-based “purchasing exchanges,” often with financial help. 

To ensure a more consistent level of benefits, the ACA requires that certain insurance plans—including those par­ticipating in the state purchasing exchanges—cover a package of diagnostic, preventive, and therapeutic services and products that have been defined as “essential” by the Department of Health and Human Services (HHS). 

This package—commonly referred to as a set of essential health benefits (EHB)—constitutes a minimum set of benefits that the plans must cover, but insurers may offer additional benefits. The EHB are intended to cover health care needs, to promote services that are medically effective, and to be afford­able to purchasers. 

The ACA stipulated that HHS will define what the EHB package should include, while states will oversee day-to-day running of the exchanges.  To assist with this, HHS asked the Institute of Medicine (IOM) to recommend a process that would help HHS do two things: 1) define the benefits that should be in the EHB, and 2) update the benefits to take into account advances in sci­ence, gaps in access, and the impact of any benefit changes on cost. 

The IOM appointed a committee to meet this task. The charge of the committee specifi­cally was not to decide what is covered in the EHB but rather to propose a set of criteria and methods that should be used in deciding what benefits are most important for coverage. 

The IOM completed its task and published a nearly 300-page report, outlining its recommendations about the process the HHS secretary should use to define and update essential health benefits.  One article noted that, “America's Health Insurance Plans commended the IOM for its recommendations, particularly the one on a premium target.”

“With this thoughtful report, the IOM is urging policymakers to strike a balance between the affordability of coverage and the comprehensiveness of coverage,” AHIP President and CEO Karen Ignagni said in a news release. “We agree that this balance is critical to ensuring that individuals, working families and small employers can afford health insurance. The recommendation that the initial EHB package reflect the scope of benefits and design provided under a typical small employer plan is an important step toward maintaining affordability.” 

In an e-mailed statement, HHS Secretary Kathleen Sebelius said she appreciated the IOM's work and looks forward to reviewing the recommendations.  “But before we forward a proposal, it is critical that we hear from the American people,” Sebelius said in the statement. “To accomplish this goal, HHS will initiate a series of listening sessions where Americans from across the country will have the chance to share their thoughts on these issues.”

IOM Committee 

Committee members who worked on the report combined perspectives from four areas—economics, ethics, evidence-based practice and population health—to create what the IOM called an “overarching framework” for HHS.  

In considering its task, the committee recognized two competing goals: to provide health insurance coverage for a wide range of health needs and to make it affordable. If it was not affordable, then many people would not be able to obtain it, even with government help, and this would conflict with the purpose of the ACA. Thus, the commit­tee saw its primary task as finding the right bal­ance between making coverage available for indi­viduals to get the care they need at a cost they could afford. This balance will help ensure that an estimated 68 million people have access to care covered by the EHB. 

The committee recommended that HHS develop the EHB package by keeping in mind what small employ­ers and their employees can afford. Employers who offer insurance packages make such choices now. Keeping the EHB affordable is necessary for consumers, employers, and taxpayers. To maxi­mize the number of people with insurance, the committee proposed that HHS embrace a frame­work for the entire EHB package that would: 

• consider the population’s health needs as a whole;
• encourage better care by ensuring good sci­ence is used to inform coverage decisions;
• emphasize the judicious use of resources; and
• carefully use economic tools to improve value and performance.  

The ACA requires that the EHB include at least 10 general categories of health services and have benefits similar to those currently provided by a typical employer. The 10 categories include: 

• Ambulatory patient services
• Emergency services
• Hospitalization
• Maternity and newborn care
• Mental health and substance use disorder ser­vices, including behavioral health treatment
• Prescription drugs
• Rehabilitative and habilitative services and devices
• Laboratory services
• Preventive and wellness services and chronic disease management
• Pediatric services, including oral and vision care  

The committee also identified a set of crite­ria for considering the content of the whole EHB package and specific components within it, as well as methods for defining and updating the EHB (see Criteria).  Current state insurance man­dates—requirements that had previously been established by state law—should not automatically be included in the EHB package but reviewed in the same way as other potential benefits. 

In both defining and updating the EHB package, IOM recommended that the methods used by HHS should be highly visible and allow for current and future enrollees to help define priorities for coverage. As envisioned by the committee, the public deliberation process would enable individuals— working in small group meet­ings around the country—to participate in a pri­oritization process, where different elements of coverage–specific services, types of cost-sharing, degree of provider choice, approval requirements, etc.–are discussed and debated. Learning from these groups will help HHS understand potential enrollees’ priorities when tradeoffs are necessary. A small number of these meetings would comple­ment the process HHS would normally use for receiving public comment. 

IOM stated that, “Consumers, employers, insurers, care provid­ers, and government have a shared responsibility for improving health and using resources wisely. Only medically necessary services should be covered, and decisions by insurers about what is “medically necessary” should depend on the cir­cumstances of an individual case. Under the ACA, when patients are denied care by their insurer, they have the right to appeal to an external review by experts.” 

IOM also noted that, “Some flexibility in defining the contents in the EHB will help encourage innovation at the state level. Proposed state-specific variations should be consistent with the ACA statute, abide by the selection criteria in this report, produce a benefits package that is equivalent in value to the EHB, and utilize meaningful public input.” 

The report also states that, “HHS should update the EHB package annually, beginning in 2016, to promote better health out­comes for both individuals and the broader pop­ulation. The benefit package needs to be based on credible evidence of effectiveness.  A National Benefits Advisory Council, appointed through a nonpartisan process, should be established to offer external advice on updates, data require­ments, and the research plan.” 

The IOM commit­tee recommended that HHS immediately begin developing a plan for identifying data needs and a research agenda that will support the EHB updat­ing process. The ability of the states to provide consistent and usable information to HHS will be enhanced if data needs are outlined at the start. 

In updating the benefits, IOM noted that “HHS should con­sider the projected cost of the current package, taking into account medical inflation, utilization of health services, and other factors. Any adjust­ments to the content of the EHB package should be made within that projected cost.

IOM noted that, “without serious attention to rising health care costs across all sectors, the EHB will become unaf­fordable over time. Thus, HHS, working with oth­ers, including the private sector, should develop a strategy to reduce the rate of growth in health care spending, bringing it in line with the rate of growth in the economy. This will help preserve what benefits are covered by the EHB package.” 

Ultimately, the IOM committee recognized the need to balance cost with the breadth of benefits covered in the EHB, otherwise, “we may never achieve the health care coverage envisioned in the ACA.”  IOM noted that, “If accessing benefits is too difficult, people will not get the care that they need. And if health care spending continues to rise so rapidly, the benefits covered under the EHB will begin to erode, eventually resulting in minimal coverage for the people who need it most.”

 

Institute of Medicine Report Medical Devices and the Public’s Health: The FDA 510(k) Clearance Process at 35 Years

 
Today, the Institute of Medicine (IOM) Committee on the Public Health Effectiveness of the FDA 510(k) Clearance Process released its long awaited, 245-page report, entitled, “Medical Devices and the Public’s Health The FDA 510(k) Clearance Process at 35 Years.”

The premarket notification, or the 510(k) clearance process, named after Section 510(k) of the authorizing legislation passed by Congress in 1976, is the primary pathway to market for Class II devices. More than 80% of 510(k)-cleared devices are categorized as Class II device types. Some Class I and Class III device types also are cleared through the 510(k) process.

The report and recommendations are focused on strengthening the science base needed to make better-informed regulatory decisions and on giving FDA the tools that it needs to identify and remove problematic devices from the market. The committee believes that taking the recommended steps will generate the information needed to design a robust regulatory framework for Class II devices. The new framework would increase the public’s confidence that safe and effective medical devices are being made available in a timely manner.

As described below, the IOM report on the 510(k) process contains broad recommendations that may have significant consequences and impacts on the medical device industry.  Specifically, IOM concluded that the 510(k) process “cannot be transformed into a premarket evaluation of safety and effectiveness as long as the standard for clearance is substantial equivalence to any previously cleared device” and that “the 510(k) clearance process is not intended to evaluate the safety and effectiveness of medical devices with some exceptions.”

Moreover, the report asserts that, FDA's finite resources would be better invested in developing a new framework that uses both premarket clearance and improved postmarket surveillance of device performance to provide reasonable assurance of the safety and effectiveness of Class II devices throughout the duration of their use, the committee said.  The report also maintains that FDA should ensure that the new process allows devices to reach the market in as rapid and least burdensome a fashion as possible. 

"It's not clear that the 510(k) process is serving the needs of either industry or patients, and simply modifying it again will not help," said committee chair David Challoner, emeritus vice president for health affairs, University of Florida, Gainesville.  "The 510(k) process cannot achieve its stated goals -- to promote innovation and make safe, effective devices available to patients in a timely manner -- because they are fundamentally at odds with the statutes that govern how FDA must implement the process.  While current information is not adequate to immediately start designing a new framework, we believe the agency can get the necessary data and establish a new process within a reasonable time frame."

As we noted yesterday, there is legal concern mounting already about the composition of the IOM committee as it pertains to the Federal Advisory Committee Act and the lack of fair balance representation.    

Without clear evidence the committee is recommending that the 510K process of approving substantially equivalent devices be scrapped in favor of adding additional studies and potentially delaying approval of competitive devices.

Background

In September 2009, the 510(k) Working Group and the Task Force on the Utilization of Science in Regulatory Decision Making were formed to address critical challenges facing the Centers for Devices and Radiological Health (CDRH),  and external constituencies.

