To Contribute to a Goal ofJAPAN AS THE BESTPLACE TO LIVE AND AGE
VISION REPORT OF THE INNOVATIVE BIOPHARMACEUTICAL INDUSTRYMay 2018 | Pharmaceutical Research and Manufacturers of America
1TABLE OF CONTENTSEXECUTIVE SUMMARY

3Introduction

3

Pillar 1: Innovation

4

Pillar 2: Sustainability

5

Pillar 3: Evidence

6

HOW TO REALIZE THE VISION

7

Innovation

8

1.
2.
3.
4.

8
9
10
11

Strengthen translational researchHarmonizing to global clinical trial guidelines
More flexible use of expedited approval pathways
Use data infrastructure to streamline pharmacovigilance

Sustainability

12

5.
6.
7.
8.

12
13
14
15

Innovation as an investment in healthier lifespans
Maintain Japan as a priority destination for innovation
Re-invest savings available through off-patent reforms
Encourage primary and secondary prevention

Evidence

16

9. Create a comprehensive electronic medical record system
10. Develop a patient-centered value assessment mechanism
11. Include broad stakeholder input into decision making

16
17
18

NOTES

19

ABOUT

20

2

INTRODUCTIONABOUT OUR VISIONThe Innovative Biopharmaceutical Industry is committed to working as a true partner of theJapanese people in order to promote pro-growth and pro-innovation policies for the benefit ofJapanese patients and the economy. Our industry works to solve unmet me dical needs ofpatients and families endeavoring to develop new medicines quickly and safely and in doing so
provides economic benefits to Japan. We seek to uphold the highest ethical standards and comply
with all applicable domestic and foreign laws and regulations, consistent with the trust placed in our
industry by patients and the broader society.
We will work together with the Japanese government in order to achieve the best possible healthfor citizens while maintaining the sustainability of the Universal Health Care Coverage system.
Our industry will contribute to these goals through sustainably advancing innovation
underpinned by the best possible evidence to inform decision-making. At the same time, we
identify barriers and challenges to achieve this goal and propose recommendations as to how
the government might address these issues so together we can deliver better patient outcomesand economic gain.

Patient centricity is at heart of what we do and therefore is thefoundation on which our vision is built.
Our vision is based on the three key beliefs of patient centricity:
1. We believe it is crucial to deliver innovative medicines to patients in need , in a safe andtimely manner.
2. We believe patient centricity can drive better health outcomes, as well as direct andindirect cost savings.
3. We believe it is crucial to include the patient viewpoint into decision -making about theirhealth care.

3

PILLAR 1: INNOVATIONADVANCING INNOVATION WITH A WORLD LEADINGREGULATORY ENVIRONMENT TO DELIVER DRUGS TO
PATIENTS IN A SAFE AND TIMELY MANNER
Promoting innovative pharmaceutical discovery and translation into
medical innovation in Japan.
•

Overcoming the ongoing issues in the area of Japanese translational research sothat Japan can capitalize on its strengths to become a world leader in medicines
discovery and global provision.

•

Further harmonizing with international guidelines to enable greater Japaneseparticipation in multi-regional clinical trials.

Improving the competitiveness of Japan as a regional and global
leader through regulatory excellence.
•

Delivering innovative medicines to patients in the region much earlier through more
flexible use of expedited approval pathways.

•

Utilizing IT infrastructure to maintain the highest safety standards and streamline
the pharmacovigilance and post-marketing surveillance system.

4

PILLAR 2: SUSTAINABILITY

SECURING TIMELY AND AFFORDABLE PATIENT ACCESSTO NEW INNOVATION IN A SUSTAINABLE UNIVERSALHEALTH CARE SYSTEM
Delivering innovative medicines and vaccines that contribute to an
extension of healthy lifespans under rebalanced NHI that secures
timely and affordable patient access to innovation.
•

Delivering innovations to Japanese patients that contribute to an extension of healthy
lifespans and that help citizens to remain active throughout their lives.

•

Maintaining Japan as a priority destination for cutting-edge technology andinvestment to achieve sustainable promotion of innovation and timely and affordable
patient access.

