Biomarkers are measurable characteristics that indicate a normal or abnormal biological process, condition, or disease.1 They are increasingly accepted by global regulators as surrogate endpoints to substitute and predict patient-relevant outcomes in a clinical trial.2 In 2024, the EMA has authorised 11 out of 114 (10%) approved treatments under the conditional marketing authorisation (CMA) and accelerated assessment (AA) pathways, which enables drugs treating conditions with a high unmet need or major public health interest to gain approval based on incomplete benefit-risk data.3,4 Similarly, in 2024 the FDA authorised 7 out of the 50 (14%) novel drugs under its Accelerated Approval Program, which grants conditional approval of drugs using surrogate endpoints or intermediate clinical endpoints that are likely to predict benefit.5 For both the EMA and FDA programs, companies must conduct post-approval studies to confirm the drug’s clinical benefit for the approval to remain unconditional. Despite the increasing approval of treatments based on surrogate endpoints, patient access remains dependent on the HTA’s assessment of a treatment’s clinical and economic benefits. In recent years, HTA agencies have developed methodological guidelines in response to the increased need to assess treatments utilising surrogate endpoints in their clinical evidence. However, there is great variation in how HTA agencies consider surrogate endpoint evidence in submissions. This is particularly regarding the level of scrutiny applied to surrogate endpoint validation, with NICE requiring the greatest validation, while HAS and NIPN demand the least level.6 IQWiG takes an even stricter stance, as it does not explicitly recognise any surrogate endpoints and valid.6 Due to the lack of harmonisation across HTA agencies, having an in-depth understanding of individual HTA perspectives on surrogate endpoints is necessary. Variation in the steps of validation of surrogate endpoints across HTA agencies Despite differences among HTA agencies, assessments that successfully demonstrate a link between surrogate and patient-centered outcomes were more likely to gain acceptance from HTA bodies.6 Choosing an appropriate surrogate endpoint is therefore crucial to ensuring the development of treatments that benefit patients and ultimately secure reimbursement. With our team of experts, and our value-based pricing methodology, GPI helps companies access the likelihood that certain surrogate endpoints will be accepted by payers in different HTA markets. By leveraging our unique combination of data, technology, and professional services we create tailor-made solutions to help you navigate the complexities of new legislation related to surrogate outcomes, and the divergent perspectives of payers to enable you to make informed go/ no-go decisions as clinical data evolves. Book a meeting here.Background and objectives
Biomarkers and Surrogate Endpoints Are Gaining Regulatory Acceptance
Methodological Guidelines Vary Across HTAs
Helping You Connect Surrogate Endpoints to Clinical Value
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