EFPIA Japan’s Basic Position on
Cost-Effectiveness Assessment in Japan“Delivering Patient-Centered Innovation”

EFPIA JapanJanuary, 2020

EFPIA Calls For Continuous Review of
Cost-Effectiveness Assessment
In April 2019, the Government of Japan officiallyintroduced “cost-effectives assessment” (CEA)
for pharmaceuticals and medical devices after
its pilot program was completed. As Japan’s version of health technology assessment (HTA), thenewly introduced system is aimed to adjust reimbursement prices of pharmaceuticals (drugprices) after product launch.
Until recently, drug prices in Japan were decidedonly according to evaluations of innovativenessand other factors, based on efficacy and safety
data. However, since April 2019, the new system
also reviews drug prices from an economic viewpoint that examines “cost” and “effect” relative
to the comparator, when the government’s criteria are met or the government recognises theneed for the review. (See Figure 1)
Japan has limited experience in HTA, and therefore needs to enrich its expertise in health economics. Since the official introduction of thenew system, European pharmaceutical companies and officials of the government’s HTA body
have held a series of discussions on selected
medicines for CEA. However, there have beendifferent views on academic expertise and due
processes between the pharmaceutical industry
and the government HTA body. We believe
Figure 1. CEA process for new medicines (if selected)

more transparency in the CEA process andbetter systems are needed for objective discussions.

Ethical concerns inrare disease treatment
The Ministry of Health (MHLW) has set a basic
rule that excludes from CEA treatments only forrare diseases with small patient populations orwith paediatric indications. However, it also allows that “the Chuikyo may make decisions onwhat should be selected for CEA” if the expectedsales forecast is large and the unit price of thedrug is significantly high1. The Chuikyo (Central
Social Insurance Medical Council) is an advisoryboard for the health minister, whose officials aredrawn from healthcare professionals groups andpayer association representatives.
The MHLW has selected several new medicines
for CEA since April 2019, which includes “orphandrugs” for leukaemia and other rare disease.2
This means “cost-effectiveness” in treatments
for diseases with small patient populations andhigh medical needs is requested to be proven.
We believe rare diseases need to be completely
excluded from CEA for ethical reasons, because
Japan’s universal healthcare coverage highly values the basic principle of equality in healthcare
services.

“Drug lag” concerns again;
denial of drug reimbursement?
Some stakeholders in Japan recently proposedthat reimbursement decisions for medicines
should be made after CEA. The Ministry of Finance, for example, seeks to use CEA for reimbursement decisions in the future.3 A
representative of a payer association also shows asimilar position.4
The MHLW, however, still maintains its traditional position that in principle all the pharma-

Figure 2. Patient Access to Medicines in Europe and Japan
Length of Market Access Delays:2018 study: 20152017. The average time between marketing authorisation and patient access, measured by the number of dayselapsing from the date of EU marketing
authorisation (or effective marketing authorisationin non-EEA countries) to the day of completion ofpost-marketing
authorisation
administrativeprocesses. Waiting times reflected in the Patients
W.A.I.T. Indicator include any delay, whether
attributable to companies or to competent authorities. Fundamentally the “waiting time” in Japan is60 days and as only the timing of coverage by
reimbursement for price revision is 90 days, theweighted average of these values are used in thisanalysis.
Rate of Availability: measured by the number of
medicines available to patients in European countries as of 2018: for most countries this is the point
at which the product gains access to the reimbursement list.
Source: EFPIA Patient W.A.I.T. Indicator 2018
Surveyceuticals should be reimbursed in the public insurance system after marketing authorization.
The ministry says it is difficult to gain public
support for changing the principle.5 A major
physicians’ group also shares a similar position.6
Japan has a principle to make decisions within
60 days on reimbursement of medicines newly
authorized to market, making patient accesspossible in a timely manner. However, in othercountries, patients cannot always access new
medicines in a timely manner and fail to enjoy
the best treatment due to reimbursement denial after HTA.7
The waiting time for patient access to new medicines after marketing approval varies country tocountry in Europe (See Figure 2). If Japan introduces a similar policy to make reimbursement
decision based on CEA for new medicines, its
world’s best healthcare services where everyone can benefit from the latest and quality
Figure 3. Composition of National Medical Expenditure (%)

treatments may drastically change in a negative
way. The drug lag issue, which has been solvedby the greatest efforts by the government8, mayemerge again in Japan.

Efficiency in healthcare,
total optimization needed
Japan’s national medical expenditure has now
reached 43 trillion yen9 and is expected to continue to grow. The government has been working on social security reforms to control the increasing expenditure in Japan’s aging society.
Pharmaceutical spending accounts for about
20% of the total national medical expenditure,
which is an unchanged trend for years in Japan.10 The other expenditures come from inand out-patient services as well as pharmacy
costs (See Figure 3).
We believe continuous efforts to balance thevalue of innovative medicines and the sustainability of Japan’s universal healthcare coveragethrough total healthcare expenditure optimisation are important. All the stakeholders inhealthcare such as patients, healthcare professionals, policy makers and the pharmaceuticalindustry need to seek the best solution based on
accurate and scientific evidence.