Optimizing expenditure through an increase in efficiency of the offpatent sector and investment in primary and secondary prevention.
•

A sustainable pro-innovation environment could be achieved through re-investingsome of the savings available through off-patent reforms.

•

Developing new policies that encourage and reward primary and secondary
prevention measures that contribute to a reduction in the total NHI budget.

5

PILLAR 3: EVIDENCE

ENSURING POLICY DECISIONS ARE EVIDENCE-BASED AND
INCORPORATE A BROAD STAKEHOLDER VOICE
Prioritizing establishment of data infrastructure as a key driver for
further optimization of the health care system and resources.
•

Building a foundation for health care decision-making of the future through the creationof a comprehensive electronic medical record system and ensuring key stakeholders
can access such data whilst protecting patient privacy.

Building a world-leading value assessment framework to ensurepredictability and provide a sound process to assess the value ofinnovation.
•

Utilizing a well-designed, evidence-based value assessment framework broadly acrosshealth care services, including new technology, to support well-informed, patient-centeredhealth care.

•

Including patients, industry, providers and government in early and inclusive dialogue toenable comprehensive evaluation of a therapy’s value.

6

HOW TO REALIZE OUR VISIONINNOVATION
Promoting innovative pharmaceutical discovery and translation into medical innovation in
Japan.
1. Overcoming the ongoing issues in the area of Japanese translational research so that Japan cancapitalize on its strengths to become a world leader in medicines discovery and global provision.
2. Further harmonizing with international guidelines to enable greater Japanese participation in
multi-regional clinical trials.
Improving the competitiveness of Japan as a regional and global leader throughregulatory excellence
3. Delivering innovative medicines to patients in the region much earlier through more flexible use of
expedited approval pathways.
4. Utilizing IT infrastructure to maintain the highest safety standards and streamline thepharmacovigilance and post-marketing surveillance system.

SUSTAINABILITY
Delivering innovative medicines and vaccines that contribute to an extension of healthy
lifespans under rebalanced NHI that secures timely and affordable patient access toinnovation.
5. Delivering innovations to Japanese patients that contribute to an extension of healthy lifespans
and that help citizens to remain active throughout their lives.
6. Maintaining Japan as a priority destination for cutting-edge technology and investment to achievesustainable promotion of innovation and timely and affordable patient access.
Optimizing expenditure through an increase in efficiency of the off -patent sector andinvestment in primary and secondary prevention.
7. A sustainable pro-innovation environment could be achieved through re-investing some of thesavings available through off-patent reforms.
8. Developing new policies that encourage and reward primary and secondary prevention measuresthat contribute to a reduction in the total NHI budget.

EVIDENCE
Prioritizing establishment of data infrastructure as a key driver for further optimization of
the health care system and resources.
9. Building a foundation for health care decision-making of the future through the creation of acomprehensive electronic medical record system and ensuring key stakeholders can access suchdata whilst protecting patient privacy.
Building a world-leading value assessment framework to ensure predictability andprovide a sound process to assess the value of innovation.
10. Utilizing a well-designed, evidence-based value assessment framework broadly across healthcare services, including new technology, to support well-informed, patient-centered health care.
11. Including patients, industry, providers and government in early and inclusive dialogue to enable
comprehensive evaluation of a therapy’s value.

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INNOVATION - 1

Strengthen translational research

Overcoming the ongoing issues in the area of Japanesetranslational research so that Japan can capitalize on its
strengths to become a world leader in medicines discovery
and global provision.
Japan is one of only a handful of countries that has the scientific capability, regulatory rigor andmanufacturing expertise to be able to undertake the entire process of drug development frombasic research through clinical development to commercialization. While Japan has a large
pharmaceutical industry, including some global leaders, much of this industry is focused onservicing the domestic market. In addition, given world leading levels of basic research, there
are issues for the bridge between Japanese basic research and pharmaceuticalcommercialization; therefore, strengthened translational research is necessary to fill the gap.
At the same time, innovation may represent the best bet for Japan to manage the challenge ofbeing the world’s most rapidly aging population and, if viewed as an investment rather thansolely as a cost, to use innovation to reduce overall health care costs and increase productivity.

