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24 posts from January 2012

January 31, 2012

Patient Centered Outcomes Research Institutes National Priorities for Research and Research Agenda – Laying a Foundation for Comparative Effectiveness Research

Foundation
One of the bigger pieces of the Patient Protection and Affordable Care Act (PPACA) was the idea to lower healthcare costs through comparative effectiveness research (CER).  To achieve this goal, PPACA (Section 6301 and Section 10602, Public Law 111-148), created the Patient-Centered Outcomes Research Institute (PCORI), an independent, non-profit health research organization.  PCORI will have an estimated $3 billion over the next decade to fund CER. 

PCORI was created to conduct research to provide information about the best available evidence to help patients and their health care providers make more informed decisions. PCORI’s research is intended to give patients a better understanding of the prevention, treatment and care options available, and the science that supports those options.  Its mission is to fund research that offers patients and caregivers the information they need to make important healthcare decisions.  PCORI focuses on CER that compares options for preventing disease and providing treatment and care by: 

  1.  Identifying national priorities for research.
  2.  Creating a research agenda based on identified priorities.
  3. Funding research consistent with these priorities and agenda.
  4. Providing patients and their caregivers with useful research information. 

Consequently, PCORI recently issued its draft list of National Priorities for Research and Research Agenda.   PCORI will provide a 53-day public comment period on its first National Priorities for Research and Research Agenda. The comment period begins on Monday, January 23, 2012 and concludes at 11:59 p.m. ET on Thursday, March 15, 2012. 

Responses received through www.pcori.org will be displayed for public view on the website.  Click here to fill out the online form to provide comments on the draft priorities and research agenda.  Public comments can also be mailed to:

Patient-Centered Outcomes Research Institute
Public Comments
1701 Pennsylvania Ave. NW,
Suite 300
Washington, DC 20006 

PCORI will also hold a National Patient and Stakeholder Dialogue on Monday, February 27, 2012, from 10:00 a.m.-5:00 p.m. EST, at the National Press Club in Washington, D.C.  The event will dedicate three and a half hours to receiving public comment, and include presentations by PCORI and a roundtable discussion involving patient advocates, clinicians and others from the health care community. Individuals may attend in person or remotely via webcast and teleconference. 

PCORI will conduct additional forums in cities across the United States, which will include focus groups, involving patients, caregivers and clinicians, to obtain and incorporate more public input and feedback on the draft national priorities for research and research agenda.   

After incorporating public input, the first National Priorities for Research and Research Agenda will be adopted by the PCORI Board of Governors before primary research funding announcements are issued.   A report will be published on www.pcori.org that summarizes the input received with an explanation of how the input led to any changes in the draft priorities and agenda. The revised National Priorities for Research and Research Agenda will be adopted by PCORI’s Board of Governors during a special public meeting in April. 

Subsequent priorities and agendas will be achieved through a “due diligence” process that includes dialogue with a broad range of stakeholders, input through a formal public comment process and additional forums, including focus groups, PCORI presentations to various audiences, outreach through PCORI’s website and other vehicles, advisory panels and stakeholder meetings. 

Nine different Priority Setting Organizations contributed to the formulation of PCORI’s draft research priorities/agenda, including AHRQ, IOM, National Quality Forum; and HHS; many of which received significant public input.  The priorities and agenda will inform the development of PCORI’s first primary research funding announcements, which will be made in May.  "These priorities and agenda give a framework to and identify the broad questions that must be addressed so that patients can make better and more personalized decisions in partnership with their clinicians across all areas of health," said PCORI Executive Director Joe Selby, MD, MPH. 

The institute plans to announce an initial round of about 40 grants, drawn from 856 applications, in May. A second round of grants, for projects linked to the research agenda, will be issued in December, he said. PCORI is a learning organization.  Once the initial priorities and agenda are adopted, PCORI will work closely and continuously with all stakeholders to refine the agenda and to identify specific areas and questions where PCORI’s research can have the greatest impact. 

This process will include analysis of PCORI’s Pilot Projects Grants Program and the applications in response to PCORI’s first primary research funding announcements. Any future revisions to the priorities and research agenda will include a formal public comment process and additional forums. 

