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.
“CGTs are groundbreaking therapies for patients suffering from rare and serious diseases with few treatment options,” said Dr. Yang. “By understanding the unique complexities of rare diseases, our HEOR experts are playing a pivotal role in making these breakthrough therapies more accessible to patients.”
The researchers conclude that the use of HEOR has provided tools to enable a more holistic assessment of therapies like CGTs, inform pricing and reimbursement strategies, enhance the efficient delivery of CGTs to patients, improve health outcomes and affordability of treatments, and reduce treatment uncertainties for patients with rare diseases. They emphasize the continued need for collaboration among researchers, payers, and regulators to refine HEOR approaches that support both innovation and sustainability in rare disease care.
“Advances in CGTs have broader societal benefits beyond just those patients who receive such treatments, and these are not always well-quantified,” remarked Professor Garrison. “HEOR innovations will continue to be essential in the development of holistic assessments for CGTs, allowing us to better quantify less tangible – but real – benefits that emerge for patients and their families over time.”
The article, “Evolving Concept of Value in Health Economics and Outcomes Research: Emerging Tools for Innovation and Access to Cell and Gene Therapies for Rare Diseases,” was published by Value in Health in May. Lead author Dr. Walter Toro coauthored the study with Analysis Group researchers Dr. Min Yang and Dr. Su Zhang, Analysis Group affiliate and University of Washington Professor Lou Garrison, and Dr. Anish Patel and Dr. Omar Dabbous from Novartis. The study was funded by Novartis.