Points of View Ripple Effects of Pharmaceuticals on Caregivers' Quality of Life and Burden of Care From the Viewpoint of Outcome Evaluation

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Yosuke Nakano, Senior Researcher, Research Institute for Pharmaceutical Industry Policy
Mariko Hirozane, Researcher, Graduate School of Pharmaceutical Sciences, The University of Tokyo
Visiting Associate Professor, Graduate School of Pharmaceutical Sciences, The University of Tokyo
Associate Professor, Health and Social Medicine Unit, Faculty of Medicine, Yokohama City University
Igarashi, Naka

1. Introduction

In the previous issue of Policy Research Institute News No. ⁵⁹¹⁾, the authors investigated the UK NICE Guidance on Health Technology Evaluation, and confirmed that in addition to QALY and Cost, which are used to calculate ICER, an indicator of cost-effectiveness, there are other value factors that were specifically mentioned during the evaluation and came into consideration within and outside of cost-effectiveness. Among them, the evaluation of the improvement of QOL and the reduction of caregiver's burden on the caregivers themselves, such as family members of the patients, was considered to be very important from the viewpoint of social value. Therefore, in this paper, we conducted a survey and analysis of the current status of outcome evaluation and examples of outcome improvement in actual clinical trials on the ripple effects of drug use on caregivers' own QOL improvement and caregiver burden reduction. This paper focuses on caregivers at home, such as family members (hereafter referred to as "family caregivers"), rather than professional caregivers (caregivers, etc.).

2. current situation of caregiving by family members, etc.

It goes without saying that improvement of the patient's own medical condition and quality of life is important in drug treatment. However, family caregivers of patients with diseases or conditions that make it difficult for them to live independently and require support and care in their daily lives bear a significant physical and emotional burden, and are sometimes referred to as "hidden patients ^. The concept of caregiver burden was first defined as "the burden of caring for a loved one.

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

Specifically, family caregivers are greatly burdened by the fact that the family member they are currently caring for and supporting is not getting any better, by anxiety about the future of the disease, and by the fact that they have no free time for themselves because they spend too much time caring for the family member. In addition, such excessive caregiving burdens can eventually wear down the physical and mental health of caregivers, leading to mental fatigue and limitations, sometimes resulting in abuse of those in need of care, and even the breakdown of home care ^.3) The National Institutes of Health ⁽ NIH) has published a report on the effects of excessive caregiving burdens on the elderly and the mentally ill.

According to a ^report4) on the current status of family caregivers by country and disease, the percentages of diseases for which family caregivers actually provide care differ 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.

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 use of nursing homes, are available, and various studies have been conducted on the effects of these measures. However, it is said that there are still few studies on the ripple effects of drug treatment, such as the reduction of caregiving ^burden5).

In light of the above, this paper 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 cases of outcome improvement.

Investigation of outcome evaluation in clinical trials

3) Using the Clinical Trials Registry System (ClinicalTrials.gov) operated by the National Institutes of Health (NIH) and others, we investigated the number of trials and outcome measures that have outcome measures for family caregivers in clinical trials of pharmaceutical products.

The search conditions in the database were as follows: ⁶⁾

3-(1). Number of studies with caregiver outcome measures

A total of 26,145 clinical trials were extracted using the search condition a) (all clinical trials that matched the target period, intervention method, etc.). From the descriptions in the Outcome Measures of the 554 trials, we found that "caregiver surrogate" was the most common response when the patient was a child or had difficulty responding to the questions. The 554 studies were screened for 554 Outcome Measures. Finally, 66 trials (about 0.25% of the total) were identified as trials in which outcomes related to the caregivers themselves were assessed. 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 the EU5. Of these 14, 13 were global clinical trials.

3-(2). Comparison by target disease

Figure 2 shows the number of trials with caregiver outcome measures by target disease. Dementia accounted for the largest number of trials (27, or approximately 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 other diseases requiring mid- to long-term care and support from childhood were identified. In addition, from the viewpoint 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 UK NICE ^HST7) evaluation guidance, were also included.

3-(3). Comparison of outcome assessment tools

Outcome assessment tools listed in the Outcome Measures section were then extracted and shown in Figure 3. If more than one outcome measure was mentioned in a single 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 frequently used.

The ZBI is the most frequently used scale in Europe and the U.S. to measure caregiver burden, and the Japanese version of the ZBI, the J-ZBI, is also used in Japan. The ZBI has also been used for conditions other than dementia, although in many cases it has been used for dementia. Most of the outcome assessment tools listed in Table 2 evaluate the caregiver's burden level or QOL, but RUD-Lite, which surveys the use of medical resources (e.g., time required for care), and WPAI:CG, which surveys the caregiver's own labor productivity (absenteeism and presenteeism), were also used. were also used.

4. examples of improvement in outcomes such as caregiving burden

As a case study of improved outcomes for family caregivers' caregiving burden, we present the work of Kitten A et al. ¹⁶⁾ Kitten et al. organized and presented the results of clinical trials of Pimavanserin, a treatment for psychiatric symptoms associated with Parkinson's disease (hallucinations and delusions, etc.), One of these trials reported significant improvement 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 caregiving burden. Kitten et al. also noted that reducing caregiving burden is very important in that it may reduce the need for nursing home use.

In a phase III trial of aripiprazole for the treatment of irritability associated with childhood autism spectrum disorder, Owen et al ^. reported a significant improvement in caregiver burden compared with placebo (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.

Furthermore, in Japan, a prospective cohort study was conducted by Yamori et al. ⁵⁾. 5) Yamori et al. examined the effects 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, a significant improvement in caregiver burden was also observed (p-value < 0.001 for both). The EQ-5D, a comprehensive quality of life assessment tool, was used for caregiver QOL assessment in this study, and the ZBI and NPI-D were used for caregiver burden.

5. summary

In this paper, we focus on family caregivers and investigate the current status of outcome assessment of caregiver burden and other outcomes of drug interventions, as well as cases 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 were incorporated in actual clinical trials. In addition, the characteristics of diseases incorporating outcome measures for caregivers and frequently used outcome assessment tools were identified.

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

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

Although it is most expected that drug therapy will improve the patient's own disease condition, quality of life, and activities of daily living, and reduce or avoid conditions requiring nursing care, it is also 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, it may be important to reduce the burden of caregiving for family caregivers. For example, a reduction in the burden of caregiving on family caregivers may lead to an increase in the labor productivity of the caregivers themselves, and may even lead to a reduction in the use of nursing care facilities, which would have no small impact from the perspective of the overall economics of the social system. In fact, in Japan, it is estimated that approximately 100,000 people leave the workforce annually (as of 2017) ¹⁸⁾ due to caregiving. In addition, it is estimated that approximately 20% of the elderly (about 7 million people) will have dementia by ^202519), and 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. In view of such future issues and from the perspective of contributing to society as a whole, it may be meaningful to focus on the value of the ripple effects of pharmaceuticals on the QOL of caregivers and the burden of caregiving.

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