Although pharmacoeconomic studies are used for policy decision-making such as reimbursement and pricing decisions in several industrialized countries, they have not yet been used in an explicit way in Japan. In this article, the general methodology to interpret the results of cost-effectiveness analyses, the present situation of the usage of those analyses in the government policy of other countries, and the potential and problems of their application to policy making in Japan, are discussed.
It should be noted that other factors are considered in the decision-making process, such as clinical need, equity of access, the “rule of rescue”, and the total cost to the health care system. However, it is inappropriate to ignore the viewpoint of cost-effectiveness with regards to the budget constraint. The establishment of guidelines for the submission of pharmacoeconomic studies is necessary to ensure their validity, which leads to improved transparency of the process of health policy making.

Objective: In this work the cost-effectiveness of smoking cessation in Japan was investigated. Five groups of subjects, simulating cohorts wishing to quit smoking, were chosen as follows: (i) naturally quitting smoking, (ii) nicotine patch OTC medicine ( OTC patch), (iii) nicotine gum OTC medicine ( OTC gum), (iv) prescribed nicotine patch ( prescribed patch), and (v) prescribed bupropion.
Design: A Markov model study
Method: Using a Markov model, the cost per life-year saved associated with pharmacotherapy for quitting smoking cohorts of 30 · 40 · 50-year-old men and women was estimated. The expenses associated with smoking cessation were calculated based on either current retail prices of the medications in a community pharmacy practice or a fee schedule ; the costs and benefits were discounted by 3 % annually. Sensitivity analyses for the treatment effectiveness were performed.
Results: The incremental cost per life-year saved for the OTC patch method ranged from 518,826 yen to 652,282 yen for men and from 351,317 yen to 725,109 yen for women. For the case of the OTC gum the values were 871,442 yen to 1,205,142 yen for men and 592,558 to 1,282,263 yen for women. The prescribed patch values ranged from 504,373 yen to 603,371 yen for men and 340,734 yen to 685,626 yen for women. The corresponding values for prescribed bupropion were 562,564 yen to 670,768 yen for men and 379,960 yen to 763,283 yen for women. Sensitivity analyses indicated that changes in the treatment effectiveness had the strong influence on cost-effectiveness.
Conclusions: Smoking cessation utilising pharmacotherapy in Japan was found to be cost-effective in comparison with natural methods of quitting smoking. OTC patch and prescribed patch were the two most cost-effective treatments.

The interest has been officially growing in introducing health economics approach to the issues of pricing and reimbursement for new drugs and medical devices. In the Social Security Reform Plan of the Cabinet Office, the Ministry of Health, Labor, and Welfare proposed an idea for further investigating a role of socio-economic evaluation to assess the innovation of medical technology. To address this new circumstance, the government must develop and implement an official guideline for economic evaluation of medical technology. Responding to the emerging need for a socio-economic evaluation guideline, we illuminate an outline of the socio-economic evaluation guideline drafted by experts' consensus and proposed to Ministry of Health, Labor, and Welfare in March 2007. Also the potentials and the issues to substantiate the guideline by government were identified for future discussions between government, academics and industry.

Health economics evaluations are affected by uncertainty when estimating their parameters. Therefore, it is important that we use a sensitivity analysis to determine the robustness of these evaluations. Most countries' guidelines recommend using a probabilistic sensitivity analysis (PSA), which enables us to evaluate the uncertainty of multiple parameters at the same time, based on a joint probability distribution. In this article, we first introduce the Monte Carlo simulation and Bootstrap method as PSA methods. Then, we review how various guidelines incorporate the PSA. Finally, we review Japanese health economics studies to determine the level of PSA use in Japan. Guidelines published before 2008 recommend conducting a sensitivity analysis, but do not specify a method. In contrast, both the French, American and English guidelines, which were published after 2011, specifically recommend using a PSA. In Japan, the “Guideline for economic evaluation of healthcare technologies in Japan” recommends conducting a PSA, “if possible”. However, PSA methods are not widely used in Japan. Of 49 Japanese health economics studies based on quality-adjusted life years, only six conducted a PSA (12.2%), although 35 (71.4%) did conduct other types of sensitivity analyses. If PSA methods are accepted as a good way to determine the robustness of an evaluation, then we need to foster their use. This, in turn, means we need specific guidelines on how best to use these methods.

As the Japanese health insurance system is reimbursed on a fee-for-service basis, the claims data are useful sources for quantitative analysis on practice and prescription patterns as well as the health care resource use. However, the claims database has limitations such as incomplete description of the patients' condition and severity, and the lack of prescription data for low-priced drugs.
For use in pharmacoepidemiologic studies, patient information from medical charts, prescription forms, and/or hospital information systems may need to be included to compensate for the limitations of the claims data.

