Rare Disease Report

Will FDA Commissioner Scott Gottlieb Be Good For Rare Diseases?

MARCH 10, 2017
James Radke
Scott Gottlieb, MD, is President Trump’s choice to head the FDA. If his nomination gets confirmed, it could be very good news for the rare disease community.
Dr Gottlieb spent many years at the FDA, including stints as a deputy commissioner of medical policy development (2003-2005) and deputy commissioner for medical and scientific affairs (2005 – 2007).
Dr Gottlieb is currently a clinical assistant professor at the New York University School of Medicine and is involved with numerous publications, think tanks, and law firms associated with the healthcare industry.
And his position about the FDA is pretty straight forward - It needs to improve. One change he would like to see is how the FDA views the drug approval process. He believes the agency's current focus on statistics rather than good medicine is making it too difficult for drugs, especially orphan drugs, to get approved.
For example, in a debate from 2013, Dr Gottlieb took the stance that the FDA’s caution is hazardous to our health. During the debate, Dr Gottlieb used examples familiar to the rare disease community in which he argued that the FDA is more focused on the mathematics of a clinical trial rather than the patients. He used examples of clinical trials for Gaucher disease and Hunter syndrome to illustrate how it is becoming increasing too long and cumbersome for companies to conduct clinical trials. Instead of embracing new technologies that use biomarkers to test a drug’s efficacy, he argued the FDA still relies on methods such as the 6-minute walk test that greatly limit the number of patients eligible for a trial. 

Dr Gottlieb also discussed the Hunter syndrome pivotal clinical trial in a 2012 National Affairs article in which he stated the design of the clinical trial demanded by the FDA were, in Dr Gottlieb’s view, unnecessarily strict for such a rare disease.
Dr Gottlieb wrote:

“In an effort to satisfy an increasingly unreasonable hunger for statistical certainty on the part of the FDA, the trial imposed extraordinary hardships on the children and families involved. In order to approve the drug for use, the FDA required the trial to involve 96 patients with Hunter syndrome — some 20% of all Americans afflicted with the disease. Moreover, for the first time in such a study of enzyme-replacement therapy, the FDA also insisted that patients be randomly assigned to receive either the experimental drug or an inert placebo. The course of Hunter syndrome is well documented and follows a very regular pattern in most afflicted children; the results for patients who got the experimental therapy could easily have been compared against readily available historical databases that track the normal course of the disease. It is hard to see why a placebo was necessary in such circumstances, especially when the requirement for a placebo group meant that some of the kids involved wasted a full year of the most able portion of their short lives effectively going untreated.
“These and several other requirements meant that the Elaprase trial took longer, and was far more complex and difficult, than trials of similar drugs in the past, thus delaying the drug's approval.
If the tone of his argument is any indication, his nomination to head the FDA could be very good news for the rare disease community as Trump has made his desire to overhaul the FDA, very obvious. In January, he told a group of pharmaceutical executives that he planned to slash regulations and speed up drug approvals at the agency.
Stay tuned. 

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