Early Endpoints Portfolio
Early endpoints that are reasonably likely to predict long-term outcomes can help streamline clinical trials and accelerate the evaluation of new treatments, but evidence is needed to support their reliable use in regulatory decision-making.
Friends Early Endpoints Portfolio generates evidence supporting the use of early endpoints in regulatory decision-making.
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What is the unmet need and why does it matter?
Scientific innovation and therapeutic advancements over the past two decades have helped patients with cancer live longer. While this progress benefits patients, it also means that clinical trials may take longer to generate readouts on traditional clinical endpoints, such as overall survival (OS). As a result, efficacy assessments, regulatory approvals, and patient access to new therapies may take longer while endpoint data mature, highlighting the need for reliable early endpoints to provide timely insights into treatment benefit.
How are we helping to find solutions?
The research partnerships in the Friends Early Endpoints Portfolio are collaborative and data-driven. We bring together stakeholders to aggregate and analyze data across clinical trials. These data generate the evidence and frameworks necessary to support reliable use of early endpoints and novel tools for regulatory decision-making.
In collaboration with industry representatives, patient advocates, government officials, and academics, we define the endpoint, create an analysis plan, identify data, and perform aggregate analyses to demonstrate associations between the endpoint and long-term outcomes.
How does this impact patients?
Drug sponsors incorporate early endpoints such as response rate and progression free survival (PFS) into clinical trials to assess therapies before OS data become available, allowing expedited approvals for promising therapies in areas of unmet need. Other innovative approaches to assessing treatment efficacy—including evaluating changes in circulating tumor DNA (ctDNA) and employing artificial intelligence (AI)-based technologies for tumor assessments—are emerging methods that may provide even earlier results or be more accurate than current tools. These novel tools hold promise for facilitating efficient efficacy assessments and supporting providing timely access to novel therapies, but robust evidence is needed to show they reliably predict clinical benefit. Our Early Endpoints Portfolio helps to demonstrate their value, establish their reliability, and validate their use for regulatory decision-making.
How does this work inform federal policy?
Friends uses insights from our Early Endpoints partnerships to inform evidence-based policy recommendations with the goal of modernizing regulatory frameworks to reflect the latest science. The FDA’s Accelerated Approval Program provides a tool to expedite approval a new treatment for a serious illness based on an early endpoint reasonably likely to predict long-term benefit.
- Hurry Up and Wait: Timelines and Takeaways from the Biomarker Qualification Program
- Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics: Draft Guidance for Industry
- Use of Circulating Tumor Deoxyribonucleic Acid for Early-Stage Solid Tumor Drug Development; Draft Guidance for Industry
Read more about each of our Early Endpoints partnerships at the links below.
Circulating Tumor DNA for Monitoring Treatment Response (ctMoniTR) Project:
(ctMoniTR Project)
Do reductions in ctDNA levels post-treatment correspond with therapeutic outcomes?
Artificial Intelligence-Enabled Measurement of Response Evaluation Criteria in Solid Tumors (ai.RECIST) Project:
(ai.RECIST Project)
Can AI-based imaging tools improve tumor measurement and accelerate clinical trials?