Drugs

Rare Cancer Seamless Clinical Trials

A new Friends of Cancer Research white paper, “Seamless Clinical Trial Designs in Rare Cancers: Leveraging Operational and Adaptive Strategies to Accelerate Drug Development,” concludes that when thoughtfully designed, such trials can accelerate timelines, reduce redundancy, and generate robust evidence from limited populations without compromising scientific integrity. The paper is drawn from the efforts of a multi-stakeholder working group that included experts from FDA, patient advocacy organizations, drug companies, academia, and the National Cancer Institute to identify critical design considerations and explore strategies for operationalizing the efficiencies offered by the seamless design framework in rare cancer development.

Strategies explored by the working group were:

seamless trial designs;
endpoints and adaptive features; and
regulatory and patient engagement.

The paper defines seamless clinical trials as “a clinical trial integrating multiple, sequential stages of drug development, such as dose escalation, dosage optimization, cohort expansion, and efficacy framework within a single framework.”

By consolidating the various stages, the paper says, seamless trials can enhance efficiency, ensure consistency, and maximize the scientific and evidentiary value of each patient’s participation.

Seamless clinical trials represent a paradigm shift in rare cancer drug development, the paper says, addressing the limitations of conventional phased approaches by integrating multiple stages of clinical evaluation into a single protocol. It suggests that realizing the full potential of seamless trials “requires collaboration among sponsors, investigators, regulators, and patient advocates. As experience grows, stakeholders can iteratively refine trial elements, learning from successes and challenges to improve efficiency, adaptive decision making, and endpoint selection. Sharing best practices and particularly seeking FDA guidance further ensures that adaptive designs remain feasible, meaningful, and aligned with regulatory priorities.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome