Family MembersNCT05141318
Evaluation of FROM-16 in ATMP Patients & Families
An observational study of Family Members and Cancer, run by Swansea University at 5 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Family Members, Cancer
- What is being tested?
- FROM-16 Questionnaire and Semi-structured two-part interview
- How many people
- 18 participants
- Where
- 5 sitesUnited Kingdom
- Study dates
- 9 Dec 2021to 31 Mar 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Show all 11Show fewer
- 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.
Who cannot join
- 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:
Show all 10Show fewer
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United Kingdom5 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2GW
University Hospitals Bristol and Weston NHS Foundation Trust1
Bristol, BS1 3NU
Cardiff & Vale University LHB
Cardiff, CF14 4HH
University Hospitals of Leicester NHS Trust
Leicester, LE1 5WW
Swansea University School of Management
Swansea, SA1 8EN
About this study
Official title: 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.
- Conditions
- Family Members, Cancer
- Being tested
- FROM-16 Questionnaire, Semi-structured two-part interview
- Keywords
- Family members, ATMPs, Family reported outcome measures, Value-based healthcare, Severe disease, Familial impact, Societal value, Advanced Therapy Medicinal Products
- Medical subject headings
- Neoplasms, Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FROM-16 QuestionnaireOther
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 interviewOther
A telephone/video interview to provide additional context to the FROM-16 responses
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: November 2021 - March 2022
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2021
- Study start9 Dec 2021
- Primary completion31 Mar 2022
- Study completion31 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Family MembersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swansea University
- Sponsor type
- University, hospital or other
- Collaborators
- Cardiff University, University of Hertfordshire, Cardiff and Vale University Health Board, University Hospital Birmingham NHS Foundation Trust, University Hospitals, Leicester, University Hospitals Bristol and Weston NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Hamish Laing, Principal investigator, Swansea University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05141318
- Sponsor's protocol ID
- WBS-ITT-43652
- Status last verified
- Nov 2021
- First submitted
- 22 Nov 2021
- Last updated
- 28 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to share IPD with other researchers outside of the study team
Publications
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.
PubMed 23797564Golics 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.
PubMed 23759884Golics 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.
PubMed 24092994Goula 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.
PubMed 33447311Chantarasap 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.
PubMed 30736795Basra 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.
PubMed 17300244Brittain 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.
PubMed 33430175
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Family Reported Outcomes in the Estimation of Societal Value of Advanced Therapy Medicinal Products (ATMPs)
- ClinicalTrials.gov ID
- NCT05141318
- Acronym / sponsor ID
- WBS-ITT-43652
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Swansea UniversityUniversity, hospital or other
- Collaborators
- Cardiff University, University of Hertfordshire, Cardiff and Vale University Health Board, University Hospital Birmingham NHS Foundation Trust, University Hospitals, Leicester, University Hospitals Bristol and Weston NHS Foundation Trust
- Study start
- 9 Dec 2021 (actual)
- Primary completion
- 31 Mar 2022 (actual)
- Study completion
- 31 Mar 2022 (actual)
- First posted
- 2 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (10 listed)
- 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 under 18 years of age.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FROM-16 QuestionnaireA 16-item questionnaire to provide information about the impact of a patient's disease/condition the participants (family member of patient) quality of life | Other | – |
| Semi-structured two-part interviewA telephone/video interview to provide additional context to the FROM-16 responses | Other | – |
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2023.
- Background: PubMed 23797564Golics 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.
- Background: PubMed 23759884Golics 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.
- Background: PubMed 24092994Golics 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.
- Background: PubMed 33447311Goula 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.
- Background: PubMed 30736795Chantarasap 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.
- Background: PubMed 17300244Basra 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.
- Background: PubMed 33430175Brittain 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.
- Description of Value-based healthcareLink provided by the sponsor.
- Individual participant dataSharing planned: No. No plan to share IPD with other researchers outside of the study team
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hamish Laing principal investigator
Swansea University
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Family Members and Cancer, run by Swansea University at 5 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Validity of the FROM-16 to the ATMP population (time frame: November 2021 - March 2022). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05141318, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2023.