FDA’s accelerated approval process is good enough for cancer and AIDS. Why not Alzheimer’s? 

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The U.S. Food and Drug Administration’s (FDA) approval of the first-ever disease-slowing therapy for Alzheimer’s has given hope to millions of Americans, their families, and caregivers who are all dealing with the brutal impacts of this disease and, before this decision, had not a single treatment option available. However, the Inspector General recently launched a formal investigation to not only look at the approval process for the Alzheimer’s drug, but also the full FDA accelerated approval pathway. This investigation is dangerous, reckless, and impacts the future not just of Alzheimer’s patients and their families, but the millions of people suffering from diseases not yet cured.

Full disclosure: My organization UsAgainstAlzheimer’s has received financial support from Biogen, the maker of the drug, as well as from Biogen’s competitors.

Ultimately getting this drug to market relied on an FDA process known as “accelerated approval.” Accelerated approval has long been an important regulatory tool to facilitate patient access to new medicines that treat life-threatening illnesses for which there are no available therapies.  

There is a false narrative that the FDA acted prematurely and in an unprecedented manner in approving aducanumab (now being marketed as Aduhelm by Biogen). I do not believe this is true.  

Accelerated approval is the tool the FDA has used frequently to tackle other major diseases. It started in the era of AIDS, when many believed that the FDA was too slow to act to fight that disease as it waited on elusive “perfect” data. 

Accelerated approval is used often for cancer drugs to ensure oncology patients are able to capture as much potential benefit from new therapies as possible. In fact, more than 40 percent of new cancer treatments were brought to market via accelerated approval. 

And it works for patients. Just last year, researchers from the National Cancer Institute showed that lung cancer survival rates are up in the last decade — but only in the type of cancer for which there are several FDA-approved therapies. The turning point in that battle came with an accelerated approval in 2003. Thousands of Americans are alive today because of that decision.  

Alzheimer’s patients and those who love them deserve no less.  

This latest Alzheimer’s approval provided a new treatment for patients. While there is more work to be done — and the Alzheimer’s patient community wholeheartedly hopes forthcoming studies expeditiously confirm the long-term benefit of this new therapy — the wise decision in favor of accelerated approval will allow more rapid generation of added data and access to a treatment that, without this action, would have required years of additional clinical study before being made available to patients.  

The FDA has an affirmative role in the process of finding a means of prevention and effective treatment against life-threatening diseases of high unmet need. The FDA should be commended for its thoughtful approach to the use of the accelerated approval mechanism to do just that. We recognize that this is only the first step in a longer process to battle Alzheimer’s, but it is an important step forward that hasn’t been able to be made for decades, until now. 

The approval of the first disease-slowing drug is a turning point in the fight against Alzheimer’s — an “opening gun” in the race for a therapy to prevent this disease and, in the meantime, a catalyst for other innovators to accelerate interventions even earlier in the disease course. This gives tangible reason for hope to the 6.2 million Americans living with Alzheimer’s disease, their families, and the 16 million caregivers who support them, including communities of color and women, who are disproportionately affected by this disease.  

The result of this accelerated approval also gives patients more of a say in the process of discovering the cure for the disease that affects them. Patients, more than anyone else, have the greatest interest in treatments that are shown to be effective and safe based on science.  

There is a scientific basis in the clinical trials to believe that this drug’s amyloid-lowering ability is producing precisely the clinical benefit so many study participants have anecdotally described in press reports, particularly its ability to maintain patients’ ability to conduct a normal life. How long that benefit will extend will be determined in the ongoing studies of Aduhelm as well as in the new Phase 4 study mandated by the FDA. 

Patients and caregivers want to be partners in the discovery process. Patients and caregivers are study participants and partners in the clinical trial process, at great personal and financial sacrifice but with a passion for life, for future generations of our families and for the millions of other families experiencing this disease. We will partner on future studies of this drug as well as others in the pipeline. 

The FDA here made a decision that was right on the science, right for patients, and right for future Alzheimer’s treatment innovations. In order to have a best-in-class drug, we must have a first-in-class drug. And — thanks to accelerated approval — that’s exactly what we’ve got.

George Vradenburg is chairman and co-founder of UsAgainstAlzheimer’s, which has received financial support from thousands of individuals, institutions and companies, including Biogen and its competitors.

Tags Accelerated approval Aducanumab Alzheimer's disease Alzheimer's drug Alzheimer's treatment BIOGEN Clinical trial Drug discovery FDA Food and Drug Administration Health sciences Hope for Alzheimer's Pharmaceutical industry

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