Recommendation
We recommend the further establishment of a public-private collaboration between AMED, academia,
bio-ventures and the pharmaceutical industries in Japan to tackle barriers to translational research inJapan and to accelerate research and development of new medicines to address both Japan’s
specific health needs as well as to improve global health.

Our Contribution
The innovative industry is contributing by promoting awareness of translational research in Japan,
growing the abilities of young researchers and expanding the opportunities for research collaboration.
Our activities include support of the Mansfield-PhRMA Research Scholars Program, Researcher Flyins and co-hosting of the Young Scientists Symposium.

8INNOVATION - 2

Harmonizing to global clinical trial guidelines

Further harmonizing with international guidelines to enable
greater Japanese participation in multi-regional clinical trials.
Japan has increasingly been included in global development programs that reflect the positive
incentives resulting from pro-innovation government policies over the last decade. However, clinical
trials in Japan tend to take longer and are more costly than in other countries, which acts as asignificant disincentive to including Japan in multi-regional clinical trials.
Through further harmonization with international guidelines, Japan should become considerably morecompetitive for multi-regional clinical trials, especially given the size of the market. In turn, this would
provide positive benefits for Japanese patients who would receive new medicines earlier than atpresent.

Recommendation
To achieve this vision, we recommend rapid implementation of international guidelines,
especially ICH-E17 4, in order to promote more multi-regional clinical trials including Japan.
Implementation of ICH-E17 will set the pre-conditions for Japan’s inclusion in more multiregional clinical trials. To help achieve this goal, we suggest greater engagement in international
capacity building workshops on ICH E17 principles, such as the Internati onal Council for
Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and AsiaPacific Economic Cooperation (APEC) programs. As part of this implementation, we would
suggest ensuring that current high cost and time-consuming aspects are addressed to further
increase Japan’s competitiveness compared with other countries that are competing for multiregional clinical trials.

Our Contribution
The innovative industry stands ready to work with the PMDA to implement ICH-E17 in a mannerthat is aligned with implementation in the U.S. and Europe. Implementation of ICH-E17 by Japan
will stimulate industry to include Japan in more multi-regional clinical trials.

9

INNOVATION - 3

More flexible use of expedited approval
pathways

Delivering innovative medicines to patients in the region muchearlier through more flexible use of expedited approval
pathways.
Over the past ten years, the PMDA has made very significant advances in improving its review
processes. This has led to a dramatic acceleration in review times and PMDA regulatory reviews are
now among the fastest in the world.
The PMDA has implemented expedited approval pathways for first-in-class innovative medicines.
However, at present, the “conditional early approval system” and Sakigake (“Pioneer”) Designation
are under-utilized in promoting domestic R&D and clinical innovation.
It is important for patients with unmet needs to have timely access to promising new medicines thatcan offer substantial benefits over existing standards of care. New expedited pathways in the U.S.
and EU are enabling accelerated clinical development and review for such medicines. Therefore, we
consider that the PMDA needs to ensure sufficient regulatory flexibility to allow rapid patient accessto medicines undergoing expedited approval pathways.

Recommendation
To achieve this vision, we recommend that the Sakigake designation system should be formally
implemented from the current trial basis as soon as possible, and to designate all innovative drugs
that meet the requirements. Additionally, the conditional early approval system should be equivalent
to the U.S. and EU systems in terms of the eligible products and number of designated products. This
will assure the global competitiveness of the Japanese early approval system compared with U.S.
and European expedited approval pathways such as Breakthrough Designation, PRIME andAccelerated Approval, and enable early access of innovative drugs to Japanese patients.

Our Contribution
The innovative industry is willing to work closely with the PMDA through sharing U.S. and European
regulatory best practices and experiences in order to help improve the Japanese system further.

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INNOVATION - 4

Use data infrastructure to streamline
pharmacovigilance

Utilizing IT infrastructure to maintain the highest safety
standards and streamline the pharmacovigilance and postmarketing surveillance system.
A common position between industry, the government, physicians and patients is that safety isparamount. Japan has a broad pharmacovigilance and post-marketing surveillance system in order toensure patient safety. However, much of this system is unique to Japan and has evolved slowly overtime with a result that the current system is cumbersome, very expensive to maintain and includes
elements that could be refined or are redundant.
The industry believes that Japan needs to modernize its pharmacovigilance and post-marketingsurveillance system in order to improve safety standards through using all the data potentiallyavailable and to improve the existing system with a more efficient and effective approach that aligns
to international standards.
The approach also has the potential to increase the early detection of possible safety signals in
medicines being launched in Japan under expedited approval pathways such as Sakigake or otherpathways.