However, Robert Dubois, chief science officer for the National Pharmaceutical Council noted that, if PCORI gets “1,000 grants submitted on 400 different topics, who is going to make the decision on, say, whether they should fund a project on low back pain, but not one on multiple sclerosis?”  Dubois says the thematic approach taken by PCORI will lend itself to one of the institutes’ goals – creating a self-sustaining and robust research environment.  But he says a portion of the funding should be directed “toward actual, unanswered clinical questions.”

Additional challenges facing such research also include political controversy and the difficulty of translating research findings into changes in medical practice. Republican opponents of the law say the institute will lead to government-directed rationing as it judges treatments. Representative Thaddeus McCotter, a Michigan Republican, has introduced legislation to eliminate the agency.

Over time, more money should be shifted to comparing treatments, said U.S. Representative Kurt Schrader, an Oregon Democrat who wrote legislation to create a comparative effectiveness research agency in 2009. The provision of the health law that created Selby’s institute echoes Schrader’s bill, which had some Republican support at the time. The research plan is “a little broader in scope” than he envisioned when he wrote his bill, Schrader said in a telephone interview. “The worry I would have is they are not going to get down to specific projects.”

Ten CER priorities areas from existing literature were identified. The first five priority areas (prevention, acute care, care coordination, chronic disease care, and palliative care) encompass the complete health cycle from staying healthy to treating conditions to reducing pain and suffering. The second five (patient engagement, safety, overuse, information technology (IT) infrastructure, and the impact of new technology) include issues that are systemic in nature to healthcare. All but one of the 10 priorities (impact of new technology) appear in at least three of the seven processes. Prevention appears in all seven and patient engagement in six. 

Research List and Agenda 

PCORI prioritized five research areas for patient-centered comparative clinical effectiveness research: 

Comparative Assessments of Prevention, Diagnosis, and Treatment Options. The research goal is to determine which option(s) work best for distinct populations with specific health problems. Comparing the effectiveness and safety of alternative prevention, diagnosis, and treatment options to see which ones work best for different people with a particular health problem.  40% of funding. 

Improving Healthcare Systems. Focuses on ways to improve healthcare services, such as the coordination of care for patients with multiple chronic conditions. Comparing health system-level approaches to improving access, supporting patient self-care, innovative use of health information technology, coordinating care for complex conditions, and deploying workforce effectively.  20% of funding. 

Communication and Dissemination. Looks at ways to provide information to patients so that they, in turn, can make informed healthcare decisions with clinicians.  Comparing approaches to providing comparative effectiveness research information and supporting shared decision-making between patients and their providers.  10% of funding. 

Addressing Disparities. Assures that research addresses the healthcare needs of all patient populations. This is needed as treatments may not work equally well for everyone.  Identifying potential differences in prevention, diagnosis or treatment effectiveness, or preferred clinical outcomes across patient populations and the healthcare required to achieve best outcomes in each population.  10% of funding. 

Accelerating Patient-Centered and Methodological Research. Includes patients and caregivers in the design of research that is quick, safe, and efficient.  Improving the nation’s capacity to conduct patient-centered outcomes research, by building data infrastructure, improving analytic methods, and training researchers, patients and other stakeholders to participate in this research. 20% of funding. 

PCORI’s Proposed Research Agenda

Comparisons of Prevention, Diagnosis, and Treatment Options. Research should focus on 1) clinical options with emphasis on patient preferences and decision-making, 2) biological, clinical, social, economic, and geographic factors that may affect patient outcomes.

Improving Healthcare Systems. Research should focus on 1) ways to improve access to care, receipt of care, coordination of care, self-care, and decision-making, 2) use of non-physician healthcare providers, such as nurses and physician assistants, and the impact on patient outcomes, 3) system-level changes affecting all populations, diseases, and health conditions.

Communication and Dissemination. Research should focus on 1) strategies to improve patient and clinician knowledge about prevention, diagnosis and treatment options, 2) methods to increase patient participation in care and decision-making and the impact on health outcomes, 3) communication tools that enhance decision-making and achieve desired outcomes, 4) ways to use electronic data (“e-health records”) to support decision-making, 5) best practices for sharing research results.

Addressing Disparities. Research should focus on 1) ways to reduce disparities in health outcomes, 2) benefits and risks of healthcare options across populations, 3) strategies to address healthcare barriers that can affect patient preferences and outcomes. 

