Since President Donald J. Trump was sworn into office, there has been much concern over the impact that his administration may have on the FDA. In fact, many predicted that President Trump’s victory would be a “game changer” for the FDA.
In a May 2017 webinar titled “The Trump Administration and the FDA’s New Commissioner,” Norma Skolnik, Director, Regulatory Affairs, Cadbury Adams, Parsippany, NJ, discussed the new appointment, on May 9, 2017, of Scott Gottlieb, MD, as FDA Commissioner, as well as other potential changes to the FDA that can be expected under the Trump administration, such as the impact of a federal hiring freeze on the FDA, an overhaul of the FDA, and slashing of FDA regulations.
According to Ms Skolnik, the appointment of Dr Gottlieb as the new FDA Commissioner may provide some semblance of stability to the FDA amidst potentially radical changes.
Dr Gottlieb Offers Hope
Dr Gottlieb is former FDA Deputy Commissioner, a physician, Clinical Professor at New York University School of Medicine, former resident fellow at the American Enterprise Institute, and has strong connections to the pharmaceutical industry as a former consultant to many pharmaceutical companies.
“Scott Gottlieb’s nomination conforms to the tradition of having a trained physician in the commissioner’s office,” said Ms Skolnik. “He would also be coming in as a reformer with an understanding of the workings of the FDA, able to swiftly execute a series of changes that could meet Trump’s stated goal of accelerating approvals and lowering some drug prices,” she added.
Dr Gottlieb similarly contends that the FDA is overly prudent when it comes to approving new drugs and medical devices, and he has criticized the FDA’s efforts to punish companies for off-label promotion, said Ms Skolnik.
At his confirmation hearing, Dr Gottlieb expressed his support for the FDA drug approval review as being the gold standard, but he also noted that it was important to expedite the drug review process in certain disease states, or even weaken randomized protocols in some cases. In addition, Dr Gottlieb underlined that the FDA maintains regulatory barriers that may keep drug prices high, said Ms Skolnik.
Nevertheless, Dr Gottlieb vowed “to be an absolutely objective regulatory watchdog and wouldn’t do anything that could besmirch the agency or undermine public confidence in the FDA’s work,” noted Ms Skolnik. He also said he would recuse himself if necessary in matters related to companies for which he served as a consultant in the past.
Dr Gottlieb will be tasked with implementing the 21st Century Cures Act, which was signed by President Obama to help speed the development and approval of drugs and medical devices; in his recent congressional hearing he pointed out that he was committed to executing this act to the depth of his ability. In addition, he seeks to address the nation’s opioid epidemic and make that a top priority, according to Ms Skolnik.
Federal Hiring Freeze and the FDA
On January 23, 2017, President Trump signed executive orders calling for a federal hiring freeze in an effort to cut federal agency spending by billions of dollars, said Ms Skolnik. Fortunately, the hiring freeze was lifted in April 2017, “but is being replaced with a narrower halt on new government employment,” she reported.
For the FDA, the hiring freeze was concerning for several reasons. First, there was apprehension that it would weaken health and safety protections.
In addition, drugs cannot be approved in a timely manner if there is inadequate staff, said Ms Skolnik. During the drug development process, FDA staff provides valuable feedback to pharmaceutical companies regarding what studies are needed for drug approval; this information helps speed up the drug development process, but cannot be accomplished if there is insufficient staff.
Even more concerning was that the hiring freeze would delay the implementation of the 21st Century Cures Act by taking into consideration real-world evidence and biomarkers, in addition to evidence from clinical trials.
Budget Cuts and the Future of Healthcare Research
In addition to the hiring freeze, President Trump proposed significant budget cuts for several federal agencies, including an 18% budget cut for the US Department of Health & Human Services (HHS) and a 19% budget cut for National Institutes of Health (NIH) research programs, reported Ms Skolnik. Researchers and the medical community reacted strongly to these budget cuts, voicing that reductions in research funding could be “devastating,” said Ms Skolnik. But the Trump administration asserted that by eliminating unnecessary overheads, it could cut the budget while funding research. Fortunately, Congress chose to increase the budget for the NIH by $2 billion.