Recommendation
To achieve this vision, we recommend that the Government of Japan leads the establishment of data
infrastructure and conducts necessary change of related regulations. We believe the government
should integrate the National Database (e.g. MID-NET) and similar large-scale medical information
databases utilizing ICT (Information and Communication Technology) to help evolve the current
systems uniquely developed in Japan into the global standard method incorporating signal
management.

Our Contribution
The innovative industry supports the highest safety standards for our medicines. We are willing tosupport the creation of a new system and engage with the Government of Japan on the creation ofthis system.

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SUSTAINABILITY - 5

Innovation as an investment in healthierlifespans.

Delivering innovations to Japanese patients that contribute toan extension of healthy lifespans and that help citizens toremain active throughout their lives.
The long-term sustainability of Japan’s world leading health care system is threatened by the growing
demographic burden of a rapidly aging society. The number of elderly are forecast to represent
35.3% of the population by 2040 while the number of working-age people supporting each elderly
person will have dropped from 7.4 in 1980 to two by 20205. In general, the elderly have a higher
demand for care, with per-capita direct medical care expenditure approximately four times that ofyounger patients6. Indirect costs are also significant. To help address this demographic challenge, thegovernment has set a policy goal of extension of healthy lifespans, including a focus on prevention ofchronic diseases from worsening and preventing people from being forced to prematurely leave theirjobs.
In an aging society, medicines represent an excellent option to improve healthier lifespans while
reducing expensive downstream health-related costs. Medicines are transforming the treatment ofmany difficult chronic diseases including cancer, cardiovascular disease, diabetes and rheumatoid
arthritis. Continuous development and diffusion of innovative medicines and vaccines that improvehealth outcomes relating to the diseases and conditions of the elderly will be essential to support the
government’s goal of healthy lifespan extension.

Recommendation
To realize our vision, it is necessary that the government and stakeholders recognize the value ofinnovation as a key investment in achieving a healthy and productive society. In order to achieve thisgoal, we recommend that all stakeholders should work to build a social health care system thatvalues and properly uses innovative medicines and vaccines.

Our Contribution
In addition to discovering new medicines, the innovative industry is contributing by providing the best
possible evidence to allow our medicines to be appropriately valued and utilized. Under a health caresystem that values innovation, the industry will be incentivized to continue to develop and diffuse
innovative medicines and vaccines that contribute to an extension of healthy lifespans and that help
citizens to remain active throughout their lives.
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SUSTAINABILITY - 6

Maintain Japan as a priority destination
for innovation

Maintaining Japan as a priority destination for cutting-edge
technology and investment to achieve sustainable promotion of
innovation and timely and affordable patient access.
Prior to 2010, Japanese patients experienced significant delays, often in years, in accessing cuttingedge innovations that were available in the U.S. and Europe. The delay was caused by a
combination of clinical trial barriers, long regulatory review times and an unfavorable reimbursement
environment, including regular price cuts of medicines while on-patent. Between 2010 to 2015, proinnovation government policies encouraged a rapid decline of the drug lag and catch-up withmedicines that had been available in other markets. From industry’s perspective, two improvements
were key: reforms to the PMDA review process and the introduction of the so-called Price
Maintenance Premium (PMP)9.
In the 2018 pricing reform package, GOJ stated that the balance between “sustainability of NHI” and“promotion of innovation” had been achieved. However, in our view, the reforms targeting innovative
medicines have gone too far while alternative approaches could have achieved the same cost-saving
objective without damaging pro-innovation policies.

Recommendation
A drastic review of the pricing reforms implemented in 2018 is necessary. In order to maintain Japanas a global leader, we believe that pro-innovation policies need to be restored, including meaningful
consultation with industry in advance of future policy changes and fair and predictable rules thatappropriately reward innovation while still ensuring the sustainability of the NHI.

