Advancements in Health Economics and Outcomes Research for Gene Therapies in Rare Diseases

In recent developments, the field of health economics and outcomes research (HEOR) is making waves in the medical community, especially regarding cell and gene therapies (CGTs) for rare diseases. The collaborative efforts of scholars from Analysis Group and pharmaceutical giant Novartis have shed light on the vital role HEOR plays in addressing the challenges related to evaluating and providing access to these innovative therapies.

As CGTs transition from experimental stages to approved treatments, healthcare systems are confronted with pressing issues on how to assess their effectiveness, set reimbursement rates, and ensure equitable access for all patients. The article published in Value in Health emphasizes the need for innovative approaches in HEOR to overcome these hurdles. The authors highlight five key areas of HEOR innovation that are critical in generating real-world evidence (RWE), which supports new endpoints and informs future models of affordability and access for CGTs.

A significant challenge faced by CGTs for rare diseases stems from the limited size of patient populations and the variability of disease presentations. Traditional randomized double-blind controlled trials may not be suitable to determine the efficacy and safety of these treatments due to a lack of adequate alternative therapies. Consequently, conventional economic models fall short in capturing the comprehensive value of CGTs. For instance, for patients with rare hereditary conditions, early access to CGTs can lead to considerable gains in life expectancy, emphasizing the need for a different evaluative framework.

Led by Managing Principal Min Yang, the Analysis Group team worked with Novartis' researcher Dr. Walter Toro to explore HEOR advancements that facilitate the assessment of CGTs. This includes the design and collection of long-term real-world data for efficacy extrapolation, justifying the use of novel endpoints, and incorporating patient preferences into the evaluation process. The integration of advanced statistical methodologies, sometimes enhanced by artificial intelligence (AI), further enriches the research's insights, demonstrating a shift towards a more patient-centered approach in evaluations.

With regulators and health technology assessment (HTA) agencies becoming increasingly receptive to RWE and patient-centered endpoints, there is a clear movement away from traditional standards that may not be relevant for CGTs. The authors of the study note that new value assessment frameworks and reimbursement models provide flexibility for stakeholders, enabling a better management of health equity and treatment affordability.

Dr. Yang points out that CGTs are revolutionary therapies for individuals facing rare and serious illnesses, highlighting the critical function of HEOR experts in making these state-of-the-art treatments more accessible. The study posits that utilizing HEOR tools allows for a more comprehensive evaluation of CGTs, aiding the development of pricing and reimbursement strategies while enhancing health outcomes and affordability for patients with rare diseases.

The importance of collaboration between researchers, payers, and regulators is crucial, as emphasized by Professor Lou Garrison. He remarks on how advances in CGTs benefit not only patients but society at large, suggesting that HEOR innovations are essential for developing assessments that capture these broader societal impacts. Through this evolving process, researchers aim to quantify both tangible and intangible benefits that accrue over time for patients and their families.

The article, titled "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 in May and stands as a collaborative effort among esteemed researchers from both Analysis Group and Novartis, funded by Novartis.

For further insight into Analysis Group’s capabilities in health economics and outcomes research, visit Analysis Group’s website.