Makary: FDA to ease trial requirements for approvals

Food and Drug Administration Commissioner Marty Makary tells STAT exclusively that the agency will shift its long-standing default for the number of clinical trials required for drug or device approvals from two to just one.

He argued the agency was being more practical, and that no standards would be lowered. In practice, requiring one trial and confirmatory evidence has become relatively commonplace.

But internal reviewers and outside experts expressed concern. The change contributed to CDER chief Richard Pazdur's abrupt decision to retire, according to agency sources who said scientific staff weren't consulted, STAT's Lizzy Lawrence reports.

"We are moving in a deregulatory direction and each day, continually think about ways that we can reduce red tape, reduce uncertainty, and reduce onerous requirements," Makary said.

personnel

FDA gets a new CDER chief, and ousts OTC regulator

Tracy Beth Høeg, a top lieutenant to Food and Drug Administration Commissioner Marty Makary, will be the next leader of the Center for Drug Evaluation and Research, according to an announcement from the FDA.

Høeg's appointment as the agency's top drug regulator comes after CDER Director Richard Pazdur filed paperwork to retire at the end of the month. Pazdur accepted the role less than a month ago at the urging of Makary. Høeg will be the fifth person to lead the center this year.

Read more here.

Separately, the FDA has abruptly removed Theresa Michele, the longtime and widely respected head of its Office of Nonprescription Drugs, STAT's Lizzy Lawrence reports exclusively. She told staff she is being transferred to the devices center.

Commissioner Marty Makary has been pressuring CDER leadership to replace career staff and accelerate prescription-to-OTC switches — a policy he believes will boost affordability. Companies, however, often resist such moves because they're more profitable on the prescription side.

alzheimer's

New data confirm Novo drug did not slow Alzheimer's progression

From my colleague Elaine Chen: Alzheimer's patients on Novo Nordisk's obesity drug semaglutide experienced virtually no difference in cognitive and functional decline than patients on placebo in two Phase 3 trials, according to new data presented last night at the Clinical Trials on Alzheimer's Disease conference.

Novo last week announced that the trials failed. The slides from yesterday's presentation show that the curves for treatment and placebo groups overlapped on measures including the Clinical Dementia Rating-Sum of Boxes and the Alzheimer's Disease Cooperative Study - Activities of Daily Living.

These trials were long seen as long shot studies, but Novo decided to run them after seeing retrospective data showing that patients taking GLP-1 drugs had a lower risk of getting a dementia diagnosis.

Observers have wondered whether the studies would have worked if Novo focused on specific subgroups, such as patients with diabetes or obesity. In the trials, semaglutide didn't have an effect in people who specifically had type 2 diabetes, though researchers are continuing to run sub-group analyses, said Peter Johannsen, international medical vice president at Novo.

It's not clear whether Novo will continue to study semaglutide or other obesity drugs in Alzheimer's. "What the company in the end decides, I don't know," Johannsen said.

clinical trials

Paradigm expands AI trial network with Roche deal

Paradigm Health has raised another $77 million, and with it has acquired a clinical-trial matching business from Roche's Flatiron Health. It's betting that artificial intelligence can make drug studies faster, cheaper, and more equitable.

Backed by ARCH and General Catalyst, Paradigm aims to use AI-driven tools and Flatiron's oncology network to identify eligible patients in community and rural settings. The hope, they say, is to address the long-standing barriers that keep many out of lifesaving studies.

"I realized that fundamentally this is a health equity issue," Ken Frazier, the former CEO of Merck who now holds a strategic role at General Catalyst, told STAT's Matthew Herper.

hair loss

Cosmo's hair growth drug succeeds in trial, but prompts some skepticism

Cosmo Pharmaceuticals says its topical androgenetic alopecia drug clascoterone spurred striking hair growth in two Phase 3 trials — reporting 168% and 539% greater target-area hair counts compared to placebo after six months — sending its share price to a decade high and setting up regulatory filings next year.

But dermatologists questioned the large variability and the company's choice to release only percentage differences rather than raw hair counts, STAT's Allison DeAngelis writes — calling the data presentation more suitable for investors than clinicians.

Cosmo defended the approach and plans to present full results at the upcoming American Academy of Dermatology meeting in March.

Read more.

VACCINES

Former FDA chiefs warn of vaccine overhaul

Twelve former FDA commissioners issued an extraordinary joint warning in the New England Journal of Medicine yesterday, arguing that sweeping vaccine-regulation changes proposed by Vinay Prasad — the agency's current biologics chief — threaten the safety, availability, and scientific integrity of U.S. vaccines, STAT's Matthew Herper writes.

The group, spanning Republican and Democratic administrations, said an internal memo written by Prasad that was leaked recently — and that citied 10 child deaths allegedly tied to Covid vaccines — relied on VAERS reports likely misinterpreted and offered no justification for "wholesale rewriting" of vaccine policy.

The former commissioners also criticized FDA leadership for sidelining outside advisory committees.

"Americans' safety depends on a culture in which evidence is reviewed openly and staff can surface concerns, challenge leadership, and engage with external scientists without fear of reprisal," they wrote.

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