Objective: To explore the data sources used in economic evaluations performed on new drugs, and to propose an improved data infrastructure in Japan.
Design: A systematic review.
Methods: We systematically reviewed economic evaluation studies of the new drugs which were launched in Japan between April 2006 and March 2011, and have been priced by the cost calculation method. The “Ichushi” and Pubmed databases were used to find the published articles.
Results: 198 drugs were priced under cost calculation methods in the last 5 years in Japan. 14 published articles (9 drugs) were found: 5 CUAs and 10 CEAs (including one that was both CUA and CEA). In all studies, several data from different sources were incorporated. Cost data were estimated by using standard treatment protocols and national price lists for drugs and medical services, or obtained from limited number of claims data. Efficacy data were obtained from RCTs or clinical trial data mostly conducted in Japan. In 4 out of the 5 CUAs, utility data were used from other studies conducted on non-Japanese samples. Other data, such as epidemiological data, were adopted from overseas as well as Japanese studies.
Conclusion: In order to increase quality and efficiency to conduct economic evaluations in Japan, three steps need to be taken in the data environment: increased accessibility to large cost databases such as the national claims database; establish an epidemiological database; and collect and accumulate utility data in Japanese samples.

Acetaminophen is commonly used as the global standard of analgesics. For example, the WHO lists acetaminophen as an essential drug and various clinical guidelines in many countries include acetaminophen as a first-line drug for pain relief because of it's efficacy and safety profile. In particular, there is not significant risk of such as gastrointestinal disorders, renal dysfunctions, bleeding, or cardiovascular events, and it is considered to be a safer option than non-steroidal anti-inflammatory drugs(NSAIDs). In Japan, however, NSAIDs are widely used to treat pain while the use of acetaminophen for pain relief is quite limited. This difference could be attributed to the low approved dose of acetaminophen in Japan, which is less than half of that used elsewhere. This lower approved dose causes difficulty in obtaining analgesic effect with acetaminophen. In January 2011, however, the approved dose of acetaminophen in Japan was increased to the world standard dose, making it easier to obtain an analgesic effect. In the near future, an increase in the use of acetaminophen for pain relief can be expected in Japan. NSAIDs are common drugs for pain in Japan, but often require co-prescription of a gastric mucosal protective agents, H₂- blockers, or proton pump inhibitors(PPI) to prevent gastrointestinal disorders. On the other hand, acetaminophen has much less risk of such adverse reactions and there is no need for co-prescription of digestive medicines. Thus, increased use of acetaminophen could decrease the cost for pain relief in Japan. (Jpn J Pharmacoepidemiol 2012; 17(2): 75-86)

Guidelines for the usage of psychoactive drugs for behavioral and psychological symptoms of dementia (BPSD) were issued by the Japanese Ministry of Health, Labour and Welfare in July 2013. Using the Anatomical Therapeutic Chemical (ATC) classification with prescription data, we surveyed the usage of psychotropic drugs in patients with dementia. N05C (hypnotics and sedatives) was the most frequently prescribed class of drugs [9,920 (19.7%) individuals]. In addition, there are few prescription ratios of risperidone in comparison with 5.6% and the survey in the UK. Although anti-anxiety drugs should not be used as per BPSD guidelines, etizolam was prescribed in a considerable proportion of patients (6.2%). In addition, with respect to prescription of combinations of antipsychotics in the same month, the highest rate was found for risperidone and tiapride [209 (2.4%) individuals]. In addition, 39 individuals were prescribed antipsychotics that are contraindicated for patients with diabetes. When the number of the clinical departments is as above 2, the ratios to become the contraindicated prescription, careful administration prescription of the antipsychotic increase with significant difference (p<0.01). The increased of the number of the clinical departments tended to increase the ratio of the contraindications and careful administration prescription. Thus, the need for a family doctor to prevent such situations was suggested, which was introduced by the 2014 Medical Treatment Fee Revisions.

Quality of Life (QOL) is attracting attention as a concept which pursues the humanity of patients in contrast to conventional treatment which mainly consists of treatment for sustaining life. In particular, as senile dementia is one of the diseases for which a complete cure is not available to date and for which the main goal of medical treatment and care is improvement in QOL, appropriate evaluation of the effectiveness of treatment for elderly people with dementia cannot be employed without evaluating their QOL.
As well, in the field of pharmacoepidemiology, with the development of antidementia drugs for Alzheimer's disease (AD), utilization of a QOL scale in evaluating drug efficacy has become an important subject. Considering the characteristics of dementia, it is not necessarily easy to evaluate how much QOL improvement antidementia drugs bring about, because many patients have difficulty in evaluating themselves. Therefore, in addition to QOL evaluation, utilizing objective evaluation by observing patients'behavior or the scale measuring the patients'preferences should be examined.
Furthermore, maintaining QOL of caregivers is an important issue as this is closely related with QOL of patients themselves. To care for elderly people with dementia, a caregiver such as a patient's family member shoulders significant mental/physical burden, which could lead to “collapse by caring”. Therefore, in evaluating antidementia drugs, it is necessary to take QOL of caregivers as well as the patients themselves into consideration.