Kolasa Katarzyna, Zwolinski Krzysztof M, Kalo Zoltan, Hermanowski Tomasz
Department of Health Economics, Nicolaus Copernicus University Collegium Medicum, Sandomierska 16, 85-830, Bydgoszcz, Poland.
Advanced Management Training Programme in Pharmacoeconomics, Pharmaceutical Marketing and Law, Warsaw University of Technology Business School, Warsaw, Poland.
Orphanet J Rare Dis. 2016 Mar 10;11:23. doi: 10.1186/s13023-016-0388-0.
The objective of this study was to assess the potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement (P&R) process with regard to orphan drugs.
A four step approach was designed. Firstly, a systematic literature review was conducted to select the MCDA criteria. Secondly, a database of orphan drugs was established. Thirdly, health technology appraisals (HTA recommendations) were categorized and an MCDA appraisal was conducted. Finally, a comparison of HTA and MCDA outcomes was carried out. An MCDA outcome was considered positive if more than 50% of the maximum number of points was reached (base case). In the sensitivity analysis, 25% and 75% thresholds were tested as well.
Out of 2242 publications, 23 full-text articles were included. The final MCDA tool consisted of ten criteria. In total, 27 distinctive drug-indication pairs regarding 21 drugs were used for the study. Six negative and 21 positive HTA recommendations were issued. In the base case, there were 19 positive MCDA outcomes. Of the 27 cases, there were 12 disagreements between the HTA and MCDA outcomes, the majority of which related to positive HTA guidance for negative MCDA outcomes. All drug-indication pairs with negative HTA recommendations were appraised positively in the MCDA framework. Economic details were available for 12 cases, of which there were 9 positive MCDA outcomes. Amongst the 12 drug-indication pairs, two were negatively appraised in the HTA process, with positive MCDA guidance, and two were appraised in the opposite direction.
An MCDA approach may lead to different P&R outcomes compared to a standard HTA process. On the one hand, enrichment of the list of decision making criteria means further scrutiny of a given health technology and as such increases the odds of a negative P&R outcome. On the other hand, it may uncover additional values and as such increase the odds of positive P&R outcomes.
本研究的目的是评估多标准决策分析（MCDA）的实施对波兰孤儿药定价和报销（P&R）流程的潜在影响。
设计了一个四步方法。首先，进行系统的文献综述以选择MCDA标准。其次，建立孤儿药数据库。第三，对卫生技术评估（HTA建议）进行分类并进行MCDA评估。最后，对HTA和MCDA结果进行比较。如果达到最大点数的50%以上（基础情况），则MCDA结果被认为是积极的。在敏感性分析中，还测试了25%和75%的阈值。
在2242篇出版物中，纳入了23篇全文文章。最终的MCDA工具由十个标准组成。总共使用了涉及21种药物的27对独特的药物-适应症对进行研究。发布了6项负面和21项正面的HTA建议。在基础情况下，有19项积极的MCDA结果。在这27个案例中，HTA和MCDA结果之间存在12项分歧，其中大多数与HTA对MCDA负面结果的积极指导有关。所有HTA建议为负面的药物-适应症对在MCDA框架中均被积极评估。有12个案例提供了经济细节，其中有9项积极的MCDA结果。在这12对药物-适应症对中，有两对在HTA过程中被负面评估，但MCDA给出了积极指导，还有两对评估结果相反。
与标准的HTA流程相比，MCDA方法可能导致不同的P&R结果。一方面，决策标准列表的丰富意味着对特定卫生技术的进一步审查，因此增加了P&R结果为负面的几率。另一方面，它可能发现额外的价值，从而增加P&R结果为积极的几率。
