抄録
In order to evaluate the Orphan Drug Development Support Program established in April 1993, we sent questionnaires to sponsors who developed or are developing orphan drugs and surveyed the reasons for applying for the orphan drug designation and the benefits of the Program they obtained after designation was granted.
The results show that shortening of the evaluation period and sufficient guidance and advice to sponsors have created great incentives for orphan drug R & D. On the other hand, the extension of the reexamination period, grants or tax deductions were considered less of an incentive than expected.
Of 87 sponsors, 73.6% were motivated by third parties such as MHW's research group, medical associations or patients' associations. This means that there is strong social need for orphan drugs and sponsors have developed such drugs in view of their responsibility in society.
It can be said that the Orphan Drug Devlopment Support Program has been successful to some extent from the viewpoint of providing necessary drug to medical practices. However, a more flexible opration of the Program is needed for efficient development, and further consideration would be necessary in order to enrich the guidance and advice, promote further shortening of the evaluation period, create priority distribution of the grants to small or middle-sized companies or to orphan drugs with very small markets, and to simplify the granting procedure.
