Benefit management for high-cost drugs, internalization of regulatory-reimbursement linkage system and cost-effectiveness exemption system

A policy proposal has emerged suggesting the need to strengthen the regulatory and reimbursement linkage system and urgently improve the cost-effectiveness evaluation exemption system for high-cost drugs. Mention of introducing a separate fund was also made as a long-term measure.

The Health Insurance Review and Assessment Service (HIRA) released a report on "Comparison of Domestic and Foreign Reimbursement Management Systems for High-Cost Drugs" that it conducted internally on the 5th. The lead researcher was Kim Yoo-jung, with Shin Hanna, Kim Ye-seul, and Cho Ssang-eun participating as co-researchers.

The researchers first pointed out the need for a new definition of high-cost drugs and manipulative definitions of domestic high-cost drugs, especially given the emergence of ultra-high-cost drugs such as gene therapy at the current juncture where the definitions of high-cost drugs domestically and internationally are ambiguous and inconsistent. They suggested that a valid criterion for defining high-cost drugs would be those with an annual per capita medication cost exceeding 100 million won (approximately 94,000 USD). However, they also emphasized that, considering the characteristics of future high-cost drugs, it would be desirable to utilize internal criteria rather than explicit ones.

The characteristics of high-cost drugs were summarized by referencing previous studies. The researchers noted that most are biological drugs such as cell therapy and gene therapy, and inherent uncertainty exists in clinical evidence due to limitations in clinical trial design. They also mentioned that in 2021, the best-selling drugs worldwide were mainly COVID-19 treatments like Comirnaty injection, with the majority of the rest being treatments for rare diseases or anticancer drugs.

Key issues related to high-cost drugs were also presented. Specifically, these include increased patient and financial burden due to high drug prices, pressure from pharmaceutical companies for listing, high demands from patient groups, the need for post-evaluation of clinical uncertainty when listed in health insurance, and the application of various exceptional criteria such as risk-sharing schemes for listing. There is also controversy over fairness in spending resources and the need for social consensus.

Regarding these issues, the researchers emphasized the need to improve exceptional systems without undermining the principles of drug selection and inclusion. They particularly stressed the importance of post-management of drugs that enter without clinical utility and cost-effectiveness evaluations.

Suggestions for dealing with high-cost drug reimbursement evaluations were presented in the short and medium to long term. From a short-term perspective, they highlighted the importance of solidifying the regulatory and reimbursement linkage system introduced domestically and expanding the effectiveness evaluation of high-cost drugs and the risk-sharing scheme based on performance. They emphasized that building a post-management system for high-cost drugs is a prerequisite for this.

Specifically, they emphasized the urgent need to improve the cost-effectiveness exemption system, operate it as a future cost-effectiveness evaluation deferral system, and reevaluate drugs introduced under the existing cost-effectiveness exemption system.

They also mentioned the need for improvement in the pre-approval system while considering ways to reduce practical burdens while increasing the effectiveness of the system.

In the medium to long term, they suggested considering the introduction of foreign early access schemes or bundled payment models. They stressed the need to increase early access to treatments for rare diseases or anticancer drugs through separate funds outside of health insurance and to create evidence to confirm the effectiveness and safety of new drugs.

HIRA evaluated this study as significant, stating that it provides insights into reviewing domestic and foreign high-cost drug-related systems, analyzing cases of domestic and foreign listing of high-cost drugs, and proposing policy directions for future high-cost drug reimbursement management systems by gathering opinions from relevant stakeholders through in-depth interviews.