"Faster Introduction of Orphan Drugs"…Designation Criteria Expanded

Ministry Allows Prompt Orphan Drug Designation Without Comparative Data on Alternatives

On December 4, the Ministry of Food and Drug Safety announced that it has issued an administrative notice for the revision of the 'Regulation on the Designation of Orphan Drugs,' which expands the criteria for designating orphan drugs to provide patients with rare diseases with more treatment opportunities.

Rare diseases are defined as conditions with a patient population of 20,000 or fewer, or diseases for which the number of patients is unknown due to diagnostic difficulties. These are designated and announced by the Korea Disease Control and Prevention Agency.

This revision expands the criteria so that if a drug is used for the treatment or diagnosis of a rare disease, it can be promptly designated as an orphan drug without the need to submit data proving that it is significantly safer or more effective than alternative drugs required for orphan drug designation under the current system.

Additionally, the requirements for data that companies must submit when applying for orphan drug designation have been clarified for each designation criterion, increasing predictability.

Since July, the Ministry has operated the 'Orphan Drug System Improvement Council' to discuss various improvement measures, including the relaxation of designation criteria, in order to enhance the orphan drug system.

A Ministry official stated, "We expect that this revision will help create an environment where patients with rare diseases can receive stable treatment and contribute to building a 'robust society,' which is a key government policy objective."