Brilliant Charles D, Finlay Andrew Y, Salek Sam M, Shah Rubina, Bacon Emily, Laing Hamish
Value-based Health & Care Academy, School of Management, Swansea University, Swansea, SA1 8EN, UK.
Division of Infection and Immunity, Cardiff University School of Medicine, Cardiff, CF14 4XN, UK.
Qual Life Res. 2025 Apr;34(4):949-962. doi: 10.1007/s11136-024-03880-0. Epub 2025 Jan 25.
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.
基于结果的定价模型考虑了医疗保健领域以前未考虑的价值领域，如社会结果，这对医疗保健系统越来越有吸引力。社会结果可以包括家庭报告的结果指标（FROMs），用于衡量疾病对患者家庭成员的影响。FROM-16是一种通用且易于使用的家庭生活质量工具，但从未在接受先进治疗药品（ATMP）治疗的患者中使用过。由于成本高昂且符合条件的患者数量较少，潜在治愈性ATMP的使用受到限制。使用FROM-16收集ATMP患者疾病和治疗对家庭的影响，可能会证明ATMP干预创造的额外价值，并加强其使用的理由。
这项可行性研究旨在测试FROM-16在ATMP患者家庭成员中的有效性，作为其用于ATMP价值评估的前奏。患者及其家庭成员（n = 24）从英格兰和威尔士的ATMP治疗中心招募。家庭成员完成FROM-16，并被邀请参加简短的汇报访谈。
FROM-16显示出高有效性，内部一致性强（Cronbach's alpha = 0.917）和组内相关性高（0.803，95%）证明了这一点。访谈发现，虽然FROM-16涵盖了参与者经历的生活质量影响的大多数领域，但一些人解释说，他们在个人健康和未来前景方面也经历了其他影响。
这项可行性研究提供了证据，表明FROM-16可作为一种结构化系统方法的一部分，用于衡量ATMP患者的家庭生活质量影响。
