An impact analysis of the implementation of health technology assessment for new treatment of orphan diseases in Japan

ABSTRACT

Objectives: In Japan, a cost-effectiveness evaluation (CEE) for pricing was introduced in April 2019 and potentially covers orphan drugs (ODs) within its scope. The purpose of this study was to explore a reasonable approach to evaluate the utility of health technology assessment (HTA) for ODs in Japan.

Methods: We extracted ODs that were approved in Japan from 2009 to 2018, and investigated their appraisals in the United Kingdom, Canada, and Australia, where HTA and different frameworks on ODs have been implemented.

Results: Overall, 76 ODs were identified, with high recommendation rates in the three countries (80.6%-90.9%). The major reason for negative recommendation was uncertainty regarding clinical effectiveness, with actual decisions varying across countries. This indicates difficulties in setting an objective evaluation for the uncertainty of clinical effectiveness. The results of the CEE were mainly used to adjust prices.

Conclusion: As Japan’s CEE is expected to be used only for price adjustment after reimbursement is secured, the approach seems to be similar to the other countries. However, pre-launch clinical data are limited and the peak sales of ODs vary in Japan. Therefore, the careful introduction of CEE and multifaceted measures referring to the policies for ODs in other countries should be considered.

KEYWORDS:

• Comparative research
• cost effectiveness evaluation
• Health Technology Assessment (HTA)
• Japan
• orphan drugs
• rare diseases

Article Highlights

• Regulators in Japan and other countries have set up incentives to encourage manufacturers to develop orphan drugs and now they are more profitable option to the pharmaceutical companies.

• However, high price of orphan drugs and their justification have been controversial and one of the most debated topic in the health policy arena.

• Our analysis found that the orphan drugs approved in Japan were mostly recommended under the national fund in the countries where economic evaluation has been implemented, such as the United Kingdom, Canada, and Australia.

• Their decision for the recommendation was mainly based on the clinical effectiveness of the drug, and the economic evaluation was reflected in the price adjustment.

• The evaluation for ‘uncertainty of clinical effectiveness’ varies, making it difficult to set a transparent standard.

• As the new ‘cost-effectiveness evaluation’ regulation in Japan utilizes the evaluation of cost-effectiveness only for price adjustment after launch, there is a limited impact on access as long as the current scheme is maintained.

• However, pre-launch clinical data are limited and the peak sales of ODs vary in Japan; therefore, careful introduction of CEE and multifaceted measures referring to the policies for ODs in other countries should be considered.

Acknowledgments

We thank Y Kojima from Otsuka Pharmaceutical for providing important background information on the orphan drugs approved in Japan.

Author contributions

K Kogushi developed the concept, designed the study, contributed to analysis and interpretation of data, and wrote the initial draft of the manuscript. All other authors have contributed to data interpretation and critically reviewed the manuscript, and agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Declaration of interest

K Kogushi is an employee of Otsuka Pharmaceutical Co., Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Data availability statement

NICE database: https://www.nice.org.uk/guidance/published?type=ta

CADTH Common Drug Review Reports: https://www.cadth.ca/about-cadth/what-we-do/products-services/cdr/reports

pCODR: https://www.cadth.ca/pcodr/find-a-review

PBAC: http://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/psd

Chuikyo (Japanese): https://www.mhlw.go.jp/stf/shingi/shingi-chuo_128 154.html

Correction Statement

This article has been republished with minor changes. These changes do not impact the academic content of the article.

Additional information

Funding

This paper was not funded.