Orphan Drug Designation (ODD) is regulated by Ministry of Food and Drug Safety (MFDS) in South Korea under the Pharmaceutical Affairs and Orphan Drugs Act. ODD is granted to drugs that treat life-threatening diseases or rare diseases with no alternative available in South Korea or proven superiority to available treatment options in South Korea.
Learn more about our Regulatory Intelligence Services and how Artixio supports global submissions, including ODD applications in Asia-Pacific markets.
Eligibility Criteria and Population Threshold
The expected disease prevalence population size is required to be less than 20,000. If the patient population is more than 20,000, MFDS does not consider the application under ODD but as normal New Chemical Entity application. ODD in reference countries is usually not accepted in South Korea. The acceptance of application is based on the prevalence and superiority of the drug against the currently available treatments in South Korea.
Independent and Combined ODD Applications
In South Korea, ODD applications (innovators or manufacturers) can be submitted independently or MFDS regulations provides options for submitting ODD applications clubbed with fast-track application requests. This enables significant reduction in review and approval timelines. Where an ODD application takes close to two years for review by MFDS, a combined application with fast-track request can help reduce the timeline to close to one year.
Conditional Approval and Data Requirements
International biotech companies often enquire is there is a possibility for conditional approval. In South Korea, MFDS can review ODD applications using data from upto phase 2 studies, therefore, there is no requirement for a separate conditional review application.
MFDS, however, considers ODD for approvals if the drug in scope is proven superior to the comparator drug / treatment available in South Korea. Therefore, either the trial design may include comparative studies to prove superiority or literature review need to be performed comparing the drugs with comparator treatment option available for proving the superiority. While MFDS considers patient population for accepting ODD application, superiority of the drug is considered more promising indicator for successful approvals.
International manufacturers, biotech companies can request pre submission meeting with MFDS to consult regarding their application. However, in practice, applicants often face restrictions due to language barriers and may find it difficult to schedule a meeting with MFDS. However, using a local representative can help overcome these challenges to facilitate detailed discussions with MFDS in common language.
Recently, MFDS has further clarified the requirements for conditional approval of orphan drugs.
The key points are as below:
- Before approval, applicants need to submit a plan to MFDS which includes the expected submission date of the Phase 3 protocol and the expected date of the final study report.
- After approval, once the Phase 3 study is completed, the results must be submitted to MFDS.
- In addition, every year by the end of March, applicants must report the progress or interim results of the Phase 3 study to MFDS
Artixio Support for ODD Applications in South Korea
If you are seeking consultation for orphan drug designation in South Korea, our team based on Seoul will be happy to assist you. Write to us at info@artixio.com
To explore regulations, guidelines related to orphan drug desgination in South Korea and receive periodic regulatory updates, please join our regulatory intelligence platform QuriousRI.