Industry had raised concerns that the 510(k) program had become less predictable, consistent and transparent thereby stifling innovation and sending companies and jobs overseas, and that CDRH reviewers had become less responsive and more risk averse. Jeffrey Shuren, MD, JD, Director of CDRH noted that “an unpredictable environment discourages investment and stifles innovation, which, in turn, can spur companies to go overseas.”

Consumers, third-party payers, and some healthcare professional groups were concerned that, for some devices, the 510(k) program did not provide adequate assurances of safety and effectiveness nor did it provide sufficient information for healthcare providers and patients to make well-informed treatment or diagnostic decisions. 

Several high-profile mass-media reports and consumer-protection groups have profiled recognized or potential problems with medical devices cleared through the 510(k) clearance process. The medical-device industry and some patients have asserted that the process has become too burdensome and is delaying or stalling the entry of important new medical devices to the market.

CDRH employees expressed concerns that the current 510(k) program failed to adapt to the increasing complexity of devices, and that reviewers’ ability to make well-informed decisions was undermined by the poor quality of 510(k) submissions. In addition, an increasing workload was straining an already overburdened program.  

In August 2010, FDA’s CDRH released for public comment the preliminary reports from these committees.  At the time, CDRH proposed three key changes:

• A streamlined pathway for lower-risk denovo devices;
• A new subclass (Class IIb) for moderate-risk devices; and
• The creation of an internal CDRH Science Council

In addition to these changes, CDRH asked IOM to carry out a parallel, complementary assessment of the 501k changes they proposed in their report.  Subsequently, in January 2011, CDRH released its new actions for the 510(k) program, which included:

• Streamlining the review process for innovative, lower-risk products, called the “de novo” process;
• Publishing guidance for industry to clarify when clinical data should be submitted to  increase predictability and transparency;
• Developing a network of external experts who can use their knowledge and  experience to help the agency address important scientific issues regarding new  medical device technologies;
• Establishing a new Center Science Council of senior FDA experts within the  agency’s medical device center to assure more timely and consistent science based decision making; and
• Improving training for CDRH staff and industry

The FDA also said it would:

Establish a public database of important device information, such as medical  device labeling and summaries of the basis for the FDA’s decision to clear  specific devices; and

Require a brief description of scientific information regarding the safety and effectiveness known to the manufacturer for select higher-risk devices on a case by-case basis through device-specific guidance. 

At this time, FDA also released an implementation plan that outlined 25 specific actions and the timelines for completion or reaching a major milestone in 2011 to make the 510(k) program a blueprint for smarter medical device oversight; one that drives innovation and brings important technologies to the public.    

Consequently, FDA received comments on seven of the recommendations that stakeholders found problematic. As a result, FDA asked IOM to offer comments on the following recommendations:

• CDRH should consolidate the terms “indication for use” and “intended use” into a single term, “intended use”;
• CDRH should expand its statutory authority to consider off-label use when determining the intended use of a device;
• CDRH should issue guidance on when a device should no longer be available for use as a  predicate; 
• CDRH should issue a regulation on its rescission authority;
• CDRH should require manufacturers to keep one unit of a device available;
• CDRH should issue guidance to create a “Class IIb”; and 
• CDRH should seek greater authorities to require postmarket surveillance studies as a condition of clearance for certain devices.

Specifically, FDA stated to the IOM that the goals of the 510(k) clearance process are to “make available to consumers devices that are safe and effective” and to “promote innovation in the medical device industry.”  Accordingly,  FDA asked IOM to review the 510(k) clearance process and to answer two questions:

Does the current 510(k) clearance process optimally protect patients and promote innovation in support of public health?

If not, what legislative, regulatory, or administrative changes are recommended to optimally achieve the goals of the 510(k) clearance process?

FDA and Medical Device Regulation Background

In reviewing the legislative and regulatory history of the 510(k) program, the committee found that it was designed in 1976 to provide only a determination of the substantial equivalence of a new device to an already marketed (predicate) device; it was not designed to determine whether a new device provides a reasonable assurance of safety and effectiveness or whether it promotes innovation.

Prior to the Medical Device Amendments (MDA) of 1976, medical devices generally had not undergone premarket review by FDA. The MDA created three classes of device types on the basis of the risks posed and the ability of postmarket controls to manage them. Each device type includes a number of devices.

As originally contemplated, the high-risk category (Class III) would require FDA review and approval, through the premarket approval (PMA) process, before a device could be marketed. The moderate-risk and low-risk categories (Class II and Class I, respectively) would not require as rigorous a review. Rather, device manufacturers would be required to notify the FDA at least 90 days before they marketed such a device. This premarket-notification requirement was contained in Section 510(k) of the Federal Food, Drug, and Cosmetic Act and is commonly referred to as 510(k) notification or 510(k) clearance.

A device not on the market at the time of the enactment of the MDA (a “postamendment” device) would be required to demonstrate through a 510(k) notification that it was “substantially equivalent” to a “preamendment” device and, for Class II devices, complied with the applicable performance standard promulgated by the FDA.

Congress’s interpretation of substantial equivalence, as stated in the Safe Medical Device Amendments (SMDA), is that: the term “substantially equivalent” or “substantial equivalence” means, with respect to a device being compared to a predicate device, that the device has the same intended use as the predicate device and that [FDA] by order has found that the device –

Has the same technological characteristics as the predicate device, or

Has different technological characteristics and the information submitted that the device is substantially equivalent to the predicate device contains information, including clinical data if deemed necessary by [FDA], that demonstrates that the device is as safe and effective as a legally marketed device and (II) does not raise different questions of safety and efficacy than the predicate device.

The term “different technological characteristics” means, with respect to a device being compared to a predicate device, that there is a significant change in the materials, design, energy source, or other features of the device from those of the predicate device.

If the new device were substantially equivalent, it would be cleared for marketing subject to the same terms and conditions as that preamendment device (for example, conformance to a performance standard). Postamendment devices that were not substantially equivalent to any preamendment device would be automatically placed in Class III and would require either PMA or reclassification by the FDA to Class I or Class II to enter the market.

The SDMA of 1990 permitted substantial equivalence to be established to marketed postamendment, in addition to preamendment, “predicate” devices, except for devices approved by PMA.

The Food and Drug Administration Modernization Act (FDAMA) of 1997 eliminated the requirement for 510(k) clearance for most Class I and some Class II device types, narrowed the array of issues that the FDA may consider in a 510(k) review, and directed the FDA to limit, to the “least burdensome” level, the scientific evidence requested to determine substantial equivalence of devices that involved new technologies.

In 2002, in an effort to address insufficient resources of the 510(k) and PMA programs, Congress passed legislation to create a 5-year user-fee program—the Medical Device User Fee and Modernization Act (MDUFMA). The user-fee program was renewed in 2007 for another 5 years.

IOM’s Conclusions

In a letter to CDRH Director Dr. Shuren, Dr. Challoner, Chair of the IOM Committee, noted that because of time constraints, the IOM committee was unable to study fully the seven recommendations referred to it by FDA. 

Dr. Challoner did note however, that the committee was able to address many of the broader issues related to CDRH’s 510(k) and Science Report Recommendations. The committee evaluated components of the 510(k) process and other relevant factors, including:

• The legislative history of the 510(k) program.
• The 510(k) regulatory framework that resulted from legislation.
• How the 510(k) process fits into the larger medical-device regulatory framework. (The 510(k) clearance process is an integrated component of the larger medical-device regulatory framework. The committee evaluated the 510(k) program in the context of this larger framework.)
• How the 510(k) process is implemented by the FDA.
• Available postmarket information on the safety and effectiveness of 510(k)-cleared devices.
• Other factors that affect medical-device regulation (for example, the process of innovation and the environment in which medical devices are developed and commercialized).

On the basis of its review and evaluation of legislative, regulatory, and administrative components of the 510(k) clearance process and other related components of medical-device regulation, the committee came to two major conclusions.

Conclusion 7-1 The 510(k) clearance process is not intended to evaluate the safety and effectiveness of medical devices with some exceptions. The 510(k) process cannot be transformed into a premarket evaluation of safety and effectiveness as long as the standard for clearance is substantial equivalence to any previously cleared device.

By law, the 510(k) process uses substantial equivalence to any previously cleared device as the standard for clearance with some exceptions. In practice, the assessment of substantial equivalence generally does not require evidence of safety or effectiveness of a device. According to the FDA and the Supreme Court, when the FDA finds a device substantially equivalent to a predicate device, it has done no more than find that the new device is as safe and effective as the predicate.

Importantly, devices that were on the market before the enactment of the 1976 MDA—the origin of all predicate devices for the 510(k) process—have never been systematically assessed to determine their safety and effectiveness. Because the preamendment device to which equivalence was established was not itself reviewed for safety or effectiveness, the committee found that clearance of a 510(k) submission was not a determination that the cleared device was safe or effective.

The 1990 SMDA permitted the FDA to require evidence of safety and effectiveness, including clinical studies, when necessary to determine whether a difference in technologic characteristics between a new device and its predicate renders the new device less safe or effective than the predicate or raises different questions of safety and effectiveness from the predicate.

If, despite the change in technologic characteristics, the new device is as safe and effective as the predicate, it will be found substantially equivalent. Nearly all 510(k) submissions for devices that have new technologic characteristics receive a determination of substantial equivalence.

The committee found that available information on postmarket performance of devices does not provide sufficient information about potential harm or lack of effectiveness to be a useful source of data on the safety and effectiveness of marketed devices.

