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Potential FDA pick favors approving drugs before efficacy demonstrated
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President Donald J. Trump is said to be considering picking former HHS official Jim O’Neill to lead the FDA, a move that would signal the administration is serious about loosening regulations on the drug approval process.
Trump has yet to name a replacement for Robert M. Califf, MD, who stepped down as FDA commissioner when the new president was inaugurated, according to tradition. He would likely find an ally in O’Neill, who appears to favor the approval of drugs before their efficacy has been established.
O’Neill held several positions at HHS from 2002 to 2008 and has been named by several media outlets as a likely candidate to lead the FDA under Trump. His views on deregulation were evident during a 2014 speech at an anti-aging conference in Santa Clara, California, when he said FDA rules should be changed so that drugs can be approved after they have demonstrated safety but before they are proven to be effective so that patients can “start using them at their own risk, though not much safety risk.”
“Let’s prove efficacy after they’ve been legalized,” O’Neill said during a keynote address.
This stance, and comments made by Trump himself, seem to strike at the heart of what the FDA has been doing since 1962, when Congressional legislation spurred by the thalidomide disaster in Western Europe gave the agency broader authority over the drug approval process by mandating proof of efficacy in addition to safety.
Any alteration to the current drug approval process would require a change in that legislation, which is where having an FDA commissioner aligned with the goals of the White House would be beneficial, according to Susan F. Wood, PhD, associate professor of health policy and management in the Milken Institute School of Public Health at George Washington University.
Wood was the FDA’s assistant commissioner for women’s health from 2000 to 2005 and directed the agency’s office of women’s health before resigning over the continued blockage of emergency contraception as an over-the-counter medication.
“If the commissioner comes in with an entirely different view of the FDA’s mission, they are the person who is going to be helping to convince Congress that this might be a good idea to change the mission, to change FDA’s role, to lower the standards for review,” Wood told Infectious Disease News.
‘A red herring’
Wood argued that the FDA’s drug approval process is already fast and maintains higher standards and enforces a more rigorous approval process than other regulatory bodies in the world. She said approaches instituted over the past 2 decades have made it even faster, but further changes could risk ruining the agency’s standards.
“The focus on speed, at this point, is a red herring,” Wood said.
The FDA has pushed back against criticism of its policies, releasing a report in January detailing 22 drugs, vaccines or medical devices that showed promising phase 2 results but failed in phase 3 development. The agency said the examples, all since 1999, were chosen from a large pool of similar cases to illustrate how the current approval process of increasingly rigorous testing at each stage can be effective.
Trump has been a vocal critic of the agency, both before and after his inauguration. Following his surprise election, his transition website indicated that he would move to loosen FDA regulations by reforming the agency “to put greater focus on the need of patients for new and innovative medical products.” Since taking office, Trump told a group of pharmaceutical industry leaders that his administration would “get rid of a tremendous number of regulations,” and signaled support for a national “right to try” law. Trump continued this line of critique, calling the FDA approval process “slow and burdensome” during his first address to Congress.
Wood said the FDA commissioner is not there to make political decisions that override scientific expertise.
“The FDA and the reviewers within it have always followed this combination of what the law instructs them to do … combined with high scientific rigor and high scientific standards. That’s why FDA is the gold standard worldwide,” she said. “If the law was changed such that it directs FDA to not follow or look at efficacy data, not follow high scientific standards, then … that would be a disaster.”
‘There are other ways’
Excluding efficacy data from the drug approval process could negatively impact the treatment of patients, according to Helen W. Boucher, MD, FACP, FIDSA, infectious disease physician at Tufts Medical Center and professor of medicine at Tufts University School of Medicine.
Boucher said treating patients with the wrong antibiotic can have “serious consequences” not only for the patient, but for the public at large because misuse of antimicrobials drives antibiotic resistance. Speeding up patient access to urgently needed new therapies should not mean sacrificing efficacy data, Boucher said.
“There are other ways,” she told Infectious Disease News.
These include the “limited population” approval mechanism for antibacterial drugs that is part of the 21st Century Cures Act signed by President Barack Obama in December, which Boucher said could lead to smaller, more rapid clinical trials. Over the past several decades, the FDA has instituted several programs such as priority review, breakthrough therapy, and the fast-track designation, that were meant to accelerate the process.
“Treating patients with serious infectious diseases is highly complex — particularly since pathogens have increasing resistance against available antimicrobial drugs,” Boucher said. “Efficacy data provide us with some level of assurance that a drug will be effective in treating a particular disease. Without it, it would be much more difficult for clinicians to make appropriate choices for our patients.”
There is some evidence to suggest that O’Neill would not be a popular industry pick to lead the FDA. Just 8% of the 53 drug company executives surveyed recently by Mizuho Securities USA Inc. thought Trump should nominate him as FDA commissioner. Among the four choices in the survey, former FDA Deputy Commissioner Scott Gottlieb, MD, currently a resident fellow at the conservative American Enterprise Institute, received 72% of the votes.
In another informal survey, a number of drug developers told Endpoint News that they supported the current science-based FDA approval process and did not want to see the changes apparently favored by O’Neill and Trump.
Wood said keeping ineffective drugs out of the market is good for everybody, even the drug companies.
“The idea that the market will somehow make determinations while companies are theoretically allowed to market and advertise a product for a particular indication for which it has not been shown to be effective [goes] straight back to snake oil salesmen,” she said. “It’s actually quite frightening. It’s not good for companies, it’s not good for patients and it’s not good for prescribers.” – by Gerard Gallagher
References:
Endpoints News. Dear President Trump: don’t destroy the FDA we know and respect. 2017. https://endpts.com/dear-president-trump-dont-destroy-the-fda-we-know-and-respect/. Accessed March 1, 2017.
FDA. Promoting safe and effective drugs for 100 years. 2006. https://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/PromotingSafeandEffectiveDrugsfor100Years/. Accessed March 1, 2017.
Mizuho Securities USA Inc. Survey results: who should President Trump nominate as the next FDA commissioner? 2017. https://msusa.bluematrix.com/sellside/EmailDocViewer?encrypt=35a97756-c4c1-42b1-924f-7c03603ff49d&mime=pdf&co=msusa&id=. Accessed March 1, 2017.
White House. President Trump works to make drugs more affordable, create jobs. 2017. https://www.whitehouse.gov/the-press-office/2017/01/31/president-trump-works-make-drugs-more-affordable-create-jobs. Accessed March 1, 2017.
YouTube. Fight aging with a durable business – Jim O’Neill. 2014. https://www.youtube.com/watch?v=9Y7oazjaSyE&t. Accessed March 1, 2017.
Disclosures: Boucher is the treasurer for the Infectious Diseases Society of America and reports being on a data monitoring committee for Actelion Pharmaceuticals. Wood reports no relevant financial disclosures.