American Institutes for Research To Help Implement Federal Program Aimed at Increasing Access to Transformative Cell and Gene Therapy Treatments

The American Institutes for Research (AIR) has been awarded a federal contract to help implement and monitor a new program that seeks to make transformative cell and gene therapy (CGT) treatments more accessible to Medicaid recipients. Under the $28 million contract from the Center for Medicare and Medicaid Innovation (CMMI) in the Centers for Medicare and Medicaid Services (CMS), AIR experts will support the implementation of the CGT Access Model.

CGTs are a growing class of one-time medicines designed to treat previously intractable diseases. The CGT Access Model seeks to test whether a CMS- and state-led approach to negotiating and administering outcomes-based agreements (OBAs) will improve access and health outcomes for people on Medicaid, reduce health care costs, and foster health equity.

The CGT Access Model will focus initially on Sickle Cell Disease (SCD), a genetic blood disorder that affects more than 100,000 people in the U.S., the majority of whom are Black. People with SCD have an average lifespan that is more than 20 years shorter than average life expectancy in the U.S., and often experience episodes of excruciating pain, which can cause multiple hospitalizations.

Through this contract, AIR will serve a pivotal role in program implementation and monitoring, and data development and analysis. AIR has extensive experience in the implementation of CMMI models and an institution-wide commitment to the advancement of health equity. Through this contract, AIR will:

• Monitor the implementation and outcomes of the CGT Access Model;
• Develop and implement a robust data collection and analysis plan to assess health outcomes and financial indicators; and
• Present at conferences to share findings and promote innovation.

Daniela Zapata, a principal researcher at AIR, will serve as the project director. AIR's partners in the project include Deloitte and Wally Smith, the Florence Neal Cooper Smith Professor of Sickle Cell Disease and Director, Adult Sickle Cell Program at Virginia Commonwealth University.

"The Cell and Gene Therapy Access Model has the potential to make groundbreaking treatments more widely available to those who need them and reduce long-term healthcare costs," said Timothy Hill, AIR senior vice president, who leads the institution's Health Division. "We look forward to working with CMS and our partners to test innovative solutions that can improve people's lives and create a more equitable world."