FDA’s Aduhelm Decision is Permanent, Aduhelm will Not be an Exception
(Thursday, July 15, 2021)
Despite the media circus around FDA’s approval of Biogen’s Aduhelm, FDA’s decision is legal and permanent. And it will be repeated for other drugs in similar situations. The controversy around this decision is being fueled by misperceptions or misunderstanding of the FDA review process. The regulatory processes and FDA interactions that preceded the FDA decision are frequently used and well-established systems. In fact, the same processes have always been encouraged by the FDA for rare diseases with few or no treatment options. Here are the three major reasons why all the hoopla should not discourage other developers from following the same route. First, accelerate approval pathway that allows a developer to rely on surrogate markers has a history of about 30 years with FDA. The use of surrogate markers was critical for the approval of all anti-HIV drugs in the 90s and several oncology drugs. Biogen’s mistake was to use the surrogate markers late in the game. Records show that Biogen waited till the end of its pivotal trials, where the direct endpoints could not be met, to discuss options with the FDA, where FDA advised the use of surrogate markers to salvage the program. It is insane to imagine a situation where FDA’s holds back critical advice to a company asking for help. FDA gave the right advise that led to the approval decision. The lesson here is that developers should plan to use surrogate markers sooner than later. Second, patients played a big role in influencing FDA about the need for the treatment and approval of the drug despite sketchy data. Something is better than nothing. In an article justifying their decision, the FDA officials emphasized the importance of patient hearings where patients were emphatic to “accept the trade-off of some uncertainty about clinical benefit in exchange for earlier access to a potentially effective drug”, as the basis for FDA’s decision. This is the not the first time that the patient’s opinion superseded scientific or medical opinion by the Advisory Committee and won’t be the last. Lesson two, read the patient opinions and use it. Third, once the decision is made, there is no mechanism for FDA to reverse it, i.e., withdraw the approval, without major safety issues with the use of the drug. Since, it is almost certain that there was nothing illegal about the FDA’s process, FDA cannot reverse its decision. Many patient advocate groups have welcomed the FDA decision, and now the discussion for patients has shifted to about the price of the drug or who would pay for it rather than if FDA should have approved the drug in the first place. All of that is outside of FDA’s scope of responsibilities or concerns. So, when the hoopla dies away, stories disappear from the media, the drug will be still there in the market and Biogen will reap the rewards. The narrowing of the patient population announced by the FDA is likely not going to affect the market much as doctors are free to prescribe the drug for any of their patients who can afford it or get it covered. Other developers of Alzheimer’s drugs have already announced that they plan to follow the same path as Biogen for their product approvals, because it works.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC