In the questionnaire investigation conducted on 59 member companies by the pharmaceutical affairs committee of the Japan Pharmaceutical Manufacturers Association, it became apparent that in Japan there have been cases where development was discontinued, such as abandonment of efficacy addition, because of failure to acquire designation as orphan drugs. In Japan, there have been many cases where the drugs were not designated because the data showing efficaciousness and safety significantly higher than those of existing drugs did not exist at the time of consulting. On the other hand, in Europe and U.S., they found that there was a tendency to give designations widely at an early stage based on the data from Phase I trial and Phase II trial. The committee is demanding the regulatory authority to make the designation criteria to be equivalent to that of Europe and U.S.
At the Annual Scientific Meeting of the Japanese Society of Clinical Pharmacology and Therapeutics held in Fukuoka on the December 5th, Mr. Yuji Kashitani (Takeda Pharmaceutical Company Limited Japan Development Center Pharmaceutical Department) presented the investigation results. The investigation was conducted in September this year. 58 out of the 59 companies affiliated with the Pharmaceutical Affairs Committee replied back.
In order to be designated as an orphan drug under the Japanese system, the following 3 prerequisites must be met: ▽Target patient number must be lower than 50,000 or the illness must be designated as an intractable disease. ▽The medical need for the drug must be especially high. ▽The possibility for development must be high.
The results of the investigation showed that the number of drugs which were unable to acquire designation because they did not meet the prerequisites at the time of consulting the Pharmaceuticals and Medical Devices Agency (PMDA) reached 86 items total in the past 10 years.
When asked for the reason, many pointed out “medical need” far above all other reasons. Among the specific reasons, the highest was “It cannot be concluded that it is showing efficaciousness and safety significantly higher than those of existing drugs at the time of consulting with the data submitted,” followed by “There is no trial data directly comparing it to existing drugs” and “There is a need to show significantly higher efficaciousness and safety than those of existing drugs in verification tests.”
When asked for the impact that the failure to receive designations had on development plans, here were some of the comments: ▽”Although we had considered an efficacy addition based on the advantages of designation, we ended up abandoning the plan.” ▽”We discontinued the development.” ▽”Japan became unable to join the International Collaboration.” ▽”Our development plan was considerably delayed.” ▽“The priority in development became lower.”
On the other hand, we compared the evidence data used at the time of designation for the 73 items out of 105 items designated in the past 10 years which also acquired designation in Europe and U.S.
As a result, even though the discussion on advisability of designation is held based on Phase I trials and Phase II trials in Europe and U.S., many discussions in Japan are held based on Phase III trials.
In Japan, items with a time period of less than 5 months from the time of designation acquirement to application for approval occupy 60% of the entirety, and it became apparent that many items got designated at the point of application.
From the member companies, opinions were raised, such as “If efficaciousness and safety significantly higher than those of existing drugs are required, then designation for subsequent items with similar efficaciousness and safety will never be given, resulting in development stalling, with a possibility that treatment choice would be limited to only one item for an extended period of time.” and “The designation of orphan drugs is important when companies are making a judgement on whether to launch the development, and it is difficult to show efficaciousness and safety compared to existing drugs at that phase.”
Mr. Kashitani expressed concern, saying “Since the designation is done at an early stage in Europe and U.S., the company can make a judgement on investment. Since Japan is the only country where designation is done only at late stages of development, development in Japan will not progress,” and insisted that “designation at the earliest stage possible is important.”
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