Social values of medicines;
Future cost reduction effect
The value new medicines bring to patients is not
limited to clinical outcomes. Their social values
such as improvement in Quality of Life (QoL)
Source: MHLW “Summary of National Medical Expenditure in FY2017”and reducing caregivers burden also contributeto reduce the total disease burden that benefit
both patients and society.
Some cancer medicines have easier administration than conventional ones, as well as provenefficacy to extend patients’ survival. As the cancer population increases while survival rates
have been improving in Japan11, new cancer
treatments are to expected to allow more patients with cancer live longer and to remain active in the workforce .
Medicines for stroke prevention have benefit
not only for patients’ health outcomes but also
for reducing the care-giver burden because thetreatments can reduce the risk of being bedridden after stroke. It is encouraging that the government legislated a new law for stroke and cardiovascular disease prevention12 because stroke,
the 3rd biggest cause of death in Japan13, and its
prevention are one of the most prioritised social
issues in Japan.
COPD (chronic obstructive pulmonary disease),
whose primary cause is smoking, is a lifestyledisease for people in middle age14. It is also reported that the lost weekly working time in
COPD patients is significantly longer than for
healthy people15. Treatments for diseases withnegative impact to people’s socio-economic activities like COPD are expected not only to improve patient health status but also reduce social loss.
New medicines with improved efficacy, safety
and utility profiles will contribute to solve social
issues and offer improved QoL for patients. We
believe that these significant additional values

new medicines bring about need to be assessed
appropriately and transparently.

EFPIA’s Principles
We, EFPIA Japan, call for continuous efforts toreview the CEA scheme by the government, andpropose the following principles.

1. CEA should NOT be used for reimbursement decisions.
2. CEA should be used only as acomplementary tool for drug pricing and reimbursement systems.
3. The ethical and social values ofpharmaceuticals should be evaluated from a long-term perspective.
4. All stakeholders in the CEA processes should be involved in atransparent manner.
Reference:
1. 厚生労働省通知「医薬品、医療機器及び再生医療等製品の費用対効果評価に関する取扱いについて」（2019年３月29日 ）2. 中央社会保険医療協議会総会資料（2019年5月15日及び8月28日）
3. 日刊薬業WEB「費用対効果、既収載含め対象拡大検討を 財務省・吉野主計官 償還可否「最終的な目標」（2019年4月19日）
4. 健保ニュース「高額薬剤を再評価で適正化 国民・患者の納得する仕組みが必要」（2019年11月15日）
5. 日刊薬業WEB「費用対効果、患者アクセス最優先に 厚労省・古元医療課企画官 日本に合った仕組み、今後も模索」（2019年4月23日）
6. 日刊薬業WEB「あくまで薬価制度を補完、費用対効果に「一定の評価」 日医・松本氏 継続的な検討は必要」（2019年3月6日 ）7. Boseley, Sarah (Dec 29, 2016). Breast cancer drug rejected for NHS use on cost-benefit grounds. The Guardian.
https://www.theguardian.com/society/2016/dec/29/breast-cancer-drug-kadcyla-rejected-for-nhs-use-on-costbenefit-grounds
8. 医薬品医療機器総合機構「ドラッグ・ラグの試算（平成25～29年度）」
9. 厚生労働省「平成29年度 国民医療費の概況」（2019年9月26日）
10. 中央社会保険医療協議会総会資料4-1（2019年6月26日）
11. 国立がん研究センター「がん情報サービス」（2017年6月14日）
12. 参議院法制局「健康寿命の延伸等を図るための脳卒中、心臓病その他の循環器病に係る対策に関する基本法（平成30年12月14日法律第105号）」
13. 厚生労働省「平成29年（2017）人口動態統計（確定数）の概況 」（2018年9月7日）
14. 日本呼吸器学会「呼吸器の病気 B01気道閉塞性疾患 慢性閉塞性肺疾患（COPD）」（2014年3月）
15. 日本医療政策機構「我が国における慢性閉塞性肺疾患(COPD) の課題および対策」（2014年1月）

European Federation of Pharmaceutical Industries and Associations, Japan
EFPIA Japan was established in 2002, and represents 23 R&D-based European pharmaceutical companies operating in the Japanese market. We are the voice of the European innovative pharmaceutical industry in Japan. Our mission is to contribute to
the welfare of the Japanese population through the provision of innovative pharmaceuticals and vaccines without delay.
http://efpia.jp

Contact EFPIA Japan at info@efpia.jp