Presentation (CTI)

OBJECTIVES: Gene therapies (GTs) offer the potential to substantially improve the lives of patients; however, evaluating their benefit from the patient perspective in clinical trials presents unique challenges. Although patient experience data is commonly suggested by regulators in approval applications, its inclusion in GT applications is limited. This research aims to summarize key challenges for GT trials and opportunities for using patient experience data.
METHODS: A review of select regulatory guidances and literature was conducted to identify measurement challenges related to GT evaluation. Based on the identified challenges, opportunities for integrating the patient voice into trials in alignment with scientific and regulatory best practices were summarized.
RESULTS: Key challenges identified for GT trials include: limited insights into natural history and disease progression for rare disease; small sample size; patient samples including children or vulnerable populations (e.g., patients may have physical or mental capacity limitations); existing clinical outcome assessments may not be suitable given the unique intersection of potential treatment benefit, adverse events, heterogeneous patient experience, and degenerative nature of many diseases; and complexities inherent to the experimental nature of GTs, e.g., accelerated approval (e.g., using surrogate endpoints) and need to understand long-term effects of treatment. Opportunities for using patient experience data to address some of the key challenges include conducting qualitative interviews with patients and/or caregivers as part of natural history and treatment intervention studies; careful selection or development of clinical outcome assessments based on research in the target population; and engaging patients post-approval to evaluate long-term effects and quality of life improvements.
CONCLUSIONS: Collecting patient experience data as part of GT development can provide important insights into disease experience and treatment benefit that is otherwise challenging to capture. These insights have the potential to help demonstrate the value of treatment and aid in the design of future studies.