The FDA is now accepting applications for its Expedited IND Pilot, a program designed to shorten the time between scientific discovery and first-in-human testing. Rather than submitting a complete IND application and waiting for the agency’s standard 30-day review clock to run, sponsors paired with qualified research institutions will submit components on a rolling basis during the pre-IND phase so that any issues can be identified and resolved before a clinical hold is considered. Applications for the pilot close October 30.

The pilot is a component of Operation TrialBlazer, the HHS initiative launched in June to accelerate drug development across all trial stages. The competitive context is direct: first-in-human trials that currently take up to two years in the U.S. are completed substantially faster in China and Australia. Acting FDA Commissioner Kyle Diamantas framed the stakes plainly: “Too often, the U.S. pathway can be measured in years, while other countries’ pathways are measured in months.”

On the rare disease side, the FDA is soliciting outside input on how to make randomized trials more workable for patients and sponsors navigating small, difficult-to-enroll populations. In advance of a September 29 workshop, CDER Director Michael Davis has indicated that the approval playbook for ultra-rare genetic diseases is not one-size-fits-all, and that sponsors may have more flexibility on evidence standards than they currently assume.

Among the approaches under consideration: guaranteeing better-than-standard-of-care for patients in the control arm and offering more favorable efficacy analyses to boost orphan drug trial enrollment. The agency is also asking stakeholders to identify the greatest unmet needs in endpoint selection.

Both developments are playing out against a backdrop the agency has been working to address. The rise of Chinese biotech is increasingly felt across FDA divisions, and compressing U.S. development timelines has become explicit administration policy.

– Leslie Isenegger, Head of Policy, Public Affairs and Access, RC Resolve

The White House this afternoon announced that all 50 U.S. states, the District of Columbia and Puerto Rico have agreed to participate in a Most Favored Nation drug pricing scheme for all state Medicaid programs.

The model, titled “GENErating cost Reductions fOr U.S. Medicaid (GENEROUS),” will see at least 26 drugmakers provide discounted pricing on certain medicines to state Medicaid programs. The program is voluntary both for states and manufacturers who have agreed to provide discounts.

GENEROUS is projected to generate $65 billion in Medicaid drug savings over the next decade, according to the Trump administration. However, independent analyses have raised concerns that the program will not actually yield significant savings, due to the steep drug discounts Medicaid already receives.

President Trump, flanked by HHS Secretary Kennedy and CMS Administrator Oz, promoted the model in an Oval Office event with the governors of Arkansas, Mississippi and South Dakota. Semafor was the first to scoop today’s announcement.

As the Trump administration continues to promote lower drug prices ahead of the 2026 midterm elections, we expect Congressional Democrats and watchdog groups like Public Citizen—which filed a FOIA lawsuit to access confidential MFN agreements—to continue their push to view more detailed accounting of the proposed savings.

– Leslie Isenegger and Andrew Wishon, Real Chemistry

A new modeling study published in The Lancet raises significant questions about whether the Trump administration’s MFN drug pricing strategy will deliver meaningful savings.

The administration’s MFN approach was intended to bring U.S. drug prices closer to those paid by 19 other wealthy countries. Under the proposed GLOBE and GUARD models, manufacturers would pay additional rebates when Medicare prices exceed the lowest international benchmark. The White House has said the effort could save Medicare $26 billion over several years.

However, the new analysis found that negotiated deals with more than two dozen pharma companies could reduce projected Medicare savings by as much as 80%. Without the exemptions secured by these deals, the study estimated savings of $5.2 billion through GLOBE and $6.4 billion through GUARD. But because the administration has not released the agreements, implementation could be “totally confounded.”

The research also warns that manufacturers may respond by increasing prices in benchmark countries or delaying international launches, potentially exporting America’s pricing pressures rather than lowering costs at home. European officials are examining how to mitigate potential impacts on their healthcare systems. Some countries are exploring closer coordination on pricing and procurement strategies, and the European Commission has said it is too early to assess the full impact of U.S. reference pricing policies.

– Megan Hickey, Managing Director

The Senate was busy this week, holding hearings to vet several nominees to fill long-standing vacancies at HHS.

