How Donald Trump can benefit patients by reducing red tape at the FDA
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President-elect Trump has called for reform of the Food and Drug Administration (FDA) to put greater focus on the need of patients for new and innovative medical products.

I outlined a method by which this could be accomplished in great part simply by returning the FDA to its lawful Congressional mission of approving drugs based on safety and effectiveness.

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This prompted an attack of my proposal and a declaration that “even if Donald trump changed the FDA drug approval process, patients wouldn’t benefit.”

Due to fear of public and congressional ridicule when side effects occur with approved drugs, the FDA has progressively migrated from the safety and effectiveness standard, now dictating to drug developers the appropriate endpoints of pre-approval studies and mandating that clinical utility be proven.

I proposed FDA reform that would define four distinct categories of biologic activity that would satisfy the effectiveness requirement, consistent with the Federal Food Drug and Cosmetic Act (FD&C) including: (1) biomarker changes; (2) improvements in clinical signs and symptoms; (3) disease modification; and/or (4) long term outcomes, including survival.

The criticism that I received for suggesting that the product labels be color-coded to facilitate communication to doctors regarding the expected effects of using the drugs misses the entire point of my proposal for tiered orders of approval. So, too, does the attack on biomarker changes as proof of biologic activity.

Clinical utility and clinical benefit are highly subjective and personal experiences, best judged by patients and their doctors.

Whether to use a drug (based on the potential benefit and risk tolerance of the patient), or to continue using a drug given the benefit-risk profile in the individual patient, is a personal health decision.

The FDA makes public health decisions; the FD&C did not intend for the FDA to practice medicine from Washington, DC, or to establish best practices and treatment algorithms. Rather, the FD&C intended the FDA to adjudicate safety and effectiveness in allowing entry of new drugs and biologics into the medical armamentarium.

The practice of medicine is reserved for doctors, not the FDA. To further reinforce the separation, consider that the FD&C does not give the FDA authorization to restrict the use of devices.  No, the FDA’s job is to enter safe and effective drugs, biologics, and devices onto the medical marketplace for physicians to use in treating individual patients.

The FDA has increasingly added new hurdles for pre-approval requirements by demanding long-term outcomes and survival. A great example of this is in the approval of Praluent and Repatha, medicines that lower LDL cholesterol.

Last year, the FDA restricted their approval for patients with cardiovascular disease who need more help getting their cholesterol under control, including sufferers of a rare genetic disorder called familial hypercholesterolemia.

Broader approval must wait for the results of long-term outcomes studies. They did not even allow its use in patients who cannot tolerate statins. Why not? Similarly, serum glucose reduction and hemoglobin A1c lowering should be the bases of approval for new drugs for diabetes — why must long-term cardiovascular outcomes studies be undertaken as the bases of approval?

LDL, serum glucose, and hemoglobin A1c are the most widely studied biomarkers — they have been correlated with clinical endpoints in innumerable studies. Why must the wheel be reinvented with for every new drug? This stifles innovation, raises development costs and drug prices, delays the introduction of new products to patients, and dissuades companies – especially small companies, the cradles of medical innovation – from focusing on diseases that affect millions upon millions of Americans.

The reason that the FDA has moved away from safety and effectiveness toward clinical utility and clinically meaningful benefit as the bases of approval is to offset potential ridicule in the event that an approved drug is shown to have undesirable toxicities. The FDA has also made post-approval study requirements more onerous. What used to be exercises to obtain safety information when a drug is used in less controlled settings than pre-approval trials have turned into re-adjudications of safety and effectiveness. This potential double and triple jeopardy also increases drug development costs, time, and price, and impedes innovation.

The mission of the Food and Drug Administration (FDA), as stated in the FD&C is “to promote health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of regulated products in a timely fashion.” This includes “ensuring that… (B) human and veterinary drugs are safe and effective; (C) there is reasonable assurance of the safety and effectiveness of devices intended for human use.”

Returning the FDA to its proper role and refocusing it on its lawful mission will reduce red tape and provide great benefit to patients.

Joseph V. Gulfo MD, MBA is the executive director of the Lewis Center for Healthcare Innovation and Technology at Fairleigh Dickinson University and author of "Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances" (Post Hill Press). He has more than 25 years of experience in the biopharmaceutical and medical-device industries and is the former CEO of Mela Sciences. Follow him on Twitter @josephgulfo.

The views expressed by contributors are their own and not the views of The Hill.