Clinical trial NCT05141318: Evaluation of FROM-16 in ATMP Patients & Families
Official title: Family Reported Outcomes in the Estimation of Societal Value of Advanced Therapy Medicinal Products (ATMPs)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Family Members
Cancer
Keywords: Family members
ATMPs
Family reported outcome measures
Value-based healthcare
Severe disease
Familial impact
Societal value
Advanced Therapy Medicinal Products
Lead sponsor: Swansea University
Sponsor class: OTHER
Collaborators: name: Cardiff University
name: University of Hertfordshire
name: Cardiff and Vale University Health Board
name: University Hospital Birmingham NHS Foundation Trust
name: University Hospitals, Leicester
name: University Hospitals Bristol and Weston NHS Foundation Trust
First posted: 2021-12-02
Enrollment: 18
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Validity of the FROM-16 to the ATMP population
timeFrame: November 2021 - March 2022
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust1
city: Cardiff
country: United Kingdom
facility: Cardiff & Vale University LHB
city: Leicester
country: United Kingdom
facility: University Hospitals of Leicester NHS Trust
city: Swansea
country: United Kingdom
facility: Swansea University School of Management
Study officials:
affiliation: Swansea University
name: Hamish Laing
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.