MFDS offering regulatory support for orphan drug discovery

The Korean ARPA-H initiative aims to facilitate communication as part of regulatory response strategies

Requirements for Orphan drug designation (ODD) will be eased, starting this year…flexibility of clinical guidelines will be established

The Ministry of Food and Drug Safety (MFDS) plans to accelerate the commercialization of government-led orphan drug (treating rare diseases) development by providing regulatory support from the early stages of development.

The MFDS has initiated support for the orphan drug sector through the "Overcoming Unconquered Diseases" project, the Korean ARPA-H initiative led by the government.

Through this support, the MFDS aims to accelerate the commercialization of orphan drugs and improve patient access to treatment for ultra-rare diseases.

In observance of "Overcoming Rare Disease Day" on the 28th, the MFDS held a briefing for the medical and pharmaceutical press to explain the current status of support and policies for rare disease treatments.

In observance of "Overcoming Rare Disease Day" on the 28th, the MFDS held a briefing for the medical and pharmaceutical press. (from left) Project Manager Misun Park of the Korea Health Industry Development Institute (KHIDI); Hyeon Jin Yim, Head of the Regulatory Science Policy Promotion Division; and Division Heads Chun-rae Kim, Jae-hyun Park, and Mi-ryeong Ahn.

"World Rare Disease Day" falls on February 29th, the rarest day of the year that occurs once every four years. Under the Rare Disease Management Act, the Korean government commemorates the last day of February each year (typically February 28th or 29th) as Rare Disease Day. The goal is to enhance public understanding of rare diseases and improve access to treatment and medical support for patients and their families.

While South Korea has designated 1,380 rare diseases, drug development remains challenging due to the small patient population and the difficulty of attracting corporate investment.

The government began treatment development through the Korean-style ARPA-H project. Benchmarked after the U.S. "ARPA-H (Advanced Research Projects Agency for Health)," this project is a "high-risk, high-return" national R&D initiative that began last year.

In the field of rare diseases, projects currently underway include the development of a tailored, innovative treatment platform for pediatric rare disease patients, the N-of-1 (single-patient) clinical trial project (HEART), and patient-customized gene therapy to overcome visual impairment in hereditary eye diseases (BEACON). A national budget of KRW 17.5 billion is allocated for investment over 4.5 years.

Typically, national R&D projects often conclude with researchers registering papers or patents. However, this project aims for commercialization. Consequently, regulatory support from the MFDS is considered crucial.

Misun Park, Project Manager of the K-Health Future Promotion Division at KHIDI, explained, "To meet regulatory requirements after development has significantly progressed can lead to irreversible losses in time and cost. It is essential to communicate with the MFDS from the early stages of development to collaborate on clinical trial design and the direction of data preparation."

Furthermore, due to the nature of rare diseases, patient administration cannot be cancelled even if any issues arise during the Investigational New Drug (IND) process. Therefore, the aim is to minimize trial-and-error by consulting with the MFDS from the start of development.

The MFDS provides early-stage regulatory support through the "Regulatory Adequacy Review System," which has been in place since last year. This system analyzes and supports regulatory requirements during the initial stages of national R&D projects.

The system reviews how products under development are classified and which laws apply to them, and provides consultations on evaluation criteria and methods for demonstrating safety and efficacy. Through this, regulatory response strategies and the necessity of joint research with the MFDS are also reviewed.

Last year, the system supported commercialization by classifying stem-cell-based artificial blood projects as advanced biopharmaceuticals and recommending GRADE changes for digital therapeutic development projects for developmental disabilities.

Hyeon Jin Yim, Head of the Regulatory Science Policy Division at the MFDS, stated regarding the system, "Researchers often develop innovative technologies but face difficulties at the commercialization stage because they do not fully understand regulatory procedures. The Ministry provides guidance from the beginning on which laws apply, what data needs to be prepared, and what strategies are necessary to increase the likelihood of approval."

Yim added, "Preventing delays in commercialization is possible if the time required for supplementation is shortened by analyzing regulatory targets in advance. We are currently in discussions regarding eight projects under the ARPA program."

The MFDS is also working to improve accessibility by easing designation requirements for orphan drugs and implementing expedited reviews. Starting this year, drugs can receive orphan drug designation (ODD) even without submitting data demonstrating significantly improved safety or efficacy compared to existing alternatives.

Chun-rae Kim, Head of the Pharmaceutical Policy Division at the MFDS, explained, "Previously, for rare disease drugs announced by the Korea Disease Control and Prevention Agency (KDCA), data proving improved safety and efficacy had to be submitted. However, through consultations with the pharmaceutical industry last year, we decided to omit this regulatory requirement."

This year, the Ministry plans to discuss with the industry measures to ease regulations on orphan drug designation cancellations.

When designated as an orphan drug, rapid approval can be expected through expedited regulatory reviews. The MFDS selects drugs for expedited review through the "GIFT" (Global Innovative products on Fast Track) system. Currently, of the 50 items approved as GIFT products, 42 are orphan drugs.

Jae-hyun Park, Head of the Expedited Review Division at the MFDS, stated, "According to the disease groups of treatments designated as GIFT, half are recurrent or intractable cancers with small patient populations. For these patients, clinical trials themselves can be a treatment opportunity. In the future, we plan to consult with the public and private sectors to ensure that patient opinions are reflected in GIFT reviews."

For orphan drugs not designated under GIFT, the MFDS also offers flexibility in reviews, granting approval based on Phase 2 clinical trials, with the requirement to submit Phase 3 clinical data post-market. Mi-ryeong Ahn, Head of the Oncology and Antibiotics Division, added, "We continue to develop guidelines related to clinical trial design to increase flexibility at the approval stage."