May 3, 2017

SWHR CEO Policy Watch Report – FDA Developments

By Amy M. Miller, PhD, SWHR President and CEO
Congress Moves One Step Closer to New Commissioner for FDA

The Senate Committee on Health, Education, Labor, and Pensions (HELP Committee) recently voted in favor of Dr. Scott Gottlieb to be the next commissioner of the FDA. Dr. Gottlieb’s nomination will now go to the full Senate for a final vote.

Dr. Gottlieb’s extensive work as a senior policy advisor to the Administrator at the Centers for Medicare & Medicaid Services, and as a former deputy FDA commissioner during the George W. Bush administration makes him a thoughtful nomination for the post. He has significant experience in understanding the realities and nuances of federal policy, and will likely be a strong steward of the science that backs FDA’s regulatory decisions.

SWHR looks forward to working with the FDA under his leadership once the confirmation process is completed.

User Fee Legislation Begins Moving Through Congress
Congress has begun reauthorizing the mechanism by which the FDA funds its regulatory work, releasing a draft bill from the HELP Committee; and the House Energy and Commerce Committee.

The reauthorization would cover the FDA user fee programs for pharmaceuticals, generic drugs, biosimilar medicines, and medical devices for fiscal years 2018 through 2022. In return for agreeing to specific performance goals to help support efficient reviews, the FDA would collect $879 million in prescription (pharmaceuticals) user fees, $494 million in generic drug fees, $45 million in biosimilar fees, and $183 million in medical device fees in FY2018.

SWHR recognizes the critical role that user fees play in maintaining efficient, predictable, and transparent FDA review processes of new innovative treatments and medical products that support women’s health. That is why SWHR participated in the discussions the FDA conducted with healthcare stakeholders and industry in 2015 and 2016, regarding performance goals and procedures in the reauthorization for the user fee programs.

We applaud the Senate and House Committees for advancing efforts to ensure a timely reauthorization of these vital funding mechanisms and recommend that Congress pass the user fee legislation as negotiated.

Drug Trials Snapshots: The Result of Successful User Fee Policy
Until recently, information about individuals participating in clinical drug trials was not readily available or accessible. Patients were left wondering if a drug had been tested on people like them and, if so, what were the benefits and side effects. Healthcare stakeholders who were involved in ensuring that the data collected was relevant and meaningful also had difficulty making such determinations.

Now, the situation is changing with the FDA’s Drug Trials Snapshots. The website provides information about who participated in clinical trials for every novel drug (i.e., a new medicine that has never been used in the U.S. marketplace) that receives FDA approval. The “Snapshot” for each drug identifies participants by the demographic subgroups of sex, race, and age; and outlines the differences in the drug’s benefits and side effects among the various subgroups.

Our work, in partnership with the American Heart Association and WomenHeart: The National Coalition for Women with Heart Disease, facilitated the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) that was signed into law in July 2012 (Public Law 112-144). It included a provision (Section 907) that directed the FDA to report on which demographic subgroups are included and analyzed in clinical trials, and also required the agency to develop an action plan on how it will address any shortcomings identified in this reporting. To address this mandate, the FDA’s Center for Drug Evaluation and Research created the Drug Trials Snapshots initiative.

The Snapshots website enhances communication with the public on drug safety and efficacy, and it gives healthcare stakeholders an important tool to use when addressing the issue of including sufficient diverse patient populations in drug trials to enable data analysis that is clinically relevant and meaningful.

SWHR commends the agency for its efforts and is committed to working with the FDA and other healthcare stakeholders to advance the conversation regarding clinical trial demographics.