'What Use Is a New Drug'... Rare Disease Treatments That Can't Exceed Reimbursement

Assemblywoman Choi Hye-young Hosts Discussion on 'Rare and Intractable Disease Treatments Stalled by Health Insurance Hurdles'

Cold Agglutinin Disease, Sanofi's 'NJMO'
Neurofibromatosis, AZ's 'Koselugo'
Approved but Health Insurance Reimbursement Discussions Stalled

Choi Hye-young, a member of the National Assembly's Health and Welfare Committee from the Democratic Party of Korea, announced on the 13th that she will hold a discussion forum titled "Rare and Intractable Disease Treatments Facing Health Insurance Hurdles: Focusing on Cold Agglutinin Disease and Neurofibromatosis" on the afternoon of the 14th at the 6th Conference Room of the National Assembly Members' Office Building in Yeouido, Seoul.

Promotional Poster for the Forum on 'Rare and Intractable Disease Treatments Facing Health Insurance Hurdles'
Photo by Choi Hye-young's Office

This forum was organized to directly hear the difficulties experienced by patients with cold agglutinin disease and neurofibromatosis, which are classified as rare and intractable diseases, from diagnosis to treatment, and to discuss ways to improve patient accessibility focusing on currently developed treatments.

Patients with cold agglutinin disease and neurofibromatosis will attend the forum in person to share their experiences battling the diseases. During the subsequent presentations, Professor Jang Jun-ho of the Hematology and Oncology Department at Samsung Seoul Hospital will present on "Current Status of Cold Agglutinin Disease Treatment and Ways to Improve Treatment Accessibility," and Professor Lee Beom-hee of the Pediatrics Department at Asan Medical Center will present via video on "The Necessity of Rapid Reimbursement for Neurofibromatosis Treatments."

In the following panel discussion, chaired by Choi Young-hyun, Special Professor at Hankyong National University, participants will include Lee Eun-young, Director of the Korea Patient Organization Federation; Kim Jin-ah, Secretary General of the Korea Rare and Intractable Disease Association; Lee Ha-rim, Officer of the Health Insurance and Pharmaceutical Affairs Division at the Ministry of Health and Welfare; Jo Woo-mi, Officer of the Rare Disease Management Division at the Korea Disease Control and Prevention Agency; and Kim Guk-hee, Head of the New Drug Listing Department at the Health Insurance Review and Assessment Service. They will engage in designated discussions and Q&A sessions.

The diseases to be discussed at this forum, cold agglutinin disease and neurofibromatosis type 1, have recently had treatments introduced domestically and approved by the Ministry of Food and Drug Safety, but they have yet to be covered by the National Health Insurance.

Sanofi's cold agglutinin disease treatment 'Enjaymo (active ingredient sutimlimab)'
[Photo by Sanofi]

Cold agglutinin disease is a condition in which the body's immune system attacks its own red blood cells, causing continuous and repeated destruction of red blood cells. Due to red blood cell destruction, patients suffer from severe anemia, fatigue, shortness of breath, hemoglobinuria, peripheral cyanosis, and thrombotic complications. There is no disease code for it in Korea yet, making it difficult to accurately count the number of patients, but it is estimated that there are about 100 patients.

Previously, there was no treatment, so temporary measures such as blood transfusions or steroid therapy were used. However, in July, Sanofi's cold agglutinin disease treatment "Enjaymo" (generic name: sutimlimab) was approved domestically. Enjaymo is the first-in-class humanized monoclonal antibody targeting the C1 protein that activates the classical complement pathway. Clinical trials showed that 73% of patients treated experienced an increase in hemoglobin levels. Fatigue caused by cold agglutinin disease also decreased.

AstraZeneca's neurofibromatosis treatment 'Koselugo (Selumetinib)'
[Photo by AstraZeneca]

Neurofibromatosis type 1 is a rare disease characterized by abnormal cell proliferation throughout the body. It is usually diagnosed before the age of 10, and as the body grows, lesions continue to enlarge, often accompanied by complications such as speech disorders, scoliosis, and severe pain. As of 2020, the estimated number of patients in Korea is about 4,000.

AstraZeneca's "Koselugo" (selumetinib), a treatment for pediatric patients aged 3 and older with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1, was approved in May 2021, but it is also not yet covered by insurance. However, on the 7th, it finally passed the Drug Reimbursement Evaluation Committee, and discussions on reimbursement have started to gain momentum again.

Assemblywoman Choi Hye-young said, "There are still many ultra-expensive drugs without alternatives, and even when treatments exist, I often meet patients suffering because they cannot afford them." She added, "Reducing the actual burden on patients and improving accessibility to treatments for severe and intractable diseases that are directly linked to patients' lives are challenges that the government and the National Assembly must continue to consider and resolve."

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