Accelerating Patient-Centered and Methodological Research. Research should focus on 1) ways to improve the quality and usefulness of clinical data in follow-up studies, 2) methods to combine and analyze clinical data that follow patients over time, 3) use of registries and clinical data networks to support research about patient-centered outcomes, including rare diseases, 4) strategies to train researchers and enable patients and caregivers to participate in patient-centered outcomes research. 

Establishing the Scope of the Research Agenda 

By design, the agenda did not cover all the activities that PCORI sees as part of its mission. For instance, it does not describe how PCORI intends to invest in efforts to maintain active engagement with patients and all other stakeholder groups over time. It does not describe how PCORI, in partnership with the Agency for Healthcare Research and Quality (AHRQ) and the U.S. Department of Health and Human Services (HHS), will invest in efforts to disseminate research findings or build capacity for patient-centered outcomes research through training programs or infrastructure development. 

This document is the foundation upon which the first set of PCORI funding announcements will be developed. Taken together, the series of funding announcements that PCORI will produce, beginning in mid-2012, will create a portfolio of research that builds from this agenda and is consistent with Congress’ intended purpose. 

The Research Agenda by design did not specify a restricted set of conditions or treatments that PCORI will study. Although some previous prioritization efforts have focused on specific high-prevalence or high-cost conditions, PCORI’s priorities and agenda do not place such limits on the scope of research that will be supported. 

Rather than focusing on specific conditions or treatments, PCORI’s initial approach specifies a set of questions and topics PCORI believes are most in need of attention – across a range of conditions and treatments. Within any topical area, studies may focus on specific diseases, conditions and interventions, or they may be cross-cutting, including broader study populations or examining interventions or other questions that apply across multiple diseases and conditions. 

This approach recognizes the likelihood that as PCORI begins its funding program, researchers partnered with stakeholders are well-positioned to present a range of compelling questions. Ultimately, decisions about funding will depend on the quality of applications – with special attention to the likelihood that the research may lead to improvement in patient outcomes, as determined by alignment with PCORI criteria. 

Targeted opportunities may focus on specific conditions or diseases, treatment modalities, outcomes or on themes that are cross-cutting. PCORI will work diligently to avoid redundancy, coordinate with other research entities, and will seek opportunities for collaboration with these entities. 

The portfolio of research supported by PCORI will be balanced based upon the characteristics of study populations such as age, gender, race/ethnicity, socio-economic status, and disease or conditions. PCORI has a commitment to include studies of patients with rare conditions as well as those with more common illnesses. Especially needed are studies to improve care and outcomes for patients faced with multiple conditions. All funded studies will have a strong orientation to the patient perspective and all will have patients involved in the development of the research, its governance and oversight, and its dissemination strategy. 

The Research Agenda does not specify preferred study designs or analytic approaches.  Instead, it recognizes that various PCOR questions might be investigated by a variety of scientifically-valid methods and approaches. In accordance with Act (Section “(C) FUNCTIONS”), research focused on developing new methods and/or improving the science and methods of patient-centered outcomes research (PCOR) is specifically included in the Agenda. PCORI’s Methodology Committee is currently developing standards for the design and conduct of different types of PCOR. These standards will be disseminated and used in the evaluation of PCORI applications. 

Discussion 

Overall the selection of topics by PCORI shows that they understand that in order to successfully transform the health care system, one has to start at the foundation and ask the big questions.  Much of the focus of PCORI support and criticisms has been focused on PCORI studying treatments, but if the system is inefficient, then studying treatments may not necessarily be the best use of public funds.  

If one really gives thought to the problem subjects such as compliance, socio economic access to therapies and social customs such as men delaying seeing a physician play a much larger role in the outcomes of patients than the treatments themselves.

January 30, 2012

Physician Payment Sunshine: Media Missing the Mark - This Could be Sunset for Innovation

Sunset
In the weeks following the issuance of the proposed rules to implement the Physician Payment Sunshine Act (“Sunshine Act”), there has been a tremendous amount of debate and media coverage of the impact these regulations will have on physicians and the life sciences industry.  Some believe that the regulations were “badly needed,” because it will “allow patients to decide whether they need to worry about any possible conflicts of interest.” 

