US FDA's accelerated approval for drugs needs more transparency, says research firm

Reuters

Drug pricing watchdog calls for increased transparency into FDA’s accelerated approval decisions

BioSpace

CDC Head Pulls Study From MMWR, Refueling Vaccine Policy Debate

Inside Health Policy

The Centers for Disease Control and Prevention’s acting leader reportedly held back a study showing Covid-19 vaccines were effective in avoiding hospitalization during the most recent respiratory virus season because of concerns over the study’s observational methodology -- marking the latest move by HHS to advocate a stringent approach to vaccine data standards despite a more lenient approach on other regulated products. The news of the study came after ICER published a Draft Evidence Report on vaccines for Covid-19. ICER’s report will be open to public comment until May 4th.

Inside Health Policy spoke with ICER’s David Rind, MD, MSc about the CDC’s concerns:

“David Rind, ICER’s chief medical officer, told Inside Health Policy that while the test-negative methodology isn’t an ideal method of measuring overall vaccine effectiveness, it can be a good measure of whether a vaccine is effective in preventing severe disease, which is what the CDC study sought to assess. ICER’s analysis found the evidence that Covid-19 vaccines are effective in preventing severe disease was strongest for adults 65 and older, children ages 6-12 months and pregnant women. The evidence was less definitive for other groups, particularly boys and men ages 12-24, and there wasn’t enough evidence to say whether any of the four available vaccines were superior.

Rind said the best study available to ICER was a study using electronic health records from the Department of Veterans Affairs, which directly compared patients who were vaccinated against both flu and COVID-19 and patients who were only vaccinated against flu.”

Should People Who Want GLP-1s Have to Work for Them?

Tradeoffs (podcast)

This CEO wants to cover weight loss drugs for employees. They're just too expensive.

NBC News

The Tradeoffs podcast and NBC News reported on employer struggles with covering weight loss medications like Wegovy and Zepbound.

Both pieces mention one or both of ICER's recent obesity management reports, including 1) a white paper on affordable access to GLP-1s and 2) last year's Final Evidence Report assessing the comparative clinical and cost effectiveness of semaglutide (branded Wegovy) and tirzepatide (branded Zepbound).

MUST READS

CMS proposes easing prior authorizations for prescription drugs

Modern Healthcare

Health insurance companies and states would have to resolve prior authorization requests for drugs more quickly and publicly disclose their denial rates under a proposed rule the Centers for Medicare and Medicaid Services published Friday.

The regulation would require Medicaid and Children’s Health Insurance Program insurers, along with state Medicaid and CHIP administrators, to respond to non-urgent prior authorization requests for prescription drugs within 24 hours after receiving a request. Insurers operating on the Affordable Care Act exchanges, selling policies on Healthcare.gov, would need to respond to standard requests for drugs within 72 hours and urgent items within 24 hours.

FDA pushes drugmakers to report missing clinical trial results

STAT

In a bid toward greater transparency, the Food and Drug Administration sent reminder letters to more than 2,200 companies and researchers that they are required to report clinical trial results to a federal government database or they may face fines.

FDA officials disclosed that an internal analysis found results were not submitted for nearly 30% of studies that were “highly likely” to fall under mandatory reporting requirements. The agency also noted that the letters were sent to companies and researchers associated with more than 3,000 registered trials, some of which were publicly funded.

White House policies are ‘incredibly worrisome’ for small biotechs, Rhythm CEO says 

Endpoints News

Biotechs with single products in the market are likely on edge about how President Donald Trump’s pharma tariffs and “most favored nation” pricing will play out, Rhythm Pharmaceuticals CEO David Meeker told Endpoints News in an interview.

The Boston-based rare disease biotech markets Imcivree for weight management in patients with certain rare genetic disorders. Meeker said similar companies with one commercial product may also have concerns that if they launched their drugs overseas first, that typically lower price would also become the list price in the US under potential MFN pricing negotiations.

Maryland state affordability board sets its first price cap for a medicine   

STAT

Seeking to cap the cost of medicines, the Maryland Prescription Drug Affordability Board set an upper payment limit for a widely prescribed diabetes treatment, marking only the second time a state panel has taken such a step.

The board, which is designed to function like a state utility commission, will now oversee a process to lower the cost of the type 2 diabetes medicine Jardiance for the state and local governments by January 2027. At that time, the price will be capped at $204, or $6.80 a pill, for a 30-day supply. The move is estimated to save $320,000 a year.

OTHER HEADLINES

• NBC News: Drugmakers raised prices on hundreds of drugs despite Trump deals, Senate Democrats report finds
• Axios: HHS brings on an affordability czar
• Axios: States' efforts to rein in PBMs hit a legal roadblock
• Inside Health Policy: Cassidy Touts Vague Affordability Plan To Expand TrumpRx, Rein In PBMs & Push Price Transparency
• Fierce Healthcare: Bill would force payers to apply DTC drug purchases to patient deductibles
• The Wall Street Journal: Around 14% of Enrollees in ACA Plans Failed to Make Payments, Data Shows

Key Upcoming Dates

• 4/29: Narcolepsy -- revised Evidence Report
• 6/8: Narcolepsy -- Final Evidence Report

Learn more about ICER’s ongoing and recently completed reports:

• Covid-19
• Hypertension
• IgA Nephropathy
• Narcolepsy
• Parkinson's Disease

Email info@icer.org for more information.

When you make a gift to ICER, you help keep ICER independent.