高價基因治療藥物的健保定價挑戰與策略：Zolgensma案例分析

Abstract

本研究探討了高價基因治療藥物Zolgensma在台灣健保定價挑戰與策略，以對脊髓性肌肉萎縮症（Spinal Muscular Atrophy, SMA）的案例進行分析，並參考韓國經驗做比較。 隨著人口老齡化和醫療技術的進步，台灣的全民健康保險面臨巨大財務壓力，高價新藥的引進尤為突出。本研究旨在分析兩國在Zolgensma健保核價過程中的共通點與差異，並探討相關利益者（政府、藥廠、病友）在核價過程中的角色與影響。 研究方法包括文獻分析法及個案研究法，透過系統性地分析台灣與韓國的醫療保健統計資料、學術文獻和政策法規文件，來比較兩國在Zolgensma核價過程中的政策框架和法規要求。 研究發現，台灣和韓國均面臨高價基因治療藥物對健保基金的重大財務衝擊，兩國在核價過程中均採用醫療科技評估（HTA）及風險分擔協議（RSA）來降低財務風險。然而，台灣的審核時程較長，影響了新藥核准的速度，韓國則透過更快速的核價程序及付款方式以減少財務衝擊。此外，相關利益者在核價過程中的角色和影響存在差異，政府政策、藥廠的市場策略以及病友的權益訴求均對核價結果產生重要影響。 本研究提出了政策建議，包括提升基因治療的認識和支持、加強國際合作及多方利益相關者參與，以促進基因治療技術的應用和發展，確保患者能及時獲得所需的治療。預期研究結果將為政府决策者、醫療機構及藥廠在制定和執行核價策略時提供實證依據，進一步完善高價藥物的支付和核價政策。

This study explores the pricing challenges and strategies for the high-cost gene therapy drug Zolgensma in Taiwan's National Health Insurance (NHI) system, focusing on the case of Spinal Muscular Atrophy (SMA) and drawing comparisons with South Korea's experience. With the aging population and advancements in medical technology, Taiwan’s NHI faces immense financial pressure, particularly regarding the introduction of high-cost innovative drugs. This research aims to analyze the similarities and differences in Zolgensma pricing processes between Taiwan and South Korea and to examine the roles and impacts of key stakeholders (government, pharmaceutical companies, and patient groups) in these processes. The research methodology includes literature analysis and case study methods, systematically analyzing healthcare statistics, academic literature, and policy documents from Taiwan and South Korea. These data were used to compare the regulatory frameworks and policy requirements for Zolgensma pricing in the two countries. The findings reveal that both Taiwan and South Korea face significant financial challenges from high-cost gene therapies in their healthcare systems. Both countries utilize Health Technology Assessment (HTA) and Risk-Sharing Agreements (RSA) to mitigate financial risks. However, Taiwan’s longer review timelines delay new drug approvals, whereas South Korea adopts faster pricing procedures and payment methods to alleviate financial pressure. Additionally, the roles and impacts of stakeholders in the pricing process differ: government policies, pharmaceutical market strategies, and patient advocacy significantly influence the pricing outcomes. This study provides policy recommendations, including increasing awareness and support for gene therapies, strengthening international collaboration, and enhancing multi-stakeholder engagement to promote the application and development of gene therapy technologies. These measures aim to ensure timely access to treatment for patients. The findings are expected to offer empirical evidence for policymakers, healthcare institutions, and pharmaceutical companies in formulating and implementing pricing strategies, thereby improving policies related to the reimbursement and pricing of high-cost drugs.