Validation of the FROM-16 in Family Members of Patients Receiving Advanced Therapy Medicinal Product (ATMP)

Abstract

Purpose: Outcome-based pricing models which consider domains of value not previously considered in healthcare, such as societal outcomes, are of increasing interest for healthcare systems. Societal outcomes can include family-reported outcome measures (FROMs), which measure the impact of disease upon the patient’s family members. The FROM-16 is a generic and easy-to-use family quality of life tool, but it has never been used in the context of patients undergoing advanced therapy medicinal product (ATMP) treatment. The use of potentially curative ATMPs is limited due their high cost and the low number of eligible patients. Using the FROM-16 to collect evidence of family impact in ATMP patients may demonstrate the potential value created by an ATMP intervention. Methods: This feasibility study aimed to test the validity of the FROM-16 in family members of ATMP patients. Patients and family members (n = 24) were recruited from ATMP treatment centres in England and Wales. Family members completed the FROM-16 and were invited to a short debriefing interview. Results: The FROM-16 showed high validity demonstrated by strong internal consistency (Cronbach’s alpha = 0.917) and intraclass correlation (0.803, 95%). Interviews identified that whilst the FROM-16 covered most areas of quality of life impact experienced by the participants, some explained that they also experienced other impacts upon their personal health and future outlook. Conclusion: This feasibility study provides evidence that the FROM-16 could be used as part of a structured systematic approach to measure family quality of life impact in ATMP patients.