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Trends in Oncology Regulation: FDA Draft Guidance on OS and Radiopharmaceuticals

Over the past two decades, the FDA has steadily increased the number of guidance documents it publishes each year. The agency issued about 101 guidance documents annually from 2005–2010, rising to 173 per year between 2011–2019, and more than 190 in FY 2023.

Two recent draft guidances on Approaches to Assessment of Overall Survival (OS) in Oncology Clinical Trials and on Radiopharmaceutical Dose Selection are examples of how the agency is clarifying guidance to address the rapid expansion and complexity of cancer research.

FDA's Stance on Overall Survival

The draft guidance underscores what has long been recognized: OS remains the gold standard endpoint for assessing clinical benefit in evaluating new oncology treatments. When feasible, the FDA recommends OS as a primary or key secondary efficacy endpoint in randomized trials. Even when OS is not included as an efficacy endpoint, sponsors are still expected to collect OS information to establish evidence that the novel product is not harmful to patients.

Professional groups such as ASCO have affirmed this position, reinforcing the central role OS will continue to play in cancer drug development.

Veristat's Perspective

At Veristat, the counsel that we have been providing our clients for years is already reflected in this draft guidance. FDA guidances grow from real-world trial conduct, based on the FDA perspective of what works, what doesn’t, and applying common sense to codify those lessons into policy. We value that approach and encourage sponsors and their partners alike to see these guidances as opportunities to strengthen trial design and regulatory strategy.

Where Exceptions Apply

The FDA acknowledges that OS may not always be practical as a primary endpoint in an oncology trial, for example, in diseases with long survival times, where extended follow-up can delay sufficient results for statistical analysis, or in certain single-arm trials where no comparator exists. In these cases, surrogate endpoints such as progression-free survival for randomized studies or overall response rate for single-arm studies may support accelerated approval, with OS follow-up expected later to confirm benefit. Importantly, OS can also be evaluated in interim analyses to identify futility or potential harm.

Accelerated Approval Pathways

Accelerated approval has transformed oncology product development, with 80% of the accelerated approvals granted for cancer therapies. Between 2013 and 2023, the FDA approved 129 oncology drugs under this pathway, a testament to the use of the accelerated approval pathway as an important mechanism for timely patient access to new treatments while confirmatory data mature.

The OS guidance reinforces that while surrogate endpoints can support accelerated approval, confirmatory trials with survival outcomes remain essential. For sponsors, accelerated approval should not be seen as the finish line but rather as a milestone that demands careful planning for OS follow-up to secure traditional approval.

Radiopharmaceuticals and Optimal Dosing

The FDA’s recent draft on radiopharmaceutical dose selection reflects both progress and ongoing challenges within the research area. Radiopharmaceuticals deliver radiation directly to tumor cells by linking a radioactive component to a targeting antibody or molecule. While highly precise, this approach makes it difficult to balance safety and efficacy. Unlike chemotherapy, dose escalation is not straightforward, and off-target effects can raise significant toxicity concerns.

The FDA’s guidance acknowledges these issues, but additional clarity will be needed to establish consistent parameters for developing radiopharmaceuticals. Veristat advises that it will not be sufficient for sponsors to simply invoke a vague reference to the “totality of the data” in their justification of dose selection in radiopharmaceutical research; further scientific work is required to determine optimal dosing strategies. At Veristat, we remain in close contact with the FDA, stay current on evolving guidance, and share insights from our experience to help shape practical approaches in this challenging area.

Turning Guidance into Action

Many oncology guidance documents, including those on OS and radiopharmaceutical dose selection, are shaped by the FDA’s Oncology Center of Excellence (OCE). This initiative unites cross-disciplinary expertise to advance greater precision in clinical studies and to promote regulatory clarity in cancer drug development.

FDA’s draft guidance on OS and radiopharmaceuticals reflect clear trends in oncology regulation: codifying common-sense practices, advancing the role of the Oncology Center of Excellence, and reinforcing the importance of survival follow-up in accelerated approval pathways.

The FDA is working to promote good science and research, and one way it does so is by providing sound, practical guidance. Veristat will submit feedback on these draft guidances as part of the agency’s comment process, ensuring that insights from our work with sponsors and investigators are reflected. We encourage other sponsors and clinical research organizations to do the same so that final guidances are informed by real-world experience.

We fully support not just this effort, but the FDA’s broader mission to advance science through clear and practical guidance. Our company is built on the same foundation of scientific rigor. Whatever challenges sponsors face, our regulatory and clinical development experts provide clear, strategic, and flexible approaches to tackle the most complex aspects of program design, clinical trial conduct, and regulatory approval. For sponsors, the takeaway is clear: guidance alone does not deliver success—it’s how you interpret, plan, and execute against it that determines outcomes.

Veristat brings the expertise, foresight, and hands-on partnership to translate evolving FDA expectations into actionable strategies that accelerate progress and minimize risk.


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