Experts Discuss Efficacy, Safety and Affordability in the New FDA

04.18.17

“Our ability to deliver value to the millions of patients who rely on us is contingent on the most efficient and effective FDA possible,” noted CVS Health Chief Health Strategy Officer and General Counsel Tom Moriarty in his opening remarks at the April 2017 POLITICO Pro Healthcare Briefing.

But why? And how do we achieve more efficiency and efficacy while ensuring drugs are safe? A panel of experts tackled these questions, highlighting changes that could help promote safety, efficacy and affordability in the new Administration’s Food and Drug Administration (FDA). Their discussion built on an insightful working group meeting of policymakers and other thought leaders, covering many of the same themes.

FDA Positioned to Evolve and Address Challenges

Panelists pointed to a history of collaboration between the FDA and industry stakeholders to address public health challenges. The agency has demonstrated potential to evolve and speed approvals under certain circumstances, such as when it implemented the breakthrough therapy designation in 2012. Experts noted this indicates continued potential for the FDA to evolve to better meet patient and public health needs.

Balancing Speed with Safety

Given the critical nature of drug safety and efficacy, panelists explored how to balance informed decision making on drug approvals with getting drugs to market more quickly. They debated the benefits of using data from surrogate markers, which are used to predict a clinical outcome, versus clinical trials, which measure actual clinical outcomes. Although the experts were mixed about which data should be evaluated and acted upon by the FDA, they leaned toward a balanced approach: looking at surrogate markers and real-world, post-approval studies for indicators of how well a drug may work, to be validated by clinical trials.

Increasing Efficiency and Competition to Drive Affordability

Some experts underscored the benefit of advancing more drugs to market, especially generics, which drive down prices through free-market competition. In fact, it was noted that by the time a second generic drug enters the market, the branded price drops 50 percent. However, increasing competition in the drug market will require addressing the generic backlog, which amounts to more than 4,000 pending applications at the FDA.

Panelists also touched on other aspects to increase efficiency and competition, such as modernizing technology to facilitate sharing real-world data – either after drugs come to market or potentially as part of the approval process – as well as evolving payment models based on value and outcomes presented to FDA.

For more information about the issues related to prescription medication costs, visit our Rising Drug Prices Information Center

Panel participants included:

  • Adriane Fugh-Berman, M.D., Professor of Pharmacology and Physiology and Director of PharmedOut, Georgetown University Medical Center

  • Joseph Ross, M.D., Associate Professor of Medicine and Public Health, Yale University

  • Eduardo M. Sotomayor, M.D., Director, George Washington University Cancer Center

  • Joel Zinberg, M.D., Visiting Scholar, American Enterprise Institute; Associate Clinical Professor of Surgery, Mount Sinai Medical Center