20th April 2021
EFPIA Japan

EFPIA Recommendations on the Cost-Effective Assessment System in JapanTOKYO – 20th April 2021 –Following the series of decision of the cost-effectiveness assessment
results in the Central Social Insurance Medical Council (Chuikyo) by 14th April, 2021 for innovative
new drugs marketed by EFPIA Japan member companies, which was the first case after the costeffectiveness assessment system was formally introduced in April 2019, EFPIA Japan believes that we
can have constructive discussions by sharing the issues that the member companies have experienced
in the system, and would like to make the following recommendations and express our awarenessof the issues. In addition to ensuring the scientific validity of the cost-effectiveness assessment
system, we expect the system to mature by ensuring the transparency so that the system can bereviewed post-hoc by third parties through detailed records and information disclosure of thediscussions held between the companies and the public analysis team, as well as the content of theevaluation by the Cost-Effectiveness Assessment Expert Panel.
1. Inclusion of the clinical perspective
Recommendation:
In the pre-analysis consultation between the company and the public analysis team, scientificevaluation must be ensured, taking into account the clinical characteristics of the items to beevaluated. To this end, we request that the clinical perspective, which is essential to reflect realworld medical practice, be reflected in the framework of the cost-effectiveness analysis by actively
utilizing the participation of clinical experts from both company and public analysis team.
Issue:In the cost-effectiveness analysis for EFPIA member companies’ cases, we had the impression thatclinical perspectives, which are essential to reflect real world medical practice, were notsufficiently taken into account. In order to ensure the scientific validity of the cost-effectivenessanalysis framework, we believe that it is necessary to reflect not only the health economics
perspective, but also knowledge of clinical experts, such as defining the added usefulness of the
product from a clinical point of view.
2. Agreement on a realistic time frame for company analysis
Recommendation:
We call for a reconsideration of the system whereby the time required for companies to submit
their analysis results is agreed at the end of the pre-analysis consultation.

EFPIA Japan
ThinkPark Tower, 2-1-1, Osaki, Shinagawa-ku, Tokyo 141-6017, Japan
Tel: +81-3- 6417-2032
Fax: +81-3- 6685-4870

Issue:In the cases of the member companies, the predetermined time frame of nine months from thepre-analysis consultation to the submission of the analysis results does not reflect the actual
process and workload required to conduct a cost-effectiveness analysis, as the time required fora company to conduct an analysis depends largely on what has been agreed at the pre-analysis
consultation. We also call for consideration of a further extension, particularly when there is asignificant difference from the analytical framework prepared in advance by the company (e.g.
requiring additional data analysis beyond clinical trial data).
3. Accurate records of pre-analysis consultations
Recommendation:
We call for prompt provision of minutes of pre-analysis consultations between the company andthe public analysis team immediately after the completion of each consultation, detailing thepresentation of data necessary for the determination of the analytical framework and thebackground to the discussions that have taken place.
Issue:
Because the content of the records of the pre-analysis consultations was inadequate, thecompanies were not able to start the analysis work promptly based on the results of theconsultations. Further, the records were not useful as reference information for post-analysis
verification and system review. We believe that it is necessary to prepare detailed minutes of themeetings, rather than a summary (abstract).
4. Disclosure of information including scientific evidence
Recommendation:
In order to ensure the transparency of the cost-effectiveness assessment system and for thirdparties to conduct qualitative review of the pre-analysis consultation and the evaluation contentsof analysis results, we request the public disclosure of "Expert Panel decisions" prepared by theExpert Panel.
Issue:
Based on these companies’ cases, in addition to guaranteeing the quality of the cost-effectivenessassessment, it is necessary that information is disclosed in order to assess whether validity ismaintained in all steps from the pre-analysis consultation to the end of the analysis by the expert
panel. We believe that open qualitative review by experts will lead to future improvement of thesystem. EFPIA Japan welcomes the publication of the outline of the cost-effectiveness assessment
results at Chuikyo and the publication of the cost-effectiveness assessment report by both thepublic analysis team and the company.

EFPIA Japan
ThinkPark Tower, 2-1-1, Osaki, Shinagawa-ku, Tokyo 141-6017, Japan
Tel: +81-3- 6417-2032
Fax: +81-3- 6685-4870

Unless all issues in the current system, including but not limited to the aforementioned
recommendations and issues, are examined and corrected, EFPIA Japan cannot accept discussions
towards expanding the system, such as expanding the number of the products subject to costeffectiveness assessment and using it to make decisions on the reimbursement of the products.

About EFPIA Japan (http://efpia.jp/)
Established in April 2002, EFPIA Japan represents 20 R&D-based European pharmaceuticalcompanies operating in Japan. In 2019, combined sales from the member companies accounted forroughly 23% of the pharmaceutical market in Japan. The mission of EFPIA Japan is to “Contribute tohealthcare and patients in Japan through the early introduction of innovative medicines andvaccines”. EFPIA Japan aims to strengthen dialogue with decision-makers in order to improve
Japanese healthcare for all.
About EFPIA (European Federation of Pharmaceutical Industries and Associations)(http://www.efpia.eu)
EFPIA, EFPIA Japan’s partner organisation, is headquartered in Brussels and represents thepharmaceutical industry operating in Europe. Through its direct membership of 36 nationalassociations and 39 leading pharmaceutical companies, EFPIA provides the voice of companies
committed to researching, developing and bringing new medicines to improve health and quality oflife around the world.

Contact:
Yuko Kidoguchi
EFPIA Japan Messaging & Communications Committee
1-6-5 Marunouchi Chiyoda-ku, Tokyo 100-8268
Bayer Holding Ltd.
Tel: 03-6266-7757
Email: yuko.kidoguchi@bayer.com

EFPIA Japan
ThinkPark Tower, 2-1-1, Osaki, Shinagawa-ku, Tokyo 141-6017, Japan
Tel: +81-3- 6417-2032
Fax: +81-3- 6685-4870