Quarterly Advocacy Newsletter – Q3 2025

In this quarter’s issue, we are excited to share updates on some of the innovative work happening at Friends, including our latest efforts in advancing AI in oncology, upcoming events, and important developments in cancer research.

What’s Happening at Friends?: Dive into our ai.RECIST Project, a cutting-edge initiative exploring the use of artificial intelligence (AI) for tumor assessments in cancer clinical trials.

Did You Know?: Learn about the use of interim overall survival (OS) data in oncology trials and efforts to improve its interpretation through a structured, multi-step approach that supports better-informed decisions for advancing cancer treatments.

Mark Your Calendar: We’re excited to announce that our 2025 Annual Meeting will take place on November 4th in Washington, DC and a follow-up Advocate Webinar!

Important News In Cancer Research: Stay up to date on the latest news impacting cancer research and drug development including upcoming discussions that are critical to funding the FDA and recent oncologic drug advisory committee meetings weighing benefits and risks of new treatments.
In Case You Missed It: Catch up on Friends’ recently released publications.

What's Happening at Friends?

Friends continues to lead the way in AI-driven oncology advancements. Our ai.RECIST Project recently kicked off, aiming to transform tumor response assessments through AI integration. AI-enabled tumor assessments have potential to accelerate clinical trials and improve patient outcomes, but additional data are needed to understand whether these approaches are reliable.

To learn more about how our ai.RECIST Project is helping generate data necessary to advance AI-enabled tumor response evaluation and accelerate clinical trials, visit the ai.RECIST page.

Did You Know?

The Problem: Accelerated Approval often relies on early endpoints (e.g., response rate) as a proxy for overall survival (OS) to assess therapy efficacy. Interim OS data, assessed alongside early endpoints, can be challenging to interpret because of the small number of events, limited follow-up, and added complexity when interim OS trends appear inconsistent with early endpoint readouts.

Friends’ Solutions: Because these data play a critical role in supporting expedited oncology approvals and timely patient access to new therapies, Friends worked with stakeholders to identify ways to improve the reliability of interim OS data. Our 2024 Annual Meeting white paper outlines a structured approach to assessing interim OS, combining qualitative analyses to provide clinical context (e.g., event counts, coexisting conditions, adverse events) with quantitative analyses using predictive models to estimate the likelihood of long-term benefit.

Why It Matters: Stronger approaches to interim OS evaluation can make these data more actionable, enabling better-informed decisions about treatment efficacy and safety. This work helps advance methods to optimize oncology clinical trials and ensure patients gain access to the most effective therapies as quickly and safely as possible.

Mark Your Calendars

⬇️ = Reduced Advocate Registration | 🟩 = Free | 🔷 = Friends’ Event

🟩 🔷 November 4, 2025	Friends 2025 Annual Meeting
🟩 🔷 November 17, 2025	Advocates Webinar: Annual Meeting 2025

Important News in Cancer Research

Prescription Drug User Fee Act (PDUFA) Reauthorization

PDUFA provides critical funding that allows the FDA to conduct timely approvals, potentially fast-tracking access to cancer treatments.
PDUFA also includes new programs focused on advancing innovative regulatory tools such as model-informed drug development and novel patient-reported outcome (PRO) measures.
Every five years, Congress reauthorizes user fee programs to ensure FDA remains at the forefront of regulatory science. The next reauthorization, PDUFA VIII, is scheduled for 2027.

Read our blog on the history of PDUFA and why it’s critical for advancing key regulatory and scientific initiatives.

Oncologic Drugs Advisory Committee (ODAC)

The ODAC is a key mechanism that provides independent expert advice to guide FDA’s decision making. Their evaluations are instrumental in shaping the future of oncology drug approvals.
The most recent ODAC hearing, held July 17, focused on clinical trial data supporting two proposed combination regimens for treatment of multiple myeloma. The committee was asked whether the benefit-risk profile of these combinations was favorable at the proposed dosage.
- Benefits: The combinations slowed disease progression and early results suggested potential survival benefits.
- Risks: Concerns focused on high rates of ocular toxicities at the proposed dose, a lack of data exploring a lower and potentially more tolerable dose, and the limited applicability of the trial to U.S. patients and clinical care.
- Ultimately, the risks were considered to outweigh the benefits.

Read our blog about the recent ODAC here

Friends’ Comment on the National Cancer Institute’s (NCI) Request for Information (RFI) on AI in Cancer Research and Care

We recently responded to the NCI RFI on benchmarks for AI in cancer research and care. Our comment highlighted the need for high-quality, independent reference datasets to ensure AI tools are accurate, and clinically meaningful.

Our recommendations included:

Prioritizing benchmarks for high-impact use cases like digital pathology, tumor imaging, and real-world data extraction;
Ensuring datasets reflect real-world complexity and support regulatory alignment;
Leveraging existing resources and fostering collaboration to accelerate progress.

Read our full comments here.

Friends Annual Leadership Awardee

Each year, Friends recognizes two visionary leaders in the field of public health who have made significant contributions to cancer research and patient access. These awardees are instrumental to the achievements of Friends and our collective work to advance the field of oncology. This year, we are honoring Mark McClellan and Peter Marks.

Mark McClellan, MD, PhD

Recipient of The Marlene A. Malek Public Service Award. The Marlene A. Malek Public Service Award celebrates Marlene A. Malek’s lifelong commitment to science, public policy, and service that has proved invaluable to the organization.

Dr. Mark B. McClellan is the Director of the Duke-Margolis Center for Health Policy and the Robert J. Margolis Professor of Business, Medicine, and Health Policy at Duke University. A physician and economist, he has held pivotal roles in U.S. health policy, including serving as Commissioner of the U.S. Food and Drug Administration (FDA) and Administrator of the Centers for Medicare & Medicaid Services (CMS). His work has focused on improving healthcare quality, payment reform, and the development of real-world evidence. Dr. McClellan is being honored with The Marlene A. Malek Public Service Award for his exceptional leadership and enduring contributions to public health.

Peter Marks, MD, PhD

Recipient of The Ellen V. Sigal Advocacy Award. The Ellen V. Sigal Advocacy Award celebrates Dr. Ellen Sigal’s lifelong commitment to science, research, and patient advocacy that she has brought to our work.

Dr. Peter Marks is a hematologist and oncologist who served as the Director of the FDA’s Center for Biologics Evaluation and Research (CBER) from 2016 to 2025. He played a critical role in the development and approval of cell and gene therapies that improve outcomes for people with cancer. Dr. Marks is being recognized with The Ellen V. Sigal Advocacy Award for his steadfast commitment to advancing public health through scientific integrity and regulatory excellence.

ProgressforPatients.org

Interested in learning more about drug development and how the FDA functions?

ProgressforPatients.org (PFP) and Project TEACH are online advocacy education programs and communities working to help patients, advocates, and caregivers acquire the necessary tools to effectively communicate with drug researchers, drug developers, and regulators.