Project Summary

Background: Novel therapies are often approved after accelerated FDA reviews. Associated costs can be high. Patient communities have high expectations for access, but payers are poised to impose stringent controls on use. Patient communities are eager to communicate with payers, yet few formal processes are in place as helpful guardrails. Case in point: patients prize improvements in quality of life and functional status in assessing new therapies, but payers often view clinical trial evidence on patient-centered markers as underdeveloped or underreported. Patient community and payer communication could be improved if both sides held a shared understanding of principles and best practices for evidence-based communication. Patient community-payer communication is an emerging trend that will influence research priorities for patient-centered outcomes research and real-world evidence and is consistent with PCORI’s new mission to support evidence development relevant to payer decision making.

Proposed Solution to the Problem: The proposed solution is a consensus-based statement of principles for good, evidence-based, two-way communication among patient communities and payers regarding novel therapies. NEHI’s patient-payer advisory committee will develop a statement, achieve consensus with a larger, representative group, release a final document, and actively promote it for endorsement and uptake. The advisory committee will include patient leaders from communities with active stakes in novel therapies, in patient-centered outcomes research and measure development, as well as payer representatives with expertise in drug evaluation and coverage decision-making processes. NEHI will seek consensus at a multi-stakeholder meeting. A final statement will be approved by the advisory committee. The advisory committee will work with NEHI to devise a detailed plan for dissemination, endorsement by patient and payer organizations, and continuing evaluation of uptake of the principles of communication.

Objectives: The primary objective is development and release of stakeholder-supported principles of communication practice between patients and payers to educate these communities on evidence-based practices for effectively sharing information about novel therapies that will be widely disseminated, endorsed, and utilized in communication between patient communities and payers as novel therapies reach the market and are subject to evaluation by payers and subsequent decisions on the terms and conditions of coverage. 

A second document, a summary of findings from NEHI’s multi-stakeholder meeting, will provide consensus-based recommendations on addressing significant opportunities for further patient engagement in PCOR that will support patient community-payer communication, and opportunities for further dissemination of existing PCOR findings to support better communication.


  • Research: Literature review and key informant interviews will support drafts of the statement of principles of good communications practices. 
  • Online survey: Invitees to the multi-stakeholder meeting will respond to an online survey on patient-payer communications and novel therapies. Findings will inform the draft statement presented to the multi-stakeholder meeting.

Multi-stakeholder meeting: Facilitated discussion and consensus development. After facilitated discussion, participants will vote on consensus-backed positions. Minority views will be noted for inclusion in a meeting summary, as will opportunities for further PCOR research that enables good patient-payer communication.

NEHI and the advisory committee will develop a dissemination, endorsement, and evaluation plan, identifying opportunities for uptake of good communications principles by patient groups and payers, building on the network of relationships established during the project. 

Projected Outcomes and Outputs: The project’s primary deliverable (principles of good, evidence-based, two-way communication among patient communities and payers as they pertain to novel therapies) will serve as a code of conduct for patient communities and payers to reference, endorse, and incorporate as standards of practice within their organizations. 

The evaluation plan (see below) outlines short-term outcomes to be tracked as statistics under the rubric of the PCORI Evaluation Reporting Tool: for example, statistics on the volume of project documents disseminated or downloaded, tracked by sector (segments of patient communities and payer organizations).

More substantively, within a long-term impact plan, NEHI and its advisory committee will identify opportunities for endorsement and adoption of the principles among patient and payer groups. This effort will build on the network of relationships built among patient and payer groups over the course of the project.

Patient and Stakeholder Engagement Plan:

  • Advisory committee: NEHI will create an eight-person advisory committee that will meet monthly to offer direction, review, and approve every aspect of the project. Memberships will include three patient community representatives (a PCORI grantee and a non-PCORI grantee from communities in which novel therapies have been launched or are anticipated, and a third representative active in patient-focused drug development); three representatives from payer organizations, all experts in payers’ drug evaluation and coverage processes; and two researchers active in patient-centered outcomes research. 
  • Participants in multi-stakeholder meeting: With advisory committee direction, NEHI will reach out to approximately 30 organizations among patient community and payer organizations, seeking representation largely reflecting the composition of the advisory committee. Participants will take part in an online survey and vote on consensus positions of the project’s statement of good communication principles.

Project Information

Thomas Hubbard
NEHI-The Network for Excellence in Health Innovation

Key Dates

13 months


Project Status
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: January 20, 2023