OBJECTIVES: With the continuous emergence of new technologies filling high unmet medical needs in oncology, accelerated time to market for earlier patient access has gained importance. Increasing costs of drug development and intense competition between treatment options have enhanced the desire for early market access using surrogate endpoints. No study has recently assessed the use of surrogate endpoints in oncology and how this affects a new product in development. This research aims to identify the key challenges associated with surrogate endpoints in oncology from a payer perspective and provide recommendations to ensure an optimal Health Technology Assessment (HTA) submission.

METHODS: Available literature using PubMed database and HTA reports were reviewed, to explore the use of surrogate endpoints in oncology, and the evidence generation requirements including different payers’ perspectives.

RESULTS: The targeted literature review included 16 articles and 19 HTA reviews evaluating PFS and DFS/iDFS as surrogate endpoints. Results demonstrated that validation of a surrogate outcome specific to the population and technology type under consideration, and therefore trial design, plays an important role in. As emerging technologies become more targeted to specific patient populations, including treatments with different classes, there is often a lack of evidence to support the relationship between the surrogate endpoint and the final outcome. HTA agencies frequently require more evidence than regulators, given that they focus on a longer-term perspective. Innovative approaches, leveraging information across trials and real-world evidence, can be used to address current challenges and support evidence generation.

CONCLUSIONS: Surrogate endpoints are often associated with challenges in assessing the value of new treatments influencing HTA outcomes and negotiable prices. Evidence generation to support surrogacy should be aligned with payers’ expectations. Cross-functional collaboration allowing for early assessment of trial design is therefore crucial when incorporating surrogate endpoints, to ensure successful market access.