Patient and Caregiver
Investigator
Regulator
IRB
Tech, Device,
PayerTrial Design Stage
Published Date: October 6, 2023
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Helpful Terms and Definitions
*This list is not exhaustive, nor does CTTI officially endorse the resources created by other organizations.
Study Concept
Questions To Ask
- Does the study align with established best practices, clinical guidelines, or standards of care for the target disease or condition?
- If there isn't a proven standard of care, how will this study demonstrate the effectiveness of the investigational treatment over other existing treatments?
- What is the benefit for patients if the investigational treatment is approved? Will it contribute to better care practices, improved clinical outcomes, or more effective disease prevention strategies?
- What populations are affected by the target disease or condition? Are there social risk factors that could affect access to care?
- How can payers and sponsors work together to ensure equal access to the investigational treatment across the affected population?
Why involve payers now?
Early payer engagement at the study concept phase helps ensure clinical trials generate evidence that supports downstream access, reimbursement, and formulary decision‑making. Building these relationships early fosters trust between sponsors and payer groups and maximizes the long‑term value of the clinical development program—engagement that should continue throughout trial design.
Payers provide insight into outcomes that are critical for reimbursement decision-making, including cost-effectiveness, quality of life, and patient-reported outcomes. Their perspective helps ensure appropriate comparators are considered and that trial designs reflect how new therapies may be evaluated relative to existing standards of care.
In addition, payers can advise on health economic models that best demonstrate budget impact and value, help define patient populations that mirror real‑world use, and recommend data collection approaches—such as real‑world evidence and long‑term follow‑up—that align with payer evidence expectations. Their guidance on evidence expectations, value demonstration, and result communication further strengthens the credibility, relevance, and acceptance of trial outcomes.
Engagement is most impactful when initiated early and maintained throughout development. Without this input, trials may miss key comparators, endpoints, or data needed for reimbursement.
How to locate a payer group
Payers include commercial insurers, government programs, PBMs, and integrated delivery networks with differing evidence needs.
What can payers contribute to the clinical research enterprise?
- Facilitating or limiting access to technologies through coverage and reimbursement decisions that have been proven safe and effective
- Encouraging the implantation of evidence-based clinical guidelines and best practices
- In some cases, supporting evidence generation through real-world data collaborations independently and through sponsor partnerships
- Helping put clinical research findings into practice through disease management efforts and provider education initiatives
- Disseminating health information through health education programs
Engagement should follow applicable regulations governing pre-approval communications.