Similarly, HHS Secretary Thomas E. Price, MD, also expressed his unwillingness to fund new medical research unless funding can be slashed in other areas, said Ms Skolnik. Every year, HHS awards billions of dollars in research grants to scientists through the NIH, FDA, and the Centers for Disease Control and Prevention, and agency officials decide how these grants are used. Sentiments by President Trump and Dr Price about research funding could have far-reaching consequences for the future of research. For example, the Trump administration may only choose to support funding for research that does not include fetal tissue or embryonic stem cells.
In addition, research is crucial for understanding a drug’s complete efficacy and safety profiles. Lack of funding for research can potentially compromise patients’ and physicians’ knowledge of what does and does not work.
Overhauling the FDA
Since President Trump took office, he has vowed to overhaul the FDA and to slash FDA regulations in an effort to streamline and expedite the approval of drugs and medical devices. He views the FDA drug approval process as lengthy, cumbersome, and keeping life-saving drugs from reaching patients who need them.
During a meeting with pharmaceutical executives in January 2017, President Trump said, “We are going to be cutting regulations at a level that nobody’s ever seen before.”
Using the Congressional Review Act, which can overrule a regulation by a simple majority vote, Republican lawmakers have sought to cut dozens of regulations, with 37 separate resolutions since President Trump took office—more than any Congress has previously attempted, noted Ms Skolnik.
“Now if such proposals are adopted, many would require an act of Congress. And this would reverse decades of FDA policy and consumer protections dating back to the early 1960s,” she said.
President Trump also issued an order requiring that 2 regulations are cut each time a new regulation is introduced. However, a lawsuit was filed under the basis that this executive order surpasses President Trump’s authority to repeal regulations that help protect the health and safety of the American people, said Ms Skolnik.
FDA regulations are necessary to safeguard people from dangerous drugs and misleading claims, and that has been the standard since 1962. “But it’s possible that Trump may be interested in changing back the regulation to focus just on safety versus efficacy,” suggested Ms Skolnik.
Nevertheless, controlled clinical trials are important for thoroughly assessing the safety of drugs, including their metabolism, interactions with other drugs, and potential differences in their safety and effectiveness between sexes and among ages and races, she said.
Therefore, the pharmaceutical industry is not especially excited about slashing regulations to accelerate the drug approval process if there is insufficient evidence to prove the drug’s safety and efficacy.
“A first-to-market advantage does not mean much if there is not enough of a market to capitalize on. And that is what has raised concerns among several prominent drug company executives who feared that insurance companies would be loath to cover an unproven treatment,” Ms Skolnik pointed out.
This was the case with Sarepta Therapeutics, which won a controversial FDA approval for its drug eteplirsen (Exondys 51) for the treatment of patients with Duchenne muscular dystrophy, a rare, life-threatening, genetic degenerative muscle disease. Unfortunately, the drug is not being covered by several insurance companies, because there are insufficient data to prove its efficacy.
When it comes to patients having access to potentially life-saving agents, compassionate use is an often overlooked pathway that allows patients to obtain drugs that have not yet been approved by the FDA. The FDA does not stand in patients’ way if they choose to receive these therapies, said Ms Skolnik. In 2015, the FDA released a draft guidance to streamline the compassionate use program application process, reducing the time physicians need to complete an application from approximately 100 hours to 45 minutes.
People may question whether the US drug approval process is inherently slow. Ms Skolnik cited a December 2016 report from the FDA, which showed that the United States surpassed Japan, Canada, and the European Union in how quickly drugs are approved. In fact, the FDA is making strides to accelerate the drug review process. In 2009, it took at least 13 months to hear back from the FDA regarding a drug application; in 2015, this process was reduced to 10 months.