Life Science Compliance Update

December 13, 2017

FDA Issues Guidance on Real-World Evidence for Medical Devices

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Earlier this year, the United States Food and Drug Administration (FDA) finalized guidance on the use of real world evidence to support regulatory decision-making for medical devices. The final guidance follows a July 2016 draft guidance on the topic in which the FDA discussed potential uses of real world data and the various factors evaluated to determine whether that data can support a regulatory decision. The FDA believes that this guidance is a “cornerstone” of its strategic priority to build a national evaluation system for health technology.

The final guidance includes clarity as to what it means for companies moving forward, “While FDA encourages the use of relevant and reliable real-world data, this guidance neither mandates its use nor restricts other means of providing evidence to support regulatory decision-making.”

In the final guidance, FDA recognizes that “traditional” clinical trials may sometimes be impractical or overly challenging, and appropriate real world data can sometimes provide comparable information. While clinical trials are controlled and require monitoring and data auditing, they may be narrow in scope. Real world data may be able to generate information on a broader patient population, but studies must be carefully designed, regardless of whether the real world data has already been collected or will be collected in the future. Protocols and analysis plans for real world data “should address the same elements that a traditional clinical trial protocol and statistical analysis plan would cover.”

The final guidance also notes that due to the “rapidly advancing methodology for generating and interpreting real world data, this guidance will not elaborate on the methodological approaches that can be used.”

The FDA believes that real world data drawn from clinical practice settings can provide greater insight into the benefits and risks of medical devices, including how they’re used by health care providers and patients, and how they perform under the conditions of routine medical practice. The guidance does not change the evidentiary standards that are required to make those decisions.

Given the expansion in the availability and use of health data derived from registries, billing claims, and electronic health records, the FDA is taking these steps in hopes of clearly describing the appropriate characteristics of real world evidence that meet the agency's standards of evidence to support regulatory decisions.

To assess reliability of data, the FDA is primarily concerned with how the data were collected (data accrual), and whether there were people and processes in place to provide “adequate assurance” that the data quality and integrity are sufficient. The real world data source should have an operational manual (or similar document) in place specifying the methods and extent of data collection and aggregation. FDA provides a list of factors that it will consider the when assessing data accrual, many of which are also considerations for any clinical trial.

Several weeks after the release of the guidance, FDA Commissioner Scott Gottlieb stated that the agency will need to work with the healthcare system to change the way clinical information is collected.

"We need to close the evidence gap between the information we use to make FDA's decisions and the evidence increasingly used by the medical community, by payers, and by others charged with making healthcare decisions," Gottlieb told attendees of the National Academy of Sciences' workshop on the impact of real world evidence on medical product development.

While noting the current uncertainty among sponsors on the role real world evidence plays in regulatory decision making, Gottlieb said FDA needs to think of itself as an information curator, rather than an arbiter of information, "where a single truth standard is secured to a fixed orthodoxy."

Rachel Sherman, principal deputy commissioner at FDA, added later in the day, "We want a better evidence base to make product approval decisions" and she noted that more guidance will be forthcoming on real world evidence and real world data. "The goal is not to define real world evidence and real world data but to get better information and in a more sensible way," she added.

For more information, we encourage you to read the final guidance and access the FDA webinar on the topic here.

December 12, 2017

Senate Appropriations Committee Takes on the Opioid Epidemic

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On December 5, 2017, the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies held a hearing to discuss the opioid epidemic and the possible role that Congress could play in the prevention, treatment, and recovery.

Senator Roy Blunt, the Subcommittee Chairman, opened the hearing by discussing the fact that overdose related deaths outnumber the deaths at the peak of the AIDS/HIV epidemic. Overdose deaths have also overtaken automobile accident fatalities to become the number one cause of accidental death in the United States. Senator Blunt also spoke about the three proposals he believes the Subcommittee needs to focus on moving forward. Those three proposals are: (1) understanding the best options for treating an opioid use disorder, including recognizing that behavioral health issues should be treated like any other physical health ailment; (2) stemming the number of individuals who become addicted in the first place, including improving surveillance to better understand where the problems are and where they are the most severe; and (3) developing new pain treatments as adequate alternatives to opioids.

Senator Patrick Leahy, the Senate Appropriations Committee Ranking Member, also mentioned the high rate of opioid-related deaths and heavily criticized the Trump Administration for not budgeting more money to fight the epidemic, noting that the president’s declaration of a public health emergency was “all talk and no action.”