Our Contribution
In order for Japan to remain a global leader, it must maintain or restore pro-innovation policies. Boththe NHI and industry need a predictable, fair and stable environment. To the extent that reforms are
required, industry stands ready to work with the MHLW to find constructive solutions.

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SUSTAINABILITY - 7

Re-invest savings available through offpatent reforms

A sustainable pro-innovation environment could be achieved
through re-investing some of the savings available through offpatent reforms.
Japanese policymakers are facing a challenge in ensuring the long-term sustainability of universal
coverage. Industry acknowledges that we have a role to play in helping reduce the fiscal burden of
the NHI on the Japanese population while also providing innovations that help reduce the burden on
the overall system through improving the health of society.
Despite the government’s own data, showing that expenditure on medicines was well controlled, inrecent years, policy has shifted from supporting pro-innovation to imposition of cost-containment
measures. Over 80% of the 440 billion yen reduction target for social security expenditure from 20162018 was raised by drug price reduction. The government’s focus on suppression of social security
expenses primarily through NHI drug price reform is an unbalanced response that does not address
the actual drivers of the total NHI increase.
In comparison with peer markets, in general Japan pays comparatively less for innovation across theon-patent period and considerably more for off-patent medicines. If Japan were to reinvest part of thesavings available through off-patent reforms to promote innovation, we can achieve a sustainablepro-innovation environment.

Recommendation
Further reforms of the off-patent sector could release savings to support the broader health carebudget, cover pro-innovation policies and remove the need for additional cost-cutting measures.

Our Contribution
Innovative companies are willing to contribute further savings from their off-patent portfolios provided
that a portion of such savings are used to support innovation in an integrated manner.

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SUSTAINABILITY - 8

Encourage primary and secondary
prevention

Developing new policies that encourage and reward primaryand secondary prevention measures that contribute to a
reduction in the total NHI budget.
The Japanese government’s goal of promoting healthier lifespans is a prevention strategy aimed at
reducing the rate of increase in health care expenditure as the population ages. In comparison tosome other mature health care markets, Japanese prevention strategies are relatively undeveloped
which leads to unnecessary downstream costs in the system.
Significant medical and social costs can be avoided or mitigated through improved use of preventive
medicines and strategies, such as smoking prevention. Medicines and vaccines for primaryprevention can avert or slow disease progress in its earliest stages. Medicines for secondary
prevention can prevent or delay disease progression or the emergence of complications, and cansubstantially reduce subsequent morbidity and mortality.

Recommendation
To realize a goal of achieving healthier lifespans through prevention, we believe that the government
should adopt pro-vaccination policies and give greater recognition to the value of incremental
innovation relating to prevention. Of particular importance would be policies that incentivize
innovations where their acquisition cost is less than cost-savings for the total health system and tothe broader economy.

Our Contribution
The industry wants to bring innovations that contribute to primary or secondary prevention to Japan.
Our industry also wants to help ensure that these medicines have the best possible beneficial impact
and would like to help address patient needs through improvement of delivery devices and utilizationof ICT (Information and communication technologies), for example to help achieve high levels ofadherence.

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EVIDENCE - 9

Create a comprehensive electronic medical
record system

Building a foundation for health care decision-making of the
future through the creation of a comprehensive electronic
medical record system and ensuring key stakeholders canaccess such data whilst protecting patient privacy.
To support the optimal use of health care services in order to derive the best treatment for the patientand also to utilize available resources in the most efficient way, a strong data system andinfrastructure needs to be put in place. Japan’s current use of electronic health care records is limited
and the collection, linkage and analysis of health data is relatively undeveloped13.
The foundation of evidence-based medicine, and indeed all health care policy, is use of quality datafrom a range of sources. In a patient-centered health care system, it is important to have integrated
data across a patient’s entire treatment journey in order to better understand physician and patient
behavior and to optimize the entire care pathway. Quality data in health care can be leveraged bydifferent stakeholders and improve medical treatment for patients overall. Key areas for potential
data-driven improvements include:•

Identification and targeting of unmet medical needs: ranging from identification of undertreated populations in Japan through to optimizing trial design.