However, the Centers for Medicare and Medicaid Services (CMS) nor any other empirical study we are aware have has shown data indicating how many people will actually use this information—of course whether they understand it is a whole different story.  The public will have until Feb. 17 to comment on the proposals. After considering the comments, Medicare officials will issue final rules with the force of law. 

The Sunshine Act requires that manufacturers of drugs, medical devices and medical supplies disclose all payments over $10 they make to doctors or teaching hospitals. The information, which would be posted on a government Web site.  Manufacturers will have to report if they pay a doctor to help develop, assess and promote new products — or if, for example, a pharmaceutical sales agent delivers $25 worth of bagels and coffee to a doctor’s office for a meeting. Royalty payments to doctors, for inventions or discoveries, and payments to teaching hospitals for research or other activities will also have to be reported. 

Manufacturers could be fined up to $150,000 a year for failing to report payments and up to $1 million a year for “knowingly” failing to report.  Moreover, top executives are potentially liable because a senior official of each company — the chief executive, chief financial officer or chief compliance officer — must attest to the accuracy of each report. 

The Obama administration estimates that more than 1,100 drug, device and medical supply companies will have to file reports, generating “large amounts of new data.” Federal officials said they would inspect and audit drug company records to make sure the reports were accurate and complete. 

The web site where payments would be posted by CMS must be searchable and understandable so that patients and advocacy groups can see which doctors are being paid and how much. Some believe that the public posting of such payments could deter some questionable payments and it could help patients decide which doctors to rely on.  Yet again, there is no evidence to support this claim or to show just how many patients will actually 1) use this information 2) understand this information and 3) change physicians.  Moreover, there is no discussion of the fact that some patients my actually want their physicians to work with industry becomes it demonstrates expertise and clinical experience that is clearly valued. 

While a recent article from the New York Times correctly recognized that “such payments can be for legitimate research and consulting,” it overemphasized and frankly attacked physician-industry collaboration by claiming that, “there is also a lot of cash being spread around to pay for doctors’ travel and entertainment or for gifts or modest meals for a prescribing doctor’s staff.”  The article failed to provide any evidence to substantiate such claims. 

The Times only claimed that, “Many researchers have found evidence that such payments can influence doctors’ treatment decisions and contribute to higher costs by encouraging the use of more expensive drugs and medical devices.”  But the article failed to mention that over 75% of drugs prescribed in America are generics. 

Consequently, the Times claimed that it “has found that doctors who take money from drug makers often practice medicine differently from those who do not and that they are more willing to prescribe drugs in risky and unapproved ways, such as prescribing powerful antipsychotic medicines for children.”  But just exactly how did the Times find this information?  How many doctors?  Did they reach out to these physicians to determine what the payments were for, or to determine in each case why the physician chose the course of treatment or procedure?  It is sad that the paper fails to meet scientific accuracy. 

While legal advocates and officials believe that disclosures may increase the likelihood that doctors would make decisions in the best interests of patients, without regard to the doctors’ financial interests, this claim ignores what’s best for patients.  

Doctors should have the freedom to choose whatever medication they believe is medically necessary for a patient based on objective factors, not the threat of publishing their name on a website that no patient will use or understand, and that only media and politicians will use to sell newspapers and get attention. 

There is no question that such practices were rampant in the past, over the last several years, industry and various healthcare stakeholders have implemented significant firewalls, rules and regulations to prevent these kinds of arrangements from happening.  Whether it is the PhRMA or AdvaMed Codes of Conduct, the various guidance’s for HHS OIG or FDA, the large settlements with DOJ, or the changes in institutional and provider policies such as NIH or Harvard, improper practices such as “gifts” are the exception today, not the practice. 

Despite limited research on the true nature of physician-industry collaboration, the article cites a previous New York Times article, which discussed “some prominent doctors and researchers who have receive hundreds of thousands or even millions of dollars a year in exchange for providing advice to a company or giving lectures on its behalf.”  Once again, the author fails to provide a fair analysis of such payments or discuss the “nature” of such payments—a requirement that even the Sunshine Act will post on their website. 

