Beyond Overall Survival: The Value of Oncology- Relevant Endpoints in HTA Body / Payer Decision-Making

Introduction: HTA bodies/payers have focused on overall survival (OS) to evaluate oncology medicine's efficacy during decisionmaking.While OS remains important, reliance on it poses three challenges: potential delayed patient access to medicines due to extended time to measure OS in some indications and settings, poor ability to capture impact on quality of survival, and vulnerability to confounding.Better value recognition of oncology-relevant endpoints (OREs), which includes all endpoints used in oncology clinical trials, including patient-reported outcomes, may address these challenges.This work explores the value of OREs and their role in HTA body/payer decision-making.Methods: This work is based on a literature review, interviews and three roundtables with 13 stakeholder group representatives (physicians, patient, former HTA bodies/payers) in US and Europe.Results: OREs beyond OS can be standalone efficacy measures through capturing outcomes beyond survival that are important to patients and clinicians.They can also act as surrogates by providing earlier measure of a medicine's efficacy and are less influenced by confounding than OS.Value recognition of OREs beyond OS varies across stakeholders and should be considered per cancer type and stage.Conclusions: Clinicians, patients and regulators recognize the value of OREs beyond OS but HTA bodies/payers can be skeptical, driven by concerns about how ORE value translates to meaningful outcomes for patients and healthcare systems.Stakeholders should build a portfolio of fit-for-purpose OREs by cancer type and stage based on shared understanding of their value to improve their acceptance in decision-making and advance patient access to novel therapies.