Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.
Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.
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DOI：
10.1111/cts.13027
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发表时间：
2021-07
期刊：
影响因子：
--
通讯作者：
Mantel-Teeuwisse AK
中科院分区：
文献类型：
--
作者：
Bloem LT;Vreman RA;Peeters NWL;Hoekman J;van der Elst ME;Leufkens HGM;Klungel OH;Goettsch WG;Mantel-Teeuwisse AK
我们的目的是确定欧洲药品管理局(EMA)确定的不确定性是否与国家卫生技术评估(HTA)机构的负面相对有效性评估(REA)和负面总体补偿建议有关。因此，我们确定了Haute Autoritéde Santé(HAS；法国)、国家健康与护理卓越研究所(NICE；英格兰/威尔士)、苏格兰医药联盟(SMC；苏格兰)和ZorgInstitute ut Nederland(Zin；荷兰)的所有HTA报告，这些报告涉及EMA在2009年至2010年批准的一组创新药物(不包括疫苗)。关于关键试验方法学、临床结果及其临床相关性的不确定性被结合起来，以反映低、中、高水平的不确定性。我们通过计算风险比(RR)和95%可信区间(CI)，以及REA和总体报销建议结果之间的一致性来评估相关性。我们确定了36种药物，在2009年9月至2018年7月期间，HTA机构发布了121项报销建议。高不确定性和低不确定性与负面原因和负面总体补偿建议的风险增加相关：RRS 1.9(95%可信区间0.9-3.9)和1.6(95%可信区间0.7-3.5)，这得到了进一步的敏感性分析的支持。我们发现33(27%)REA和总体补偿建议结果之间缺乏一致性，这些结果大多是限制性建议，在低或中等不确定性的情况下遵循负面原因。总而言之，EMA确定的高度不确定性与消极的理由和消极的总体补偿建议有关。为了减少不确定性并最终促进患者的有效接触，监管机构、HTA机构和其他利益相关者应该讨论如何在创新治疗的药物生命周期早期权衡和解决不确定性。
We aimed to determine whether uncertainties identified by the European Medicines Agency (EMA) were associated with negative relative effectiveness assessments (REAs) and negative overall reimbursement recommendations by national health technology assessment (HTA) agencies. Therefore, we identified all HTA reports from Haute Autorité de Santé (HAS; France), National Institute for Health and Care Excellence (NICE; England/Wales), Scottish Medicine Consortium (SMC; Scotland), and Zorginstituut Nederland (ZIN; The Netherlands) for a cohort of innovative medicines that the EMA had approved in 2009 to 2010 (excluding vaccines). Uncertainty regarding pivotal trial methodology, clinical outcomes, and their clinical relevance were combined to reflect a low, medium, or high level of uncertainty. We assessed associations by calculating risk ratios (RRs) and 95% confidence intervals (CIs), and agreement between REA and overall reimbursement recommendation outcomes. We identified 36 medicines for which 121 reimbursement recommendations had been issued by the HTA agencies between September 2009 and July 2018. High versus low uncertainty was associated with an increased risk for negative REAs and negative overall reimbursement recommendations: RRs 1.9 (95% CI 0.9–3.9) and 1.6 (95% CI 0.7–3.5), respectively, which was supported by further sensitivity analyses. We identified a lack of agreement between 33 (27%) REA and overall reimbursement recommendation outcomes, which were mostly restricted recommendations that followed on negative REAs in case of low or medium uncertainty. In conclusion, high uncertainty identified by the EMA was associated with negative REAs and negative overall reimbursement recommendations. To reduce uncertainty and ultimately facilitate efficient patient access, regulators, HTA agencies, and other stakeholders should discuss how uncertainties should be weighed and addressed early in the drug life cycle of innovative treatments.
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影响因子：
5.6
作者：
Malinowski KP;Kawalec P;Trabka W;Sowada C;Pilc A
通讯作者：
Pilc A
影响因子：
120.1
作者：
Eichler, Hans-Georg;Pignatti, Francesco;Breckenridge, Alasdair
通讯作者：
Breckenridge, Alasdair
影响因子：
6.7
作者：
Lipska, I.;Hoekman, J.;Hoevels, A. M.
通讯作者：
Hoevels, A. M.
影响因子：
5.6
作者：
Allen, Nicola;Liberti, Lawrence;Salek, Sam
通讯作者：
Salek, Sam
DOI：
10.1136/bmj.j4530
发表时间：
2017-10-04
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Davis C;Naci H;Gurpinar E;Poplavska E;Pinto A;Aggarwal A
通讯作者：
Aggarwal A