The article claimed that about a quarter of all doctors take some cash payments from drug or device makers and nearly two-thirds accept meals or food gifts. Analysts contend that even seemingly trivial gifts can influence doctors to prescribe expensive drugs that may not be best for a patient’s health or pocketbook.  But where is the evidence?  The only evidence we are aware of is an old study conducted by Wazana et al, which did not use patient outcomes and is therefore completely invalid.  Moreover, social science experiments and the use of brain imaging equipment is hardly an evidence-based method. 

Despite offering no fair balance of the issue, or discussing the important role and impact physician-industry collaboration has had on increased longevity of life and improved outcomes in heart disease, cancer, and hypertension, the author unequivocally claims that, “The new rules should give a welcome boost to otherwise spotty efforts by some companies, medical centers, scientific journals, states and ethical codes to eliminate, minimize or at least disclose financial interests that might cloud medical judgments.”  The suggestion that policies such as those at major medical centers or the AdvaMed and PhRMA Codes, or the various mechanism that companies have in place due to corporate integrity agreements as “spotty” is misguided. 

Discussion 

Allan J. Coukell, a pharmacist and consumer advocate at the Pew Charitable Trusts, said: “Patients want to know they are getting treatment based on medical evidence, not a lunch or a financial relationship. They want to know if their doctor has a financial relationship with a pharmaceutical company, but they are often uncomfortable asking the doctor directly.”  While CMS noted in its preamble to the proposed rules that physician-industry relations can “lead to conflicts of interests that may affect clinical decision-making” and “threaten the underlying integrity of the health care system,” CMS also recognized that “patients can benefit when doctors and the industry work together to develop life-saving drugs and devices.” 

Moreover, even if such relationships pose a risk or potential for conflict, CMS clearly asserted that it has “has no empirical basis for estimating the frequency of such problems (inappropriate use), the likelihood that transparent reporting will reduce them, or the likely resulting effects on reducing the costs of medical care.”  In fact, the Congressional Budget Office (CBO) does not even predict immediate savings, which is problematic considering the regulations will cost over $224 million to implement, which is a rough estimate.  Instead, CBO believes that, “over time, disclosure has the potential to reduce spending,” by reducing instances of overprescribing.  Yet, CMS has no evidence to substantiate this claim. 

Christopher L. White, executive vice president of the Advanced Medical Technology Association, which represents makers of medical devices, said the payment data could be used by federal law enforcement agencies, plaintiffs’ lawyers and whistleblowers.

“Some companies fear that doctors may no longer want to engage in consulting arrangements, and such reluctance could chill innovation,” Mr. White said. 

Matthew D. Bennett, a senior vice president of the Pharmaceutical Research and Manufacturers of America (PhRMA), said the industry “supported transparency of physician payment information.” However, he said, it is important that payment data be presented in a proper context, emphasizing that interactions between doctors and drug companies played a critical role in improving care, educating doctors and fostering appropriate use of medicines. 

Alan E. Reider, JD, MPH, an attorney in the healthcare practice at Arnold & Porter noted that the Sunshine regulations have a considerable chance for a negative effect on eye care professionals.  His “concern is that the information is going to be misunderstood and abused by the press, by enforcers and by whistleblowers.”  He is “concerned about the potential adverse impact this may have on the relationship between physicians and industry, which is so critical to developing new technology.”

Reider further emphasized the potential “chilling effect” the provision may have, leading to fewer legitimate financial ties between physicians and industry, along with the openness potentially leading to a surge of whistleblower lawsuits. “What you really need to do is make sure that you're comfortable with your financial relationships ... because you may find that this is going to be in the press, and you need to be sure that you're comfortable and you can defend what you're doing,” Mr. Reider said.

Consequently, a recent presentation on physician-industry collaboration highlighted some of the significant problems the Sunshine Act will impose on physicians and the healthcare system overall.  The presentation, given by Stefanos A. Zenios, PhD Professor of Healthcare Management Stanford University first looked at the crucial role physicians play in medical device innovation. He noted that “Physician involvement is a necessary part of maturing the technology so that it can be applicable to patients” and that, “The industry cannot do anything without physicians and physicians could do very, very little without industry.” 

