Cures for Life: Long-Term Follow-Up Data Collection for Cell and Gene Therapies

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Through significant advancements in biomedical innovation, new promising gene and cell therapy treatments may be the key to unlocking the cure to debilitating genetic, oncologic, and other diseases that were once deemed incurable. The first cell therapy treatment was approved in the US in 2017. Currently, four products are approved in the US, with six products approved in Europe. Moving forward, the US Food and Drug Administration (FDA) expects those numbers to grow exponentially.

Because currently approved gene and cell therapy treatments target rare diseases, the FDA does not require a large patient population, as it does for traditional drug approvals. This means a shorter time to market and earlier access for patients; but, it also means more uncertainties about the benefits of the treatment versus adverse effects. As a result, in some instances, the FDA requires a post-approval data collection for up to 15 years. Following patients for extended periods of time and collecting data on patient outcomes can help resolve many of the uncertainties presented by cell and gene therapies, but the mechanisms for data collection and the types of data collected can vary based on different stakeholders’ perspectives.

This report explores the data needs of different stakeholder groups, the strengths and limitations of current models for data collection, and future considerations relevant to each stakeholder group. Multi-stakeholder collaborations and partnerships are crucial and should strive to center patient preferences and unmet needs.

Updated/Published July 10, 2020