Research on the Current Status of Orphan Drug Designation Requirements in Japan A Study Based on a Questionnaire Survey of Pharmaceutical Companies
Ryuta Asada (Associate Professor, Center for the Advancement of Advanced Medical Sciences and Clinical Research, Gifu University Hospital)
Katsuichi Shibukawa (Senior Researcher, Pharmaceutical and Industrial Policy Research Institute)
(No. 70: Published in March 2017)
Although an increasing number of pharmaceutical companies are now actively developing drugs for rare diseases, there are still many diseases for which no development has yet taken place and for which no effective treatments exist. In order to further promote the development of orphan drugs in Japan, we believe that it is necessary to clarify the requirements for designation as an orphan drug, thereby improving the possibility of development as an orphan drug and encouraging designation at an early stage of development.
Therefore, we surveyed information related to the criteria for designation of items already designated as orphan drugs, and also requested cooperation from 73 companies that are members of the Japan Pharmaceutical Manufacturers Association (JPMA) and participate in the Drug Evaluation Committee, and conducted a questionnaire survey regarding the designation system and preferential measures desired by pharmaceutical companies.
The results of the survey on already-designated orphan drugs,
-
(i)(i) When "a prefix, a suffix, etc., to the name of the disease" is added to the indication or the target disease in the application for designation, one point is that the number of subjects should not exceed 50,000 for the disease as a whole, and if the number exceeds 50,000, a rational reason for limiting the disease based on pharmacological effects, etc. is considered necessary. (i) The number of subjects should not exceed 50,000.
-
(ii)Regarding the possibility of development, the current situation suggests that it may be difficult to obtain designation if there are no data in Japan or overseas that suggest efficacy at least in patients subject to designation.
-
(iii)Regarding the period between designation and submission of an application for approval, 55% of the items that received designation took less than one year, suggesting that some of the supportive measures may not be effectively utilized.
From the results of the questionnaire survey, we were able to obtain a certain level of understanding of the views of pharmaceutical companies regarding the support measures after the designation of orphan drugs that they consider beneficial, the criteria for designation that they consider problematic, the support measures that should be implemented in the future, and the status of utilization of the designation consultation. We were able to obtain a certain degree of understanding of the views of pharmaceutical companies regarding the "supportive measures that should be implemented" and the status of utilization of designated consultation. Among them, the following noteworthy comments on supportive measures and designation criteria were found to be particularly noteworthy.
- Many companies cited "extension of reexamination period," "priority review," "guidance and advice by PMDA," and other measures directly related to early approval, as well as the extension of the post-marketing development cost recovery period, as beneficial support measures,
- Many companies also felt that "medical necessity" and "development potential" in the designation criteria make it difficult to designate a product at an early stage of clinical development.
In addition, many respondents pointed out the need for separate designation criteria for Ultra Orphan, the need for post-marketing support measures, the creation of a PMDA consultation framework regarding the applicability of designation, and issues related to the uncertainty of NHI drug prices.
In the future, it will be necessary to request the government to consider support measures that can be utilized after marketing approval (e.g., subsidies for post-marketing surveillance) and to establish a consultation framework regarding designation, such as "consultation on the applicability of orphan drugs for new drugs" in PMDA's face-to-face advice.