Unlocking Market Access: HEOR strategies for cell and gene therapies in the United States

This microcourse was developed in collaboration with experts from Cencora, an ISPOR Mission Partner organization.

Cell and gene therapies (CGTs) are transforming treatment paradigms for serious and rare diseases, but they also introduce new complexity across the market access landscape. This course explores how Health Economics and Outcomes Research (HEOR) professionals can help address payer uncertainty, demonstrate value, and support sustainable access to these innovative therapies. Learners will examine the unique market access challenges associated with CGTs, including limited data at approval, high upfront costs, evolving payer evidence expectations, and the need for long-term real-world evidence. Through practical examples and interactive activities, the course highlights strategies for evidence generation, payer engagement, value communication, and innovative reimbursement approaches. By connecting clinical, economic, and real-world evidence to strategic communication tools such as AMCP dossiers and pre-approval information exchange, this course prepares learners to contribute meaningfully to successful market access strategies for cell and gene therapies.

After the completion of this course participants will be able to:

• Describe the key market access challenges associated with cell and gene therapies, including payer concerns related to uncertainty, durability of response, and economic impact.
• Explain evolving payer expectations for evidence generation, including clinical, economic, and real-world data needs throughout the product lifecycle.
• Identify HEOR strategies used to support payer decision-making, including economic modeling, real-world evidence generation, and patient-reported outcomes.
• Differentiate between key value communication tools, including AMCP dossier types and pre-approval information exchange (PIE), and their roles in supporting payer engagement.
• Apply practical approaches for communicating the value proposition of cell and gene therapies to payers and other healthcare decision-makers.
• Recognize innovative pricing and reimbursement models used to address the financial and access challenges associated with high-cost transformative therapies.

LEVEL: Intermediate TRACK: Health Policy & Regulatory HEOR Competency: 1.2 Pricing, Reimbursement, and Access HEOR Competencies: A resource for HEOR Professionals

The access period once enrolled into the course is 30 days from the date of purchase. After this duration, you will need to re-enroll if you wish to re-enter the course.

Time to complete – approximately 1 hour