Chris Klomp, who made a name for himself spearheading the Trump administration’s MFN negotiations over the past year, bore the brunt of questioning across two Senate committee hearings as he sought to advance his nomination as HHS deputy secretary.

The most contentious moments came when Sen. Elizabeth Warren, D-Mass., sparred with Klomp on sharing the details of those MFN pricing deals with industry—a request that the Trump administration hasn’t answered for months due to “proprietary information and trade secrets.”

However, amid the growing measles outbreaks across the U.S., vaccine policy became the most prominent line of questioning facing nominees.

Both Klomp and Surgeon General nominee Dr. Nicole Saphier offered largely vaccine-supportive testimony when grilled by senators on both sides of the aisle. Both nominees denied a link between vaccines and autism and reiterated their belief in the safety and efficacy of the MMR vaccine.

Still, both Klomp and Saphier stopped short of directly criticizing President Trump’s Executive Order on splitting MMR vaccines, or recent changes to federal vaccine policy by HHS Secretary Robert F. Kennedy Jr. Klomp even went so far as to claim the EO was President Trump’s effort to reach out to vaccine-hesitant parents—an answer most Senators didn’t seem to buy.

As American public health challenges continue to mount and the midterm elections grow closer, the administration is eager to fill as many HHS vacancies as possible before the Senate adjourns next week. Saphier and Klomp seem to be on their way to confirmation, but what actions they’ll take once in power are less clear.

– Rachel Bridges, Senior Director

• Sept. 23 – conversations on “how the U.S. can maintain its global leadership in biotechnology,” Axios
• Sept. 24 – votes to advance three HHS nominees (incl. Nicole Saphier as surgeon general), Senate HELP
• Sept. 24 – hearing on the nomination of Dr. Heidi Overton to be FDA commissioner, Senate HELP

• “We take vaccines really, really seriously. As we should. It’s a matter of life and death. Somewhere along the way, though, we forgot how to celebrate. We are sorely lacking in optimism and excitement” – Adam Zimmerman, STAT
• “Children with rare diseases and patients affected by neglected diseases deserve more than a tradable regulatory asset with a poor track record of promoting innovation or securing access” – Melissa Barber et al., Health Affairs Forefront
• “Patients should not have to wait nearly two years for access to lifesaving drugs. And yet, all too often, they do — because European health systems use these delays as a pressure tactic to compel biotech companies to offer steep price discounts” – Nicole Sheahan, Washington Examiner

• HHS Secretary Kennedy named eight new members to the U.S. Preventive Services Task Force, drawing concern over the departure from primary case-focused membership. Reuters: US Health Secretary Kennedy Jr. adds eight new members to key health committee.
• Kennedy also told members of an anti-vaccine group he founded that they had a “friend at the White House,” signaling a renewed embrace of vaccine skepticism. The New York Times: Kennedy Assures Vaccine Skeptics That They Have a ‘Friend at the White House.’
• With pharma tariffs of up to 100% set to take effect on Sept. 29, federal guidance on exemptions has yet to be published—leaving biotech companies with little time to navigate the process. BioCentury: Twelve days to go and pharma tariff rules are a total mystery.
• The FDA approved Ultragenyx’s Fayuvi as the first treatment for Sanfilippo syndrome type A, a rare neurodegenerative disorder in children. Fayuvi will launch at a list price of $3.95 million. Fierce Pharma: FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder.
• The FDA’s push to hire more than 2,000 workers is mired in bureaucratic delays caused by DOGE-era centralization of HR offices, leaving hundreds of prospective employees in pre-onboarding limbo. STAT: The FDA’s rebuilding effort is running into a new challenge.
• Six UK parliamentary committee chairs have called for an investigation into the cost of the UK-U.S. drug pricing agreement struck last year. Politico: British lawmakers demand probe into cost of Trump NHS deal.
• Major pharma CEOs expressed confidence in the near-term U.S. policy environment, citing PBM and 340B reform as potential industry tailwinds. Pink Sheet: Big Pharma CEOs Bullish On US Policy Environment Heading Into Midterm Elections.
• Novo Nordisk rebranded as Novo for day-to-day operations, betting a shorter, more consumer-friendly name will help it resonate with consumers. The Wall Street Journal: Novo Nordisk Slims Down Name to Novo.