The Fight for Timely Access: Rethinking AHUS Treatment Protocols

Patient advocacy groups and medical professionals are intensifying their efforts to overhaul the existing pre-approval system for Atypical Hemolytic Uremic Syndrome (AHUS) treatments in South Korea. Citing critical delays that directly threaten patient survival, stakeholders are demanding immediate action to streamline access to life-saving therapies.

A System Under Scrutiny: Delays and Denials Plague AHUS Patients

the current system mandates a preliminary review process for expensive rare disease treatments before health insurance coverage is granted. While intended to manage healthcare costs, this process has proven detrimental in the context of AHUS, a condition characterized by rapid deterioration.Experts emphasize that treatment delays exceeding 48 hours from onset can lead to irreversible kidney damage and other life-threatening complications. Yet, the pre-approval process can take up to 14 days, leaving patients in a precarious situation.

Data reveals a concerning trend: from 2018 to 2024, the average pre-approval rate for AHUS treatments stood at a mere 18%. A notably alarming instance occurred in November of the previous year, where only one in five applications were approved. This low approval rate has prompted the Korea Federation of Rare and Intractable Diseases, along with affected patients, to file formal grievances with the National Rights Commission, the Ministry of Health and Welfare, and the Health Insurance Review and assessment Service.

Voices of Concern: Experts and Advocates Unite

A recent meeting held at the Seoul Goverment Complex, convened by the National Rights Commission, brought together key stakeholders to address the urgent need for reform. Participants included Chung Jin-hyang, Secretary-General of the Korea federation of Rare and Intractable Diseases, patient representatives, and leading medical experts such as pediatric nephrology specialists from Seoul National University Hospital and The Catholic University of Korea.

82% of 39 adult patients diagnosed with AHUS in the last five years have died of late renal failure. A government-level solution is urgently needed so that the administrative procedure is not repeated by the treatment of treatment.
Chung Jin-hyang, Secretary-General of the Korea Federation of Rare and Intractable Diseases

AHUS is a severe disease that damages the microvascular vessels of the whole body.If you miss the treatment time, you can lead to fatal complications such as kidney damage, heart failure and stroke.
Professor Kang, Pediatric Nephrology Specialist, Seoul National University Hospital

The Path Forward: Seeking Immediate Solutions

The Korea Federation of Rare and Intractable Diseases has specifically requested the reclassification of Eculizumab, a crucial AHUS treatment, to a general screening category. This change would expedite access to the drug,ensuring that patients recieve timely intervention and improving their chances of survival. The National Rights Commission, acknowledging the gravity of the situation, has pledged to develop practical measures to improve the existing system.

Park Jong-min,Vice Chairman of the National Rights Commission,stated:

We will listen to the grievances of AHUS patients through the meeting and prepare practical measures to improve the system.
Park Jong-min, Vice Chairman of the National Rights Commission

Global Context: AHUS Treatment and Access

The challenges faced by AHUS patients in South Korea are not unique. Globally,access to rare disease treatments remains a important hurdle. In the United states, for exmaple, the FDA’s Orphan drug Act provides incentives for the progress of treatments for rare diseases, but access and affordability remain concerns. Similarly,in Europe,the European Medicines Agency (EMA) offers similar incentives,but disparities in access persist across different member states. The situation underscores the need for complete policies that address both the development and equitable distribution of rare disease therapies.