Biotech leaders urge Senate to restore FDA's key functions, cite funding concerns for small companies

Original from: FIERCE BIOTECH

More than 200 biotech leaders have penned a letter voicing concerns over the FDA’s capacity after mass federal layoffs, specifically for small biotechs that depend on investor backing to meet agency standards.

Certain biotechs have already seen the effects of the FDA restructure, according to the authors, who urge the government to “quickly preserve and restore its core functions.”    

The letter, addressed to Senate Health, Education, Labor and Pensions (HELP) Committee Chair Bill Cassidy, M.D., R-Illinois, is written and supported by 207 biotech innovators, including industry heavyweights such as founding Alnylam CEO John Maraganore, Ph.D., and Nkarta Therapeutics’ CEO Paul Hastings. 

The letter, which was published last Friday, also includes signatures from patient advocates and investors like RA Capital, Blackstone Lifesciences, Sofinnova Investments and The Column Group, among others.

“While we share a desire to strengthen and modernize the FDA, we are writing to share that we are deeply concerned about the current state of the agency and its future,” the letter begins, referring to recent federal restructuring that has gutted health agencies of sites, staffers and leaders.

“Specifically, we worry that the institutional knowledge that makes the FDA the world’s leading regulatory body will be irretrievably lost due to the agency’s recent reduction in force and wave of retirements,” the leaders write. “The agency’s ability to function is compounded by a hiring freeze. As a result, American patients, American industry and American biomedical leadership will bear the consequences.”

“While we share a desire to strengthen and modernize the FDA, we are writing to share that we are deeply concerned about the current state of the agency and its future."

In late March, Department of Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. announced 10,000 layoffs across the federal health department, including 3,500 full-time FDA employees. The cuts are on top of 10,000 early retirements or “fork in the road” exits.

A few days later, RFK Jr. walked back 20% of the newly implemented cuts, saying 2,000 of the layoffs were done in error.

While the HHS said the FDA workforce reduction wouldn’t impact drug, medical device or food reviewers, the biotech leaders say it already has.

The authors explained that while problems stemming from the loss of federal staffers could take months or even years to emerge, depending on when a biotech must interact with the FDA, some companies have already run into problems. 

For example, an FDA dispute resolution process with a Massachusetts biotech was canceled, with an agency counterpart saying there may not be any senior staff to review the case.

Or take the California biotech that is now working with the European Medicines Agency “to enroll patients in geographies outside the U.S." and seeking pacts with foreign CROs to keep its existing development timelines. These decisions are “effectively ‘offshoring’ capital and investment dollars away from the U.S.," the letter authors wrote.

Another example spelled out in the letter is from a biotech that received submission feedback conducted by a seemingly inexperienced reviewer, including suggestions inconsistent with medical practice or standard approaches to trials, plus requests for information that was already included in the company's submission.

These scenarios have prompted the biotech stakeholders to urge the Senate HELP committee to restore FDA staffing levels in an effort to maintain established regulatory timelines and consistency.

The letter coincides with recent commentary from Janet Woodcock, M.D., former acting FDA commissioner and former principal deputy commissioner of food and drugs, who warned that the FDA changes were “a slow-moving catastrophe.”

“You’re not going to see everything come to a stop in the actual review of documents and development programs,” Woodcock said during an April 7 session of the Biopharma Congress. “But the whole apparatus to get a drug on the market, a lot of that is missing, and you're going to see mistakes.”

When explaining FDA reviews, Woodcock detailed routine disputes between different disciplines at the agency, noting that varying opinions arise “because there's a lot of uncertainty in development of medical products.”

Supervisors and leaders generally help quell these disagreements, hearing out all sides and determining the best course of action.

“So, I would expect that you'll see that you'll see mysterious slowdowns and you won't know what it's about, but it's about the agency doesn't have any more smooth mechanism to sort out all these disputes,” Woodcock said.

The letter to Cassidy underscores this sentiment, calling for the preservation of the FDA's “ability to make ‘judgment calls.’” This means applying regulations in a consistent and thoughtful manner, especially when considering accelerated approval pathways, product development incentives and new modalities, according to the authors.

“Critical gaps in leadership and management at the review division level have typically been filled by senior FDA leadership,” the authors write. “But with these individuals now gone, companies fear we are entering a period of years to build back up an experienced workforce.”

One biotech has already received conflicting feedback from a review team, a situation in which a senior official should have determined the agency’s position, according to the letter.

The Senate should also protect the FDA’s ability to continue collecting user fees, which help fund the agency and drug reviews, the authors wrote. 

Certain unnamed biotech leaders went so far as to request certain employee reinstatements, including Chris Joneckis, Ph.D., and Betsy Valenti, who helped maintain the user fee program.

The authors also urged for the reinstatement of Yaeming Chae and Isaac Dorfman, who worked on capacity planning, plus Andy Kish, Patrick Zhou, Emily Ewing and Josh Barton, who'd served at the Center for Drug Evaluation and Research.

Furthermore, the writers requested that the government unfreeze hiring for roles deemed critical, such as head of human tissue, head of clinical evaluations and head of cell and gene therapy.

How the FDA cuts could impact biotech fundraising

The biotech leaders underscored the importance of a well-staffed FDA specifically for small, clinical-stage biotechs. The companies are generally tight on resources and depend on investor funding to meet FDA requirements, the authors explained.

“Any delays in FDA reviews will substantially impact these companies’ ability to secure the funding they need to continue to advance to the next stage of development; the extreme turmoil in the biotech financial markets reflects that investors are worried about our ability to execute,” the leaders wrote.

For the past few years, the industry has been plagued by a bear market, with hopes initially pinned on 2025 for a return to normal. But geopolitical uncertainty and federal cuts to healthcare and research funding are hitting biotech, with companies forced to batten down the hatches even more than usual.

The current ambiguity regarding the FDA's capabilities may trigger downstream uncertainty from investors questioning whether the agency is able to do its job, the biopharma stakeholders wrote.

“Any delays in FDA reviews will substantially impact these companies’ ability to secure the funding they need to continue to advance to the next stage of development; the extreme turmoil in the biotech financial markets reflects that investors are worried about our ability to execute." 

The authors further cite efforts from the newly created Department of Government Efficiency—which has canceled leases for numerous health agency sites—and RFK Jr.’s previous claim that he would help establish “gold-standard science.” 

“We recognize and support that America must strive for government efficiency, and we believe we share with the administration a vision of a gold-standard regulatory agency that efficiently shepherds safe and effective products to the market,” the authors write.

That being said, the writers urge the administration to be cautious when “trying to centralize all functions and over-systematize a regulatory process that has to be flexible in the face of new scientific discoveries.”

“In fact, it's often the creativity and good judgment of senior people that has allowed reviewers to set aside formal guidance that, if imposed strictly, would stymie progress,” the biotech leaders conclude. “By maintaining deep agency expertise today, future reductions in force can be guided by those with the experience and vision necessary to build an efficient FDA that we and all Americans can count on.”

The letter includes a spot for other industry players to sign in support of the message.  

Source: Biotech leaders urge Senate to restore FDA's key functions, cite funding concerns for small companies