Subcommittee Ranking Member Senator Patty Murray also criticized the Trump Administration and its response to the epidemic. She did suggest a solution, however, urging Congress to focus on restricting synthetic fentanyl — a main driving force in the opioid epidemic today.

Witnesses present at the hearing were: Francis Collins, Ph.D., Director at the National Health Institute; Patrick Kennedy, former Congressman and current member of the President’s Commission on Combatting Drug Addiction and the Opioid Crisis; Elinore McCance-Katz, Ph.D., Assistant Secretary for Mental Health and Substance Use at Substance Abuse and Mental Health Services Administration (SAMHSA); and Debra Houry, Ph.D., Director of the National Center for Injury Prevention and Control in the Centers for Disease Control and Prevention (CDC).

As with most Congressional discussions, funding played a prominent role in the hearing. Mr. Kennedy expressed concern about the lack of funding to fight the crisis. He had previously called for the Federal government to provide $10 billion a year for a decade to fight the epidemic. He stated that this number was the minimum amount that should be provided and cited that when the AIDS epidemic was at its height, Federal spending was “$24 billion of today’s dollar.” Senator Shelley Capito also voiced concerns that there are not enough resources available for states that have the most need.

Senator Leahy opined that the best way to treat individuals with opioid addiction it to prevent addiction, questioning what steps the NIH has taken to treat chronic pain without using opioids. Dr. Francis Collins, the Director at the National Health Institute, stated that there are two things to take into consideration when avoiding addiction: (1) understand the transition from acute to chronic pain, as opioids work well for acute pain when the patient is exposed to the drug for only a short amount of time; and (2) the longer the patient is prescribed to the opioid, the more likely they will transition to addiction.

Education was also discussed, with Senator Capito wondering how closely prescriptions were being tracked by the CDC and questioned why those numbers were not flagged earlier. Dr. Houry responded that the CDC has been tracking numbers for a few years and they are now sending officials to communities that have exceptionally high prescription rates to educate prescribers about over-prescribing. In that vein, Senator Dick Durbin questioned whether doctors are paying attention to CDC prescribing guidelines. Dr. Houry noted that the CDC is being more rigorous with training for physicians and nurse practitioners while they are still in school. Further, for those who are no longer in school, the CDC has started online courses that outline safe prescribing practices.

Overall, throughout the hearing, members of the Subcommittee showed bipartisan support for promoting the development of effective, non-addictive pain medications.

Alliance for Continuing Education in the Health Professions Annual Conference

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January 20, 2018 through January 23, 2018, the Alliance for Continuing Education in the Health Professions (the Alliance) will hold their 2018 Annual Conference in Orlando, Florida at the Grande Lakes hotel.

The Annual Conference boasts four days of education and networking events, starting off on Saturday, January 20, 2018, with several early morning workshops followed by afternoon member section meetings. Closing out the first day are the Opening Night Awards Dinner and the President’s Dessert Reception.

On Sunday, January 21, 2018, attendees will hear the Annual State of the Alliance, followed by a keynote speech on understanding the new era of empowered, engaged e-patients. The day then breaks out into groups where topics like “Physician and Patient Beliefs About Patient Satisfaction: Aligned or Discordant,” “FAQs (and Answers!) Regarding the New CHCP Exam,” “Nurturing Effective Teams: Creating an Educational Home Where Together is Better,” and “Applying Facebook and Other Social Network Approaches into Interprofessional Continuing Education,” among many others, will be discussed. Closing out Sunday’s events is the Alliance Got Talent competition, where your peers will be performing a variety of acts.

The third day, Monday, January 22, 2018, begins with a keynote speech entitled, “Will You Be the Disruptor or the Disrupted? The Battlegrounds Over Health Care for the Next 10 Years,” before breaking off into various breakout groups throughout the day.

Our editor, Thomas Sullivan, will be part of the breakout panel discussion, “Understanding MACRA’s New QI-CME Improvement Activity: What You Need to Know to Maximize this Opportunity for Your Organization.” The program is an informal panel with three participants discussing MACRA’s physician incentives for quality care under Medicare, the Quality Payment Program and CE Opportunities. Ways to design an QI CME activity to meet the CMS MACRA definition will also be discussed.

Tuesday, January 23, 2018, the morning begins with breakout sessions discussing topics such as: integrating the patient perspective into the PM&R education process; ensuring patient voices are heard through video narratives; and how to break the silo mentality to address real world needs. The conference closes with a plenary session around noon.

If you are interested in attending this conference, you can register here.

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