Concerns have emerged that follow-up research crucial for administering customized treatments to children aged 2 to 6 with rare diseases could face setbacks. This worry stems from a nearly 28% reduction in the budget for the Korean version of the Advanced Research Projects Agency for Health (ARPA-H) project during the government’s review process.
News1 reported on Monday that the proposed government budget for the ARPA-H project next year is approximately 107.367 billion KRW (80.1 million USD), down about 41.263 billion KRW (30.8 million USD) from the initial request of 148.63 billion KRW (about 110.9 million USD).
The budget for mission-driven research and development decreased from 136 billion KRW (101.5 million USD) to 96 billion KRW (about 71.6 million USD), while the foundational budget was adjusted from 12.63 billion KRW (9.42 million USD) to 11.367 billion KRW (about 8.48 million USD).
The Ministry of Health and Welfare’s budget request was revised during the government research and development (R&D) budget review led by the Ministry of Science and Information and Communications Technology (ICT). Insufficient strategies for result dissemination and low research fund execution rates were cited as reasons for the cuts.
One area particularly at risk due to budget cuts is the development of patient-customized treatments for rare diseases. Even with treatment designs completed, the process must still navigate manufacturing, non-clinical trials, and clinical trial plan approvals before patient administration. Delays in this process could result in missed treatment opportunities as patients’ conditions progress.
The HEART project, aimed at developing a patient-customized innovative treatment platform for pediatric rare diseases, focuses on creating treatments tailored to individual genetic mutations. It assesses safety and efficacy through single-patient clinical trials.
Currently, research targets conditions such as congenital disorders of glycosylation and hereditary childhood epilepsy. Patients with congenital disorders of glycosylation are between 2 to 6 years old.
The research team has completed the design of patient-customized antisense oligonucleotide (ASO) treatments using tissue samples from these young patients. They are now preparing documentation for drug manufacturing, quality control, and non-clinical trials, while also pursuing production that meets good manufacturing practice standards.
The project timeline aims for clinical trial plan approval from the Ministry of Food and Drug Safety in 2027, following manufacturing, quality control, and non-clinical trials, with patient administration targeted for 2029. Considering potential disease progression, the team is exploring ways to expedite administration through consultations with regulatory agencies.
The main concern is that budget cuts could cause cascading delays in subsequent procedures. Manufacturing, quality control, and non-clinical trials are critical steps for entering clinical trials and constitute the majority of the second phase funding. Delays here could disrupt the timeline for translating design achievements into actual patient treatments.
While the two young patients are not currently in critical condition, treatment delays could lead to irreversible disease progression. Given the nature of patient-customized treatments, which must consider both development schedules and disease progression rates, research delays could result in lost treatment opportunities.
The BEACON project, focusing on overcoming visual impairments caused by hereditary eye diseases, has also completed patient-customized gene correction designs and is preparing for clinical trial approval. This research develops precise gene correction strategies for major causative mutations in hereditary retinal diseases among Koreans.
Rare disease treatment technologies face challenges due to small patient populations and high development costs, deterring private investment. The strategy is to secure technological foundations and clinical applicability through national R&D, then seek investments from private companies or nonprofit foundations.
The issue of budget cuts for health and medical R&D, including the Korean ARPA-H project, is expected to be addressed during the National Assembly’s review of next year’s budget. Key discussion points will likely include ongoing research continuity, justification for budget cuts, and the need to adjust research objectives.
Park Ji-won, a Democratic Party member of the National Assembly’s Health and Welfare Committee and Special Committee on Budget and Accounts, stated in a recent News1 interview that projects maintaining research objectives despite budget cuts need reevaluation. He emphasized restoring appropriate budgets for projects requiring continuity and adjusting goals for those where cuts are justified.