When FDA Plays God, Patients and Doctors Need to Step Up
[Thursday, January 23, 2020] The patients and doctors expect FDA to approve products based only on credible scientific data, but a few cases have shown that FDA does not always do that. In those cases, it falls on patients and their doctors to make sensible decisions based on facts before embarking on uncertain therapies carrying astronomical price tags. FDA holds practically unchallengeable discretion about its decisions. And it uses it to make questionable decisions which may or may not be in the interest of patients. A Complete Response Letter (CRL) issued last year presents a scathing criticism by FDA reviewers of Sarepta’s application for a new treatment for Duchenne Muscular Dystrophy (DMD). The reviewers highlighted critical safety issues with the drug under review and highlighted new safety concern from Sarepta’s previous DMD drug approved 3 years ago from commercial use. Sarepta was also chastised for not meeting its post-market commitments for the previous related drug. However, shockingly just three months after the rejection, FDA approved the same drug without Sarepta making any major changes to its application, based on promises that are hard to enforce. Sarepta promised that it would better educate the doctors to use their product and recommend monitoring patients frequently for renal toxicity. It also “committed” that “If it is concluded that the results of the confirmatory study do not support a clinical benefit (i. e., no relevant analyses find sufficient evidence of such a benefit), Sarepta Therapeutics will voluntarily withdraw golodirsen from the market.” The company that has not met its previous commitment for post-market studies and, who filed for approval of a new drug with questionable data (according to FDA’s own reviewers), is expected to keep new promises and “voluntarily” withdraw its drug at a later time. For a drug taken life-long that costs patients more than $300,000 annually (some estimates put the annual cost to almost $900,000), this decision puts patients and their doctors in a precarious situation. DMD affects children and their parents now have to deal with a conundrum of deciding to use a “FDA-approved” drug on their kids knowing that the drug as best has a minor benefit while subjecting the patients to life-threatening risks. Since FDA has decided to play God here, the responsibility now falls on the patients and their doctors to treat or not. Imagine being a parent with a decision of no treatment versus a dubious one. And all the time the company gets richer. To put in context, Sarepta has increased more than 12 folds in value since its first approval three years ago, thanks to the FDA Gods; patients probably paid for it. This is not fair to the patients, FDA. |
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