It has been a busy start to our 30th year and we’re excited to share more updates from the first half of 2026.
Here’s what you can read more about in this newsletter:
- What’s Happening at Friends?: Catch up on Friends recent public meetings and advocates webinars on External Control Arms (ECA), Next-Generation Therapies, and the Biomarker Qualification Program (BQP).
- Advocate Spotlight: Read the Advocate Insights blog written by Friends’ Advisory Advocate, Misha Mehta, who shared her son’s brain tumor journey and how it drives her continuing advocacy for critical cancer research.
- Mark Your Calendars: Register for Friends’ upcoming meetings and webinars and save the date for our 19th Annual Meeting.
- Data-Driven Insights: Gain insights into how regulatory flexibility through post market activities can enable access to precision oncology drugs relying on companion diagnostic tests.
- Important News in Cancer Research: Catch up on discussions during the latest Oncologic Drugs Advisory Committee (ODAC) meeting and other regulatory policy news.
What's Happening at Friends?
Application of External Control Arms (ECA) in Oncology Drug Development
ECAs are constructed using prior clinical trial data or real-world data and provide an external source for comparison when traditional randomized controlled arms are impractical, such as in small patient populations or settings with limited standard of care options. Our recent public meeting and advocates webinar included discussions on how ECAs can support evidence generation and regulatory decision–making. Discussions highlighted how use of ECAs can enable trials that better reflect real-world clinical practice and help generate more generalizable evidence.
Read the public meeting recap here. Watch the advocates webinar here.
Unlocking Next-Generation Therapies
Cell and gene therapies have transformative potential for treating cancer, however, access remains limited, with estimates suggesting that up to 80% of eligible patients lack access. Existing operational frameworks do not always align with the unique complexities of cell and gene therapies, creating regulatory and manufacturing hurdles that slow progress. Our recent public meeting and advocates webinar explored how innovative, flexible, and risk-based approaches to clinical trials and manufacturing can help reduce unnecessary barriers, maintain safeguards, and expand access to these transformative treatments.
Read the public meeting recap here. Watch the advocates webinar below.
Virtual Hill Briefing: Strengthening FDA’s Biomarker Qualification Program (BQP)
Biomarkers and early endpoints have the potential to modernize drug development by providing early indicators of treatment efficacy, new approaches to identify patients most likely to benefit from targeted treatments, and other information to guide decision-making. Novel biomarkers and endpoints require significant evidence before they can be used to support regulatory decision-making, such as supporting accelerated approvals. Frameworks such as the BQP were created to provide a structured process for development, validation, and qualification of biomarkers; however, opportunities remain to further optimize the process.
Friends recently worked with a group of stakeholders to propose enhancements to biomarker qualification that would support a more streamlined pathway for predictable, efficient, and evidence-driven biomarker development. Our recent virtual hill briefing explored some of these proposals and what they mean for the future of drug development.
Watch the virtual hill briefing below and read the issue brief here.
Read our analysis in DIA’s Therapeutic Innovation & Regulatory Science, which highlights the evidence-based opportunity that prompted this briefing and showed limited use and extended timelines for surrogate endpoint projects.
Advocate Spotlight
Misha recently wrote an article sharing her journey and how it drives her continued advocacy for critical cancer research. Read more in our latest Advocate Insights Blog here.
Mark Your Calendars
Please register for our upcoming events at the links below.
⬇️ = Reduced Advocate Registration | 🟩 = Free | 🔷 = Friends Event
| ⬇️ June 22 – 25, 2026 | BIO International Convention |
| 🟩🔷 July 15, 2026 | Interpreting Interim Overall Survival (OS) for Decision-Making Webinar |
| 🟩🔷 September 15, 2026 | Optimizing Dosing for Radiopharmaceutical Therapies Webinar |
| 🟩🔷 November 10, 2026 | Friends of Cancer Research Annual Meeting 2026 |
| 🟩🔷 November 17, 2026 | Advocates Webinar: Annual Meeting 2026 |
Data-Driven Insights
In recent years, an increasing number of novel oncology therapies have required companion diagnostic (CDx) testing to identify patients most likely to benefit from treatment. For rare and ultra-rare diseases, developing and validating these tests can be challenging due to small patient populations and limited available samples. In some cases, the FDA approves a drug before a CDx is available and uses postmarketing commitments (PMCs) to require sponsors to continue generating evidence supporting CDx approval after the drug reaches the market.
We examined novel oncology drugs approved by the FDA from 2012–2025 to evaluate how PMCs have been used to support the development, validation, and availability of CDx tests. We found:
Rapid recent growth in CDx PMCs: 20 of 95 novel precision oncology drugs approved since 2012 had a CDx PMC — half of those were approved in just 2024–2025, signaling a shift in the FDA’s approach.
CDx PMCs are concentrated in rare and ultra-rare diseases: CDx PMCs were mostly issued for drugs targeting small patient populations, reflecting both the real-world difficulty of developing diagnostics for rare diseases and the FDA’s willingness to use post-approval flexibility to preserve patient access.
FDA rationale for flexibility: The FDA points to limited risk of inappropriate patient selection, demonstrated therapeutic efficacy, availability of non-CDx or standard of care tests for the biomarker, rarity of the patient population, and high unmet need as reasons supporting their decision to approve the drug prior to CDx availability.
Why it matters: Cancer treatments increasingly rely on diagnostic testing to select patients for treatment, ensuring timely availability of these tests is critical. In areas of unmet need, such as ultra-rare and rare diseases, PMCs provide flexibility to support patient access to new drugs.
Grace Collins, Friends Manager of Regulatory & Data Insights, will be sharing these data at DIA Global on June 17th – follow us on LinkedIn for more details!
Important News in Cancer Research
April 30th Oncologic Drugs Advisory Committee (ODAC) Meeting
What: The ODAC evaluated two biomarker‑driven treatments—early circulating tumor DNA (ctDNA) ESR1‑triggered switching to camizestrant in HR+/HER2‑ metastatic breast cancer and a capivasertib combination regimen for PTEN‑deficient metastatic hormone‑sensitive prostate cancer—to decide if their benefits outweigh their risks.
Why it matters: The discussions emphasized the need for carefully designed trials and validated biomarker-defined treatment strategies. They also highlight that the patient voice is critical when interpreting whether progression-free survival (PFS), early overall survival (OS), and safety demonstrate “clinically meaningful benefit”; although the benefits may be “modest” on paper, they translate to real and meaningful benefits for people living with aggressive metastatic cancers.
For more on the ODAC meeting, read our recap blog here.
Plausible Mechanism Framework Guidance
What: The FDA released new draft guidance laying out a “Plausible Mechanism” framework that aims to support development of individualized therapies intended to treat a “severely debilitating or life-threatening disease or condition in a small number of patients where a randomized controlled trial typically is not feasible.”
Why it matters: The framework provides a flexible regulatory approach that maintains rigorous evidence standards while considering the unique challenges in developing individualized therapies. This can help support development and access to innovative treatments for patients with serious life-threatening diseases, including in rare and ultra-rare patient populations with high unmet need.
Friends submitted a public comment on how the FDA could strengthen this framework by providing clearer guidance on using the framework, particularly regarding the use of well-defined natural history data and how evidentiary expectations evolve across the full product lifecycle.
Read Friends’ public comment on the draft guidance here.
Resources
ProgressforPatients.org and ProgresoparaPacientes.org
Interested in learning more about drug development and how the FDA functions?
ProgressforPatients.org and ProgresoparaPacientes.org 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.