Family members caring for relatives with severe disease can experience significant impacts, which are often unrecorded. Measuring these can help to understand the wider impact that a patient’s disease can have on their family and wider society, and therefore can help to demonstrate potential greater value of treatment. The FROM-16 is a validated tool for collecting family-reported outcome measures across a broad range of medical disciplines but has not been used previously to measure the unique impacts in the heterogenous group of patients receiving ATMPs and their families. Therefore, we aimed to validate the FROM-16 in this population.
When applied to family members of ATMP patients, the FROM-16 showed high reliability, through excellent internal consistency (Cronbach’s alpha = 0.917) and intraclass correlation (0.803). Confirmatory factor analysis showed only moderate fit to a two-factor model with a high RMSEA, only slightly improved in a four-factor model, which also showed a high RMSEA. A larger study may allow the generation of a more meaningful model.
Cognitive debriefing demonstrated high content validity. All participants reported that the FROM-16 was easy to complete, and non-burdensome. Most participants felt the FROM-16 addressed all areas of their life that had been impacted: One participant described the FROM-16 as “quite generic” and commented that it could be applicable even to a person who had just had a baby. Others also considered some items not to be relevant to their specific experiences. These comments reflect a specific design principle of the FROM-16, which is for it to be applicable to families of both adult and paediatric patients with a wide range of different conditions and illnesses and family circumstances as well as lived experiences. Therefore, we do not consider these views to reflect a limitation of the FROM-16 but rather a positive attribute: quality of life impact can be variable and is dependent on the current circumstances of the individual. The FROM-16 reflects the concerns reported by 130 family members/partners of patients from a wide range of medical specialties. It is therefore encouraging that a FROM that was conceptualised as a generic instrument was perceived as one.
The majority of interviewees attempted, at least once, to downplay the impacts they felt from their family member’s illness. Reasons for this were varied, such as not ascribing importance to the impacted area, or feeling as if the impact they feel was overshadowed by more important issues. Some participants also used coping strategies to lessen their perceived impact, which require additional mental or physical efforts from the family carer. We consider this to indicate a hidden or silent impact that could lead to ‘burnout’, and even depression in the family carer. This is a potential limitation of the FROM-16, which does not ask the family member to consider how important items are to them, regardless of the scale of the impact. Therefore, if using the FROM-16 to identify areas of unmet need in family carers, especially if the goal is to direct the family carer to appropriate support, consideration must be given to the importance that the family member ascribes to the impacted area of their life. This could be achieved through the routine use of a structured systematic assessment of family carers, including use of the FROM-16, to help detect hidden or silent impacts before they progress to greater future impacts.
This is highlighted additionally by participants reporting both positive and negative impacts across multiple areas of their lives (Table 2). The FROM-16 does not have a utility for qualifying impacts experienced and may encourage a user to consider negative impacts only. Family members must have an opportunity to discuss the nature of any impacts they experience during any routine monitoring.
Participants also reported neglecting their own health or struggling more with their own health issues as a result of their relative’s illness, which is also not measured by the FROM-16 (Table 4). Any routine assessment of family impact in ATMP patients should include a measure for tracking perceived impacts upon physical health of the family members, to bring this impact to light, and prevent it being part of the hidden wider impacts of disease. This will facilitate the allocation of support/resources to family members who require assistance and may prevent worsening impacts in the future.
Participants discussed that long-term planning, or the ability to “look to the future”, which they reported as having been given back to them as a result of the ATMP, also was not captured by the FROM-16 (Table 4). This suggests that when assessing impact in families of ATMP patients, an additional dimension or measure examining their perception on future outlook may be important.
This study has added to the pool of FROM-16 validation studies, with comparable scores to previous validation and development studies, however it is of interest to note that the scores reported in the current study are slightly higher (Table 1). The majority of patients recruited to this study had already received an ATMP treatment, so a reduced impact might be expected. However, family members reported ongoing impacts such as continuing to attend hospital appointments and uncertainty about the long-term treatment success as well as feelings of relief and being able to look to the future again as a direct result of the ATMP. This may explain the slightly higher total and domain scores shown in the current study. This suggests that the FROM-16 may be equally valuable in longer term monitoring; implementing FROM-16 as part of a routine monitoring system could be preferable for tracking family impacts of ATMP treatment.
Whilst the number of participants recruited to this study was small, we do not consider this to be strong limitation of the study. We sought to validate the FROM-16 in the ATMP patient population and identify any domains of unique impacts felt by the family members. Sufficient participant numbers were achieved to demonstrate validity of the FROM-16 in this population and saturation was reached during the interviews.