Opportunities for Orphan Drugs in Japan
Creating Opportunities for Orphan Drugs
to Enter the Japanese Market
Market Size
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Population: 123 million 12th in the world (Source: UNFPA, 2023)
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One of the top 3 global pharmaceutical markets
Patient Friendly Healthcare Insurance System
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Universal coverage of medical expenses
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Deductions for medical expenses for patients with rare/intractable diseases
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Well established regulatory system for new drug approval
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Quick and firm timeline from approval to reimbursement (usually 2 months)
Accelerating Orphan Drug Development
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Median 9 months from NDA submission to approval (orphan drug)
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Additional benefits from the Orphan Drug Designation System
Market Needs：The Environment Surrounding Drugs for Rare Diseases
In Japan, there is significant demand for the development of unapproved rare disease pharmaceuticals. As of a 2020 survey, out of 176 domestically unapproved drugs, 90 received orphan designation from the FDA or EMA, accounting for 51% of the total unapproved drugs. There are signs of expansion in regards to "domestically unapproved drugs," which are approved in other countries but not in Japan.
Source: Seisaku-ken News, No.66, July 2022
Market Size
The Japan's population is 123 million, ranking 12th in the world. It is the third-largest pharmaceutical market in the world and has an attractive market size.
Healthcare System
Japan has a well-established public health insurance system, allowing essentially all citizens to receive medical care with a certain percentage of co-payment (ranging from 10% to 30%). Medical pharmaceutical products need government approval and a nationwide uniform drug price is set. Patients with pediatric chronic specified diseases or designated rare diseases are eligible for medical expense subsidies. Additionally, there is a system in place to provide financial assistance for high medical expenses.
For details, please refer to "Market Access: Drug Pricing and Reimbursement"
Support for Drug Development for Rare Diseases
When a pharmaceutical product is designated as an orphan drug, several benefits include:
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Grants for research and development expenses (to alleviate development costs)
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Priority review and consultation* (to enable faster provision of medical care)
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Extension of market exclusivity period (designation as an orphan drug extends the typical 8-year re-examination period for new drugs to a maximum of 10 years)
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Price premium (marketability premium) for NHI drug pricing
