Korea's GIFT system speeds approvals for rare and severe disease treatments

The Ministry of Food and Drug Safety (MFDS)'s “Global Innovative Product Fast-Track Review Support System (GIFT)” has yielded tangible results since its implementation three years ago.

The ministry introduced the GIFT system to enhance treatment accessibility for patients with severe and rare diseases, strengthening the global competitiveness of the Korean pharmaceutical industry.

On Tuesday, the MFDS held a briefing session for journalists covering the ministry. Park Jae-hyun, director of the Rapid Review Division at the National Institute of Food and Drug Safety Evaluation (NIFDS)’s Innovative Product Review Team, explained the system’s performance and answered reporters’ questions.

Park emphasized that GIFT has become a core system, expanding treatment opportunities for patients by enabling the early approval of numerous treatments for rare and intractable diseases, thereby driving innovation across the entire pharmaceutical industry.

GIFT: A flagship initiative of Regulatory Reform 1.0

The MFDS introduced the GIFT system in August 2022 as a flagship initiative of “Regulatory Reform 1.0.” Based on the Pharmaceutical Affairs Act, it designates treatments for severe intractable and rare diseases, as well as new drugs from innovative pharmaceutical companies, as priority review candidates. The Rapid Review Division concurrently conducts reviews in non-clinical, clinical, and quality domains.

Since implementation, the system aims for approval within an average of 90 days—25 percent faster than the standard 120-day review period. “When a company submits a designation application, undergoes supplementation, and submits the issued designation certificate along with its product license application, expedited review proceeds,” Park explained.

The GIFT system has proven particularly effective in addressing rare and intractable diseases, where patient numbers are low and treatment options are limited. According to the ministry, 55 items have been designated as GIFT targets in the three years since the system's introduction, with 39 items (23 ingredients) receiving final approval.

Among these, treatments for rare diseases, such as myasthenia gravis, accounted for the largest share at 23 items (59 percent), while treatments for severe diseases, including pediatric neuroblastoma and recurrent/refractory cancers, comprised 14 items (35.9 percent). Together, treatments for severe and rare diseases constituted 84.6 percent of the total.

Annual outcomes were also ‘prominent,’ with designations and approvals doubling

The number of designations and approvals also shows an upward trend annually. It more than doubled from 22 cases in 2022 to 46 cases in 2023.

The actual review period was also shortened to an average of 70 working days. Among these, 23 cases were completed within 70 days, and 15 cases took between 70 and 90 days to complete. “As the GIFT system becomes known to overseas regulatory agencies, Korean pharmaceutical companies are increasingly applying for product approvals swiftly, with little time lag compared to overseas.”

Of the 39 items approved so far, 18 (46 percent) are treatments for severe and rare diseases for which no existing therapies were available. This demonstrates that the GIFT system has directly contributed to meeting patient treatment needs, Park noted.

The ministry also links GIFT-designated items to the Ministry of Health and Welfare's “Parallel Approval-Evaluation-Negotiation Pilot Project” to shorten the time to insurance listing. Among the five items selected for the pilot project, three have already received approval through expedited review, and the remaining two are currently in the phase of negotiating drug prices.

“New drugs developed by innovative pharmaceutical companies are also providing momentum for the development of the Korean pharmaceutical industry by reflecting preferential drug price evaluation regulations, Park emphasized.

She added, “While only some items are currently included in this pilot project, we plan to propose expanding its scope in consultation with relevant ministries going forward, hoping more items will be included in the parallel review process.”

Regarding operational challenges with the GIFT system, Park noted, “Reviewing treatments for rare or complex diseases is never easy. Particularly under the pressure of completing reviews within such shortened timelines, the shortage of specialized personnel is one of the biggest difficulties.”

If the review workforce is strengthened, the system's operation would become much smoother, she added.

Park also added a request for pharmaceutical companies. “Corporate preparation is also crucial for expedited review. If companies submit supplementary materials in a more systematic and complete manner when applying for approval, it would help reduce unnecessary delays during the review process,” she advised.

Regularizing the ‘HOPE Forum’ to reflect patient voices and strengthen information sharing

Additionally, the MFDS plans to promote policy consensus with patient groups, the pharmaceutical industry, and the medical field by regularizing the “HOPE Forum,” which focuses on expedited review of medical products. The forum will share information such as designation status and clinical results to encourage the development of follow-up drugs

“We planned this forum with the hope that patients' voices will be reflected in the GIFT system,” Park said. “We will also provide information such as designation status and clinical results to encourage the development of follow-on drugs.”