Pharmaceutical Administration and Regulations in Japan - Nihs

disease and clinical usefulness using the
propriety review selection criteria.
Applicants are requested to submit results
of clinical studies up to late Phase II as a
rule as data for estimating the clinical
usefulness. Hearings and inquiries are
undertaken for the applicant as required
and the designation is decided after
hearing opinions of experts in the field.
The results, including reasons, are
notified to the applicant in writing.
Orphan drugs are all handled as products
for priority face-to-face advice and an
application is not required.
4.4 Restrictive Approval System
The drugs to which this system applies
are those used in emergencies to prevent the
spread of diseases that might have a major
effect on the public health. It also applies to
drugs for diseases for which the drug
concerned is the only method of treatment
and which are marketed overseas. Such
products may be granted a restrictive
approval by the Minister without going
through ordinary approval review procedures
after hearing the opinion of the PAFSC.
4.5 Orphan Drugs
Policies to promote research and
development on orphan drugs were adopted
in 1993, and a notification was issued by the
Pharmaceutical Regulations in Japan:
MHW concerning designation criteria and
measures to promote research. The criteria
for designation include less than 50,000
patients indicated for the drug concerned and
excellent usefulness of the drug from the
medical standpoint. The PAFSC gives its
opinion on the designation.
Drugs designated as orphan drugs are
entitled to certain priority measures such as
financial aid, tax relief on research expenses,
guidance and advice, priority review, and
extension of the reexamination period from
the conventional 6 years to a maximum of 10
years for drugs and from 4 years to a
maximum of 8 years for medical devices.
4.6 Drugs for Pediatric Use
Drugs used in pediatric clinics are often
considered as “therapeutic orphans”
throughout the world because they are
difficult to develop and are not provided with
sufficient information. This also applies in
Japan and very few drug products are
indicated for pediatric use. The number of
clinical trials performed in children is not
sufficient, the number of products that can be
used for children is insufficient, and
information contained in package insert
(dosage, efficacy, safety, etc.) in relation to
applications in children is also insufficient.
Therefore, “off-label use” of drugs basically
intended for adults, use of in-hospital
products without adequately verified stability,
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