Department of Health Sciences and Health Policy, University of Lucerne, Lucerne, Switzerland.
Appl Health Econ Health Policy. 2019 Apr;17(2):143-162. doi: 10.1007/s40258-018-0437-z.
The aim of this review is to assess the current state of empirical research regarding the effectiveness of national pricing regulations of the patent-protected market for prescription pharmaceuticals. Effectiveness is understood to be the capacity of policies to have a desired impact on outcomes, such as health status, patient access, healthcare expenditure, and research investments, among others.
A systematic review of the published literature on pricing regulations in OECD countries was performed. The PubMed, MEDLINE, Scopus, Web of Science, Cochrane Library and the OECD iLibrary databases were searched in September 2016 and December 2017, with an update in August 2018. Interrupted time series studies and additional empirical studies were included, as well as systematic reviews if appropriate methods were applied. The risk of bias was assessed based on the recommendations of the BMJ guidelines, Cochrane EPOC criteria, QHES instrument, HTA good practice guidelines, CRD's guidance and the CHEC criteria. The quality of evidence was evaluated using the suggestions from EPOC and GRADE.
Thirty-one publications met the inclusion criteria. Most of the assessed empirical research included therapeutic (TRP) and/or external reference pricing (ERP), with a clear majority focusing on TRP. The main outcomes that were analysed were drug prices, expenditures and drug use. For value-based pricing (VBP), only limited empirical data were found.
We found evidence that TRP may reduce pharmaceutical prices and expenditures in the short term. Furthermore, TRP may lead to substitution effects towards lower-priced pharmaceuticals. The effects of TRP on patient access, healthcare utilisation and R&D investments were found to be uncertain. No conclusions were drawn for ERP and VBP. No evidence was found for the effects on health outcomes for any of the analysed policies. There is a strong need for evidence generation regarding effective pricing policies, particularly for VBP, managed entry agreements and non-financial outcomes.
本综述旨在评估当前关于专利保护处方药市场定价法规有效性的实证研究现状。有效性被理解为政策对诸如健康状况、患者可及性、医疗保健支出和研究投资等结果产生预期影响的能力。
对经合组织国家定价法规的已发表文献进行了系统综述。2016 年 9 月和 2017 年 12 月在 PubMed、MEDLINE、Scopus、Web of Science、Cochrane Library 和经合组织 iLibrary 数据库进行了检索，并于 2018 年 8 月进行了更新。纳入了中断时间序列研究和其他实证研究，如果使用了适当的方法，则还纳入了系统评价。根据 BMJ 指南、Cochrane EPOC 标准、QHES 工具、HTA 良好实践指南、CRD 指南和 CHEC 标准评估偏倚风险。使用 EPOC 和 GRADE 的建议评估证据质量。
有 31 篇出版物符合纳入标准。评估的实证研究大多包括治疗性（TRP）和/或外部参考定价（ERP），其中绝大多数侧重于 TRP。分析的主要结果是药品价格、支出和药品使用。对于基于价值的定价（VBP），仅发现了有限的实证数据。
我们发现证据表明，TRP 可能会在短期内降低药品价格和支出。此外，TRP 可能会导致向价格较低的药品的替代效应。TRP 对患者可及性、医疗保健利用和研发投资的影响尚不确定。对 ERP 和 VBP 没有得出结论。没有证据表明任何分析政策对健康结果有影响。特别需要针对有效的定价政策（特别是 VBP、管理准入协议和非财务结果）生成证据。
