# Validation of the FROM-16 in family members of patients receiving advanced therapy medicinal product (ATMP)

## TL;DR

This study tests if the FROM-16 tool can effectively measure family quality of life impacts for patients receiving advanced therapies.

## Contribution

Demonstrates the FROM-16's validity for ATMP patients' families, a novel application of the tool.

## Key findings

- FROM-16 showed strong internal consistency (Cronbach’s alpha = 0.917) and reliability (intraclass correlation = 0.803).
- Participants felt the FROM-16 captured most quality-of-life impacts, though some noted additional unmeasured effects.

## Abstract

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.

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.

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.

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.

The online version contains supplementary material available at 10.1007/s11136-024-03880-0.