The committee did not believe, however, that there is a public-health crisis related to unsafe or ineffective medical devices. Although the safety and effectiveness of preamendment Class II devices have not been systematically reviewed, their continued use in clinical practice provides at least a level of confidence in their safety and effectiveness.

Conclusion 7-2 Information that would allow an understanding of the extent to which the 510(k) clearance process either facilitates or inhibits innovation does not exist.

The committee noted that it is not necessary for a Class II device to be demonstrated to be innovative to be cleared for marketing. The FDA should not be the arbitrator of what constitutes innovation, nor should it seek to channel device development and premarket review toward agency-determined public-health priorities.

Accordingly, the committed noted that FDA’s role in facilitating innovation in Class II medical devices through premarket review should be to create a regulatory framework that sets appropriate thresholds for bringing products to the market. Those thresholds should be stringent enough to satisfy the agency’s objective of ensuring that marketed medical devices will be safe and effective throughout their life cycles but realistic enough to permit timely entry of new devices that may offer improvements over already marketed devices. Rather than be charged with promoting innovation, the committee believes that the FDA should seek to facilitate it.

To assess how innovation is affected by medical-device regulation, the committee studied the legislative history and implementation of the 510(k) process. It is unclear—and the committee argues that it is indeterminable, given current data—whether legislative, regulatory, and administrative changes over the last 35 years have had a positive or negative effect on innovation.

The committee noted that while FDA has procedures for developing, adopting, and implementing guidance and standards, the agency however, is persistently hindered in fully developing those materials by a lack of or limitations on human, fiscal, and technologic resources and capabilities. IOM acknowledged that the lack of clear guidance affects the timeliness and consistency of 510(k) device submissions provided by industry and the ability of reviewers to evaluate them.

RECOMMENDATIONS

CDRH’s proposed recommendations 1–3 are related to FDA’s postmarket authorities for medical devices. As stated in its report, the IOM committee found that the FDA has a broad array of tools available to address safety concerns in the postmarket period but that the agency does not use these tools extensively. However, IOM asserted that FDA has not adequately explained the limitations of the tools and why it has not used them more widely.

 As a result, the IOM committee recommended in its report that the FDA identify the limitations of its current regulatory tools and seek to address the limitations.

The IOM committee supports the concept of allowing conditional clearance based on postmarket surveillance in appropriate cases and has suggested this option as a potential component of a modified de novo process.

CDRH’s proposed recommendations 4–7 are related to FDA’s authorities for the 510(k) program. The IOM committee’s report concludes that the 510(k) process generally is not intended to evaluate the safety and effectiveness of medical devices and, furthermore, cannot be transformed into a premarket evaluation of safety and effectiveness. The report’s recommendations are focused not on making improvements in the 510(k) process but rather on the steps needed to develop a more rational medical-device regulatory framework.

The IOM committee does not believe that the FDA should use its sparse resources to modify the 510(k) clearance process. Instead, it believes that the FDA’s resources would be put to better use in obtaining information needed to develop a new regulatory framework for Class II medical devices and addressing problems with other components of the medical-device regulatory framework.

The committee recognized that scarce resources in the FDA could affect its ability to implement the recommendations and, therefore, has directed the recommendations toward activities that are useful in both the short term and the long term to conserve scarce resources.

With regard specifically to CDRH’s proposed recommendation 5, the IOM committee’s report includes an extensive discussion of issues related to the lack of clarity in the phrases intended use and indications for use. It is not clear to the IOM committee how combining these regulatory phrases would lead to more consistency and transparency.

Designing a New Medical Device Regulatory Framework

Recommendation 7-1 FDA should obtain adequate information to inform the design of a new medical-device regulatory framework for Class II devices so that the current 510(k) process, in which the standard for clearance is substantial equivalence to previously cleared devices, can be replaced with an integrated premarket and postmarket regulatory framework that effectively provides a reasonable assurance of safety and effectiveness throughout the device life cycle. Once adequate information is available to design an appropriate medical-device regulatory framework, Congress should enact legislation to do so.

The committee believes that a move away from the 510(k) clearance process should occur as soon as reasonably possible but recognizes that it will take time to obtain the information needed to design the new framework.

The new regulatory framework must be based on sound science and be developed in harmony with other components of the FDA’s medical-device regulatory system. The committee does not believe that available information is adequate to inform the design of an appropriate framework.

On the basis of its findings, the committee identified several topics to which the FDA should give particular consideration as it develops a new framework. A number of existing and emerging technologies (for example, combination products, software, nanotechnology, and medical robotics) merit detailed thought as part of the development of the new framework.

The committee stated that the FDA should consider integrating some elements of the quality-system regulations, especially those related to design controls and product-release criteria, into the premarket review process of the new framework to demonstrate that devices will perform as represented by their manufacturers. The FDA should also consider, as part of the new framework, including a more extensive review of device labeling and a system of tracking of labeling changes.

The committee urged FDA to create a regulatory framework that closely matches the ideal regulatory framework outlined by the committee:

• The process should be based on sound science.
• The process should be clear, predictable, straightforward, and fair.
• The process should be self-sustaining and self-improving.
• The process should facilitate innovation that improves public health by making medical devices available in a timely manner and ensuring their safety and effectiveness throughout their lifecycle.
• The process should apply relevant and appropriate regulatory authorities and standards throughout the life cycle of devices to ensure safety and effectiveness.
• The process should be risk-based.

Postmarketing Surveillance

The committee found substantial weakness in postmarketing surveillance of medical devices. The inadequate postmarketing surveillance systems, both those in the FDA and those that are privately funded, and the resulting lack of useful, consistent, and reliable data make it impossible to draw confident conclusions about the performance of medical devices that are now on the market.

Recommendation 7-2 FDA should develop and implement a comprehensive strategy to collect, analyze, and act on medical-device postmarket performance information.

IOM recommended that FDA give priority to postmarketing surveillance as an invaluable investment in short-term and long-term oversight of medical-device safety and assessment of device effectiveness. They stated that Congress should support the capacity of the FDA’s postmarketing surveillance programs by providing stable and adequate funding.

In particular, the committee noted that FDA should develop a postmarketing surveillance strategy to meet the following objectives: providing performance information for use in the premarket review process, informing the development and use of postmarketing tools (that is, general and special controls2) to manage the risk-benefit ratio throughout the life cycle of devices better, and informing the design of a new regulatory framework.

Postmarket Regulatory Authorities

The FDA reports that there are “important limitations” on the use of postmarket authorities, but does not describe them. The committee identified the procedural requirements that the FDA must fulfill to exercise the authorities, but the requirements do not in themselves appear to explain the agency’s perception that there are limitations on its use of postmarket tools.

Recommendation 7-3 The Food and Drug Administration should review its postmarket regulatory authorities for medical devices to identify existing limitations on their use and to determine how the limitations can be addressed.

IOM stated that FDA should analyze barriers to efficient and effective use of the authorities and identify means of mitigating them. If it is required, Congress should pass legislation to remove unnecessary barriers to the FDA’s use of postmarket regulatory authorities.

A Modified De Novo Process

The de novo process is a mechanism by which the FDA can down-classify, to Class I or Class II, devices that have no predicates but are deemed to be of low to moderate risk. The process avoids having novel, low- to moderate-risk devices go through the PMA process. In the de novo process, a 510(k) submission for the device is sent to the FDA, which issues a determination of “not substantially equivalent” and indicates to the applicant that the device may be eligible for the de novo process. Special controls generally need to be developed for the device. The general and special controls must be sufficient to provide reasonable assurance of safety and effectiveness.

IOM stated that the de novo process offers a potential basis of a better regulatory model for premarket review of Class II devices. In its current state, however, the de novo process is time-consuming and difficult for both the FDA and manufacturers to navigate.

Recommendation 7-4 The Food and Drug Administration should investigate the viability of a modified de novo process as a mechanism for evaluating the safety and effectiveness of Class II devices.

A pilot program of a modified de novo process would allow the FDA to determine its feasibility as a replacement for the 510(k) clearance process. As part of a modified de novo process, IOM recommended that the FDA explore ways to expedite development of special controls, develop guidance, and adopt standards for devices. The agency also should consider expanded use of external expertise and preinvestigational device-exemption meetings with submitters and use of conditional clearances (such as postmarketing surveillance or use of registries) for devices on which there is little premarket performance information.

A Continuous Quality-Improvement Program

The committee assessed that the FDA lacks a continuous quality-assurance process for regulation of medical devices. As a result, the FDA cannot effectively address new issues as they arise.

Recommendation 7-5 The Food and Drug Administration should develop and implement a program of continuous quality-improvement to track regulatory decisions on medical devices, identify potential process improvements in the medical device regulatory framework, and address emerging issues that affect decision-making.

The committee found evidence that the inadequate information technology and management infrastructure in the FDA affects not only the 510(k) program but the agency’s other programs for regulating medical devices. For example, because data systems are inadequate, the FDA does not have the ability to trace the history of 510(k) decisions. Prior 510(k) clearances are legally binding on the FDA when making 510(k)-clearance decisions.

Thus, any unsafe or ineffective devices are embedded in the system and as both a legal and a practical matter may be used as predicates for new devices until the predicates are removed from the market. It may be difficult for the FDA to remove devices from the market because it has no systematic way to identify them. By developing a business model grounded in continuous quality improvement, the FDA will be better able to identify problems with devices and develop the information and capacity to address them in a data-driven, transparent manner.

