New Research Identifies Steps to Improve Gene Therapy Contracting and Patient Access

A new NPC study in JMCP proposes consensus-based guidance to support sustainable payment approaches and improve equitable patient access to gene therapies.

WASHINGTON, DC, UNITED STATES, May 14, 2026 /EINPresswire.com/ -- New research from the National Pharmaceutical Council (NPC) published in the Journal of Managed Care + Specialty Pharmacy (JMCP) outlines consensus-based principles and practices intended to make payment models for gene therapies in the U.S. more efficient, scalable, and patient-centered.

Gene therapies pose unique challenges to traditional reimbursement models. As interest in innovative contracting continues to grow, “Principles and Practices for Successful Gene Therapy Innovative Contracting: Insights from a Multistakeholder Convening,” brought stakeholders together to develop practices for them using a mixed methods approach. The research is authored by NPC’s Tyler D. Wagner, PharmD, PhD, and Jonathan D. Campbell, PhD, Real Chemistry’s Jacqlyn W. Riposo, MBA, Kendra M. Gould, MSPH, and Theresa Schmidt, MA, and consultant James T. Kenney, RPh, MBA.

A diverse group of stakeholders including payers, manufacturers, employers, and patient advocates came together to develop two frameworks: a framework for principles outlining the “why” behind past contracting successes and a framework for practices detailing the “how.” Through a multimethod approach combining a literature review, stakeholder interviews, and surveys conducted before and after a virtual convening, researchers identified eight principles and seven top-ranked practices that achieved stakeholder consensus (defined as at least 75% agreement).

“Innovative contracts are one tool among many, and they work best when designed well. This research outlines clear principles, such as protecting patients from additional out-of-pocket costs, streamlining data collection, and reducing complexity, that are critical to getting them right,” said lead author and NPC Director of Research Tyler Wagner. “By sharing these ideas, we hope to improve how these therapies are used and paid for, making sure all individuals who need gene therapies can access them.”

Key Insights:

· Stronger data and real-world evidence are needed to address uncertainty around long-term results.

· Reducing administrative burden is critical to make these models work in practice.

· Flexible approaches based on real-world evidence and the unique characteristics of each therapy are needed to support broad adoption and patient access.

“Gene therapies have the potential to transform how we treat serious and rare diseases, but they require new approaches to achieve equitable patient access,” said Jon Campbell, NPC Chief Science Officer. “This research builds on prior NPC work and reinforces the importance of patient-centered evidence generation.”

The study also highlights areas where stakeholders held diverging views, including methods to best incorporate patient and provider input into contract design, the appropriate use of warranty contracts, and the role of site-of-care requirements. These open questions point to opportunities for further research and dialogue.

NPC’s research library includes substantial evidence on the historical landscape of innovative contracts for gene therapies. As more gene therapies receive FDA approval, the authors note that these consensus-based principles should serve as a starting point and evolve alongside new evidence, payment innovation, and policy reform.

About the National Pharmaceutical Council
NPC serves patients and society with policy-relevant research on the value of patient access to innovative medicines and the importance of scientific advancement. We envision a world where advances in medicine are accessible to patients, valued by society, and sustainably reimbursed by payers to ensure continued innovation. For more information, visit www.npcnow.org and follow NPC on LinkedIn.

Rebekah Pepper
National Pharmaceutical Council (NPC)
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