June 26, 2024
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September 16, 2024
June 17, 2026
U.S. drug companies are accelerating licensing deals with Chinese pharmaceutical companies to take advantage of China’s clinical trials system. The Trump administration is eyeing the trend warily. New bipartisan legislation would create a vast biological database primed for AI-driven drug research. French photonics quantum computing company Quandela gets tapped for the Defense Advanced Research Projects…
Read MoreJune 11, 2026
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…
Read MoreJune 5, 2026
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…
Read MoreJune 5, 2026
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…
Read MoreJune 4, 2026
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…
Read MoreJune 3, 2026
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…
Read MoreJune 1, 2026
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…
Read MoreJune 1, 2026
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…
Read MoreMay 27, 2026
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…
Read MoreMay 27, 2026
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…
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