In The News

June 11, 2026

Pink Sheet — Tiered, Risk-Based Approach Could Increase US FDA Biomarker Qualification Utility

A Friends of Cancer Research white paper proposes statutory changes allowing different levels of biomarker evidence across drug development stages, as well as near-term steps for the FDA to make the qualification process more transparent, predictable and useful. Key Takeaways The FDA’s biomarker qualification program is a single, all-or-nothing determination out of step with evidence…

June 5, 2026

Pink Sheet — Rare Disease Groups Tell US FDA Endpoint, Biomarker, Adcomm Reforms Needed

During a meeting with acting FDA Commissioner Kyle Diamantas and other agency officials, rare disease groups discussed whether advisory committees could examine broader scientific, regulatory and development issues affecting multiple diseases more often. Key Takeaways Rare disease groups urged senior FDA officials to modernize its approach to endpoints, biomarkers, trial design and evidence generation for…

June 5, 2026

Regulatory Focus — This Week at FDA: WH reviewing candidates for FDA commissioner, Diamantas’ rare disease outreach, and more

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation, and what we’re reading from around the web. This week, the Trump administration is reviewing candidates for FDA commissioner, the agency is trying to repair relations with the rare…

June 4, 2026

Endpoints News — Acting FDA commissioner pledges to prevent political interference in rare disease meeting

Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas made it clear he’s headed in a different direction from former Commissioner Marty Makary and his team, particularly when it comes to listening to career staff on new drug approvals. The goal was…

June 3, 2026

Reuters — Acting US FDA chief meets with rare disease groups to mend fences

Acting U.S. FDA Commissioner Kyle Diamantas met with rare disease groups on Wednesday, according to groups attending and a government official, as the new chief seeks ‌to repair relations with a sector disappointed by his predecessor. Representatives for rare disease organizations including Friends of Cancer Research and the Foundation for Angelman Syndrome Therapeutics are pushing…

June 1, 2026

Pink Sheet — Match Made In Oncology: Pazdur Joins Friends Of Cancer Research Board

The long-time FDA Oncology Center of Excellence director joins former FDA and NCI officials on the FOCR board of directors. Richard Pazdur, the long-time head of the US Food and Drug Administration’s oncology review divisions, was elected to the board of the Friends of Cancer Research, one of the sector’s key advocates. Pazdur, who left…

June 1, 2026

Regulatory Focus — FDA, industry discuss need for flexibility in early phase clinical development of cell therapy products

More flexibility in early-phase development for cell therapies is needed, a panel of US Food and Drug Administration (FDA) representatives and industry members said at a public meeting hosted by Friends of Cancer Research and the Parker Institute for Cancer Immunotherapy. Vijay Kumar, acting director of the Office of Therapeutic Products (OTP) in the FDA…

May 27, 2026

Pink Sheet — US FDA’s Plausible Mechanism Guidance: CMC Expectations Too Stringent, Advocates Say

Rare disease advocates said chemistry, manufacturing and controls requirements in the FDA’s plausible mechanism framework guidance should not resemble standards for large-scale manufacturing. Key Takeaways CMC requirements in the FDA plausible mechanism guidance could limit the diseases and patient populations that could access the framework, advocates said. The FDA should clarify how manufacturing expectations would…

May 27, 2026

Pink Sheet — US FDA’s Plausible Mechanism Framework May Exclude Patients It Aims To Help, Groups Say

The plausible mechanism framework draft guidance requirement for “well-characterized natural history” could be too high a bar for many rare diseases and more clarity is needed on the patient populations that would qualify, advocacy groups said. Key Takeaways Patient advocates said the requirement for “well-characterized natural history” is vague and may preclude many rare disease…

May 15, 2026

AgencyIQ — With new incubator, FDA aims to bolster biomarker capabilities

The FDA’s biomarker qualification process has drawn criticism for its long timelines. A new incubator initiative is meant strengthen the agency’s data aggregation and evaluation capabilities, starting with a pilot project focused on drug-induced kidney injury. Background on biomarkers Biomarkers are a vital tool for disease diagnosis, patient risk assessment and drug development. The FDA describes biomarkers as…

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