NORD has submitted the following Letter to the Editor of the Journal of the American Medical Association (JAMA):
The recent study evaluating the evidence supporting FDA approval of novel therapeutic agents has generated debate about FDA exercise of flexibility and judgment in approving new drugs. FDA Commissioner Margaret Hamburg, MD, wrote a blog explaining why FDA supports a flexible approach to drug development. 
We who represent the patient community – in my case, the 30 million Americans with rare diseases – avidly support FDA’s exercise of appropriate flexibility. While all drugs must be shown to be safe and effective before approval, there are different ways to demonstrate this. The same clinical trial that might be used for patients with a well-understood, highly-prevalent disease would not be appropriate or feasible for a disease that affects few patients, or in whom the natural progression is not well understood.
FDA has done an excellent job in making thoughtful, scientifically-based and medically-rational decisions about the quality of evidence supporting new therapies. FDA clearly seeks to strike a balance between protecting patients from unsafe or ineffective drugs, and making new therapies available expeditiously. It is a delicate balance. About a third of the drugs approved in the past few years have been for rare diseases, and the patients we represent benefit from them.
Approving new drugs requires the kind of careful judgment that FDA has exercised. FDA has been listening to the patient voice more than ever before. We within the patient community support this approach and are thankful that FDA has enabled so many patients to benefit from novel therapies.
Peter L. Saltonstall
President and CEO
National Organization for Rare Disorders
 Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012, JAMA. 2014;311(4):368-377.
 Margaret A. Hamburg, MD, Why FDA Supports a Flexible Approach to Drug Development Posted on February 6, 2014 by FDA Voice http://blogs.fda.gov/fdavoice/index.php/2014/02/why-fda-supports-a-flexible-approach-to-drug-development/#sthash
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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