Policy Research Institute page Ripple Effects of Pharmaceuticals on Caregivers' Quality of Life and Burden of Care From the Viewpoint of Outcome Evaluation

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In Policy Research Institute News No. 59*1, we investigated the National Institute for Health and Clinical Excellence (NICE) Guidance on Health Technology Evaluation in the UK, and confirmed that in addition to QALY and Cost, which are used to calculate the incremental cost-effectiveness ratio (ICER), which is a measure of cost-effectiveness, there are other value factors. Among them, the evaluation of the improvement of the QOL (Quality of Life) of the patient's family members and other caregivers themselves and the reduction of the burden of caregiving were considered to be very important from the viewpoint of social value. Therefore, this paper focuses on the ripple effects of the use of pharmaceuticals, such as improvement of caregivers' own quality of life and reduction of caregiving burden, and conducts a survey and analysis of the current status of outcome evaluation in actual clinical trials and examples of outcome improvement. This report focuses on caregivers at home, such as family members (hereafter referred to as "family caregivers"), rather than professional caregivers (caregivers, etc.).

Current status of caregiving by family members and others

It goes without saying that improvement of the patient's own medical condition and quality of life is important in drug treatment. However, when a patient has a disease or condition that makes it difficult for him or her to recuperate and live independently and requires support and care in daily life, the physical and emotional burden on the patient's family caregivers can be significant, and they are also called "hidden patients. 2 Improving the quality of life of family caregivers and reducing the burden of caregiving are important aspects.

Zarit was the first to define the concept of caregiving burden, defining it as "the degree of damage suffered by a caregiver as a result of caring for a relative in terms of emotional and physical health, social life, and economic status.

Specifically, family caregivers are burdened by the fact that the family member they are currently caring for is not getting any better, that they are worried about the future of the disease, and that they have no free time for themselves because they spend too much time caring for the family member. In addition, the excessive burden of caregiving can wear down the physical and mental health of caregivers, eventually leading to mental fatigue and limitations, sometimes resulting in patient abuse, and even the breakdown of in-home care*3.

According to a report*4 surveying the current status of family caregivers by country and disease, the percentage of diseases for which family caregivers actually provide care differs among Japan, the United States, and the United Kingdom (Table 1). In particular, Japan, a super-aged society, has a higher percentage of people caring for dementia patients than other countries. In addition, although diseases that are common among the elderly are at the top of the list, a certain percentage of diseases that develop in adulthood and childhood also cover a certain percentage of the population. Furthermore, the survey reports that family caregivers' health-related quality of life and depressive symptom scores are worse than those of non-caregivers.

Table 1: Percentage of diseases for which family members provide care (%)
Note: Diseases overlap
Source: Modified from "CROSS-COUNTRY PROFILE OF ADULT CAREGIVERS" *4, ISPOR Europe 2015 poster presentation by KANTAR HEALTH

There are various measures to improve the QOL of family caregivers and reduce the burden of caregiving, such as education on caregiving, support by care managers, and the use of nursing homes. However, studies on the ripple effects of drug treatment in reducing the burden of caregiving are said to be still scarce*5.

In light of the current situation described above, this report investigates and reviews the current status of outcome evaluation of caregiving burden and other outcomes of drug intervention, with a particular focus on family caregivers, as well as examples of improved outcomes.

Investigation of outcome evaluation in clinical trials

Using ClinicalTrials.gov, a clinical trial registration system operated by the National Institutes of Health (NIH) and others, we surveyed the number of trials and outcome measures that have established outcome measures for family caregivers in pharmaceutical clinical trials.

The search conditions in the database were as follows*6 (This search condition is limited in comprehensiveness because it does not specifically define and search for all outcome assessment tools targeting caregivers.)

(1) Number of studies with caregiver outcome measures

The total number of clinical trials extracted using search condition a) (all clinical trials that matched the time period covered, intervention method, etc.) was 26,145. From the 554 trials, we excluded those that were "proxy assessments by caregivers (not a survey of the caregiver's own situation)" in cases where the patient was a child or had difficulty responding to the survey. (not a survey of the caregiver's own situation)" in cases where the patient was a child or had difficulty responding to the survey. Finally, 66 trials (about 0.25% of the total) were identified as trials in which outcomes related to the caregivers themselves were evaluated. There was no significant change in the annual trend over the last 10 years (Figure 1). Comparing the countries where clinical trials were conducted, there were 14 fewer trials conducted in Japan than in the U.S. and five European countries (U.K., Germany, France, Italy, and Spain), and 13 of these 14 trials were global clinical trials.

Figure 1: Annual changes in the number of trials including outcome assessment of caregivers themselves and comparison of countries where trials are conducted
*Europe 5: 5 countries: UK, Germany, France, Italy, and West
Source: Prepared by the author based on ClinicalTrials.gov data

(2) Comparison by target disease

Figure 2 shows the number of trials with caregiver outcome measures by target disease. Dementia was the most common (27 cases, or about 40% of the total), followed by atopic dermatitis, Rett syndrome, and autism. From the perspective of those requiring care, not only dementia, which is primarily cared for by the elderly, but also diseases requiring medium- to long-term care and support from childhood were identified. In addition, from the perspective of pharmaceuticals, there was a trend toward diseases in specialty areas. Furthermore, rare and intractable diseases (e.g., mucopolysaccharidosis, muscular dystrophy), which were also covered by the HST*7 evaluation guidance of the UK NICE, were included.