Facilitating Innovation in the Medical Device Industry

The FDA’s role in facilitating innovation with respect to Class II devices is to create and enforce a regulatory framework for which the threshold to market provides reasonable assurance that medical devices are safe and effective throughout their life cycle while permitting timely entry of new devices that may offer improvements over already marketed devices. The committee did not find assessments of how much and in what way (that is, facilitating or inhibiting) innovation is influenced by the 510(k) clearance process.

Recommendation 7-6 FDA should commission an assessment to determine the effect of its regulatory process for Class II devices on facilitating or inhibiting innovation in the medical-device industry.

The recommended study should include various ways to measure innovation beyond “time to market” or the number of devices of a particular type on the market and instead focus on a broader understanding of the relationship among regulation, innovation, and patient health and safety throughout device life cycle.

Software

Manufacturers are increasingly using software in devices, software as devices, and software as a tool in producing devices. The committee found that current guidance on software validation is insufficient for preventing serious software-based device failures.

Recommendation 7-7 FDA should develop procedures that ensure the safety and effectiveness of software used in devices, software used as devices, and software used as a tool in producing devices.

The FDA should develop a better understanding of the roles of software in medical devices, analyze its potential effects on the safety and effectiveness of the devices, and insist on evidence-based procedures that ensure device safety and effectiveness. The committee believes that the FDA should review and update its guidance on software validation.

Preamendment 510(k)-Eligible Class III Devices

After 35 years, the FDA has not completed the task of calling for PMAs for or reclassifying preamendment Class III device types. Until the FDA completes that task, those devices are allowed to enter the market through the 510(k) clearance process. Congress in 1990 directed the FDA to complete this task in a timely manner. Twenty-six Class III device types remain eligible to enter the market through the 510(k) process.

The FDA has begun a five-step process to require PMAs or to down-classify those device types. As of April 2011, the FDA had assessed the risks and benefits associated with 21 device types (step 2 of the process) and had received and reviewed public comments on five device types (step 4 of the process). The agency has not issued final rules requiring PMAs or reclassifying the devices for any of the 26 device types. The committee recognizes the resource constraints that have prolonged the process, but it nevertheless urges the FDA to give high priority to completing the task.

Recommendation 7-8 FDA should promptly call for PMA applications for or reclassify Class III devices that remain eligible for 510(k) clearance.

This recommendation though not explained in the report will potentially add years to the approval of several important devices.

Discussion

Overall the recommendations are broad and in many places of the report vague.  It is disappointing that rather than answer the questions that the FDA asked to be answered the committee for the most part went down a road of strategic vs. tactical recommendations.   Not only did they not answer the questions they focused on the legislative history of the FDA and the 510K clearance.

What this report does is require that all class II devices that are substantially equivalent will now have to go through extensive studies and additional approval process.  It is not clear that there is evidence to make these recommendations.

Much of this as previously noted can be accounted for by the lack of inventor and industry representation on the committee.   You can clearly tell from reading the report the complete lack of understanding of current 510K approval process or have spent time filing a 510K.

The report does recognize the vast majority of device manufactures are small businesses with 50 or fewer employees and “Delays during any of those phases (regulatory and insurance reimbursement) extends the “burn rate” of capital (that is, the amount of funds spent in a given period) and can create financial difficulties for companies, particularly startup companies, which often have limited financial resources”. 

Throwing out the 510K process in favor of a more rigorous review process in the absence of evidence that there is an unacceptable risk posed by current class II devices has the potential to cause considerably more harm than good.  It was clear given this restraint that additional regulations especially in the pre-market arena will result in vastly fewer devices coming to market.  This result will help neither inventors nor patients.

Institute of Medicine FDA 510K Advisory Committee Fails to Meet US Fair Balanced Standards

 
On Friday of this week, the Institute of Medicine will release their recommendations on the FDA 510K process. The IOM recommendations may determine the fate of all future medical device approvals.  Unfortunately the panel has no industry or inventor representation which may constitute a violation of federal laws on fair balance in advisory committees to the federal government.

 Recently, the Washington Legal Foundation (WLF) urged the Food and Drug Administration (FDA) not to use any advice or recommendations provided by the Institute of Medicine committee (the“IOM Committee”) assembled for the purpose of assessing whether changes are need in the 510(k) approval process, the process by which most medical devices reach the market.

In a Citizen Petition filed with FDA, WLF argued that FDA’s use of advice or recommendations from the IOM Committee would violate § 15 of the Federal Advisory

Committee Act (FACA). Section 15(a) provides that an agency may not use any advice or recommendation developed by the committee unless the committee has complied with all the requirements of § 15 – including § 15(b)(1)’s requirement that the committee membership be “fairly balanced.”

WLF charged that the IOM Committee is not fairly balanced, and thus, Section 15(a) of FACA prohibits FDA from “us[ing] any advice or recommendation provided by” any IOM committee that is not “fairly balanced.”

WLF’s Citizen Petition argued that § 15 of FACA does not permit FDA simply to rely on assurances from the IOM that the Committee is fairly balanced, but rather it must determine for itself that the Committee is indeed fairly balanced before it can consider any recommendation from such a committee.

WLF asserted that it is particularly important for FDA to ensure that the Committee meets the fair balance requirement, even if the result is to require the appointment of individuals with conflicts of interest.

“Using advice from a committee that lacks fair balance encroaches upon Congress’ mandate that each Advisory Committee should be representative of a broad range of viewpoints and should include affected individuals,” said WLF Chief Counsel Richard Samp after filing WLF’s Citizen Petition.

“The IOM Committee is not ‘fairly balanced’ under any plausible definition of that term; numerous persons and groups directly affected by the Committee’s work are totally unrepresented on the Committee’s membership,” Samp said.

Background

Courts examining FACA have concluded that the “‘fairly balanced’ requirement was designed to ensure that persons or groups directly affected by the work of a particular advisory committee would have some representation on the committee.

Congress provided a fair degree of guidance regarding what a “fairly balanced” committee entailed and what it hoped to accomplish by requiring fair balance. It stated, for example, that:

•  Advisory committees should avoid “the charge of favoritism” by ensuring that “a particular region, university, industry, company, or discipline” are not “overrepresented”;
• Rather than relying on “personal acquaintances,” agency “clientele,” or individuals who are “close” to the agency, an agency should broaden advisory committee membership to include “[i]ndividuals with ideas, knowledgeable people, and affected individuals” (emphasis added);
• Committee members should “be representative of a broader range of interests . . . than has been the case”; and
• Agencies should avoid the pre-FACA state of affairs, under which many agencies were “receiving their advice from sources which have special or limiting viewpoints.”

Congress was particularly concerned with the requirement that a broad range of viewpoints be included on advisory committees, even (and especially) unpopular viewpoints. Such diversity of viewpoints is especially important when (as here) the issues being considered by a committee are policy-oriented.

Where the functions to be performed are not “narrow and explicit,” but instead involve “diverse and far-reaching issues that affect others,” broader representation on the committee is required. This is especially true of committees whose purpose is “to study the effects of a particular type of regulation . . . on the public, which is exactly what FDA asked IOM to do regarding the 510(k) program does.

According to their press release, WLF filed its brief with the pro bono assistance of Robert Klepinski, an attorney from Minneapolis, Minnesota.

FDA and IOM Committee for 510(k)

In 2009, FDA commissioned the IOM to conduct a detailed analysis of the § 510(k) system. FDA referred several issues to the IOM Committee, including these principal questions: (1) whether the current § 510(k) process optimally protects patients; and (2) whether it promotes innovation in support of public health.  FDA asked the IOM Committee:

•  To consider defining the scope and grounds for the exercise of the Center’s authority to fully or partially rescind a 510(k) clearance.
• To seek greater authority to require post market surveillance studies as a condition of clearance of certain devices.
• To develop guidance defining “Class IIb” devices for which clinical information, manufacturing information or, potentially, additional evaluation in the postmarket setting would typically be necessary to support a substantial equivalence determination.
• To clarify when a device should no longer be available for use as a predicate.
• To consolidate the concepts of “indication for use” and “intended use” into a single term, “intended use.”
• To consider the possibility of requiring each 510(k) submitter to keep at least one unit of device under review available for CDRH to access upon request.
• To explore the possibility of pursuing a statutory amendment that would provide the agency with the express authority to consider an off-label use when determining the “intended use” of a device.

FDA has made clear that it intends to act upon any advice and recommendations it receives from the IOM Committee. It should be noted that after these seven additional questions were posed to the Committee, FDA has sought no further input from the medical device industry or patient groups regarding the questions posed. Nor have any of the IOM Committee meetings conducted during this time period been open to the public.

WLF Citizens Petition to FDA

The IOM Committee is composed of twelve members. The composition of the committee is as follows: five doctors, three lawyers, and several academics with a variety of technical backgrounds. Although each member has impressive qualifications, the committee lacks individuals with experience in areas of critical importance to the Committee’s work.

For example, not present on the committee are innovators, inventors or any product developers who are familiar with the clearance process. In particular, the IOM

Committee’s membership includes no one with any experience in seeking to use the 510(k) process to obtain FDA marketing approval for a new medical device.  There are no individuals who routinely prepare § 510(k) applications, or any professionals from the medical device industry.

Even more striking is the fact that committee does not include any representatives of patients and patient advocacy groups that have benefitted from the development of innovative medical devices. Nor does it include any representatives of patients, who have a strong interest in ensuring that the 510(k) program continues to permit the speedy approval of new, life-saving medical products.

