Five Rare Disease Drugs Selected for "Expedited Listing"; Cost-Effectiveness Evaluation to Follow

Treatments selected for generalized pustular psoriasis, Duchenne muscular dystrophy, and other conditions

The Ministry of Health and Welfare announced on November 6 that it had selected five drugs for a pilot project to expedite the listing of treatments for rare diseases, following an application process and evaluation.

The selected drugs are Spevigo injection (LEO Pharma), a treatment for generalized pustular psoriasis; Agamree oral suspension (Nexera Pharma Korea), a treatment for Duchenne muscular dystrophy; Joenja tablets (Berto Korea), a treatment for activated PI3K delta syndrome; Qalsody injection (Biogen Korea), a treatment for amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease); and Iqirvo tablets (Ipsen Korea), a treatment for primary biliary cholangitis.

The pilot project was launched to improve access to treatments for patients with severe rare diseases who have few treatment options, as the small number of patients makes it difficult to obtain sufficient clinical evidence. Previously, drugs had to undergo a cost-effectiveness evaluation before being listed for coverage under the National Health Insurance. Under the pilot project, this will be replaced with a post-listing evaluation based on actual clinical outcomes and a pre-agreed evaluation plan.

In June, the Ministry of Health and Welfare reported its plan for the pilot project to the Health Insurance Policy Deliberation Committee and invited applications for participating drugs in July and August. In September and October, it requested additional materials from pharmaceutical companies, held meetings with patient groups, and gathered opinions from relevant academic societies. On November 4, it convened a selection evaluation committee comprising experts and officials from relevant organizations, and finalized the selected drugs after reviewing presentations by the pharmaceutical companies.

The committee comprehensively considered the severity of each disease, the availability of alternative drugs, and treatment efficacy, as well as whether the post-evaluation plans were appropriate and whether plans to ensure continued treatment for patients after the evaluation were adequate.

The selected drugs must have their post-evaluation plans supplemented and submit implementation agreements before applying for National Health Insurance coverage listing, no later than January 2027. The ministry expects the listing process to proceed quickly, with the drug prices and projected claim amounts set in accordance with the pilot project announcement.

Yoo Juheon, Director General of the Ministry of Health and Welfare's Health Insurance Policy Bureau, said, "Based on the results of the pilot project, we will develop a plan to institutionalize the expedited listing system. If necessary, we will also actively consider a second pilot project to ensure access to treatments for patients with severe rare diseases."

This content was produced with the assistance of AI translation services.