An Opportunity to Improve the FDA

Kathryn Nix / Alyene Senger / February 03, 2012

This year, Congress will have to reauthorize the Prescription Drug User Fee Act (PDUFA), which funds the Food and Drug Administration’s (FDA) drug approval process. As noted by Amanda Kronquist, a Heritage Health Policy Graduate Fellow, the FDA drug approval process has become “increasingly unpredictable, uncertain, and inefficient.”

The House Energy and Commerce Subcommittee on Health held its first hearing this year on the topic yesterday. It heard from the FDA commissioner and others in the field on the PDUFA reauthorization and its effect on jobs, innovation, and patients.

In her paper, Kronquist breaks down the complex issues surrounding the upcoming PDUFA reauthorization debate and outlines four key issues to watch:

1. Enhancing the review model for drug and biologic marketing approval. Kronquist argues that one of the main areas the FDA needs to improve is in its communication with drug companies during the review process: “The FDA needs to use its time more efficiently and incorporate sponsors throughout the review process, not just at the beginning and the end as it has done in the past. This will enhance predictability and reduce total review time.”
2. Providing resources to the FDA to further develop its regulatory sciences. Rather than lower standards to speed up the development pipeline, Kronquist says that the better solution is “to incorporate scientifically based methodologies and infrastructure—such as bioinformatic systems, which collect and analyze data from a plethora of sources using computers and statistical techniques, and developing and qualifying new biomarkers, which act as indicators for a particular biological state—into the developmental and review processes.” This would advance the FDA infrastructure to accommodate the future of medicine.
3. Establishing a risk-benefit grid as a guiding tool for approval. The drug approval process needs a “more structured and systematic system” for risk-benefit evaluation of new products. Kronquist suggests that this new approach should be more patient-centered, focusing on the risk tolerance of the actual patients affected, not the population at large.
4. Standardizing risk evaluation and mitigation strategy (REMS). Like risk-benefit assessment, Kronquist explains, REMS is in need of a standardized framework: “The FDA needs to be held accountable for ensuring that its chosen REMS are effective and efficient and ultimately reduce risk.”

Congress should use the reauthorization of PDUFA to improve the drug approval process and avoid adding more complicating regulations, since maintaining and improving the United States’ global competitive edge in the production of prescription drugs and medical devices is critical to the quality of our health care system and the economy.