Additionally, there are no entrepreneurs, venture capitalists, investment bankers, or angel investors with experience financing medical device innovations, even though one current controversy surrounding the § 510(k) program is its effect on investment in new companies, and even though the second of FDA’s initial two questions to IOM related to the program’s effect on innovation.

In addition to the absence of members with the expertise/backgrounds enumerated above, the IOM Committee does not appear to include members from a wide variety of policy perspectives. The § 510(k) program has been criticized from all sides of the ideological spectrum. Some view the program as too lax from a safety perspective; they charge that the program has allowed medical devices to be marketed with insufficient attention being paid to whether the product is safe for its intended use. Others view the program as overly bureaucratic and one that unnecessarily delays the marketing of innovative products.

The former perspective is well-represented on the Committee. Indeed, one member has worked for 20 years at Public Citizen Litigation Group, a public interest law firm that has been highly critical of the § 510(k) process and has asserted that dangerous medical devices have been allowed to enter the market because devices are approved too quickly.

But no member of the Committee is publicly identified with the latter perspective. The inclusion of a Public Citizen lawyer makes all the more inexplicable the IOM’s failure to include an industry representative or anyone else with practical experience in navigating the § 510(k) program.

The IOM does not contest that such an absence exists. Rather, the IOM evidently takes the position that inclusion of individuals with meaningful experience in the development of innovative medical devices and/or navigating the § 510(k) process would risk violating FACA’s conflict-of-interest requirements.

FACA Conflict-of-Interest Rules

Numerous interested parties have contacted the IOM to express their dismay over the unbalanced nature of the IOM Committee and to urge the IOM to broaden the Committee’s composition. For example, in a February 17, 2010 letter to the IOM, the Medical Device Manufacturers Association (MDMA) stated that “the current makeup of the committee does not adequately reflect all relevant stakeholders in the process” and urged the IOM to “take the necessary steps to ensure the committee’s review of the 510(k) process is fair, balanced and inclusive by including representatives of key stakeholder groups to participate as members of the committee.” Despite such entreaties, the IOM took no steps to balance the IOM Committee’s membership by adding members with backgrounds not represented on the Committee.

WLF argued that the conflict-of-interest rules do not prevent the appointment of committee members with ties to firms doing business in the field, particularly if individuals are from a background that otherwise would go unrepresented on the Committee.

 Moreover, WLF recognized that nothing in FACA authorizes the National Academy of the Sciences (NAS) to ignore the “fairly balanced” requirement in order to avoid conflicts of interest among committee members. Indeed, FACA § 15(b)(1) indicates that in the event of a direct conflict between these two provisions, it is the conflict-of-interest provision that is to give way.

 The Academy’s own internal policies regarding committee selection recognize the need to include individuals with diverse points of view. Those policies explicitly permit committees to include members who are biased or have expressed a strong opinion on a particular issue of interest. According to the Academy guidelines governing the committee appointment process, “[a] point of view or bias is not necessarily a conflict of interest. The IOM recognizes that member bias is not only permissible, it is sometimes necessary.

 According to the NAS Policy on Committee Composition and Balance, “[f]or some studies . . . it may be important to have an ‘industrial’ perspective” if such a perspective is “vital to achieving an informed, comprehensive, and authoritative understanding and analysis of the specific problems and potential solutions to be considered by the committee.” However, the IOM must balance these perspectives to produce an overall objective committee and avoid bias or the perception of bias. If these individuals have conflicts of interest, they may nevertheless be included on the committee to provide needed expertise, knowledge, balance, or perspective. In such cases, the conflict of interest is “unavoidable” and simply must be disclosed.

Consequently, WLF pointed out that “conflict-of-interest rules have not prevented numerous other committees established by the IOM at the behest of FDA from including industry representatives and others from far more diverse backgrounds than IOM Committee members.” In fact, out of ten current or recent FDA sponsored IOM activities, at least half contain members with industry background and at least three committees contain members with disclosed conflicts of interest.

Clearly, individuals with industry background or connections are frequently deemed valuable and necessary for IOM committees to fulfill their functions, despite obvious conflicts of interest. Thus, it is not unusual that individuals with “unavoidable” conflicts of interest are included on IOM committees.

Conclusion

Ultimately, the IOM has explicitly recognized – both in its own internal policies and in its past committee appointment practices – that conflict-of-interest rules do not prevent the appointment of committee members with ties to firms doing business in the field, where such individuals’ backgrounds will provide the committee with expertise in areas that would otherwise be absent. In light of that recognition, the IOM cannot plausibly maintain that conflict-of-interest rules justified its failure to appoint a “fairly balanced” committee in this instance.

Accordingly, WLF first requested that FDA either proceed with its comprehensive review of the § 510(k) program without obtaining any input from IOM, or else request that IOM reconstitute its committee so as to come into compliance with the “fair balance” requirement.

Second, WLF suggested to FDA that it issue guidelines that would help other advisory committees comply with the requirement of “fair balance” and avoid a similar situation where FDA would not be able to use an advisory committee’s report because the committee failed to comply with § 15 of FACA. Finally, WLF requested that FDA issue a guidance document explaining FDA’s obligations under FACA, and what advisory committees must do in order to permit FDA to make use of any advice or recommendations provided to the agency.

Institute of Medicine Report - The Future of Nursing: Leading Change, Advancing Health

 
As the current debate surrounding the Affordable Care Act (ACA) continues in Congress, a number of strengths and weaknesses of the legislation are being emphasized. One specific issue is how the American health care system will treat an additional 32 million Americans.

In order to make quality care accessible to the diverse populations of the United States, many stakeholders in the health care industry believe it is necessary to expand the role of health care providers including primary care physicians, pharmacists, and especially nurses. Consequently, the Committee on The Robert Wood Johnson Foundation (RWJF) Initiative on the Future of Nursing, at the Institute of Medicine (IOM), developed a vision for a transformed health care system to address these issues.

In their report entitled “The Future of Nursing: Leading Change, Advancing Health,” IOM envisions a future where “primary care and prevention are central drivers of the health care system, interprofessional collaboration and coordination are the norm, and payment for health care services rewards value, not volume of services, and quality care is provided at a price that is affordable for both individuals and society.” To achieve these goals, IOM recognized that many of the aspects of the health care system must be changed, including the nursing profession, which, with more than 3 million members, represents the largest segment of the health care workforce.

Background

In 2008, RWJF approached the IOM to establish a 2-year Initiative on the Future of Nursing, with the primary mission of producing a report containing recommendations for an action-oriented blueprint for the future of nursing, including changes in public and institutional policies at the national, state, and local levels. To produce this report, a committee examined and produced recommendations related to the following issues, with the goal of identifying vital roles for nurses in designing and implementing a more effective and efficient health care system:

Reconceptualizing the role of nurses within the context of the entire workforce, the shortage, societal issues, and current and future technology;

Expanding nursing faculty, increasing the capacity of nursing schools, and redesigning nursing education to assure that it can produce an adequate number of well prepared nurses able to meet current and future health care demands;

Examining innovative solutions related to care delivery and health professional education by focusing on nursing and the delivery of nursing services; and

Attracting and retaining well prepared nurses in multiple care settings, including acute, ambulatory, primary care, long term care, community and public health.

Framing the Discussion

The question presented to the committee that produced this report was: What roles can nursing assume to address the increasing demand for safe, high quality, and effective health care services? IOM recognized that the new health care laws will require ways to address challenges in the management of chronic conditions, primary care (including care coordination and transitional care), prevention and wellness, and the prevention of adverse events (such as hospital-acquired infections). The report also identified the demand for better provision of mental health services, school health services, long-term care, and palliative care (including end-of-life care) is increasing as well.

Accordingly, IOM noted that “nursing brings to the future a steadfast commitment to patient care, improved safety and quality, and better outcomes. “ In fact, the report asserts that most of these challenges “speak to traditional and current strengths of the nursing profession in such areas as care coordination, health promotion, and quality improvement.” As a result, IOM emphasized that “nurses have key roles to play as team members and leaders for a reformed and better-integrated, patient-centered health care system.”

Expanding the Role of Nurses

In order to expand the role of nurses, the report first recognizes the need to allow nurses to practice in accordance with their professional training. IOM also recognized the need for nurse education to “better prepare them to deliver patient-centered, equitable, safe, high-quality health care services; engage with physicians and other health care professionals to deliver efficient and effective care; and assume leadership roles in the redesign of the health care system.” Consequently, IOM asserted that “an expanded workforce to serve the millions who will now have access to health insurance for the first time will require:

• Changes in nursing scopes of practice
• Advances in the education of nurses across all levels  
• Improvements in the practice of nursing across the continuum of care
• Transformation in the utilization of nurses across settings, and
• Leadership at all levels so nurses can be deployed effectively and appropriately as partners in the health care team.

Report Recommendations

As a result of its deliberations, the committee formulated four key messages that structure the discussion and recommendations presented in this report:

• Nurses should practice to the full extent of their education and training.
• Nurses should achieve higher levels of education and training through an improved education system that promotes seamless academic progression.
• Nurses should be full partners, with physicians and other health professionals, in redesigning health care in the United States.
• Effective workforce planning and policy making require better data collection and an improved information infrastructure.

IOM arrived at these recommendations by acknowledging the unique role that nurses play in the health care system. Because nurses have regular and close proximity to patients and scientific understanding of care processes across the continuum of care, they have a unique ability to act as partners with other health professionals and to lead in the improvement and redesign of the health care system and its many practice environments, including hospitals, schools, homes, etc.

