Orphan Drug Development for Rare Diseases
69
History
Since 1972, the Japanese national health system
has been active in implementing measures to
promote drug development for the treatment of
rare diseases. Initially, rare diseases were known
as “intractable diseases (Nan-byo).” Policies to
promote the research and development of orphan
drugs were adopted in 1993, and a notification
was issued by the Ministry of Health, Labor
and Welfare (MHLW) concerning designation
criteria and measures to promote research, e.g.,
the revised Orphan Drug Regulations, amend-
ment to the Pharmaceutical Affairs Act, and an act
establishing a fund for the relief of adverse drug
reactions and promotion of research.1-2 Table 4-1
presents the timeline of rare disease regulations/
initiatives in Japan, such as orphan drug regula-
tions, subsidization of specific medical expenses
related to intractable/rare diseases, and research
on medical care for intractable/rare diseases
Benefits3-4
The following is a discussion of the benefits
sponsors receive once a drug receives an orphan
designation:
Subsidy Payment
Sponsors can receive subsidies, including funds
for tests and research, through the National
Institute of Biomedical Innovation (NIBIO).
This subsidy is intended to help reduce the over-
all financial burden of orphan drug development.
Companies receiving the subsidy are expected to
pay a part of the profits obtained from orphan
drugs to the NIBIO. The NIBIO will allocate
payments received from companies for the devel-
opment and promotion of orphan drugs.
Guidance and Consultation
Sponsors receive guidance and consultation from
the MHLW, the Pharmaceuticals and Medical
Devices Agency (PMDA), and NIBIO on research
and development activities related to orphan drugs.
The PMDA has established a priority consultation
system with lower user fee categories for desig-
nated orphan drugs/medical devices.
Preferential Tax Treatment
Sponsors can report 12% of study expenses
incurred during the NIBIO subsidy payment
period (not including subsidies granted by
NIBIO) for orphan drugs these expenses can be
reported as a tax credit.
Priority Review
During marketing authorization application, the
designated orphan drug will be provided priority
review for faster access to the market (the review
period is nine months compared to the standard
Orphan Drug Development in Japan
4
69
History
Since 1972, the Japanese national health system
has been active in implementing measures to
promote drug development for the treatment of
rare diseases. Initially, rare diseases were known
as “intractable diseases (Nan-byo).” Policies to
promote the research and development of orphan
drugs were adopted in 1993, and a notification
was issued by the Ministry of Health, Labor
and Welfare (MHLW) concerning designation
criteria and measures to promote research, e.g.,
the revised Orphan Drug Regulations, amend-
ment to the Pharmaceutical Affairs Act, and an act
establishing a fund for the relief of adverse drug
reactions and promotion of research.1-2 Table 4-1
presents the timeline of rare disease regulations/
initiatives in Japan, such as orphan drug regula-
tions, subsidization of specific medical expenses
related to intractable/rare diseases, and research
on medical care for intractable/rare diseases
Benefits3-4
The following is a discussion of the benefits
sponsors receive once a drug receives an orphan
designation:
Subsidy Payment
Sponsors can receive subsidies, including funds
for tests and research, through the National
Institute of Biomedical Innovation (NIBIO).
This subsidy is intended to help reduce the over-
all financial burden of orphan drug development.
Companies receiving the subsidy are expected to
pay a part of the profits obtained from orphan
drugs to the NIBIO. The NIBIO will allocate
payments received from companies for the devel-
opment and promotion of orphan drugs.
Guidance and Consultation
Sponsors receive guidance and consultation from
the MHLW, the Pharmaceuticals and Medical
Devices Agency (PMDA), and NIBIO on research
and development activities related to orphan drugs.
The PMDA has established a priority consultation
system with lower user fee categories for desig-
nated orphan drugs/medical devices.
Preferential Tax Treatment
Sponsors can report 12% of study expenses
incurred during the NIBIO subsidy payment
period (not including subsidies granted by
NIBIO) for orphan drugs these expenses can be
reported as a tax credit.
Priority Review
During marketing authorization application, the
designated orphan drug will be provided priority
review for faster access to the market (the review
period is nine months compared to the standard
Orphan Drug Development in Japan
4