There is a need for surrogate endpoints. Approval and reimbursement decisions for new pharmaceuticals often need to be based on surrogate endpoints due to the following key reasons:
Efficacy data based on clinical endpoints require a large sample size and a long follow up.
This can present a challenge for therapies for chronic diseases and where there is a small expected effect size
Treatments are becoming more effective, therefore, endpoints are reached later.
Longer follow up is needed to observe the outcome
Only a small effective size for the clinical outcome when observed early
There can be ethical issues in stopping a trial early once the investigational product is proved to be superior based on other outcomes.
Unbiased clinical endpoints sometimes cannot be observed due to:
Crossover to the efficacious therapy
Emergence of many possible subsequent therapies
There is, however, wide variability regarding the validity and acceptability of surrogate endpoints across therapy areas and HTA agencies. Payer research and case studies were used to identify payer positioning on surrogate endpoints for pricing and reimbursement, and these insights will be shared in this webinar.
Key Learning Objectives
- Overview of the current regulatory and HTA landscape regarding surrogate endpoints and associated challenges for pharmaceuitcal manufacturers
- Analysis of the acceptability of surrogate endpoints to payers for supporting the incremental clinical value of novel therapies across therapy areas and healthcare systems, and consequences for pricing and market access
- Exploration of the key drivers behind the acceptability of surrogate endpoints to inform payer decision making and implications for evidence generation and value communication strategies
- Key recommendations for pharmaceutical manufacturers on situations in which a surrogate endpoint can successfully support pricing and market access, and requirements for ensuring validity within payer assessments
Andrew Satherley, MSc, Senior Consultant, Payer Strategy, Evidera; Eric Chang, PharmD, Consultant, Payer Strategy, Evidera; Sonika Awasthy, MSc, Senior Business Analyst, Payer Strategy, Evidera