ObjectivesTo provide a useful framework for improved understanding of international value drivers in the decision-making process of medical insurance access, and to explore the value assessment criteria of orphan drugs from stakeholders’ perspective.MethodsPubMed, EMbase, CINAHL Plus, ProQuest, Web of Science, CNKI and WanFang Data databases were electronically searched to collect studies from January 1st, 1983 to December 31st, 2018. Research questions were constructed based on SPIDER model. We established the inclusion and exclusion criteria to filter studies. Study quality was evaluated using the Critical Appraisal Skills Programme (CASP) checklist. A thematic synthesis was undertaken to develop descriptive themes, analytical constructs and third-level themes of value drivers by NVivo 11 software, and confidence in the findings was assessed using the CERQual method.ResultsA total of 10 studies including 20 research countries were included. Fifty descriptive themes were interpreted and embedded within 14 analytical constructs and 3 third-level themes after induction. Specifically, 3 broad themes were disease-related influence factors, which included severity, unmet requirements, disease burden, affected individuals, and moral and ethical considerations; drug-related influence factors, which included safety, efficacy, economics, innovation, quality of evidence; and some external and non-pharmaceutical intrinsic properties factors, which included reimbursement status in other countries or regions, government goals and priorities, confirmed drug supply and impact on environment.ConclusionsIn addition to conventional considerations such as efficacy and pharmacoeconomics, stakeholders are willing to take a broader perspective when in the case of the value assessment of orphan drugs. Comprehensive understanding of these value drivers is important to shape policy and enhance decision-making.
ObjectiveTo systematically review the studies on the clinical comprehensive evaluation system of drugs at home and abroad. MethodsThe PubMed, EMbase, Cochrane Library, CNKI, WanFang Data, VIP and CBM databases were electronically searched to collect the literature and policy documents of the clinical comprehensive evaluation system of drugs from inception to October 31, 2022. The evaluation organization, evaluation perspective, application scope, domains and criteria were sorted out, and the evaluation of different types of drugs was analyzed in detail. ResultsA total of 101 clinical comprehensive evaluation systems of drugs were included. The publishing time was from 2009 to 2022, and the number of published articles increased. The first author mainly came from 26 countries, including China (n=34), Canada (n=11), the United States (n=10), and Spain (n=10). Forty-seven articles reported the evaluation perspective. All evaluation systems could be used for drug evaluation, of which 43 could be used as universal evaluation tools, and 58 could be used for the evaluation of specific drugs, mainly including anti-tumor drugs (n=15), orphan drugs (n=10) and traditional Chinese medicine (n=7). The number of evaluation domains varied from 2 to 22, and the number of criteria varied from 4 to 56. Among them, economics (n=73), effectiveness (n=72), safety (n=54), disease demand/burden (n=34), and innovation (n=24) were the most frequent evaluation domains. ConclusionThe research on clinical comprehensive evaluation of drugs at home and abroad has grown up. During the implementation of clinical comprehensive evaluation of Chinese patent medicine, it is necessary to clarify the evaluation perspective, define the evaluation scope, and determine the evaluation domains.