"Even When Treatments Exist, Costs Are Unaffordable... Separate Fund Needed to Support Rare Disease Patients"

HIRA Hosts Symposium on "Treatment Directions and Social Ethics for Rare and Severe Diseases"
52 Ultra-High-Cost Treatments Exceed 43 Million Won Per Patient Annually
Alternative Proposals: Conditional Reimbursement Based on Evidence Generation and

There has been a call to establish separate funding to support patients with rare and severe diseases who lack appropriate treatments or medications, or who cannot access newly developed drugs due to prohibitive costs.

The symposium on "Treatment Directions and Social Ethics for Rare and Severe Diseases," hosted by the Health Insurance Review and Assessment Service, is currently underway on the 25th at the Catholic University Seoul St. Mary's Hospital. Health Insurance Review and Assessment Service

At the symposium on "Treatment Directions and Social Ethics for Rare and Severe Diseases," held by the Health Insurance Review and Assessment Service at Catholic University Seoul St. Mary's Hospital on the 25th, Lee Soyoung, Director of the Drug Outcome Evaluation Department at the Health Insurance Review and Assessment Service, stated, "Because the reimbursement decisions for rare and severe disease treatments have a significant impact on the national health insurance budget, it is necessary to design a sustainable and systematic system."

Rare diseases are defined as those affecting fewer than 20,000 people or those for which the number of patients is unknown due to diagnostic difficulties. As of this year, around 8,000 rare diseases have been identified worldwide, and there are 1,314 rare diseases currently managed by the Ministry of Health and Welfare in Korea.

Of these, only about 3 to 5 percent have available treatments, and even when treatments exist, many are excessively expensive or cannot guarantee effectiveness. Kwon Youngdae, Policy Director of the Korea Alliance for Rare and Incurable Diseases, appealed, "Ninety-five percent of rare disease patients cannot receive fundamental treatment and are limited to symptom management or delay. The lifelong cost of treatment and care devastates households, destroying not only the lives of patients but also their entire families."

According to the Health Insurance Review and Assessment Service, as of last year, there were 52 rare disease drugs that required more than 43 million won (approximately 32,000 USD) per patient annually. Among these are gene therapies that require only a single lifetime dose but cost several billion won. Because such costs are beyond the means of individual patients, the government sometimes partially subsidizes them through national health insurance. However, as national resources for healthcare are limited, the government must strike a balance between expanding treatment opportunities for patients and maintaining fiscal soundness.

There is also an ethical dilemma regarding how long to provide medically futile treatments to patients. Yoo Hanuk, Professor at Bundang CHA Women's Hospital, pointed out, "It is urgent to establish clear criteria for discontinuing treatment and to build a continuous monitoring system. It is also necessary to develop systematic clinical guidelines for rare diseases."

As an alternative, the "conditional reimbursement system based on evidence generation" was mentioned. Director Lee explained, "Even if the clinical usefulness and cost-effectiveness of treatments for severe and rare diseases are uncertain at the time of listing, reimbursement should be provided on the condition of post-market evaluation to ensure patient access. Subsequently, the government should evaluate and manage the uncertainty of evidence regarding these treatments."

There was also an opinion that, as in other countries, a separate fund should be established to support patients with rare and severe diseases. The proposal is to separate current exceptional health insurance coverage into its own funding source, strengthen the responsibility of pharmaceutical companies, and reduce the insurance burden based on re-evaluation results.

The United Kingdom introduced the Innovative Medicines Fund (IMF) in 2022, requiring pharmaceutical companies to bear the cost of treatments for rare diseases not covered by insurance. France operates a rare disease fund and allocates a large-scale budget every five years. In Australia, the Life Saving Drugs Program (LSDP) provides high-cost medicines for ultra-rare diseases that have clinical usefulness but lack proven cost-effectiveness. Pharmaceutical companies or physicians can apply for patient support, and the drugs are supplied free of charge, with re-evaluation after two years.

Policy Director Kwon emphasized, "With advances in medical technology, the number of rare diseases is increasing every year. To ensure sustainable fiscal management, it is essential to establish fair and transparent standards through social consensus, create a dedicated fund, and provide stable financial input."

Lee Ilhak, Professor of Medical Law and Ethics at Yonsei University, also suggested, "While treatments for rare diseases have opened new medical possibilities, high costs and limited effectiveness have deepened debates over who should be covered and to what extent by national health insurance. It is necessary to secure separate funding and prioritize within that framework."

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