Fig. 2 Number of trials by target disease
Source: Prepared by author based on ClinicalTrials.gov data

(3) Comparison of outcome assessment tools

Outcome assessment tools listed in the endpoints were then extracted and shown in Figure 3; when multiple outcome assessment tools were listed in one study, each was counted, for a total of 70. The most commonly used tool was the Zarit Burden Interview (ZBI), created by Zarit as described above. The Neuropsychiatric Inventory (NPI-D) and Resource Utilization in Dementia - Lite Version (RUD-Lite) were the next most common.

Fig. 3 Number of studies by outcome assessment tool
Source: Prepared by author based on ClinicalTrials.gov data

In addition, Table 2 summarizes the characteristics, etc. of each outcome assessment tool that was confirmed to be used in two or more cases. the ZBI is the scale most frequently used in Europe and the United States to measure care burden, and the Japanese version of the ZBI, the J-ZBI, is also used in Japan. The ZBI was also used for diseases other than dementia, although in most cases it is used for dementia. Most of the outcome assessment tools listed in Table 2 are used to evaluate the burden level or QOL of caregivers, but RUD-Lite, which surveys the use of medical resources (e.g., time required for care), and WPAI:CG, which surveys the labor productivity (absenteeism and presenteeism) of the caregivers themselves, were also used. The RUD-Lite was used to survey the caregivers' own labor productivity (absenteeism and presenteeism).

Table 2 Summary of outcome assessment tools for caregivers
Source: Authors' compilation based on information in each source.

Case examples of improvement in outcomes such as caregiving burden

Kitten A et al. One of these trials reported significant improvements in patients' SAPS-PD scores, the primary endpoint, as well as in patients' sleep quality and family caregivers' caregiving burden (p value 0.0016) (Figure 4). (Figure 4). Note that the ZBI was used as the outcome assessment tool for caregiver burden. Kitten et al. also noted that reducing caregiver burden is very important in that it may reduce the need for nursing home use.

Figure 4: Pimavanserin Clinical Trial Results
Source: pimavanserin: A Novel Drug Approved to Treat Parkinson's Disease Psychosis. Innov Clin Neurosci, Feb 1;15(1-2):16-22. 2018

In another example of improved outcomes, Owen R et al.*17 reported that in a phase III trial of aripiprazole for the treatment of irritability associated with childhood autism spectrum disorder, they found a significant improvement in caregiver burden compared to the placebo group (between-group difference -1.9 [95% confidence interval = -2.7, -1.2], p value not available). The Caregiver Strain Questionnaire (CGSQ) was used as the outcome assessment tool in this study.

In addition, a prospective cohort study conducted in Japan by Jun Yamori et al.*5 examined the effect of donepezil on the quality of life of first-time patients diagnosed with Alzheimer's disease and their caregivers. The results of their study showed that not only patient QOL but also caregiver QOL improved significantly during a 14-week pre- and post-treatment comparison, and in addition, significant improvements in caregiver burden were also observed (p-values < 0.001 for both). The EQ-5D, a comprehensive quality of life assessment tool, was used to assess caregiver quality of life in this study, and the ZBI and NPI-D were used to assess caregiver burden.

Summary

In this paper, we focus on family caregivers, and investigate and review the current status of outcome assessment of caregiver burden and other outcomes in pharmaceutical interventions, as well as examples of outcome improvement. First, a survey through ClinicalTrials.gov, a clinical trial registration system, confirmed that outcome measures such as quality of life improvement and caregiver burden for family caregivers are incorporated in actual clinical trials. Furthermore, we were able to confirm the characteristics of diseases that incorporate outcome measures for caregivers and the outcome assessment tools that are frequently used.

On the other hand, the current number of diseases and clinical trials that use outcome measures such as QOL improvement and caregiver burden for family caregivers is limited, and we need to examine the need to disseminate comprehensive QOL evaluation tools commonly used in medical technology evaluation, and how to evaluate the value of the outcomes obtained, The issue is that long-term data and knowledge on the effects of improvements in caregiving burden, etc., are not yet fully available.

Although the aforementioned ZBI scale has become somewhat popular, it is difficult to say that a standard indicator has been established for the evaluation scale of care burden itself. In order to promote consideration of not only qualitative but also quantitative care burden in the future, it is desirable to establish some standard evaluation indicators (identification of standard indicators among existing indicators and development of new, highly versatile indicators).

The most expected outcome of drug therapy is to improve the patient's own disease condition, quality of life, and activities of daily living, and to reduce or avoid conditions that require nursing care. We believe it is important from a social perspective to focus on the effect of reducing the burden not only on the patient but also on the family caregivers who provide care and support for the patient. For example, reducing the burden of caregiving on family caregivers may lead to improved labor productivity of the caregivers themselves, and may even lead to a reduction in the use of nursing care facilities, which has no small impact from the economic perspective of the entire social system. In fact, it is estimated that about 100,000 people leave the workforce annually in Japan (as of 2017)*18 due to nursing care, and it is also estimated that about 20% of the elderly (about 7 million people) will suffer from dementia by 2025*19, so the number of family caregivers is also expected to increase. Especially in Japan, where the rapid decline in the working-age population has been recognized as a problem, it will be increasingly necessary to consider ways to improve the quality of life of family caregivers, who are also expected to play a valuable role as workers, and to reduce the burden of caregiving. With such future issues in mind, it would be meaningful to focus on the value of the ripple effects of pharmaceuticals on caregivers' QOL and caregiving burden from the perspective of contributing to society as a whole.

( Yosuke Nakano, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute)

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