However, because of more rules and regulations, he noted that product development today is more difficult.  “There seems to be an ever “increasingly more complex set of barriers… the regulatory environment has gotten worse, the reimbursement environment has gotten worse, the costs to advance these projects are more expensive.”  He quoted Warren Watson who noted that one “of the greatest risks “in the nation today is the social-political environment that is attempting to separate physicians from industry.” 

Dr. Zenios presentation then went into the various roles physicians play through physician-industry collaboration.  During the early-stage of such collaborations physicians are inventors: 

  • Physician identifies “need” through clinical work
  • May patent technology and develops product in partnership with team of engineers to address it
  • Partnership with company to license technology or develop product  

Physicians are also entrepreneurs: 

  • Physician involved in every step of building a company from invention, writing patents, and taking operating roles in start-ups
  • Some physicians may continue to practice medicine after joining start-up company  

Physicians also act as product design consultants for device companies, in which they offer their expertise on design specific features for a product or to advise on modification of existing design.  Physicians are also “co-developers” who collaborate extensively with device companies to design and commercialize a product. 

In Early and Late Stage Roles, physicians act as clinical researchers.  Physicians are retained by a device company to conduct clinical trials, which can either be early-stage trials to help refine product or later stage.  Doctors can also sit on a company advisory board or board of directors of a medical device company. 

“Once the product is commercial the company has an interest in making sure that as many people as possible understand the benefits of the product and so they would do clinical studies and try to get those clinical studies, get doctors to talk about those clinical findings on the podium, etc. There maybe, those doctors may play a role as a consultant to the company again.”—Fischell. 

Accordingly, physicians also have Late Stage Roles.  Physicians act as “educators” who train and educate other physicians on the use of the product and help get other physicians “up to speed” on the new technology, thereby helping to boost adoption post-launch.  Physicians also serve as general consultants for a company to advise them about overall product development and research, and they may be hired to consult on an ongoing basis or for a stand-alone projects. Doctors also serve on at least three (3) types of panels, which may be ongoing or one-off in nature: 

  1. Advisory panel: explore new areas of interest or assess strategic goals of the company
  2. Product performance panel: analyze product performance data and help improve it
  3. Product development panel: help guide early product development         

The presentation then discussed a three step process for determining level of compensation for a physician; 

First, Ensure direct link between compensation and work.  Increasingly companies have gone away from the model of paying physicians monthly or annual retainers.

Avoid situations where physicians receive payments for companies without providing an actual service. 

Assess opportunity cost of physician’s time.  Ensure that physicians are compensated at a rate roughly equal to what they would earn from seeing patients in their practice.  Companies should also try to appropriately value physician’s time; ensuring that they are not significant incentives to spend more time consulting to companies. 

Match prevailing compensation rates.  Companies should try to match physician compensation to the prevailing rate of compensation paid by other companies in the industry because the tendency might be to over-pay otherwise. 

The presentation then gave some examples of conflicts of interest that may arise in collaborations  

  • Disputes over who actually owns the IP for a co-developed product
  • Physicians may be more likely to use the products of the company with which they have existing relationships
  • Physicians entitled to large royalties for products may be more likely to use these products in their clinical practice
  • Physicians who have a stake in a product or company are unlikely to be unbiased clinical investigators 

Accordingly, the presentation then examined key considerations for assessing conflicts of interest.  In the early stage of relationships, companies must consider whether a physician is the inventor/entrepreneur or they have some significant stake in success of the company or product.  Late stage, companies must ask whether the physician is acting in more than one role, such as an advisor or consultant while also acting as a clinical investigator. 

Companies must also consider what how physicians are compensated for their role.  Is it upfront cash or consulting arrangements or is it deferred payments through equity or royalties that are tied to commercial success of a product.  Companies should also consider at what stage of development a product is in that a relationship may be in place for.  Is it in pre or post-approval stage of development?  If the physician continues to practice, there may be some potential impact on his prescribing behavior depending on his “ties” to the product. 

While these factors are important to consider, the presentation noted that for major conflicts are likely to center on two main parties: Clinical investigators who receive royalties or equity from device companies and Clinicians in daily practice who receive cash payments from companies with approved products. 