In addition, the recommendations reflect the ability of nurses to help bridge the gap between coverage and access, to coordinate increasingly complex care for a wide range of patients, and to enable the full economic value of their contributions across practice settings to be realized. This includes nurse’s crucial role in preventing medication errors, reducing rates of infection, and even facilitating patients’ transition from hospital to home.

Nursing practice covers a broad continuum from health promotion, to disease prevention, to coordination of care, to cure—when possible—and to palliative care when cure is not possible. However, IOM acknowledged that “many members of the profession require more education and preparation to adopt new roles quickly in response to rapidly changing health care settings and an evolving health care system.”

The recommendations also recognize how restrictions on scope of practice, policy- and reimbursement-related limitations, and professional tensions have undermined the nursing profession’s ability to provide and improve both general and advanced care. As a result, IOM asserts the need to transform the work environment, scope of practice, education, and numbers of America’s nurses by creating a health care system that delivers the right care—quality care that is patient centered, accessible, evidence based, and sustainable—at the right time.

Conclusion

Ultimately, the IOM recommendations focus on the critical intersection between the health needs of diverse populations across the lifespan and the actions of the nursing workforce. Based on these recommendations, it is clear that America will need to educate and train nurses to handle many areas of medicine and to help create a patient-centered health care system if health care reform will be successful and improving care and reducing costs.

This is encouraging, but some system changes need to happen for nurses, including changes in compensation towards procedures and productivity, the shift work mentality often found with hospital nurses needs to change.  Nurses taking on more responsibility will mean greater accountability.

The role of continuing education (CE) will be crucial in realizing the IOM recommendations. Not only will CE for nurses need to cover many more areas of training and education, it will also need to focus on team-based approaches for collaborating with other physicians, which also means that CE for physicians will have to focus on interacting with nurses. Accordingly, CE must play a critical role in realizing the potential benefits of health care reform by improving the role nurses play in the health care system and improving patient outcomes. 

IOM: Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease

In 2008, the Food and Drug Administration (FDA) asked the Institute of Medicine (IOM) to conduct a study on the evaluation process for biomark­ers, focusing on biomarkers and surrogate endpoints in chronic disease.

 

Biomarkers are characteristics that indicate biological processes, and are essential for monitoring the health of both individuals and communities.

 

According to IOM’s report released this month entitled Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease, “doctors, scientists, and other health professionals use biomarkers as tools to obtain information about a person’s health status or response to interventions.”

 

The study, which was initiated by FDA’s Center for Food Safety and Applied Nutrition, noted how “some biomarkers, called surrogate endpoints, are used as sub­stitutes for actual clinical endpoints such as incidence of disease or death.” These surrogate endpoints are “intended to predict benefit or harm based on scien­tific evidence, and they are used in practice when it is difficult to collect data based on clinical endpoints.”

 

The committee used examples of case studies using biomarkers and surrogate endpoints in various diseases, such as low-density lipoprotein (LDL) and high-density lipoprotein choles­terol levels in cardiovascular disease.  In evaluating these case studies, the report’s authoring committee recommended that the “FDA adopt a consistent scientific process and framework for biomarker evaluation in order to achieve a rigorous and transparent process for all stakeholders.”

 

IOM believes it is important to create such a framework because although “LDL cholesterol level is an excellent biomarker in many situations, it does not always fully predict cardiovascular disease outcomes; in other words, it cannot be assumed to be a surrogate endpoint.”  This led them to assert that “no surrogate endpoint is a perfect substitute for a clinical endpoint.”

 

FDA and Surrogate Endpoints

 

FDA has the responsibility of examining data and making deci­sions about whether biomarkers or surrogate endpoints can be used for regulatory reviews. The FDA sometimes uses surrogate endpoints such as LDL to make decisions about health claims and drugs. When manufacturers present evidence to the FDA that a product reduces LDL levels, the FDA considers the evidence in relation to the sur­rogate endpoint and makes decisions about car­diovascular health claims or drugs based on that evidence.

 

As a result of this process, the committee recommended that the “FDA use the same degree of scientific rigor for evaluating biomarker use across regulatory areas, includ­ing drugs, medical devices, biologics, foods, and dietary supplements.” In carrying out this proposal, IOM noted that Congress may need to strengthen FDA authority to accomplish these goals.  While legislative action has not been taken yet, IOM noted that the biomarker evaluation process should consist of the following three steps:

 

-   Analytical validation – Biomarker tests need to be reliable, reproducible across multiple laboratories and clinical settings, and main­tain adequate sensitivity and specificity before data based on them can be used in subsequent evaluation steps;

 

-   Qualification – Qualification requires: (1) eval­uation of the nature and strength of evidence regarding whether a biomarker is associated with the disease, and (2) assembly of available evidence demonstrating that interventions targeting the biomarker impact the clinical endpoints of interest; and

 

-   Utilization – Decisions to use biomarkers depend on the specific use proposed in addi­tion to the strength of the available evidence. Strong evidence and a compelling context are needed for the use of a biomarker as a surro­gate endpoint.

 

In addition to this process, IOM recommends that for biomarkers with regulatory impact, the FDA convene expert panels to evaluate biomarkers and bio­marker tests. The committee noted that the initial evaluation of analytical vali­dation and qualification should be conducted separately from a particular context of use, and then expert panels should reevaluate ana­lytical validation, qualification, and utilization on a continual and a case-by-case basis. Other recommendations included the need for:

 

-   Congress to strengthen the FDA’s authority to request and enforce post-market surveillance across drugs, devices, and biologics when approvals are initially based on putative sur­rogate endpoint data;

 

-   Congress to grant the FDA authority to request studies and suffi­cient authority to act on the results of studies on consumer understanding of claims on foods and supplements.

 

-   HHS to facilitate a coordinated, department-wide effort to encour­age the collection and sharing of data about bio­markers for all uses, including drugs, biologics, devices, and foods; and

 

-   FDA, in coordination with other federal agencies, to build data infrastructure and surveillance systems to handle the information necessary to gain sufficient under­standing of the effects of biomarker utilization.

 

Conclusion

 

IOM’s recommendations underscore their own acknowledgement that “biomarkers are important in that they can enable faster clinical trials for interventions, improve understanding of healthy dietary choices, assist public health professionals in identifying and tracking health concerns, and help health care practitioners and patients make decisions.”  These obvious benefits from allowing a smooth and simple process for biomarkers overwhelmingly outweighs any risks.

 

While IOM seems to downplay the critical role biomarkers play, they do recognize that scientific information is always evolving and “allows for the introduction of new, life-saving health interventions.”  As a result, they correctly assert that “modern medicine depends on biomarkers.” Yet by suggesting a seemingly burdensome process to evaluate bio­markers in general, and surrogate endpoints spe­cifically, IOM has essentially proposed to limit innovation. Moreover, IOM’s report did not address the feasibility or cost of these recommendations, which will likely divert important resources and staff away from helping patients.

 

With thousands of drugs in the pipeline being developed, hundreds more waiting approval at FDA, and a severe shortage of staff and resources at numerous health agencies, does IOM really want to make it harder for drugs to get approved? Patients are presently waiting for treatments for severe and chronic illnesses, such as cancer and heart disease. Does making those individuals wait longer, so that FDA can increase its “degree of scientific rigor” actually protect patients or harm them?

IOM Report: National Cancer Clinical Trials System for the 21st Century

“Advances in biomedical research continue to create significant opportunities for improving the detection, treatment, and prevention of cancer,” according to a recent Institute of Medicine (IOM) report titled “A National Cancer Clinical Trials System for the 21st Century: Reinvigorating the NCI Cooperative Group Program.”

These significant opportunities have resulted from collaboration with industry and clinical trials that have tested the safety and therapeutic benefit of promising treatments.  

Such clinical trials are essential in translating new knowledge into tangible benefits for patients with cancer—the second leading cause of death in the United States, behind heart disease. This collaboration with industry is also important because it has played a key role in developing new and improved cancer therapies.

Although NCI’s “efforts complement the clinical trials that pharmaceutical and biotechnology companies conduct,” the program has “been falling short of its potential to conduct the timely, large-scale, innovative clinical trials needed to improve patient care,” according to the report.

IOM Report

IOM reviewed the roles of the various stakeholders involved in cancer clinical trials and recommended a series of changes across the board to improve clinical trials by:

-   Using a system that efficiently responds to emerging scientific knowledge;

-   Involving broad cooperation of stakeholders; and

-   Leveraging evolving technologies to provide high-quality, practice-changing research.

IOM feels that by adopting these changes, “clinical trial participation would be desirable for patients and physicians because it would provide access to innovative therapies that reflect patient preferences and are reimbursed.” The report also “emphasizes the need to maintain a robust, standing cancer clinical trials network by preserving the historical strengths of the Cooperative Group Program while:

·   Improving the speed and efficiency of the design, launch, and conduct of clinical trials;

·   Making optimal use of scientific innovations;

·   Improving selection, prioritization, support, and completion of clinical trials; and

·   Fostering expanded participation of both patients and physicians

Improving Speed and Efficiency

Since the average time required to design, approve, and activate a cancer clinical trial is two years, IOM recommended that protocol development be coordinated and streamlined by implementing the processes proposed by the Operational Efficiency Working Group.

They proposed reorganizing clinical trial structures and operations into a truly national trials network, expanding drug distribution, and implementing standardized case report forms and remote data capture systems to aid trial efficiency.

