CRISPR And The Commercialization Of Gene-Based Therapies: Learning From The Recent Past To Change The Near Future

By Andrew Kuhlman, MS, Clinical Research Methodology; Sean D. Kennedy, MPH, Real-World Evidence Strategy; and Michael F. Murphy, MD, PhD, Chief Medical and Scientific Officer

CRISPR-based gene editing techniques have the potential to revolutionize the development of gene-based therapies, offering groundbreaking possibilities for treating a wide array of diseases. However, the recent history of approved cell and gene therapies serves as a cautionary tale for pharmaceutical companies investing in CRISPR-based innovations. While gene-based therapies show tremendous promise, they also face significant barriers to access, such as high costs and reimbursement challenges imposed by payers.

Traditional health technology assessment methods often fall short when applied to these novel therapies, creating uncertainties and prompting the development of new approaches for evaluating their value. Payer coverage decisions are increasingly shaped by a comprehensive assessment of the therapy's value, considering factors like benefits, cost-effectiveness, affordability, budget impacts, and long-term outcomes.

To better determine the value of these therapies, advanced statistical techniques, such as survival modeling and multi-criteria decision analyses, have been proposed. Additionally, a variety of innovative payment plans are being explored to balance risks and maximize benefits. The importance of early collaboration between drug developers, manufacturers, and payers is becoming more evident, as it aids in the creation of meaningful clinical endpoints and the formulation of a viable market access strategy, ultimately facilitating the successful commercialization of these cutting-edge treatments.