Analysis Group Co-Authors Examine the Advancements in and Application of Health Economics and Outcomes Research to the Evaluation of Cell and Gene Therapies for Rare Diseases

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5 days ago

BOSTON, July 22, 2025 /PRNewswire/ -- In a new article published in Value in Health, researchers from Analysis Group, a global leader in health economics and outcomes research (HEOR), collaborated with Novartis to examine how HEOR is transforming the evaluation and access pathways for cell and gene therapies (CGTs) for rare diseases. As CGTs move from experimental to increasingly approved treatments for rare diseases, health systems face urgent questions about how to evaluate, reimburse, and scale their access equitably. In their article, the authors spotlight five key HEOR innovation areas that are enhancing the generation of real-world evidence (RWE), supporting novel endpoints, and shaping future models of affordability and access.
Because of small patient populations, heterogeneous disease presentations, a lack of effective alternative treatments, and hence, ethical concerns, gold standard randomized double-blind controlled clinical trials are often not suitable for establishing efficacy and safety for CGT treatments. Consequently, traditional economic models are not able to capture the holistic value of CGTs, given that the true benefits associated with such treatments are subject to a much longer time outside of the trial periods. For example, for patients with rare hereditary genetic disorders, timely access to CGTs can yield real differences in meaningful gains in life years. To address these challenges, researchers are increasingly relying on innovations in HEOR to inform proper clinical and economic evaluation of CGTs.
An Analysis Group team led by Managing Principal Min Yang, Manager Su Zhang, and affiliate Lou Garrison collaborated with researchers from Novartis, led by Dr. Walter Toro, to examine the advancements in the evaluation of CGTs made possible by HEOR innovations, including the design and collection of long-term real-world data (RWD) for CGT efficacy extrapolation, justification for use of novel endpoints, integration of patient voices and preferences, and application of sophisticated statistical methodology, sometimes augmented with artificial intelligence (AI) or natural language processors. These advances are reflected in regulators' and health technology assessment (HTA) agencies' growing openness to the acceptance of RWE and patient-centered endpoints in their evaluations, marking a shift from traditional standards that may not suit CGTs. The authors note that emerging value assessment frameworks and novel reimbursement models offer solutions and adaptability to decision makers with enhanced ability to manage health equity and affordability.