Update: Since the writing of this article, Donald Trump has picked Scott Gottleib as FDA commissioner.
-SITN editorial staff
by Linda Honaker
figures by Rebecca Clements
Donald Trump will soon pick a new commissioner for the Federal Drug Administration (FDA). The choice will likely be someone who will try to make the administration’s drug approval requirements less rigorous in order to get drugs on the market more quickly.
In the development of new drugs, FDA approval is actually one of the faster steps. The basic biomedical research and clinical trials that precede it take years, often decades. The approval of generic drugs, while not bottlenecked by research, is slowed by approval backlogs caused by limited funding to the Office of Generic Drugs and poor cooperation from drug manufacturers.
New Administration, New FDA Commissioner
During the campaign, Trump’s plan for his first 100 days in office included the pledge that, “Reforms will also include cutting the red tape at the FDA: there are over 4,000 drugs awaiting approval, and we especially want to speed the approval of life-saving medications.” In his recent address to a join session of Congress, he blamed the FDA’s approval process for preventing “too many advances … from reaching those in need.”
The president’s rumored picks for FDA commissioner have all been outspoken on how to speed the drug approval process. One candidate, venture capitalist Jim O’Neill, would do away with the requirement that drugs need to be shown to be effective as well as safe. “Let people start using them at their own risk,” he said in a 2014 speech. Another candidate, biotech and Bitcoin entrepreneur Balaji Srinivasan, has also been vocal about the FDA as standing in the way of innovation, suggesting it be replaced with ‘Yelp for Drugs.’
Should O’Neill or Srinivasan be appointed, it will be the first time an entrepreneur has lead the FDA, as well as the first time in 50 years that someone who is not a physician or biomedical researcher held the position. But Trump’s other two candidates, who are physicians, have been critical of the FDA as well. Scott Gottlieb, MD, has accused the FDA of breaking the law by not approving drugs fast enough. (Interestingly, Gottlieb was deputy commissioner of the FDA from 2005 to 2007.) And Joseph Gulfo, MD, has written of how mismanagement of the FDA cripples medical advances.
The decisions that the FDA commissioner makes about drug safety can have life or death consequences. Thus, it is worth examining whether these claims made by potential FDA commissioners about the FDA bear any truth by considering the approval processes of both novel and generic drugs.
New Drug Approvals are Fast – the Bottleneck is Basic Biomedical Research
The FDA’s approval rate of novel drugs (i.e., drugs that are the first of their kind on the market – the development timeline is seen above) has steadily increased over the years, according to a recent Forbes analysis. The rate grew from 50% in 2008 to 88% in 2014, and the FDA claimed that rejecting drugs had become a rare thing at the agency. Furthermore, a study in the New England Journal of Medicine found that the FDA outpaced its international equivalents serving similar populations. The study compared the FDA to the European Medicines Agency (EMA) and Health Canada (HC) in the time each took to review and approve novel drugs. The study found that the FDA spent less average time on both the initial application and the total review. This was despite the FDA making more requests for further data collection or analysis of the industry applicant than either the EMA or HC. Additionally, the majority of novel drugs included in this study were first approved in the US.
These data show that the FDA has come a long way since the 1980s, when it was accused of exacerbating the AIDS epidemic by not allowing patients early access to drugs still under investigation. In response, the agency developed programs to expedite the approval of new drugs in cases where no other treatments are available. These programs are still very much in use today – 60% of the novel drugs approved in 2015 and 73% in 2016 benefitted from expedited approval programs.
The FDA approval is only the final step in the lengthy, trial-and-error filled process of drug development. Researchers today design drugs to target specific pathways involved in diseases, so the less the cause of a disease is understood, the harder it is to target specifically. Finally, even when the mechanism of a disease is known and a drug is designed against it, the clinical trials needed to determine the drug’s safety and efficacy take years.
Funding for biomedical research leads to medical breakthroughs. The new FDA commissioner must understand that it is more important than ever to fund the scientific endeavors at universities, medical schools and research hospitals. The discoveries made at these institutions are essential for advancements in medicine.
The Approval of Generic Drugs is Backlogged
The FDA regulates the approval of not only novel drugs but also generic drugs (sometimes called copycat drugs). The generic drug approval process evaluates whether the drug is equivalent to its brand name counterpart in efficacy, quality, purity and stability. Because generic drugs are sold at lower costs than their brand name equivalents, increasing the number available would allow people access to treatments they might not otherwise be able to afford. When a treatment has no generic competition, the manufacturer may charge exorbitant prices, as in the recent case of EpiPen, the delivery system for a treatment for life-threatening allergic reactions. The price of a one-time use EpiPen was recently raised to $400, representing a 400% markup and sparking public outrage.
There is a longstanding backlog of thousands of generic drug applications awaiting FDA approval. This backlog has been reduced since the passing of measures to increase the funding for the FDA’s Office of Generic Drugs. However, while the time for initial review has shortened, the number of applications that await approval remains high. The FDA blames generic drug manufacturers for bogging down the process by submitting incomplete applications. The drug manufacturers in turn blame the FDA for too often changing its requirements.
Increasing the public’s access to affordable drugs is a worthy goal. To accomplish it, Trump and his FDA commissioner must look beyond cutting red tape. To make more generic drugs available, what the FDA needs is not less regulation, but continued funding to take on the generic drug approval backlog. And rather than changing the new drug approval process in what could be dangerous ways, the administration must continue to fund the basic biological research, the foundation of all medical breakthroughs.
Linda Honaker is a third year graduate student in the department of Molecular and Cellular Biology at Harvard University.