This would entail consolidating many of the administrative functions and processes within the Cooperative Group Program, streamlining government oversight of trials, and enhancing collaboration among stakeholders. In doing so, IOM called on federal agencies, academic centers, community practices, and the pharmaceutical industry to work together to improve the system.

Incorporating Innovative Science

To advance progress in clinical trials, IOM recommended that biospecimens collected from patients in the course of Cooperative Group trials be submitted to standardized central biorepositories supported by a national inventory and a defined peer-review process for accessing specimens for study.

This strategy will allow trials to focus more on the treatment strategy most likely to benefit individual patients, and will allow for more systematic, multidisciplinary, and dynamic approaches when developing standards for new scientific methods and technologies used in trials, to ensure appropriate and consistent use.

Prioritizing and Supporting Trials

To increase the speed of advances in oncology care, IOM recommended that NCI focus on the facilitation of Cooperative Group trials by prioritizing, selecting, and supporting clinical trials and for enrolling patients quickly after a trial is launched. They also want NCI to enroll patients in any high-priority trial, and eliminate sites with low patient accrual.

Patient and Physician Participation

To get the most participation out of trials, IOM recognized the need for adequate reimbursement for physicians and patients, such as reducing career and financial concerns and providing salary support for protected research time. They also called for the system to reward more collaborative work, and to accord more clinical trials at academic medical centers.

Patients also need more consistent coverage from insurance for costs in clinical trials, and IOM asked that CMS establish consistent payment policies to cover patient care costs (except for specific study-related costs that should be paid for by the drug or device manufacturer) in clinical trials approved through the NCI prioritization mechanism.

Conclusion

It is disappointing that the IOM which is generally favors all new regulations, has pointed out that our regulatory and clinical trials system is unnecessarily slow.

Given the pace at which new scientific findings are emerging, a trial concept may become outdated in the two years it is being tested, and patients simply cannot afford this setback.

If improved treatments for cancer are delayed, patient lives will be lost unnecessarily unless the efficiency and effectiveness of the clinical trials system improves.

In order to improve this system, all stakeholders, including physicians, patients, and health care insurers, as well as NCI, FDA, other federal agencies, academia, foundations, and industry, must work together.

Institute of Medicine: Redesigning Continuing Education in the Health Professions

 

The Institute of Medicine (IOM) issued a report titled Redesigning Continuing Education in the Health Profession.  This is the outcome of the IOM Planning a Continuing Health Professional Education Institute which was funded by the Josiah Macy Foundation.

 

The report consisted of a 12 month study, 3 face-to-face meetings, 2 public workshops with 17 speakers, an “extensive literature review,” and 16 external reviewers.

 

Overall the report is ambitious, involving research and regulation for a diverse group of professions including physicians, nurses, pharmacists, physical therapist, social workers, dieticians, dentist and all other health professionals. 

 

It is a top down driven report calling for a central system for research and regulation of all continuing education in the health professions (CE).  It somewhat falls into a belief that a small group of 13 people should decide what is best for educators and clinicians, potentially missing the marketplace and innovation.

 

The report states that the purpose of CE is to enable health professionals to keep their knowledge and skills up to date, with the ultimate goal of improving performance and patient outcomes. Consequently, the report suggests that there are major flaws in the way CE is conducted, financed, regulated, and evaluated.

 

They report flaws that exist in the current CE system include:

 

· Meeting regulatory requirements rather than identifying personal knowledge gaps

· Concerns about conflicts of interest in CE activities

· Regulations that vary widely by profession, specialty, and state, leading to inconsistent learning

 

The report also claims that the science underpinning CE is fragmented and underdeveloped, which leads to “didactic learning methods (e.g., lectures) in traditional settings (e.g., auditoriums); little specific information about how to best support learning; and health professionals lacking a dependable basis for choosing among CE programs.

 

Such a claim is somewhat overstated as any heart surgeon, oncologist, or other specialist would contest to, as CE programs have helped keep these specialists up to date on the newest treatments and breakthroughs.

 

From these claims, the report calls for a new, comprehensive vision for CE that prepares all health professionals to perform to their highest potential, which they call Continuing Professional Development (CPD). In CPD, learning opportunities:

 

· Stretch from the classroom to the point of care

· Shift control of learning to individual practitioners

· Adapt to individuals’ learning needs

 

The CPD system offers promise to:

 

· Advance evidence-based, inter-professional, team-based learning

· Strengthen the research workforce, particularly through academic institutions

· Engender coordination and collaboration among the profession

· Provide higher quality for a given amount of resources

· Lead to improvements in patient health and safety

 

To achieve this CPD system, the report recommends that the Secretary of the Department of Health and Human Services should commission a planning committee to develop a public-private institute for continuing health professional development. The role of this institute would be to coordinate and guide efforts in content and knowledge of CPD; regulation across states and professions; financing of CPD; and strengthening of a scientific basis. What this boils down to is simple: government controlled continuing education. One obvious problem is: where are the resources (money and staff) to support such a drastic change?

 

Financing CE

 

The report rightly asserts that CE will need the necessary resources (money) because of the innovations in learning, specifically about treatments, drugs and devices created by academic-industry-government collaboration. The report notes that at present, 58% of CE funding comes from industry, and out-of-pocket cost to physicians is 42%, or $1,200 per physician per year.

 

In addition, IOM notes how medical schools provide the most hours of continuing medical education (45 percent of the total). Almost another quarter of all CME hours comes from professional societies and organizations, who are also important providers outside of medicine (ACCME, 2008). Other sources of CME hours include employers of health care professionals, such as the Department of Veterans Affairs (VA), and health care institutions, such as hospitals and insurers.

 

Other providers of CME, who are crucial for medicine and physician include medical education and communication companies (MECCs). These companies which are accredited by the ACCME, provide tremendous service to physicians and health care providers, even though the report believes their motivations to sometimes have the potential for bias.

 

This potential however is minimal, because as the report states, “CE funding comes from numerous sources, including health professionals themselves, employers, commercial entities, and the government.” Bias can easily be avoided by the fact that because many physicians have to pay for CE “they are likely to choose an activity based on its perceived value.” In other words, they know who is funding the program, and as highly trained professionals will be able to judge the content and clinical benefit accordingly.

 

Ideas for new ways to fund CE are obviously important but, as the report notes, “the federal government provides little direct financing for CE for health professionals.” Although some federal money is spent on CE for professionals in the VA or DOD, “no consistent investment for a large majority of non-government related health professionals exists.”

 

The lack of government funding, and thus the increased reliance on private industry funding, causes critics to believe conflicts may arise. Such claims are also contradictory considering the report itself states that “MECCs can be significant resources, for example, by supplying well-trained staff who provide high quality CE.” Moreover, the potential for bias has also been significantly reduced over the past five years by ACCME and PhRMA, who have ensured that commercial interests are kept separate from learning activities and course content. For example, the ACCME requires CME providers to give a balanced view of therapeutic options and encourages the use of generic names of therapies, rather than promoting specific proprietary names (ACCME, 2006).

 

With organizations already regulating CE payments, it is hard to see what is wrong with the current system. Today, CE does “directly align with the goals of driving improved quality of care and patient safety and it does support a mix of activities that are effective both in terms of performance and cost.” To safeguard CE from making a profit goes against the very nature of freedom of enterprise and numerous American principles. Investing in CE requires a significant risk and investment, and profits do not reflect the impact such programs have on saving lives and training physicians.

 

IOM notes that if all industry funding were removed, the average physician could fund his own CME and continue attending the same types of CME activities by investing about $3,500 annually. But the report states that this is an “estimate and does not take into account the nuances of the various sources of CME funding.” The report also states that ‘it is not even clear how the content would change in the event” that industry funding is removed.

 

Another reason why removing industry funding of CME is problematic is because physicians want to learn and be motivated in a network of their peers to be updated on new practices. In fact the report states that “a mix of cost-effective CE could be achievable in the current financing environment.”

 

In the absence of evidence, the committee assumes that physicians could maintain participation in activities similar to the ones in which they currently participate (e.g., a hybrid of low-cost, journal-based CME with occasional participation in higher quality, higher cost activities) without paying more out of pocket.

 

Since IOM clearly states that “no evidence exists to predict how physicians would respond to changes in CE financing, and a determination cannot be made of whether the costs of an ideal system would be greater than the costs of the current system,” why make any changes?

 

Summary

 

Overall there are lots of interesting recommendations dealing with continuing education in this report.  They have truly identified a problem in the system that the focus of CE is often on meeting regulatory requirements rather than identifying personal knowledge gaps.

 

There is a tread of national control throughout the report, and one of the presenters on the conference call stated that they realize the next committee to report on this (always more committees) will temper their recommendations.  Also requiring more regulations may not necessarily help the problem of over regulation.

 

The report which would like to set up some type of national system for all CE in the health professions is ambitious project but not a current national priority given the expenses that will go into healthcare reform.    To accomplish great things one must think big thoughts, this report is not lacking in any big thoughts. It will be interesting to see if any of those thoughts are implementable.

 

For details on the recommendations the report and slide set are available online.  The report provides details on the nature of the recommendations, the data and science the members of the committee hope to collect from this effort.

 

Institute of Medicine:  Redesigning Continuing Education in the Health Professions 12/04/09

 

Full Report

Slide Presentation

Summary

 

Macy Foundation-IOM: Planning a Continuing Health Care Professional Education Institute: Recommendations from the Institute of Medicine Webcast

The Macy Foundation funded Institute of Medicine (IOM) report on Planning a Continuing Health Care Professional Education Institute is expected to be released sometime mid to late November.

