The FDA’s most polarizing regulator is heading for the exit. Vinay Prasad, director of the Center for Biologics Evaluation and Research, will leave the agency at the end of April after a turbulent year regulating vaccines, cell and gene therapies and more. His tenure has been defined by unusually public clashes with industry and a tendency to intervene directly in regulatory decisions.

For biotech investors, the reaction was swift and largely positive. Shares of several rare disease and gene therapy developers rose following the announcement, reflecting hopes that a new CBER chief could restore a more predictable regulatory environment.

This news comes amid a backdrop of mounting scrutiny of the FDA’s regulatory decisions. Over the past year, the agency has faced criticism after denying or discouraging multiple experimental drug applications – especially in the rare disease space – while in some cases reversing earlier guidance to companies.

Those tensions are now spilling into Washington. Sen. Ron Johnson, R-Wis., has launched an investigation into recent FDA rejections of rare disease therapies and is seeking internal documentation from the agency.

Prasad’s departure may remove a lightning rod, but it does not necessarily resolve the underlying uncertainty at the FDA. Leadership turnover has accelerated, and political pressure surrounding vaccines and rare disease approvals remains intense. Much will depend on who takes the helm at CBER and whether the agency can reestablish clearer, more consistent expectations for biotech companies developing some of the most cutting-edge therapies in medicine.

– Addison Bortz, Senior Director

There’s no shortage of public health crises currently keeping us up at night. But antimicrobial resistance is facing a particularly unique (and terrifying) problem: While microbes are constantly evolving to outsmart our current tools, companies are not investing in antimicrobial R&D to help us keep pace.

A new analysis from the Access to Medicines Foundation found that over the last five years, the number of potential antimicrobial treatments being developed by the world’s largest biopharma companies dropped by 35%, leaving only 60 candidates remaining. While 39 of those 60 pipeline projects are focused on the WHO’s priority pathogens, just five are being developed for children under age five.

This doesn’t reflect the state of AMR, which continues to be one of the largest threats to global health.

• AMR directly causes more than a million deaths annually.
• In the U.S. alone, there are more than 2.8 million antimicrobial-resistant infections each year.
• Gaps in access to antimicrobials continue to grow in low- and middle-income countries, where the highest burden of AMR exists.

The fundamental problem: Investment in antimicrobials doesn’t make financial sense for most companies.

Policymakers and NGOs are seeking to assist through market-shaping initiatives, with mixed results thus far. The AMR Action Fund, the UK’s antimicrobial subscription model, and the EU’s Partnership on One Health AMR have shown catalytic funding promise. But other efforts, such as the PASTEUR Act in the U.S., have stalled.

R&D investments can’t wait for policy to catch up. Without urgent action, The Lancet estimates that AMR will cause more than 39 million deaths by 2050 and ultimately threaten modern medicine as we know it.

– Rachel Bridges, Senior Director

More than 100 biologic medicines are projected to come off patent in the next decade; however, about 90% of those medicines are not yet facing competition from biosimilars in development. The FDA, through new guidance, hopes to steer the U.S. away from the “biosimilar void” and help lower costs for developers and patients.

The draft guidance, shared Monday, provides updated recommendations in two areas:

• Companies may now rely on clinical data generated with a non-U.S.-licensed comparator product. Previously, developers were required to conduct an additional three-way study comparing the biosimilar candidate, the U.S.-licensed reference product and the non-U.S. comparator.
• The agency revised a recommendation that developers conduct at least one clinical study that directly compared a proposed biosimilar with the U.S.-licensed reference product. Now, companies can cite existing data using a comparator product approved outside the U.S. if “scientifically justified.”

Announced through an agency release and a JAMA editorial, FDA leaders say the moves are intended to “increase the feasibility of biosimilar programs” by saving drugmakers tens of millions in development costs. Trade groups such as AAM and the Biosimilars Forum praised the FDA for using “commonsense reforms” that help “modernize” the development process.

With new guidance in hand, the biosimilars industry seeks to continue momentum in uptake in the U.S. health system, particularly among hospitals. New research published in JAMA found hospitals’ adoption of biosimilars for three oncology treatments nearly tripled in a recent five-year span – coinciding with higher markups on the medicines.

– Andrew Wishon, Senior Manager

• March 18 – committee hearing on lowering health care costs in the U.S. provider landscape, House Energy & Commerce
• March 18 – committee hearing on improving kidney health through better prevention and innovative treatment, House Ways & Means
• March 18-19 – meeting of the Advisory Committee on Immunization Practices (ACIP), CDC

• “China has no scientific edge over the United States, and it is unlikely to soon replicate the mix of venture capital, biotechnology start-ups and academic research that bolsters discovery here. China has only a policy edge, which derives from its deliberate economic strategy.” – Scott Gottlieb, Forbes
• “Drug pricing remains complicated, and responsibility for high costs extends well beyond PBMs. Manufacturers, insurers, regulators and policymakers all play a role. But sunlight is a good place to start.” – Andrew Langer and Jerry Rogers, RealClearHealth
• “Credit where credit is due: Prasad is right that the FDA, in the case of rare disease treatments, needed to demand clearer evidence before issuing approvals. The paradox is that when it came to battles over clinical data, he pulled out a bazooka when he needed a scalpel.” – Matt Herper, STAT

• In an unexpected ripple effect of President Trump’s most-favored nation pricing push, Astellas secured a more generous reimbursement level in Japan for its eye medicine Izervay. Bloomberg: Trump’s Drug Cost Push Helps Astellas Win Higher Price in Japan.
• A new survey from Phil found that six in 10 patients never fill a prescription even after seeing an ad for a drug they’re interested in, with price uncertainty cited as the top barrier. Fierce Pharma: Patients want price transparency, e-commerce experience from pharma DTP platforms: survey.
• China leads the world in developing competitors to the 10 highest-priced gene therapies, with 48 candidates in development – more than double the U.S. pipeline – at prices as low as one-tenth that of American rivals. Endpoints: China is going after the world’s most expensive drugs.
• The Inflation Reduction Act’s Medicare drug provisions reduced cost-related medication nonadherence among Part D enrollees in the year after taking effect, with an even greater effect among those with multiple chronic conditions, according to a new JAMA study. MedPage Today: Biden-Era Law Linked to Improved Medication Adherence in Medicare.
• Hims & Hers announced a “strategic shift” to partner with Novo Nordisk to sell FDA-approved GLP-1 medicines and cease the advertisement of compounded versions, ending a legal feud between the companies. The Hill: Hims & Hers to partner with Novo Nordisk and stop selling compounded GLP-1s.
• CMS has teased a proposed rule that would designate hospitals purchasing essential medicines from domestic sources as “Secure American Medical Supplies” and make them eligible for enhanced payments, though key questions remain unresolved. Pink Sheet: US Medicare Proposes Buy American Rx Incentives, But What Is A ‘Domestic’ Medicine?
• Public Citizen says it will encourage France and other countries to pursue compulsory licensing of generic alternatives if Pfizer seeks to halt the supply of medicines to countries that refuse to pay higher prices. Inside Health Policy: Public Citizen To Push Compulsory Licensing If Pfizer Withholds Drugs From France.
• Saturday Night Live spoofed Amgen’s Otezla in a pharma ad parody featuring Ryan Gosling, portraying the medication as an unknowable, time-transcending entity beyond scientific understanding. MM+M: SNL mocks Amgen’s Otezla in bizarre sketch featuring Ryan Gosling.