OPINION:
Drug review processes at the U.S. Food and Drug Administration are not serving the American public.
Exhibit 1 illustrating this are the special FDA programs Congress has developed over the past two decades: fast-track designation, the priority review voucher program and emergency use authorization.
Earlier this year, FDA Commissioner Marty Makary went further, announcing that the FDA’s default stance on drug approval would now be a single Phase 3 or “pivotal” trial, rather than two.
Despite their best intentions, these congressional mandates have failed to overcome the fundamental problem: The cumbersome, bureaucratic and ossified regulatory practices and norms that characterize today’s FDA advance the interests of Big Pharma at the expense of innovation.
The recent history of Big Pharma innovation has been grim, with the industry typically relying on predatory acquisitions of smaller innovators or on the development of patentable copycat drugs.
The actual innovators are usually small, capital-light companies focused on research and development, and they lack the funds needed to navigate what is frequently an arbitrary and capricious FDA landscape, notoriously resistant to change and innovation. The complicated, cumbersome and time-consuming regulatory practices benefit large, cash-rich corporations, which have specialized staffing and resources necessary to comply.
All too often, these small biopharmaceutical innovators are faced with three options: sell their company at a discount to Big Pharma before their product has navigated the byzantine, expensive and often arbitrary bureaucracy; go offshore to countries such as China, which offer more flexibility and motivated regulators; or go out of business.
The consequence is that American patients and medical caregivers are denied access to truly innovative products while gaining items of questionable quality, safety, purity, potency and utility.
This must change.
Patients seek relief from symptoms, not from laboratory test results. The FDA must be guided by a commitment that drugs and biologicals must provide clinically meaningful benefits that greatly exceed the risks of harm, recognizing that all drugs and biologicals are associated with some risk in some patients. Therefore, patient-centered outcomes must drive drug development and approval.
In the past year, I have come across many small pharmaceutical innovators with products designed to address aspects of the president’s Make America Healthy Again Commission agenda. They share a common complaint: They have a product that addresses a MAHA priority, but the FDA is a burdensome roadblock.
One case study involves a small molecule product (L1-79) that shows great potential for relieving central nervous system symptoms associated with autism, which afflicts more than 6 million Americans. L1-79 may provide significant improvement in patient-reported outcomes in a market where nothing is currently available for the core symptoms of autism.
L1-79, which is being developed by Yamo Pharmaceuticals, is a traditional oral drug and has already received FDA “Fast Track” designation in 2018. Preliminary clinical data indicates that L1-79 may help relieve the psychological barriers that inhibit social function in adolescent and young adult autism patients and, once released, can help begin persistent functional improvement.
Many parents and doctors of adolescents and young adults with autism who participated in the Phase 1, Phase 2a, and Phase 2 trials for L1-79 were amazed by the positive results. In many cases, parents witnessed positive social interactions that had been absent before the trial.
Despite these positive results and Health and Human Services Secretary Robert F. Kennedy Jr.’s focus on finding prevention and treatments for autism, the FDA has now required that the company run clinical development studies in young children before it can obtain approval for adolescents and young adults. The estimated price tag? More than $200 million.
The FDA has also rejected Yamo’s request for “breakthrough therapy” designation, which would have facilitated raising the funds required for the program to move forward.
That is a massive hurdle.
Lost in the weaponized, partisan conflict over the President’s Make America Healthy Again Commission efforts to change the FDA culture is the fact that the quality of American patients’ lives is being compromised by the current system. The result is that American pharmaceutical innovators are being forced offshore.
It is long past time to dial back the rhetoric and focus on solving the problems that have plagued the FDA for decades. American citizens, children and patients deserve no less.
• Dr. Robert W. Malone is an American physician, biochemist and clinical researcher who conducted early foundational work in mRNA vaccine technology.
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