Should patients have a say in which new drugs the FDA approves?
“Do Neurology Patient Advocacy Groups Wield Too Much Power?” This was the headline of a recent article in Medscape Medical News which supplied a number of reasons why the FDA should be wary of listening too uncritically to “regular people” who want to see a certain drug authorized. If everyone is being bribed, who can be trusted to provide unbiased scientific advice?
Who could be more pure-minded than the patients themselves?
The FDA (Food and Drug Administration) was founded in 1906 with the stated purpose of protecting American consumers from unsafe foods and medications. Over the past few decades, it has fallen into disrepute among many who claim that the agency has been “captured” by the pharmaceutical industry — that its members are bribed with money and/or prestige to approve drugs and medical devices that do more harm than good. It is no secret that many FDA members either come from drug companies or end up working there following a stint at the federal agency in what is often referred to as the “revolving door.” But whose are the voices, untainted by corruption, that could be overseeing the FDA’s activities and ensuring that it really does act in the public interest?
Some might think that consumer advocacy groups are perfectly situated to fill that gap. Such groups are usually focused on a single disease or condition, such as Alzheimer’s, ALS, or Multiple Sclerosis, and they supposedly have a single interest at heart: ensuring that new drugs or treatments that offer hope and real promise in treating disease are approved and made available to sufferers.
That is certainly true in some cases, and many advocacy groups do very good and important work. As Holly Fernandez, associate professor of Medical Ethics and Health Policy at the University of Pennsylvania points out, members of consumer groups bring a unique perspective to the FDA table — they can provide insight on what it actually feels like to live with the condition under question and which symptoms are subjectively most distressing.
Pure minds are wonderful, but smart brains are essential
Fernandez also stresses, however, that the risk-benefit analysis made by a suffering patient may differ significantly from that made by a medical professional with access to and a deep and nuanced understanding of the research:
Listening to the patient's voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try. People who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.
Nonetheless, their voices are being heard, and heeded, at FDA meetings, and this has been the case ever since the 1980s, according to Professor Matthew S. McCoy of the University of Pennsylvania, Philadelphia, who traces this development back to the activism of AIDS sufferers during that period.
These days, the FDA usually grants advocacy group representatives around an hour to make their presentations at advisory committee meetings. These representatives could be patients themselves, or caregivers (for instance, relatives of people with Alzheimer’s disease). In addition to the presentations they give, members of advocacy groups frequently write hundreds or even thousands of letters to the FDA in advance of its review of a new drug.
When desperation leads to denial
What Fernandez warns about has transpired on many occasions. To give one example, the Alzheimer’s Association lobbied the FDA intensely for years, pressuring its members to approve a new drug called aducanumab (Aduhelm). Aduhelm’s manufacturers, Biogen/Eisai, claimed that the drug slowed the degeneration of Alzheimer’s patients. The drug’s clinical trial results, however, did not support their claim strongly enough to justify the associated risks, particularly that of potentially fatal brain swelling, and the FDA advisory panel’s expert members refused to vote in favor of authorizing Aduhelm.
That was in 2020. Then, in 2021, the FDA nonetheless decided to approve Aduhelm, following many more months of intense pressure from advocacy groups. Their members were determined to see the drug on the market despite its astronomical price tag (which would be covered by Medicare). Two other similar drugs were also subsequently approved against the initial advice of the FDA’s expert advisers and in line with the demands of Alzheimer’s carers. These drugs were ultimately taken off the market when their failings became too obvious to ignore.
Who needs clinical trial results when weeping patients make the case better?
In 2016, the FDA approved Exondys 51, promoted by Sarepta Therapeutics (its manufacturer) as “the first FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene...”
Exondys 51 was licensed under “accelerated approval,” meaning that the FDA allowed it to be marketed even before its clinical benefit was fully proven:
Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.
As with Aduhelm, the FDA expert advisory board initially voted against approving Exondys. Nonetheless, the FDA — or rather, Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research — ultimately did approve it. Dr. Aaron Kesselheim, one of the three FDA advisers who resigned in protest of the agency’s authorization of Aduhelm, excoriated Marks for his decision, writing in The Washington Post that it “undermined public trust” as well as the incentive for drug companies to conclusively prove the merits of their products.
If you're a poor farmer from Alabama, how will you travel to the FDA to plead your case without Pharma-funded transportation?
Sarepta, according to Medscape, “has a history of working with — and funding — patient advocacy groups ... When the FDA panel voted ‘no,’ Sarepta mobilized families to pressure the agency and Exondys was eventually approved.”
The Medscape article’s author then asks,
Does Taking Industry Money Equal Conflicts of Interest?
Their question is not rhetorical. After all, there are convincing reasons why advocacy groups need access to significant funds in order to do the work they do. On the most basic level, representatives need to travel to wherever the FDA advisory board is meeting in order to present their case. Transportation is expensive; days off work mean lost income; many regular patients or caregivers may need professional assistance in crafting their presentations for maximum effect. Where is all this money to come from?
Nonetheless, bribery remains bribery no matter how noble the motive. Medscape presents the essential problem as being one of disclosure, and notes that around 20 percent of advocacy groups fail to disclose the Pharma money that has covered their expenses either in part or in full. And huge amounts of money are being poured into their endeavors:
The Alzheimer's Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug. [emphasis added]
The problem is widespread. In fact, not only do Pharma companies fund advocacy groups, they also provide them with board members:
In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions.
It is by no means coincidental that so many of these organizations are focused on cancer and neurological conditions — these are areas where highly effective treatment is either non-existent or unpredictable, making patient advocates far more desperate in their pleas for new drugs and far more capable of inspiring pathos in their listeners.
Is Pharma really paying people to lobby for drugs that will harm them?
Professor McCoy stresses that advocacy groups are highly effective at what they do, evidence of which is provided by the drug companies themselves:
The sponsors wouldn't be paying for patients to show up and give these testimonies if they didn't think it made a difference. The audience isn't just panel members; it's also agency officials and maybe elected officials as well.
Advocacy groups lobby not only for new drugs but also for new laws and new federal funding for pet projects which may or may not be to patients’ benefit.
One thing is certain, however: Their actions do benefit the pharmaceutical companies, significantly.
The information contained in this article is for educational and information purposes only and is not intended as health, medical, financial or legal advice. Always consult a physician, lawyer or other qualified professional regarding any questions you may have about a medical condition, health objectives or legal or financial issues. If you are struggling with suicidal thoughts, you can call a qualified free mental health helpline or seek help from a qualified therapist.
