Patient and Caregiver
Investigator
Regulator
IRB
Tech, Device,
PayerTrial Design Stage
Published Date: October 6, 2023
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Helpful Terms and Definitions
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*This list is not exhaustive, nor does CTTI officially endorse the resources created by other organizations.
Study Concept
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 implementation 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.
A positive trial is not automatically a coverable trial.
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?
Payer Resources
- Payer Evidence Strategy & Design
These resources focus on aligning trial design (endpoints, comparators, and population selection) with the evidentiary needs of health technology assessment (HTA) and payer organizations.
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- Designing Clinical Trials with the Payer in Mind (Research Consortium): An essential tactical manual on embedding payer-relevant metrics, such as comparative effectiveness and real-world value, early in the protocol design phase.
- Payer Coverage for Clinical Trials (PMC3348601): A foundational study on the mechanisms of payer coverage for clinical trials, highlighting the need for transparency in "routine" versus "investigational" cost documentation to facilitate patient access.
- Four Key Trends in Oncology Drug Development (Applied Clinical Trials): An analysis of the evolving evidentiary bar in oncology, where regulators (FDA) and payers often have diverging requirements for trial data.
- Communication Frameworks & High-Stakes Planning
Tools for structuring the dialogue between sponsors and payers to de-risk market access.
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- FDA CDRH Payor Communication Task Force: The official FDA/CDRH platform to facilitate early interaction between device manufacturers and payers, aiming to synchronize the timeline of regulatory approval and coverage determinations.
- Preparing for High-Stakes Meetings: Conveying Value (Certara): Expert strategies for preparing internal teams to present clinical data through a "value-based" lens during pivotal discussions with payers.
- Operational Case Studies & Novel Design
Practical applications of these strategies in non-traditional trial environments.
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- Understanding Payer Perceptions of a Novel Trial Design (Lightning Health): A case study evaluating the "payer-readiness" of non-traditional designs like basket studies, specifically regarding how payers view the validity of novel clinical endpoints.