"I tried every medication without any effect, but a month after starting the new drug, my shortness of breath disappeared. I can now walk short distances almost as well as when I was healthy."
Patient A, in his 50s, has been battling pulmonary arterial hypertension for years. Despite using three medications including Remodulin injections, his breathing difficulties worsened, forcing him into intensive care late last year. Pulmonary arterial hypertension is a condition in which pressure in the arteries supplying blood from the heart to the lungs becomes abnormally high. It causes shortness of breath, chest pain and fainting, with constant fear of sudden death. Its five-year survival rate is 71.8%, lower than that of cancer. After doctors recommended combination therapy with a new drug in January, Patient A's symptoms improved enough for discharge within a month. However, he worries about how long he can continue treatment given monthly medication costs exceeding 10 million won ($7,300).
According to the medical community on Tuesday, concerns have emerged over the effectiveness of the pilot program combining "new drug approval, reimbursement evaluation and drug price negotiation" — known as Heo-Pyeong-Hyeop — introduced to ease treatment cost burdens for patients with rare and severe diseases. Launched in June 2023, the program aims to shorten the period for listing new drugs under national health insurance coverage from the existing 330 days to 150 days by running in parallel procedures previously conducted sequentially by three agencies: the Ministry of Food and Drug Safety, the Health Insurance Review and Assessment Service, and the National Health Insurance Service. However, critics say that most drugs selected over nearly three years of the program have failed to produce meaningful results, raising false hopes among patients.
Qarziba (dinutuximab beta), a neuroblastoma treatment selected as the first drug under the program, made a smooth start by receiving approval in June 2024 — seven months after its application in October 2023 — and being listed for reimbursement in December of the same year. In contrast, Bylvay (odevixibat), a treatment for progressive familial intrahepatic cholestasis, took one year and two months to be listed for reimbursement after approval. Counting from the application date, the process took well over 400 days, undermining the program's purpose. The performance of drugs selected in the second round in 2024 has been even more disappointing. Winrevair (sotatercept), a pulmonary arterial hypertension treatment, has remained at the reimbursement adequacy evaluation stage for nearly nine months since its approval in July 2025. Fintepla (fenfluramine), a treatment for Dravet syndrome, is approaching four months since approval, and Limkato (anbal-cel), Korea's first domestically developed chimeric antigen receptor (CAR)-T cell therapy, has not even received approval.
Against this backdrop, when the government in January announced measures to strengthen support for rare and severe intractable diseases — aiming to shorten drug reimbursement procedures to within 100 days by streamlining reimbursement adequacy evaluation and drug price negotiation — controversy over its effectiveness erupted within and outside the medical community. The Korea Alliance of Patients Organization and the Korean Congenital Heart Disease Patients Association issued a joint statement last month, criticizing that "the pilot program's intent of shortening the listing period by running evaluation and negotiation in parallel from before approval is not working at all," and demanded authorities "clearly disclose the causes of reimbursement delays and immediately normalize the stalled procedures." Sotatercept was launched in Japan at its insurance price in August last year, and about 100 new prescriptions are reportedly issued monthly. Thanks to Japan's "designated intractable disease" system, which sets out-of-pocket caps based on income levels, patients there pay less than those in Korea.
The government is also making persistent efforts to improve treatment access for rare and severe diseases. Effective the 15th of this month, an amendment to the "Rules on Standards for Health Insurance Benefits" was enacted, allowing the National Health Insurance Service and pharmaceutical companies to reach separate agreements when determining new drug prices for severe rare diseases designated by the Ministry of Health and Welfare. For new drugs designated by the health minister, the NHIS and pharmaceutical companies can now separately agree on ceiling prices for drugs covered by health insurance. The challenge is that it is difficult to justify the high prices of innovative new drugs working through novel mechanisms when compared with existing drugs whose prices have fallen after decades on the market. At the economic evaluation stage, the "incremental cost-effectiveness ratio (ICER)" — which divides the additional cost required to achieve improvement by how much more effective the new drug is compared with existing treatment — serves as a key indicator. However, rare and severe diseases, which affect small patient populations and require long-term treatment, inherently tend to produce high ICER values. A structural contradiction also exists: the more dramatically a drug improves patient survival, the longer the treatment period becomes, increasing health insurance spending. This means that even when approval, evaluation and negotiation are conducted in parallel, applying the same criteria as in the past inevitably prolongs reimbursement procedures.
Experts agree that an evaluation system reflecting the innovation of new drugs for rare and severe diseases must be established. "Cases showing clinically meaningful improvements after administration of new drugs are being confirmed, making the reality of limited treatment access even more regrettable," said Jeong Wook-jin, president of the Korean Society of Pulmonary Hypertension and professor at Gachon University Gil Medical Center. "More flexible reimbursement evaluation criteria need to be applied to properly reflect the characteristics of diseases and the innovation of drugs."
