Evaluation of FROM-16 in ATMP Patients & Families

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.

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Key information

Conditions

Family Members Cancer Disease Neoplasms Pathologic Processes Pathological Conditions, Signs and Symptoms

Sex eligibility

All sexes

Study type

Observational

Primary location

University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

FROM-16 Questionnaire

Other

A 16-item questionnaire to provide information about the impact of a patient's disease/condition the participants (family member of patient) quality of life

Semi-structured two-part interview

Other

A telephone/video interview to provide additional context to the FROM-16 responses

Primary outcomes

Validity of the FROM-16 to the ATMP population

Time frame: November 2021 - March 2022

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.

Sponsors and collaborators

Lead sponsor

Swansea University

Other

Collaborators

Cardiff University
Cardiff and Vale University Health Board
University Hospital Birmingham NHS Foundation Trust
University Hospitals Bristol and Weston NHS Foundation Trust
University Hospitals, Leicester
University of Hertfordshire

Registry information

Official study title

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

Important dates

Study start: 2021
Primary completion: 2022
Study completion: 2022
First posted: Dec 2, 2021
Registry last updated: Apr 28, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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