Where is PDUFA Headed in 2015?


Today's post is authored by Dr. Rob Metcalf, Vice President of Global Regulatory Affairs at Eli Lilly and Company and a member of the FasterCures Benefit Risk Advisory Council.

What's ahead for regulatory reform in 2015? I may not have a crystal ball, but after participating in a panel last week on the future of the Food and Drug Administration (FDA) and Centers for Medicare and Medicaid Service, I do have some predictions--especially when it comes to the Prescription Drug User Fee Act (PDUFA). PDUFA helps provide the FDA with the resources it needs to review and approve new medicines in a timely manner. The fifth version of PDUFA, PDUFA V, became law as part of the Food and Drug Administration Safety and Innovation Act in 2012; it will be up for reauthorization by Congress in 2017 as PDUFA VI.

The panel agreed that 2015 should be a year of progress toward PDUFA VI. Here are three areas of focus for PDUFA next year:

Increased Patient Engagement

PDUFA V gave patients new opportunities to engage in the regulatory process. This input has helped the FDA understand what people with a particular disease see as treatment priorities, as well as what they think about the pros and cons of potential new medicines. As my fellow panelist Margaret Anderson of FasterCures pointed out, "There's a hunger and a thirst to better understand patient preference in benefit-risk assessment." This robust patient engagement should continue and expand to include a broader range of patient groups in PDUFA VI.

Continued Strong Bipartisan Support

Improving the drug development and review process--ultimately, helping patients have access to safe and effective medicines--is a universal goal. Past iterations of PDUFA have earned strong bipartisan support, and I expect to see the same feeling in its next round. Engagement by advocacy groups and patients has helped members of Congress on both sides of the aisle understand the importance of this legislation to keeping people healthy.

Enhanced Scientific Expertise

To ensure efficient and rigorous review of all potential medicines, the FDA needs to keep pace with cutting-edge science. FDA needs to be able to more quickly and efficiently hire key expertise and leadership. However, in today's fast moving scientific environment, it's impossible for the FDA (or any organization) to have all the internal expertise and capacity at any point in time to manage work demands. Broader access to both capabilities and capacity to work on behalf of the FDA could be provided by external experts groups to augment the efforts of FDA personnel in areas such are tool development; for example, to accelerate biomarker qualification. This combination of capability and capacity will result in more timely and well-informed drug development and decisions on new medicines.

Of course, an open and inclusive dialogue about PDUFA is perhaps the most important movement to continue in 2015, and I was honored to be a part of that conversation last week. Thanks to the FDA/CMS Summit for having me and to my fellow panelists for a great discussion!