Long-Term Tracking of Clinical Effectiveness, Safety, and Value of Gene Therapies and Implications for EU Joint Clinical Assessment: Multistakeholder Perspectives From Patients, Manufacturers, and Payers

PURPOSE: Demonstrating long-term (LT) effectiveness, safety, and value of gene therapies necessitates linking data from diverse data sources, safeguarding data quality, privacy, and security, and minimizing burden to patients, providers, and manufacturers. When the LT value of gene therapies is demonstrated, it also creates a need to revisit past reimbursement decisions and adjust them into the future. From a multi-stakeholder’s perspective, this workshop will use real-time polling with an audience discussion about the opportunities, challenges, and policy implications, especially for the EU Joint Clinical Assessment (JCA).

DESCRIPTION: Gene therapies disrupt traditional treatment paradigms. The transformative nature of a potentially one-time treatment as the potential “next-gen therapy” creates the need to revisit how LT clinical effectiveness, safety, and value are demonstrated.

From a patient advocacy perspective, a use case by the World Federation of Hemophilia Gene Therapy Registry will demonstrate the value of incorporating technologies to maximize patient engagement in reporting LT data while minimizing the burden on patients. This creates the need to revisit the reliability and validity of current outcomes from a patient perspective and may re-shape regulatory and health technology assessment (HTAs) policies.

From a manufacturer’s perspective, challenges of collecting consistent LT real-world data (RWD) to support HTA reimbursement decisions for gene therapies will be discussed. Use cases of unique partnerships will illustrate approaches to improve real-world evidence generation using existing data infrastructures.

From a life-science service organization’s perspective, leveraging technologies to integrate RWD from active registries, patient-reported outcomes, electronic medical records, and claims data to support regulatory-grade RWE generation will be presented.

From a payer/HTA’s perspective, emerging LT RWD from gene therapies also creates the need to revisit past reimbursement/financing decisions and adjust these decisions and other relevant policies for the future. New methodologies will be needed and adapted to the unfolding EU JCA.