Consequently, he noted that “Conflicts will always be there . . . it is how you handle it. Separate the physician’s role in company creation/growth from involvement in clinical trials “If I’m allowed to be a principal investor (in a company), I can sometimes be a co-investigator in trials. I (however) cannot be the principal investigator, and that’s appropriate. “—Tim Fischell  

Tightly link compensation to services rendered “We attempted to create a very tight linkage between the work they (physicians) did and the payment that they received . . . no more retainers.” —Warren Watson.  Moreover, these relationships are constantly being managed by industry itself through a code of ethics (e.g., AdvaMed) and transparency.  There is also physician self regulation: Medical training, mentors, senior and peer review, desire not to hurt patients or professional reputation. 

Ultimately, he concluded by noting that “clinician-company collaborations are vital to the creation of new medical devices.  He noted relationships are subject to conflicts of interest especially in two dimensions: Pre-approval: Investigators compensated with equity or royalties.  Post-approval: Advisors practicing medicine compensated in cash.  This kind of increasing scrutiny increases the cost of doing business in the U.S. and pushes activities that would otherwise be done in the U.S. outside the country.  Accordingly, he asserted that industry can self regulate and avoid the outright prohibition of relationships that can benefit patients.

 

January 27, 2012

Translational Research Innovation The Way Forward

Medical-innovations
A recent Forbes article from David Shaywitz, MD/PhD, discussed a highly interactive translational research innovation symposium at Duke University put together by Dr. David Epstein at the Duke Eye Center and Chancellor Victor Dzau.  The program featured both University faculty and invited external speakers.  Shaywitz described the symposium based on four key themes.

Theme 1: Driving science into application remains difficult but desirable 

Shaywitz noted that many participants were eager to push science into the clinic, from PhD chemists and biologists to internists and surgeons (and, of course, ophthalmologists).  A vigorous entrepreneurial spirit was apparent, a determination to do something outside the usual academic practice.  To achieve this goal, he identified three necessary basics: culture, coordination, and capital. 

 

He noted that culture really matters – and academic leaders can play a vital role by either passionately encouraging and supporting entrepreneurship (Susan Desmond-Hellmann and Tom Byers stand out in my mind here), or offering only tepid lip service.  Arguably the most valuable aspect of culture are the presence of positive examples, faculty members and students who’ve actually done this – developed a product, started a company, pushed an innovation into the clinic and changed medical practice. This also suggests an opportunity to initiate a virtuous cycle – once you have a nucleus of entrepreneurs, a critical mass, the excitement can spread virally. 

Shaywitz noted that coordination is important because forming a company or developing a technology for licensing, tends to be complex, and (in the case of start-ups) typically involves far more work than the participants had originally anticipated.  It’s also clear that while there may not be a formula for innovation, there are discrete structural activities that can be done in an environment to support, nurture, guide and sustain innovation, as several faculty members from Duke’s Fuqua School of Business emphasized.  

Some learning is inevitably didactic (learning the rules of road), but most is experiential, and a crucial element seems to involve learning how to work as team, to transform the germ of an idea into something concrete.  The best learning, clearly, is real world experience; seeing and solving the real-world challenges experienced as you try to transform an idea into an actual (often commercial) product or service likely represents the most useful training of all. 

Finally, he noted that capital plays a vitally important role in driving innovation, and the availability of capital can be transformative – just as the lack of same can be a problem.  

Theme 2: Think differently 

Shaywitz noted several examples from this meeting, which suggested that there are actually approaches to deliberately think about problems in slightly different ways.  Dean Nancy Andrews, for example, presented interesting social science data highlighting the value of diversity in innovation; it turns out that heterogeneous teams tend to solve difficult problems more effectively and apparently more creatively than teams with less diversity.  This ties in nicely with the suggestion that breakthroughs tend to occur at the intersection of disciplines.  

Keynote speaker and legendary MIT bioengineer Robert Langer emphasized the value of multi-discipline collaboration as well; such interactions have been a defining characteristic of his career, and also the foundation, beginning with his decision to pursue his post-doc not in a chemical engineering division, but rather in the lab of a pediatric surgeon, the late Judah Folkman

The emphasis on unexpected ideas tied in very well with a second point Dean Andrews also made, about the unique value of students on entrepreneurial teams; not only do they tend to be energetic, of course, but they are also relatively naïve, and can ask the fundamental, vital questions overlooked by those with more experience.  

