Department of Pharmacology, Faculty of Medicine, Masaryk University, Brno, Czechia.
Value Outcomes, Prague, Czechia.
Int J Technol Assess Health Care. 2023 Feb 7;39(1):e10. doi: 10.1017/S0266462323000053.
Out of 185 orphan medicinal products (OMPs) registered in 2015-2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia.
The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients' organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial.
We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of "value-based" decision making.
在 2015 年至 2021 年注册的 185 种孤儿药（OMP）中，仅有 110 种（59%）可供捷克患者使用，仅有 54 种（29%）获得官方报销。此外，这一比例随着时间的推移而稳步下降。经过多年由这种不理想的 OMP 患者可及性引发的公众辩论，国家观点转向为 OMP 的报销创建一个特殊途径。因此，最近捷克采用了一种严格的定价和报销程序，具有严格的时间表和详细的方法。
这项创新立法遵循了罕见病价值评估和资金流程的建议，并纳入了额外的价值元素，如社会视角。首先，有临床证据、成本效益和预算影响分析的申请由营销授权持有人或健康保险公司提交给政府卫生技术评估（HTA）机构。此外，专业协会和患者组织是该程序的合法参与者，提供证据和意见。然后，HTA 机构对证据进行评估/评估。随后，它发布一份总结可用信息的评估报告。该报告随后转发给卫生部及其咨询机构，由患者、临床专家、健康保险公司和国家组成。他们对文件进行批判性评估，并根据预先设定的决策标准发布具有约束力的意见。根据这一具有约束力的意见，HTA 机构作出决定。因此，咨询机构在这一过程中的作用至关重要。
我们相信，这种新方法可以为孤儿药提供令人满意的患者可及性。此外，它为“基于价值”的决策提供了一个真实的例子。
