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.

Sex

All

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.

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