The idea of thinking differently was a key message for Shaywitz’s talk and a significant theme of the talk given by Geoff Duyk, a physician-scientist by training, and currently managing director of TPG’s in-house biotech VC fund; not surprisingly, Duyk is a big believer in the concept of intellectual “cross-training.”  

Both Duyk and Shaywitz highlighted the need to look beyond the traditional model of early stage innovation (pathway -> target -> drug), and consider other types of innovation that could improve health – including innovation in devices, services, processes, and care delivery.  Of particular interest, Duyk highlighted the unique healthcare needs and opportunities associated with countries in Central and South America, as well as Asia, providing an often underappreciated global perspective, and an important reminder of the value of thinking outside the border as well as the box. 

Theme three: Pharmascolds still have academics running scared. 

Despite enjoying the discussion of innovation, Shaywitz was “struck” by the fear that university researchers and clinicians had from the attacks on physician-industry collaboration led by the pharmascolds.  An anxiety that took many forms.  Junior faculty were concerned that working with industry in any way might jeopardize their relationship with the National Institutes of Health (NIH), and introduce a prohibitive array of complications and requirements into their already busy lives.  

Many people emphasized the need to be completely transparent and open; there was unanimous agreement here.  The problem, rather, was the seemingly excessive and gratuitously onerous requirements associated with this transparency, a level of micromanagement (one might argue nanomanagement) that was perceived at times to be absurdly heavy-handed, and more likely to inhibit university/industry collaboration than enhance it. 

There was also a strong sense that partnering with industry is still viewed in many academic circles as collaborating with the enemy, a relationship that threatens to stigmatize the researcher – a disgraceful phenomenon.  In Shaywitz’s view: we should be celebrating, not castigating, the researchers who seek to drive their science into practice, not just into papers and prestige. 

Several senior faculty members strongly believed that the media had their crosshairs fixed squarely on academics who work with industry, and any academic leader who tried forcefully to defend or support these relationships was likely to be taken down.  Shaywitz noted that a number of academic leaders at a range of institutions have highlighted the perceived ferocity of the pharmascold attacks,  as well as the courage of the rare academic leaders – such as Desmond-Hellmann – willing to stand up and forcefully make the case that driving science into application (a) is core to a research university’s public mission, and (b) can be powerfully facilitated through well-structured collaborations with industry.  (See here as well.) 

Theme four: Role of the champion and the opportunity to make a difference 

Shaywitz said that the two highlights of the symposium were the first talk of the day by Langer, and the last talk of the day, by Y.T. Chen.  Each seminar highlighted the tremendous impact of an impassioned innovator. 

Langer’s talk discussed the arc of his career, including the initial challenges he faced as he struggled to get a new technology patented.  He also highlighted the excitement and many challenges of developing new products and forming new companies, the success of which depends not only on brilliant and uncommonly creative individuals but also “exceptional persistence, and a lot of gritty work.” 

Chen’s talk, to close the symposium, “offered a poignant and modestly-presented reminder of why translation is so essential.” Chen, a physician-scientist (pediatric geneticist), developed the first treatment for Pompe Disease after being motivated by the searing experience of a patient’s memorial service.  Following the development of a enzymatic replacement strategy by Chen – and its subsequent development and commercialization by industry (Synpac, later Genzyme, now Sanofi) – many children with this disease have a significantly improved chance of living a much better – in some cases, relatively normal – life, especially if treatment is initiated very early. 

This example caused Shaywitz to think about this “model of innovation, this narrative of the intrepid, determined physician-scientist successfully shepherding a discovery from the lab into the clinic.”  He said, “It’s a compelling vision – it’s what I had in mind when I decided to pursue an MD/PhD, and I know it’s what many of my colleagues were thinking as well.”  

However, he noted his concern that this model is now the exception, not the rule.  He noted that more often, progress may involve a number of discrete, and often disjointed advances, that over time are integrated into a more complete picture, and ultimately give rise to novel therapies.  Ultimately, Shaywitz hoped that we can “continue to highlight the accomplishments of clinical champions like Y. T. Chen, like Judah Folkman, like Peter Pronovost (incidentally a nice example of a champion pursuing systems innovation)” because these individuals “offer an inspirational reminder of the power of what can be achieved with a focused mind, a committed heart, and little bit of luck.”

 

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