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We must revamp the FDA's approval process for new drugs

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By U.S. Rep. Edolphus "Ed" Towns (NY-10)

Since I first entered Congress, I have taken a keen interest in health care matters. I learned recently in listening to the concerns of patients and health care providers that there is a need to modernize the Food and Drug Administration's (FDA) accelerated approval of pharmaceuticals for certain serious diseases--while, of course, maintaining established safety standards.

Consequently, I have been the Democratic lead in the effort to help bring new, innovative drugs to market sooner to benefit patients who currently have unmet medical needs. This is a bi-partisan effort with my Republican colleague in the House, Rep. Cliff Stearns (FL-6).

Our legislation, H.R. 4132 or the Faster Access to Specialized Treatment (FAST) Act, will modernize and expand the FDA's existing accelerated approval pathway to expedite the approval of drugs for patients suffering from life-threatening diseases. This will spur innovation and encourage the development of innovative therapies.

Twenty years ago, the FDA issued the agency's Accelerated Approval regulation. It allows early approval of drugs that treat serious or life-threatening illnesses. The FDA grants early approval on the basis of clinical trials establishing that the drug product has an effect on a clinical endpoint (a laboratory measurement) that is reasonably likely to predict clinical benefit.

Even though all drugs for serious or life-threatening conditions are eligible for accelerated approval, the fact is that HIV/AIDS and oncology therapies have disproportionately been approved using this pathway. A part of the problem is that the pathway to approval has been interpreted narrowly. Furthermore, the uncertainty and lack of transparency regarding eligibility, approval standards, and endpoint selection has undercut innovation and research into new drugs.

One of my concerns is that the current Accelerated Approval process has not sufficiently benefitted rare diseases, which are illnesses that affect fewer than 200,000 Americans. According to the National Institute of Health, rare diseases affect 25 million to 30 million Americans, which include such illnesses as cystic fibrosis, muscular dystrophies, and Huntington's Disease.

According to the FDA, there are more than 400 drugs and devices approved for rare diseases. "However, we still have much to do to meet the rare diseases challenge -- there are 7,000 rare diseases and most are without a current treatment available," the agency notes in its blog.

The FAST Act would codify, modernize, and expand FDA's Accelerated Approval pathway through a series of targeted revisions, which include broadening the approval pathway, leveraging new scientific methods and tools, and introducing greater transparency and predictability in the pathway.

Expediting the approval process for neglected illnesses would not weaken the FDA's robust approval process and existing safety standards. If the FAST Act passes, it could improve the delivery of medications to millions of Americans who suffer from serious or life-threatening diseases.


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