The Alliance for Continuing Medical Education (the Alliance) announced a webcast on this report for December 9^th. 

According to the Alliance: A report from the Institute of Medicine (IOM) on recommendations for the development of a national inter-professional institute devoted to the continuing education of healthcare professionals is due to be released shortly as a result from the recommendations from the 2008 Macy Foundation report on "Continuing Education in the Health Professions: Improving Healthcare through Lifelong Learning."

The purpose of establishing such an institute is to advance the science of CE by promoting the discovery and dissemination of more effective methods of educating health professionals over their professional lifetimes, by developing a research enterprise that encourages increased scientific study of CE, by developing mechanisms to assess research applications, by stimulating new approaches to both intra- and inter-professional CE, and by being independent and composed of individuals from the various health professions.

This is more than likely a report recommending a stimulus package for educators working on CE research.   Though this is a somewhat laudable goal the Macy Foundation has made no bones about their goals in hiring the IOM ($428,177) to legitimize their Bermuda Conference.  They were actively involved in the selection of panel members, fully funded the committee, and the opening meeting started with a presentation titled: Understanding the Committee’s Charge: A Discussion with the Sponsor - George Thibault, Josiah Macy Jr., Foundation.    Other opening day activities included presentations on concurrent Macy funded Activities to Inform the CE Committee:

Dave Davis, Association of American Medical Colleges (~$300,000 Macy Funding) Introduce workshop to (1) review formal CE, (2) review a range of alternative interventions, (3) summarize the inter-professional education literature, and (4) review current knowledge in lifelong learning.

Eric Campbell, Institute for Health Policy, Harvard Medical School (~$150,000 Macy Funding) Introduce plan to (1) review what types of CME work best, (2) understand the amount of CME physicians need, (3) review current CME organizational structures, and (4) explore and model alternative CME payment methods.

Copies of the Macy Foundation Report were given out to all members of the committee at the beginning of the meeting with the Chairman stating that this is an important document and will serve as our guide in writing the IOM report.

The goals of the Macy Foundation Report were simple:

    Eliminate Commercial Support of CME in Five Years

    Eliminate all private providers of continuing medical education

    Replacing the current accreditation system with a government controlled system

Because the report will be based on the Macy paper along with committee members including Katherine DeAngelis, Editor of JAMA who is against all involvement between industry and physicians, this report promises to be a rehash of old materials with the new cover of the IOM.

A background article of this report and other Macy Supported initiatives was published in May in the National Review titled: Conflicting Interests: The effort to push the private sector out of continuing medical education.

One interesting note that with the exception of the first meeting all subsequent meetings have been behind closed doors and not open to the public.

At one closed session in May, (which at the time of the meeting was listed on the IOM website as a closed meeting not open to the public), the IOM apparently held one open presentation that featured Mike Saxton the Senior Director of Medical Education at Pfizer sharing his “personal” views on CME funding.  

Catherine Cleary, MD of Pfizer had previously testified at the open meeting last December as the only representative of the pharmaceutical industry.

There is also a webcast later in December on Macy Foundation funded initiative with the Association of Academic Medical Centers (AAMC)  Recommendations from AAMC/AACN Lifelong Learning Consensus Conference

Overall this should be interesting, for more information on the webcast go to:

 

December 9, 2009

2:00 – 3:00 PM Easter Time

Planning a Continuing Health Care Professional Education Institute

Recommendations from the Institute of Medicine

This report describes recommendations for the development of a national inter-professional institute devoted to the continuing education of healthcare professionals.

Click here to register!

 

January 13, 2010

2:00 – 3:00 PM Eastern Time

Continuing Education in Nursing and Medicine: What Does the Future Look Like?

Recommendations from AAMC/AACN Lifelong Learning Consensus Conference

This report that will discuss recommendations for delivery and format of continuing education and the development of lifelong learning skills in health professionals the delivery.

Click here to register!

FDA Hires IOM to Review Medical Device Approval System

The Food and Drug Administration (FDA) recently issued a press release announcing the commissioning of the Institute of Medicine (IOM) to study the premarket clearance process for medical devices in the U.S.

According to the release, the IOM study will examine the premarket notification program, also called the 510(k) process, for medical devices, and the FDA’s Center for Devices and Radiological Health (CDRH) will convene its own internal working group to evaluate and improve the consistency of FDA decision making in the 510(k) process.

The goals of these evaluations according to Jeffrey Shuren, M.D., acting director of CDRH, is to help “determine how the 510(k) process can be improved to better support FDA’s mission to protect and promote the public health.”

The 510(k) process was established under the Medical Device Amendments of 1976 with two goals:

• Make safe and effective devices available to consumers
• Promote innovation in the medical device industry.

The release calls for review of the 510(k) process because “technology and the medical device industry have changed dramatically.” The IOM commission will specifically try to answer two principal questions:

• Does the current 510(k) process optimally protect patients and promote innovation in support of public health?
• If not, what legislative, regulatory, or administrative changes are recommended to achieve the goals of the 510(k) process?

The $1.3 million IOM review is slated for completion in 2011. Part of the review will include two public workshops during the next nine months, as well a final report that will be published in March 2011. Dr. Shuren also noted that in addition to this review he has outlined six priorities for CDRH including:

• Creating an internal task force on the use of science in regulatory decision-making
• Developing an effective compliance strategy
• Optimally integrating premarket and postmarket information
• Increasing transparency in decision-making
• Establishing clear procedures to resolve differences of opinion.

While it is certainly important for the FDA and CDRH to monitor such changes, it is equally important not to hinder such breakthroughs—often led by collaboration with industry and academia—through burdensome evaluations that scare away scientists and researchers. The criteria IOM should focus on is ensuring that the technological breakthroughs from the medical device and drug industries are kept intact, so that we can keep treating patients and saving lives.   Simply setting up further delays in approvals does not constitute a solution.

For more information about the FDA premarket approval of devices read the GAO report on FDA system for device approval.

IOM: Report on Conflict of Interest: Maybe Best of Intentions but Overreliance on Distant Past

 

 

Despite all the best intentions, the federal Institute of Medicine is trying to make proposals for CME’s future based on facts from the distant past. In its 2009 report on Conflict of Interest in Medical Research, Education, and Practice, the IOM released a report (underwritten by the Josiah Macy, Jr. Foundation) calling for an end to educational grants from pharmaceutical and medical device manufacturers.

 

Some of the report’s conclusions (Eliminate non-educational interventions from university campuses) make sense. But when you dig into the report’s findings on CME, you find mostly stale data and arguments that do not take into account the dramatic regulatory and accreditation board rules now required to ensure that CME excludes industry influence and addresses all conflicts of interest.

 

Most important, the report generally confuses Certified CME with other forms of so-called “education” and relationships that have nothing to do with CME. To be clear, Certified CME programs only are those developed by accredited providers of CME in compliance with all ACCME standards and rules, as well as FDA and Health and Human Services Office of Inspector General Compliance Guidance requirements that ensure CME is “independent from promotional influence.”

 

But by trying to address conflicts in research, education, and practice, the IOM mistakenly blends Certified CME with non-CME interactions. The result? Confusion and outdated data regarding CME, not clarity.

 

Some examples:

•  In the report’s table 5-1, the IOM mentions that drug companies provide lunches, pens, snacks, and dinners for medical students in the third year of schooling. The report complains of “non-educational” interventions in the learning environment. Worthy complaint, but the comments deal with grad school and do not apply in the least to Certified CME activities that occur after physicians are practicing.

• The IOM report cites studies about “influence” by manufacturers that date back to 1986. Oddly enough, the report does not mention at all the academic literature survey on bias produced by the ACCME in 2008 concluding that “no empirical evidence” exists to support a connection between industry grant funding for Certified CME and bias.

• In a flagrant violation of the principle of “evidence-based” reporting, the IOM report includes a “personal account” in which an unknown person states that CME speakers were hired on the basis of their support for a “sponsor’s message.”  If this were the case, it would be a clear violation of ACCME rules and would cause the accredited provided to either be placed on probation or lose its accreditation immediately. The report fails to mention that accredited providers must collect audience evaluations regarding any possible bias and have policies to address any such issues. In addition, the ACCME has a process for handling complaints and has implemented a monitoring system to detect and address any bias. Last, nobody seems to realize that physicians are not compelled to attend any CME program (they can get a year’s worth of required credits over a few days at a single society annual meeting). Physicians attend CME because it’s useful and needed, given the current explosion of healthcare information. Physician CME attendees also are required to evaluate programs for bias, and this information is easily obtained.

• The IOM report claims that bad CME programs were developed related to the federal Neurontin and Vioxx cases. The problem? The problems cited by the IOM report are specifically addressed and prohibited by current rules (the CME enterprise has addressed past problems).

• The IOM report slams the ACCME by citing 2001 and 2003 articles stating that the ACCME Standards for Commercial Support of CME activities do not deter industry influence. Two problems: 1) the original articles did not include evidence to support the claim, and 2) the ACCME updated the Standards in 2004, as well as several new definitions, policies and rules approved in subsequent years, ensure that Certified CME is independent from promotional influence.

The CME enterprise has undergone many positive changes during the past five years. It’s time organizations, such as the IOM, understands and acknowledges them.

This commentary was written by Stephen Lewis the President of the North American Association of Medical Education and Communications Companies.  Early in his carrier Steve served as press secetary for US Senator Thimothy Worth (D-CO).