The South Korean government has finalized five drugs for a fast-track approval program aimed at providing treatment options for patients with severe rare diseases who lack adequate alternatives. This initiative allows these new drugs to enter the health insurance coverage list without undergoing the usual pre-approval economic assessment.
On November 6, the Ministry of Health and Welfare announced the selection of five drugs for the fast-track listing program. This program was introduced to enhance access to new treatments for severely ill patients, given the limited number of patients with rare diseases, which makes it challenging to gather large-scale clinical evidence.
Instead of the rigorous pre-approval cost-effectiveness evaluation that new drugs typically undergo before entering health insurance, the program allows for immediate listing. Following this, a post-evaluation will be conducted based on actual clinical outcomes and a pre-agreed evaluation plan.
In June, the Ministry presented the program to the Health Insurance Policy Review Committee. Subsequently, from July to August, the ministry called for drug submissions, followed by a review of additional data from pharmaceutical companies and consultations with patient groups in September and October. On November 4, an evaluation committee composed of experts and relevant agency officials convened to assess the presentations from participating pharmaceutical companies and finalize the five selected drugs.
The drugs selected for fast-track approval include: ▲ 'Spevigo' (Leo Pharma) for generalized pustular psoriasis, ▲ 'Agamir' (Nexera Pharmaco Korea) for Duchenne muscular dystrophy, ▲ 'Joenza' (Bertoko Korea) for activated PI3K delta syndrome, ▲ 'Calsody' (Biogen Korea) for amyotrophic lateral sclerosis, and ▲ 'Ikervoy' (Ipsen Korea) for primary biliary cholangitis.
The evaluation committee considered the severity of the diseases, the availability of alternative treatments, and the effectiveness of the therapies, as well as the appropriateness of the post-evaluation plans and patient treatment guarantees following the evaluations.
Pharmaceutical companies holding the selected drugs must complete subsequent procedures, including submitting post-evaluation plans and agreements, by January 2027 at the latest. According to the program's announcement, they will receive predetermined drug prices and expected billing amounts, which is expected to expedite the listing and supply of these drugs to patients compared to typical new drug negotiation processes.
Yoo Joo-hun, Director of the Health Insurance Policy Bureau, stated, “Based on the results of this pilot program, we will prepare a plan for institutionalizing the fast-track approval system. If necessary, we will actively consider a second pilot program to ensure access to treatments for patients with severe rare diseases.”
* This article has been translated by AI.
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