The FDA has wavered from this mission over the past two decades such that pre-approval requirements and post approval demands have gone far beyond the Federal Food, Drug, and Cosmetic Act (FD&C) imperative to approve safe and effective drugs.
Through the approval process, it has, in effect, endeavored to establish preferred treatment algorithms for physicians to follow. That is, the FDA is seemingly attempting to practice medicine from Washington, DC.
Take a look at the FDA’s response to the testimony of patients and patient advocacy groups at the Senate Committee on Homeland Security and Governmental Affairs hearing, Connecting Patients to New and Potential Life Saving Treatments. In a follow-up article, Tragic Patient Stories Don't Trump Weak Data, Official Says, the FDA officials were quoted as saying the following: “We don’t want drug developers to just study what doctors think is important…Patients often have a different focus. They want better quality of life. They may even trade off survival for a greater quality of life.”
So, the FDA believes it knows better than patients’ doctors what is important to patients. On a personal note, I find this quite amusing because my parents paid for two MD degrees (my brother and myself); but, they listen to neither — they only want to hear what their personal doctors say. That is how sacred the bond between doctor and patient truly is.
How can Donald TrumpDonald TrumpBiden says Roe v. Wade under attack like 'never before' On student loans, Biden doesn't have an answer yet Grill company apologizes after sending meatloaf recipe on same day of rock star's death MORE’s new FDA commissioner fix the problem? Here are my suggestions:
Three things that must be done. First, the behaviors that have driven the FDA to adopt a protect-the-status-quo-in-health at all costs ideology need to stop. This includes public humiliation and ridicule of the agency at Congressional hearings when unfortunate side effects emerge with approved drugs. The new FDA commissioner needs to project pride in FDA decisions and engender confidence in the American people that the FDA is more interested in the well-being of patients than its own image. We need to show the FDA love, and support FDA staff in doing their jobs and in the decisions they make.
Second, the FDA needs to get back to its lawful mission, which is to promote health. The law was actually constructed this way as it lists reasons for not approving new drug applications. The expectation in the law is clearly that products that make it through the long hard slog and gauntlet that is the clinical trials process would be approved. It is like baseball — a mediocre hitter goes up to the plate with the mentality, “I will swing if it is a strike,” but a Hall of Fame hitter goes up to the plate with the mentality, “I am swinging unless it is a ball.” Patients and doctors want the new FDA commissioner to start swinging, again.
Last, we need to make the FDA return to using safety and effectiveness as the sole basis of approval, as stated in the law. In lieu of safety and effectiveness, the FDA now preferentially mandates long term outcomes, survival, and their evaluation of clinical utility and benefit-risk — this drives R&D cost, time, and uncertainty.
For new drugs that can be used safely, I have proposed four categories of approval based on the nature of the medical evidence used to prove that the drugs have positive biological effects in patients:
(1) biomarkers (e.g., reductions in glucose, cholesterol levels)
(2) improvements in clinical signs and symptoms (e.g., pain and lung function)
(3) disease modification and modulation (e.g., reduction in flare-ups of MS and ulcerative colitis);
(4) long-term outcomes (e.g., overall survival).
Whereas the D.C. establishment has only succeeded in greatening the FDA’s size, scope, power, budget, and bureaucracy, President-elect Trump is uniquely positioned to make the FDA great again as an outsider. The way to make the FDA great again is to make it work for patients and doctors, as was the intention of the FD&C. As it stands, doctors and patients and their loved ones have experienced lackluster advancement of health relative to the scientific breakthroughs that are occurring, and they are literally sick of it.
Trump, unbeholden to the Washington bureaucracy, political class, and various special interests that have contributed to, enabled, or permitted the current situation has the ability to do this like no President-elect in history. And, if he truly wants to reform the FDA to put greater focus on the need of patients for new and innovative medical products, his new commissioner absolutely must be a strong leader who can reverse the culture of fear and inertia that has hamstrung the delivery of innovative medical products to patients.
The views expressed by contributors are their own and not the views of The Hill.