Feasibility Study Protocol: Investigating Family Reported Outcome Measures (FROMs) in the Estimation of Societal Value of Advanced Therapy Medicinal Products (ATMPs)

Abstract

Abstract Background Advance Therapy Medicinal Products (ATMPs) are a novel class of therapeutics that target chronic and severe conditions, which can have an impact beyond the patient themselves; impacting the lives of partners and other family members. This impact is a major secondary burden of disease that in the past has often been ignored. The Family-Reported Outcome Measure (FROM-16) is the first generic questionnaire designed to measure the impact of any disease on the quality of life of family members of patients with any health condition. We seek to validate the FROM-16 as an instrument for assessing family impact of patients receiving ATMPs and demonstrate a potential mechanism for measuring this impact. Methods Up to 30 patients of any age who are preparing for ATMP treatment, or who have received an ATMP treatment, and their family members aged 18 years or older, attending ATMP provider sites within the Midland-Wales Advanced Therapy Treatment Centre will be recruited into this study. Family members will be asked to complete the FROM-16 and Global Health Score questionnaires, provide basic demographic information and complete a short semi-structured interview conducted by a member of the study team. The interview will provide data to contextualise the FROM-16 responses and allow construct and content validity to be assessed. Themes of experience will be identified from interview transcripts. The FROM-16 responses will be evaluated with respect to total score, domain score, and individual item score. Reliability of responses between and within domains will be assessed by intraclass correlation. Discussion Collecting FROM data from families can be important for demonstrating the wider impact of disease. This study, if successful, will demonstrate that collecting family-reported outcomes using the FROM-16 is a valid mechanism for demonstrating this impact within the population of ATMP patients and their families, and will provide evidence of the overall quality of life impact experienced by partners and family members of such patients, and the specific impacts as identified from the two domains and 16 questions within FROM-16. Trial Registration This trial has been adopted onto the NIHR portfolio (I.D. 51376) and registered with https://clinicaltrials.gov/ct2/show/NCT05141318 (#NCT05141318) in December 2021.