•

Study the benefits of health care options: including the benefits of different treatment practices
or technologies based on a range of patient-reported outcomes and acceleration of detection
and response to safety issues.

•

Manage delivery: including optimizing supply chain operations, improving care coordination
and utilization, improving adherence, reduction of waste14 and optimization of procurement.

Recommendation
To achieve this vision, we believe the government should integrate the National Database (e.g. MIDNET) and similar large-scale medical information databases utilizing ICT (Information andCommunication Technology). We recommend that the government should set a goal for the entire
population to have electronic medical records and all hospitals and physicians to utilize digitized
health data. The integrated database should include information covering a patient’s entire treatment
journey so that health care is tailored to the needs of each patient while providing access to deidentified data to qualified stakeholders in industry, government, and academia. Safeguards shouldbe put in place to ensure patient privacy, protection of commercially sensitive information including
intellectual property, and responsible use of the database, following global standards.

Our Contribution
The innovative industry has been supporting the PMDA’s MIHARI project and MID-NET, actively
contributes to government discussions on IT infrastructure and has a common view with thegovernment regarding the future system architecture. We support strong protection for personal data
and would be willing to work with the government to ensure consistency with the best international
privacy standards.
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EVIDENCE - 10

Develop a patient-centered value assessment
mechanism

Utilizing a well-designed, evidence-based value assessment
framework broadly across health care services, including newtechnology, to support well-informed, patient-centered healthcare.
Industry supports the use of sound evidence for informed decision-making in health care andrecognizes the interest of the government in adopting Health Technology Assessment (HTA) forJapan.
HTA is a complex process and at present there is considerable diversity in how countries have
implemented HTA. Most HTA systems have shortcomings in recognizing value or can lack objectivity
due to focusing overly on cost-containment. Such issues can lead to delays in patient access toneeded medicines and misalignment between the HTA and overall health system goals. Many
countries are evolving their systems in response to shortcomings, which may help Japan to avoid theunintended consequences of a new system as some problems take time to identify.
Overseas experience has demonstrated that critical elements to developing a quality HTA framework
include (i) development of a sound process that has clear data requirements, methodology anddecision rationale; (ii) inclusiveness of patient and clinician input; (iii) a system-wide perspective
through examination of the full range of evidence and broad outcomes; and (iv) that places value on
the continued step-wise nature of scientific and medical progress.

Recommendation
To achieve this vision, we recommend that Japan continue to develop its approach to HTA to ensurethat the evaluation is patient-centered and ensures that incentives remain for needed therapies. In
order to achieve a high-quality, evidence-based outcome that is predictable, fair and transparent, werecommend that value assessment should deliver reliable, relevant information using rigorous
methods that rely on the full range of evidence.

Our Contribution
The innovative industry supports evidence-based decision-making that is designed to ensure thatpatients receive the most appropriate treatments to meet their needs. Industry has experience withmany different approaches to HTA and is willing to work with the government in designing anappropriate HTA system for Japan that aligns with the current pricing system while avoiding many of
the unintended problems that have occurred in HTA systems elsewhere.

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EVIDENCE - 11

Include broad stakeholder input into decision
makingIncluding patients, industry, providers and government in early
and inclusive dialogue to enable comprehensive evaluation of atherapy’s value.
The understanding of the value that a given innovative technology provides can vary considerablyacross stakeholders due to differences in perspective, including the relative weighting of clinical,
social, and economic factors. Therefore, it is crucial to involve broad stakeholder input, especiallyfrom clinical experts, patients and the manufacturer.
We believe that the evaluation of a therapy’s value needs to be patient-centered, which would require
substantive patient input. In order to achieve patient-centered care, health care systems should
identify and promote health technologies that are of value to patients, and allow for patientparticipation in the evaluation process—including a permanent role of patients in decision-making.
Many countries are already adopting this approach.
Furthermore, biopharmaceutical developers need to understand what authorities expect in terms oftherapeutic added benefit and what benefit they judge worth paying for—this will require dialogue
between industry and authorities prior to marketing authorization. Dialogue should focus on
determining benefits that are particularly relevant to patients and health care providers.

