The HIV epidemic was devastating, a virus that infected individuals globally but also society as a whole. The public health crisis prompted the FDA’s creation of the accelerated approval program in 1992.

This program expedites the approval of drugs which show promise in an area of unmet medical need such as AIDS or muscular dystrophy. The advantage of fast tracking is that patients can receive these drugs exponentially faster. The cost is that pharmaceutical companies aren’t required to provide rigorous evidence of their drug’s clinical success as they are with traditional approval. Instead, companies submit a datapoint that implies future success- drug makers must predict efficacy, not prove efficacy. In other words, accelerated approval has a lower standard of evidence than traditional approval so a confirmatory trial is required afterwards to confirm that the drug is indeed effective. The problem is that prediction is subjective and its only validation- a confirmatory trial- is not strictly imposed by the FDA. 

Speculation over the program’s merit heightened after 3 FDA advisers resigned after the accelerated approval of Biogen’s Alzheimer’s medication: aducanumab.  Dr. Aaron S. Kesselheim, a Professor of Medicine at Harvard Medical School with legal training, said it was “the worst approval decision the FDA has ever made” and resigned afterwards. Dr. Erik Musiek, a physician who specializes in cognitive disorders and treats many Alzheimer’s patients at Washington University, believes Aducunumab suggests a turning point where questionable evidence can fulfill FDA standards. He has publicly discussed how it is harmful for drug companies like Biogen to profit from minimally tested treatments because families “are thinking about the now; they’re not worried about a few years down the road,” and now advocates for a stricter scrutiny of its approval. 

 Aducanumab’s approval sparked debate in Washington given current studies suggest it may not be effective. Biogen has 10 years to conduct a confirmatory trial to ensure it is. However, after 10 years, the market has already been saturated with the treatment. After 10 years, Biogen has already profited and families have ingrained hope that the drug may cure their loved one from Alzheimers. 

According to the British Medical Journal, 112 of the 253 drugs approved through the accelerated approval pathway still have not been confirmed as clinically effective. The reason is that companies drag their feet if there aren’t credible threats that the FDA will withdraw approval if not given a confirmatory trial. If the Senate legislated the FDA to enforce the completion of confirmatory trials 5 years after drugs have been released via accelerated approval, we could ensure the safety of drugs for Americans and reduce health care costs for the government and U.S citizens.

Asking for results in 5 years isn’t impossible – in the past drug companies have been able to quickly prove that their medicines merit full approval or don’t. Once Gilead Sciences’ drug Trodelvy received accelerated approval in 2020, Gilead efficiently conducted trials and provided additional data that proved the drug lengthened the lives of breast cancer patients. Gilead received full approval just a year later in April 2021 and now their treatment is changing lives. On the other hand, in 2008, Gennetech’s drug Avastin was granted accelerated approval. But in 2011 the FDA Commissioner Margaret Hamburg revoked the agency’s decision after confirmatory trials discovered Avastin significantly raised the risk of serious bleeding, heart attack, and stroke. Had Genentech postponed their confirmatory trial, a life-threatening drug would have remained on the market, endangering women for much longer than 3 years. Furthermore, a 2021 research study led by Yale’s School of Medicine finds that when companies are punctual and systematic, their confirmatory trial duration can be 50 months. Less than 5 years. Evidently, in many cases a pharmaceutical company can conduct a confirmatory trial within 5 years after accelerated approval for the safety of Americans. 

In addition to drug safety, strengthening confirmatory trial requirements will financially benefit the U.S government, patients, and taxpayers. A 2021 study of Medicare spending in the Journal of the American Medical Association revealed from 2015-2019, Medicare spending on accelerated approval drugs reached 9.1 billion dollars. Yet half of these funds are spent on treatments in which the clinical benefits remain confirmed. Private insurance companies, medicare, and medicaid pay millions of dollars for drugs for which we do not know the real benefits and risks. The consequence is raised insurance premiums and wasted tax dollars. Charging vulnerable patients for drugs without evidence that those drugs will improve the patient’s quality of life can be unethical as well as an economic burden on the government and U.S healthcare system. By requiring timely confirmatory trials, the FDA can withdraw drugs that are clearly ineffective and swiftly halt their economic damage, as opposed to letting it percolate into national debt and patient bills.

FDA officials argue that stronger confirmatory trial enforcement will disincentivize pharmaceutical companies from seeking drug development in underdeveloped medical areas. However the treatment of rare and incurable diseases is a competitive, billion dollar market that companies cannot easily be deterred from. In Medscape, Dr. Gyawali of Queen’s University of Medicine states, “an unmet need doesn’t imply that the treatment void should be filled with a drug that provides nothing of value to patients.” While this reform does make it harder for companies to have their drugs approved, harder isn’t necessarily a bad thing when patient health is at stake. 

With the expiration of the FDA User Fee Act in September 2022, Congress is examining current FDA protocols with greater scrutiny in the re-authorization process. The greater urgency being given to the FDA and its policies in 2022 in Congress is a “natural vehicle for other FDA legislative reforms.” This is the time to take action to reform the FDA’s accelerated approval program.The United States Senate Committee on Health, Education, Labor, and Pensions (HELP) which has jurisdiction over the FDA will be key in these decisions. What’s notable is that the Senators, democrat and republican, in this committee have received a total of $420,791 in 2021-2022 campaign donations (Senator Tim Scott received $137,062) from pharmaceutical companies. Strengthening accelerated approval may have the effect of reducing drug company profits and consequently drug company campaign donations. The question is will Senators be hard-pressed to reform a policy that momentarily benefits their political ambitions? 

The FDA must end accelerated approval’s weakness, or accelerated approval’s weakness will end the FDA.