The joy of overcoming blood cancer through hematopoietic stem cell transplantation is short-lived for many patients, who are soon overwhelmed by another concern. That’s because severe rare complication that follow transplantation, known as graft-versus-host disease (GVHD), threatens patients' survival and restrict their lives.

The problem is that this disease is excluded from both rare disease support and special cancer treatment programs. In this institutional blind spot, patients are left with no choice but to face the despair of being unable to use the treatment available to them.

On Wednesday, a policy discussion forum titled “Talking About Life After Blood Cancer: Improving the Treatment Environment for Severe and Rare Complications” was held by Rep. Seo Mi-hwa of the Democratic Party of Korea, a member of the National Assembly's Health and Welfare Committee. The forum served as a platform to raise public awareness of the plight of these patients.

GVHD, another threat after survival

Hematopoietic stem cell transplantation is considered the last hope for a cure for blood cancer patients. However, another ordeal awaits at the end of the transplantation process. GVHD, which occurs when the immune cells received during transplantation recognize the patient's body as foreign and attack it, affecting up to half of all transplant patients. Symptoms begin with skin rashes and oral mucosal damage and spread systemically to major organs, including lungs, liver, kidneys, and muscles.

In particular, chronic GVHD, which appears 100 days after transplantation, is accompanied by fibrosis, leading to organ hardening and functional loss. Forty-two percent of patients were found to have damage to four or more organs at the time of diagnosis.

Professor Kim Hye-ri of the Department of Pediatric Hematology and Oncology at Asan Medical Center, who gave the first presentation, said, “Even if cancer does not recur, death from GVHD complications is common,” identifying GVHD as “the leading cause of non-recurrence death in blood cancer patients.”

Chronic GVHD is a complication that causes patients more suffering than cancer itself, Professor Kim said, emphasizing the necessity of early diagnosis and active treatment environment improvements.

Patients pushed out of the healthcare system

The problem lies in the ambiguous classification of GVHD. Due to its status as a secondary disease, it is excluded from both rare disease designation and cancer disease designation. As a result, patients and medical professionals have raised their voices, saying that “GVHD patients are being pushed out of the system.”

According to patient groups, many patients say that even though there is a treatment available, they cannot use it for financial reasons. In particular, the average treatment cost for GVHD patients is more than 50 percent higher than that for general blood cancer patients, and the loss of income due to long-term treatment forces them to give up treatment.

Choi Hye-kyung, a GVHD patient who participated in the forum as a panelist, said, “Even something as simple as walking is a wish for patients, as their daily lives are completely shattered. It is cruel that we cannot even access treatment that is right in front of us because it is not covered by insurance.”

Belumosudil’s monthly medication cost alone reaches 10 million won

Chronic GVHD is initially treated with steroid therapy. However, the problem is that over half of patients do not respond, leading them to move on to second- or third-line treatments. Additionally, the second-line treatment, the JAK inhibitor, “ruxolitinib” (Jakavi in trademark name), is known to fail in halting fibrosis progression—the core issue in GVHD treatment—and thus does not provide a fundamental solution.

Amid this situation, the recently developed “belumosudil” (Rezurock in trademark name) has emerged as the first targeted therapy specifically designed for GVHD, demonstrating efficacy against both inflammation and fibrosis. While it has been approved as an orphan drug in Korea, it has not yet been included in the national health insurance coverage.

“Belumosudil regulates inflammation and fibrosis, showing a high response rate of 75 percent,” said Professor Kwak Dae-hoon of the Department of Hematology at the Catholic University of Korea Seoul St. Mary’s Hospital, who presented the second paper. “Its efficacy has also been proven in the lungs and liver, where previous treatments showed no improvement.”

However, Professor Kwak noted, “Belumosudil costs nearly 10 million won ($7,153) per month,” expressing concern that “patients are unable to afford the medication and are forced to give up third-line treatment, which is a tragic situation.”

“If rare disease treatments are approved but not covered by insurance, they have no meaning for patients,” said Park Jung-suk, secretary-general of the Korea Blood Disease and Cancer Association, who also participated as a panelist. “Despite being the last hope for patients, uncovered Belomosudil forces patients to bear the full cost of the expensive medication.”

As “strengthening support for patients with rare, severe, and chronic diseases” is included in “the reduction of national medical expenses section” of the national policy agenda recently announced by the Lee Jae Myung administration, it is urgent to reflect the desperate situation of GVHD patients and improve the reimbursement system for new drugs, Park emphasized.

Collapse of quality of life and socioeconomic burden

Chronic GVHD completely destroys patients' lives. According to a survey, 75 percent of patients have lost their income, and 25 percent have been permanently disabled and excluded from the labor market. One in three severe patients suffers from depression and anxiety.

A patient from a GVHD patient support group who attended the symposium as an audience member stated, “Some patients cannot even cry due to damaged tear glands, and others cannot walk for more than a few minutes due to lung function dropping below 30 percent. If this situation persists for years, patients become completely isolated economically and mentally.”

Experts warn that the socioeconomic losses caused by GVHD pose a burden on the entire nation. The decline in patients' labor productivity and the increase in long-term treatment costs strain the health insurance budget and could become a greater risk factor in an aging society.

“Chronic GVHD threatens patients' lives due to limited treatment options and high medical costs, yet they are excluded from medical benefits such as special billing exemptions, creating a blind spot,” Rep. Seo, the forum’s organizer, said. “We will discuss practical alternatives to enable blood cancer patients, who face life-threatening complications, to return to their daily lives.”

Kim Eun-hee, a section chief at the Ministry of Health and Welfare Insurance and Pharmaceutical Benefits Division, who participated as a government panelist, also said, “We will actively review the process for listing the drug on the national health insurance list to ensure it proceeds smoothly, as we have heard the urgent voices of patients.”

Kim Guk-hee, head of the Drug Management Division at the Health Insurance Review and Assessment Service, said, “We agree that treatments with high unmet needs are essential for patients. We will explore various options, including securing temporary funds through a fund.”