Evaluation of FROM-16 in ATMP Patients & Families

Family Reported Outcomes in the Estimation of Societal Value of Advanced Therapy Medicinal Products (ATMPs)

Poor health, and its treatment, has impact beyond the healthcare system into wider society. A person's productivity, taxable earnings, benefit payments and community contribution may all be adversely affected by poor health. Family members living with or caring for a patient may suffer equally, or sometimes more than the patient themselves, but this familial burden has gone largely unrecognised by healthcare systems. The Family-Reported Outcome Measure (FROM-16) is the first generic questionnaire designed to measure the impact of any chronic disease on the quality of life of family members or partners of patients with a health condition. Advance Therapy Medicinal Products (ATMPs) are a novel & ground-breaking therapeutic approach for curative treatment of disease and/or injury where conventional treatments have been ineffective. Such disease/injury generally has an extremely high impact on the patient's quality of life, and also the quality of life of the patient's family, in particular those family members who take on the role of 'informal carer'. ATMPs usually have very high costs and this can limit their usage, especially in the context of low prevalence disease and publicly-funded healthcare systems, where healthcare providers may be reluctant to take on the cost burden of the ATMP therapy. As a result, there is a particular focus on the 'value' of ATMPs. An important component of value is 'Societal Value', where a treatment leads to societal contributions, and considering Societal Value may justify the high cost of ATMPs despite the relatively few patients cured. In this study, we will validate the FROM-16 for use as one measure of the Societal Value of very high cost, potentially curative treatments such as ATMPs.

Study Overview

• Status: Completed
• Conditions: Cancer; Family Members
• Intervention / Treatment: Other: FROM-16 Questionnaire; Other: Semi-structured two-part interview

• Study Type: Observational
• Enrollment (Actual): 18

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B15 2GW
    • University Hospitals Birmingham NHS Foundation Trust
  • Bristol, United Kingdom, BS1 3NU
    • University Hospitals Bristol and Weston NHS Foundation Trust1
  • Cardiff, United Kingdom, CF14 4HH
    • Cardiff & Vale University Lhb
  • Leicester, United Kingdom, LE1 5WW
    • University Hospitals of Leicester NHS Trust
  • Swansea, United Kingdom, SA1 8EN
    • Swansea University School of Management

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Family members of patients, of any age, with diagnosed long-term diseases who are being prepared for ATMP therapy, or have completed an ATMP therapy, in ATMP provider centres primarily within the extended Midlands-Wales Advanced Therapy Treatment Centre (ATTC) network.

Description

Inclusion Criteria:

Patients:

• Patients of any age
• Ability to read English or Welsh (where age-appropriate).
• Capacity to give written informed consent, or assent for their family member to participate
• Attending ATMP treatment centre with a family member/carer.
• Being considered for, or have recently received, ATMP therapy for any diagnosed condition.

Family Members:

• Considered by the patient to be the person most affected by the patient's condition
• Adult family members aged 18 years or older.
• A family member or partner, living with or caring for, a patient who is being prepared to receive ATMP treatment or has recently received ATMP therapy for any diagnosed condition.
• Have capacity to give written informed consent and complete the interview and questionnaires.

Exclusion Criteria:

Patients:

• Adult patients: Lacking capacity, or unwilling to give written informed consent for their family member to participate.
• Gillick competent paediatric patients: unwilling to give written informed assent and/or consent for their family member to participate.
• Non-Gillick competent paediatric patients: patient's parent/guardian refuses to give consent or is unable to give consent on their behalf.
• Not being prepared for ATMP therapy, or is currently receiving ATMPs.

