MFDS to ease orphan drug designation eligibility criteria

Comparative data related to alternative medicine will be exempt

National briefing on top 50 food and drug safety tasks…"will add security to safety"

Yu-Kyoung Oh, Minister of the MFDS, is speaking at the

As the criteria for Orphan Drug Designation are set to be significantly eased, this is expected to expand the introduction of rare disease treatments currently not approved in Korea.

 

The Ministry of Food and Drug Safety (MFDS) plans to revise the regulations by February of next year.

 

The MFDS announced this during the 'Top 50 Food and Drug Safety Tasks National Briefing Session' held on the 5th at the International Conference Hall on the first floor of the Seoul Foundation of Women and Family in Dongjak-gu, Seoul.

 

The relaxation of the Orphan Drug Designation requirements was introduced as the very first of the seven key projects.

 

Yu-Kyoung Oh, Minister of the MFDS, said, "We will ease the designation requirements for rare drugs not yet approved in Korea to expand treatment opportunities for patients." Currently, a drug is designated as an orphan drug if the prevalent patient population is 20,000 or fewer and if comparative data are available with an alternative medicine.

 

During this process, it is required to submit data demonstrating that the drug is significantly safer and more effective than existing alternatives, but the industry reports difficulty providing this documentation.

 

There have been calls to ease designation requirements to expand treatment options for rare disease patients with limited or difficult treatment options.

 

In response, the MFDS plans to revise the criteria.

 

Under the revised plan, a drug will be designated as an orphan drug simply by being used for the treatment or diagnosis of a rare disease, effectively eliminating the requirement to submit comparative data against an alternative therapy.

 

The exemption of the superiority requirement for orphan drug designation is already unnecessary in countries like the United States, Switzerland, and Taiwan.

 

The relaxation of the orphan drug designation will be implemented through the revision of the 'Regulations on Orphan Drug Designation' planned for February of next year.

 

In addition, the MFDS plans to tag medications currently imported directly by patients for self-treatment as urgently imported drugs to ensure proper inventory stockpiling and supply.

 

The policy aims to sequentially transition over 10 items annually, starting next year.

 

To achieve this, the MFDS plans to secure the necessary budget through inter-ministerial consultation and expand the scope of the related program.

 

The seven key tasks also include: ▲Operating a hotline for rapid and convenient one-stop preliminary consulting ▲Faster announcement of harmful food information via 'consumer-customized SNS' ▲Expanding treatment opportunities for innovative anti-cancer drugs by improving clinical trial participant requirements ▲Quick confirmation of safe information on dietary supplements via QR code ▲Rapid safety management of contaminants in livestock products (meat) using AI ▲Establishing clear standards for safe consumption of decaffeinated coffee.

 

The task of improving anti-cancer clinical trial participant requirements involves establishing review criteria for early-stage anti-cancer clinical trials.

 

This is intended to expand patient choice, invigorate anti-cancer clinical trials, and support the development of treatments for intractable cancers in situations where a cure is difficult.

 

The MFDS plans to issue the 'Considerations for Participant Selection in Early-Stage Anti-Cancer Clinical Trials' guideline in December, which will clearly present the review criteria for clinical trials involving patients with established alternative treatments.

 

Minister Oh emphasized that through these seven key tasks, the MFDS aims to "add security to food and drug safety so that the daily lives of the people can be healthier."