Aduhelm: Lessons From its Approval

Aduhelm: Lessons From its Approval
(Biogen via AP)
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Outrage over the Food and Drug Administration’s recent approval of a new Alzheimer’s treatment highlights several serious flaws in our nation’s drug-approval process.  We are focused on medical certainty, which can delay the availability of innovative, potentially life-saving treatments. Additionally, reports of behind-the-scenes machinations are creating authentically earned distrust in the products manufactured by our pharmaceutical industry, which leads the world in innovation. 

The FDA approved Aduhelm using an accelerated process “available under federal law when a drug intended for a serious disease is expected to provide a meaningful advantage over available treatments,” according to FDA officials

Aduhelm’s trials did not demonstrate “certain” clinical benefits, but “suggested” clinical benefits and “convincingly and consistently show safety and reduction of amyloid plaque in the brain, a defining pathological characteristic of Alzheimer’s disease.” 

The FDA’s approval has sparked a wave of protest and outrage from within the medical and research community and efforts to limit public access to the treatment.

But therein lies the issue. The FDA must balance their roles of insuring safety, efficacy and much needed innovations. Aduhelm, would give Alzheimer’s sufferers and their families the option to try an innovative treatment that is safe and reduces the hallmark finding of the disease. As the daughter of an Alzheimer patient, I would want all possible options to stop the relentless march toward cognitive darkness. 

Many suggest the primary role of the FDA is to evaluate the level of efficacy of drugs and treatments and eliminate uncertainty that those suffering from equally horrific diseases are willing to accept. 

When it comes to issues as complex as cutting-edge medicine, government’s unwieldy inexpert hand is never the best tool. Government functions slowly and with brute force. Experts work more quickly and efficiently when given fair incentives. Deciding whether utilizing new drugs or medical treatments requires the latter; these decisions must be examined with a scalpel, not a sledgehammer.

The 1962 Kefauver‐Harris Amendments established, for the first time that the FDA must demonstrate new drugs are not only safe, but also efficacious at treating a particular symptom or disorder. These amendments completely changed the role of the FDA from an entity that ensures the safety of new drugs and treatments to one that takes on the massive and time-consuming responsibility of ensuring the efficacy of new drugs before they are allowed to be legally brought to market.

Regardless of what proponents of the FDA bearing the responsibility of an entire industry suggest, medical professionals can and will use legal drugs and medical treatments demonstrating efficacy. Even with the 1962 Kefauver‐Harris Amendments, existing law exhibits this purpose; after having to prove efficacy for a specific purpose for a drug to be approved, that very same drug can be legally administered for entirely different purposes. 

The original purpose of the Food Drug and Cosmetic Act of 1938 was to create the FDA to oversee food and drug safety. The cumbersome requirements added since 1938 have at times obstructed innovative firms like Aduhelm’s developer, Biogen, preventing positive impacts on medical patients. And, Biogen has a host of innovative products including others in partnership with well-respected companies. 

The negative impacts of this additional burden placed in between new medical treatments and drugs and the American consumer are profound. According to David Dranove and David Meltzer writing in the RAND Journal of Economics, “the average time from initial human subject testing until final FDA approval of drugs approved in the late 1960s and 1970s was about 6 years . . . by the 1980s, this process took an average of 9 years. These studies and others like them have caused many researchers and policy makers to take a critical view of the 1962 Amendments.” 

In the case of cutting-edge drugs, those who are currently suffering from Alzheimer’s and other diseases also suffer from a government-imposed inability to try a new life or livelihood-saving drug for three additional years.

The FDA serves an important function – it ensures the safety of new medical treatments and drugs before they are brought to market; however, it is little merit for the FDA to determine benefit certainty. The burden of proof-of-efficacy requirement brought about by the 1962 Kefauver‐Harris Amendments only serves to prevents patients from choices in accessing treatments for horrific diseases.  Worse yet, the lack of transparency behind these efforts are creating distrust when trust is most needed.  We must restore the original and proper role of the FDA, which will in turn allow for medical innovation while promoting choices and public trust.

Marion Mass is a Philadelphia-area pediatrician who trained at Duke University Medical School and Northwestern University; she is the co-founder of Practicing Physicians of America.

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