Family Members:

• Family members under 18 years of age.
• Not considered by the patient to be a family member or carer.
• Lacking capacity or willingness to give informed written consent to participate
• Having a severe handicap or disability that prevents the completion of interview and questionnaires.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Validity of the FROM-16 to the ATMP population	Successful outcomes will be defined by this study demonstrating that the FROM-16 is a suitable tool for gathering FROMs from ATMP patient family members, through showing high reliability of the responses to the FROM-16, and showing that the FROM-16 is valid when applied to this population. Reliability, assessed by Cronbach's Alpha, should have scores in excess of 0.8, indicating high internal consistency. Validity will be assessed in three dimensions: Face, Construct and Content Validity. Face validity will be assessed by an experimenter independent to the data analytics and collection processes. Content validity will be assessed by thematic analysis of interview transcripts, and by assessing the intraclass correlation of themes. Construct validity will be assessed by measuring convergent and discriminatory validity by confirmatory factor analysis.	November 2021 - March 2022

Collaborators and Investigators

• Sponsor: Swansea University
• Collaborators: Cardiff University; University Hospitals, Leicester; University Hospital Birmingham NHS Foundation Trust; Cardiff and Vale University Health Board; University Hospitals Bristol and Weston NHS Foundation Trust; University of Hertfordshire
• Investigators: Principal Investigator: Hamish Laing, Swansea University

Publications and helpful links

General Publications

• Basra MK, Sue-Ho R, Finlay AY. The Family Dermatology Life Quality Index: measuring the secondary impact of skin disease. Br J Dermatol. 2007 Mar;156(3):528-38. doi: 10.1111/j.1365-2133.2006.07617.x. Erratum In: Br J Dermatol. 2007 Apr;156(4):791.
• Golics CJ, Basra MK, Finlay AY, Salek S. The development and validation of the Family Reported Outcome Measure (FROM-16)(c) to assess the impact of disease on the partner or family member. Qual Life Res. 2014 Feb;23(1):317-26. doi: 10.1007/s11136-013-0457-y.
• Golics CJ, Basra MK, Finlay AY, Salek S. The impact of disease on family members: a critical aspect of medical care. J R Soc Med. 2013 Oct;106(10):399-407. doi: 10.1177/0141076812472616. Epub 2013 May 10.
• Golics CJ, Basra MK, Salek MS, Finlay AY. The impact of patients' chronic disease on family quality of life: an experience from 26 specialties. Int J Gen Med. 2013 Sep 18;6:787-98. doi: 10.2147/IJGM.S45156. eCollection 2013.
• Goula A, Gkioka V, Michalopoulos E, Katsimpoulas M, Noutsias M, Sarri EF, Stavropoulos C, Kostakis A. Advanced Therapy Medicinal Products Challenges and Perspectives in Regenerative Medicine. J Clin Med Res. 2020 Dec;12(12):780-786. doi: 10.14740/jocmr3964. Epub 2020 Dec 18.
• Chantarasap P, Johns NP, Pairojkul S, Sookprasert A, Wirasorn K, Cheawchanwattana A, Salek S, Subongkot S. Validation of the Thai version of the family reported outcome measure (FROM-16)(c) to assess the impact of disease on the partner or family members of patients with cancer. Health Qual Life Outcomes. 2019 Feb 8;17(1):32. doi: 10.1186/s12955-019-1091-3.
• Brittain E, Muirhead N, Finlay AY, Vyas J. Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS): Major Impact on Lives of Both Patients and Family Members. Medicina (Kaunas). 2021 Jan 7;57(1):43. doi: 10.3390/medicina57010043.

Helpful Links

• Description of Value-based healthcare

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2021
• Primary Completion (Actual): March 31, 2022
• Study Completion (Actual): March 31, 2022

Study Registration Dates

• First Submitted: November 22, 2021
• First Submitted That Met QC Criteria: December 1, 2021
• First Posted (Actual): December 2, 2021

Study Record Updates

• Last Update Posted (Actual): April 28, 2023
• Last Update Submitted That Met QC Criteria: April 27, 2023
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Keywords: Value-based healthcare; Family members; ATMPs; Family reported outcome measures; Severe disease; Familial impact; Societal value; Advanced Therapy Medicinal Products
• Other Study ID Numbers: WBS-ITT-43652

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: No plan to share IPD with other researchers outside of the study team

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No