This Week at FDA: RFK Jr. nominated to HHS Secretary, FDA wants feedback on RWE and patient-focused drug development

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug, and medical device regulation, and what we’re reading from around the web. This week, President-elect Donald Trump wasted no time in naming his picks for crucial government leadership posts – including tapping Robert Kennedy Jr. to head the Department of Health and Human Services (HHS). In other top news, FDA will hold meetings to get feedback on using real-world evidence (RWE) and patient-focused drug development.

The selection of Kennedy surprised many. The former independent presidential candidate has taken positions that concern many in the scientific community, such as his stance that vaccines cause autism and that school shootings are linked to the increased use of antidepressants. He also advocates for addressing chronic diseases and changes to nutrition regulations.

As Axios pointed out, Kennedy has argued that FDA has aggressively suppressed the marketing of "psychedelics, peptides, stem cells, raw milk, hyperbaric therapies, chelating compounds, ivermectin, hydroxychloroquine, vitamins, clean foods, sunshine, exercise, nutraceuticals and anything else that advances human health and can't be patented by Pharma." While Kennedy rejects the notion that he is anti-vaccine, the Associated Press reports that as chair of the Children's Health Defense, he stated that there are no safe and effective vaccines. The news agency also cited a 2021 study in the Journal of Medical Internet Research (JMIR) that concluded that his Twitter account was the top “superspreader” of vaccine misinformation during the COVID-19 pandemic.

In a post-election interview with MSNBC News interview, Kennedy said that “entire departments” at FDA “have to go” and singled out FDA’s “nutrition department” for not protecting children. During the interview, he said he would “clean up corruption” and return health agencies to evidence-based science.

Earlier in the week, FDA Commissioner Robert Califf declined to comment on Kennedy's statements, stating it would not be productive. However, he said he was disappointed in the election results and stressed that FDA decisions should be based on science, not politics. Reporting from the same event organized by the Friends of Cancer Research (FOCR), STAT News noted that Califf raised concerns about the agency's ability to hire and retain skilled staff if Kennedy oversees FDA considering his hostility toward civil servants.

Kennedy has long railed against the pharmaceutical industry for high drug prices and for putting drugs on the market that he believes hurt the public. Reuters reported that pharmaceutical stock prices, especially for vaccine-makers, dropped significantly after Kennedy’s nomination.

The largest drug lobby group, PhRMA, issued a conciliatory statement and said that it wants “to work with the Trump administration to further strengthen our innovation ecosystem and improve health care for patients.” The group noted that it supports addressing the “growing epidemic of chronic disease, which is the biggest driver of health care spending,” and called for a comprehensive strategy that focuses on prevention and early intervention.

Several key lawmakers issued statements, including Sen. Patty Murray (D-WA) chair of the Appropriations Committee and member of the Health, Education, Labor, and Pensions (HELP) Committee, who condemned Kennedy’s nomination.

“Donald Trump’s selection of a notorious anti-vaxxer to lead HHS could not be more dangerous—this is cause for deep concern for every American,” said Murray. “There is no telling how far a fringe conspiracy theorist like RFK Jr. could set back America in terms of public health, reproductive rights, research and innovation, and so much else.

“The consequences here are not theoretical or superficial—health care access, coverage, research, and public health are life or death issues for people—and the COVID pandemic was an all too recent, all too painful reminder,” she added. “Confirming RFK Jr. as HHS Secretary would be nothing short of a disaster for the health of millions of families.”

On the other side of the aisle, Sen. Bill Cassidy (R-LA), slated to take over as HELP chair, said that he looks forward to learning more about Kennedy’s policy positions.

“[Kennedy] has championed issues like healthy foods and the need for greater transparency in our public health infrastructure,” he noted.

Rep. Frank Pallone Jr. (D-NJ), ranking member on the House Energy and Commerce Committee, said he has deep concerns about Kennedy's lack of experience and repeated promotion of conspiracy theories.

"Congressional Republicans regularly speak about the importance of restoring public trust in our nation's health agencies," he said. "Confirming a known conspiracy theorist is not the way to restore that trust."

Drugs & Biologics

The Duke-Margolis Institute for Health Policy and FDA will host a hybrid public workshop on 12 December on optimizing real-world evidence (RWE) in regulatory decision-making for drugs and biologics. The agency said it would update stakeholders on its efforts, accomplishments, and challenges, and interested parties are encouraged to share comments with regulators by 13 January.

On 13 December, FDA plans to host a virtual public workshop to discuss methodological and other challenges in patient-focused drug development. The meeting is part of the agency's Prescription Drug User Fee Amendment (PDUFA VII) commitment to advance patient-focused drug development.

In biologics news, PTC Therapeutics' adeno-associated virus vector-based gene therapy Kebilidi (eladocagene exuparvovec-tneq) has been approved by FDA as the first such treatment for aromatic L-amino acid decarboxylase (AADC) deficiency. ClearPoint Neuro's infusion tube SmartFlow Neuro Cannula was also authorized and is currently the only approved delivery system for the gene therapy drug.

The Center for Biologics Evaluation and Research (CBER) published lists of biological devices and supplemental biologics license applications (sBLA) that have been given marketing authorization this year. They include IT Synergetics' bexWISE API for use in blood establishments, which allows communication between 510(k) cleared Blood Establishment Computer Systems (BECS) and Pfizer's Abrysvo (respiratory syncytial virus (RSV) vaccine), which was given an expanded indication to prevent lower respiratory tract disease (LRTD) in patients between 18 and 59 years.

Medtech

Between April and August, FDA says it has received 18 complaints about certain Getinge/Maquet's VasoView HemoPro Endoscopic Vessel Harvesting (EVH) Systems where silicone has detached from the jaws of the harvesting tool, making them unusable and leading to silicone debris entering patients. The agency sent a letter to healthcare providers advising them to use alternative EVHs if possible while also updating its recall notice for the products. It also noted that it had added the products to its medical device shortage list.

Alcon Research's UNIPURE SF6 Ophthalmic Gas for use in the UNIFEYE Gas Delivery System and UNIPEXY Gas Delivery System was approved by FDA to treat uncomplicated retinal detachment. The agency noted that clinical literature review of similar SF6 gas found that the overall primary retinal reattachment rate reported 3 months after injection was more than 87%.

W.L. Gore's Gore Excluder Conformable AAA Endoprosthesis was approved by FDA to treat weakened and bulging section (aneurysm) of the infrarenal abdominal aorta. The agency said a clinical study of 175 patients found that almost 90% of participants were successfully treated without device or aneurysm-related complications up to one year after the procedure, and almost none of the patients reported a safety event.

This Week at FDA: RFK Jr. nominated to HHS Secretary, FDA wants feedback on RWE and patient-focused drug development

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