Another great conference session this afternoon: “Timing Is Everything: Balancing Access and Uncertainty.” This one was moderated by Jeff Skopek, with presentations by Shannon Gibson, Trudo Lemmens, and Efthimios Parasidis.
First, we heard from Gibson and Lemmens on “Overcoming ‘Premarket Syndrome,’” AKA the various problems associated with relying solely on premarket data for safety and efficacy determinations. These problems include the fact that industry studies have been shown to be more likely to be biased in favor of demonstrating a positive result, and premarket studies also cannot really demonstrate how a product will be used in clinical practice. Moreover, for niche market drugs (e.g., those for orphan indications or pharmacogenomics), which are becoming more and more popular, there are fewer patients available to serve as clinical trial subjects, thereby inherently limiting the data that may be generated prior to approval and increasing uncertainty around safety and effectiveness.
So what should we do? Develop an improved post-market research agenda, say Gibson and Lemmens, and explore “adaptive licensing,” by which they mean rendering regulatory decisions based on the entire body of evidence collected throughout a product’s life cycle. They argue that drug access decisions should not be binary, but instead, should be incremental and continually reassessed based on new data, including data that becomes available after a drug has been initially made available to patients. They closed by pointing out the importance of data transparency at all points in drug regulation.
Next, we heard from Efthimios Parasidis on “Innovative Regulating as a Public Health Imperative.” Parasidis focused on the ways in which FDA can leverage its existing post-market regulatory authorities granted under the Food and Drug Administration Amendments Act of 2007, including requiring post-market studies and imposing Risk Evaluation and Mitigation Strategy requirements (REMS).
