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

Overview

Journal Qual Life Res

Date 2025 Jan 25

PMID 39862357

Authors

Charles D Brilliant

Andrew Y Finlay

Sam M Salek

Rubina Shah

Emily Bacon

Hamish Laing

Affiliations

Soon will be listed here.

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 the impact on family of disease and treatment in ATMP patients may demonstrate additional value created by an ATMP intervention and strengthen the case for its use.

Methods: This feasibility study aimed to test the validity of the FROM-16 in family members of ATMP patients as a prelude for its use in ATMP value estimation. 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.

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