Recommendation
To achieve this vision, we recommend that the government meaningfully include broad stakeholder
input into all phases of technology valuation. We believe that the government should consider how to
support training patients and related organizations on how to provide relevant and objective input inthe health care decision-making process. We suggest further platforms for regular policy dialogue
between all stakeholders as equal partners. In addition, we recommend establishment of an earlyadvice process following the successful models used in other countries.

Our Contribution
The innovative industry helps to support patient-centered inputs through clinical assessments (PROs,etc.) and via supporting initiatives to enable patient groups to provide their non-clinical experiences inan unbiased way.

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NOTES
1. PHRMA (accessed April 2018) https://www.phrma.org/graphic/the-biopharmaceuticalresearch-and-development-process; PhRMA (2017) Prescription Medicines: Japan Costs inContext.
2. PHRMA and EFPIA (2016) Research in your Backyard Japan: Contribution of PhRMA and
EFPIA member companies to Japan’s society and economy through clinical trials ofinnovative medicines.
3. The per-patient clinical trial costs in Japan are two to six times higher than anywhere else in
the world. There are substantial problems with trial initiation, patient and researcher
recruitment.
4. ICH-E17 (2017) General Principles for Planning and Design of Multi-Regional Clinical Trials.
http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E17/E17EW
G_Step4_2017_1116.pdf.
5. Ministry of Health, Labour and Welfare, 2017, Annual Health, Labour and Welfare report.
6. Ministry of Health, Labour and Welfare, 2010, Summary of Health Care Expenditure
http://www.mhlw.go.jp/toukei/saikin/hw/k-iryohi/10/.
7. Endo et al., 2015, “Returning to work after sick leave due to cancer: a 365-day cohort study ofJapanese cancer survivors,” Journal of Cancer Survivorship. Note: Return to work includes
full-time and part-time work.
8. Ministry of Health, Labour and Welfare, 2015, Study on the Economic Impact of Dementia inJapan, Health Labour Sciences Research Grant.
9. The PMP’s full name is the “Premium to promote the development of new drugs and resolve
off-label use”.
10. Pharmaceuticals and Medical Devices Agency, Product approvals by FY 2009-2014; Center
for Innovation in Regulatory Science, 2015, “New drug approvals in ICH countries, 20052014: Focus on facilitated regulatory pathways and orphan designations,” R&D Briefing 59;
Yasuhiro Suzuki, Director-General, Health Insurance Bureau, MHLW, BIO Asia Conference,
14 March 2017, Tokyo; IMS “Market Prognosis 2016-2020- Asia/Australia-Japan.”
11. The novel PHiD-CV vaccine as compared to the standard of care would be expected toprevent 92,467 cases of acute otitis media (ear infection) in children born in a single year
(2013) and which would result in a net cost saving to the health care payer of JPY 1.9 billion
and indirect cost savings of JPY 3.9 billion (avoiding lost wages of parents providing care).
12. Ministry of Economy, Trade and Industry (accessed April 2018)
http://www.meti.go.jp/policy/mono_info_service/health care/downloadfiles/2017healthcare_presentation_v2.pdf.
13. OECD Reviews of Health Care Quality: Japan 2015.
14. Here ‘waste’ means wasteful medical expenditure and can result from non-adherence, overprescription, prescription error. Refer: National Institute for Research Advancement, 2015,“Towards fully-fledged reform of Japan’s medical care system, My Vision.”

19

ABOUT PhRMAThe Pharmaceutical Research and Manufacturers of America (PhRMA) represents the United States’
leading innovative biopharmaceutical research companies, which are devoted to discovering anddeveloping medicines that enable patients to live longer, healthier, and more productive lives. Since
2000, PhRMA member companies have invested more than $600 billion in the search for new
treatments and cures, including an estimated $65.5 billion in 2016 alone.

ABOUT PhRMA in Japan
Since its opening in January 1987, PhRMA’s Japan office has actively developed various activities onbehalf of its member companies in Japan. We promote activities focusing on direct dialogue with allconcerned parties such as administrative officials, medical policy officers, doctors and other medical
staff, media parties and patient organizations. PhRMA cooperates with the Japan Pharmaceutical
Federation, the Japan Pharmaceutical Industry Association, and the European Pharmaceutical
Federation to develop activities.
Publication date: May 16, 201820