Anis A H, Gagnon Y
Center for Health Evaluation and Outcome Sciences, St Paul's Hospital, Vancouver, British Columbia, Canada.
Pharmacoeconomics. 2000 Jul;18(1):55-62. doi: 10.2165/00019053-200018010-00006.
It is mandatory for drug manufacturers requesting formulary inclusion under the British Columbia (BC) provincial drug plan to submit a pharmacoeconomic analysis according to published guidelines. These submissions are reviewed by the Pharmacoeconomic Initiative (PI) of BC.
To assess the compliance of submitted studies with specific criteria outlined in the guidelines, to assess the methodological quality of individual submissions, and to demonstrate the importance of submitting guidelines-compliant pharmacoeconomic analyses.
All submissions between January 1996 and April 1999 assessed by the PI of BC were included. Submissions were reviewed according to a checklist to establish compliance with respect to choice of comparator drug, study perspective, sensitivity analysis, analytical horizon and discounting. Submissions were examined for association between analytical technique and author, and between source of submission and compliance. Association between compliance and recommendation for approval was also examined.
95 applications were reviewed. Seven submitted no analyses. There were 25 cost-comparison/consequence, 14 cost-effectiveness, 11 cost-minimisation, 9 cost-utility/benefit and 29 budget-impact analyses. 65 of these 88 submissions failed to comply with guidelines. Of these, 45% used an inappropriate comparator drug, 61% lacked a sensitivity analysis, 73% used a third-party payer and excluded a societal perspective, 66% did not provide a long term evaluation and 25% did not specify any time horizon. 80% of noncompliant studies were cost-comparison/consequence or budget-impact analyses (p < 0.001, Fisher's Exact). Of 25 cost-comparison/consequence and 29 budget-impact analyses, 19 (76%) and 24 (83%), respectively, were industry-conducted, whereas cost-effectiveness (11 of 14) and cost-utility/benefit (6 of 9) analyses were mostly subcontracted to private consultants or academics (p < 0.001, Fisher's Exact). 74% of all submissions (compliant and noncompliant) were not recommended by the PI for listing as a provincial drug plan benefit, 16% received approval for restricted benefit and 9% were recommended as full benefit. 80% of the noncompliant submissions were not recommended (p = 0.06, Fisher's Exact test). Moreover, a strong association between type of analysis and type of recommendation was found (p = 0.03, Fisher's Exact test). Cost-comparison/consequence and budget-impact analyses were less likely to be recommended. IMPLICATIONS OF FINDINGS: Our findings show poor compliance with guidelines, especially among industry-conducted studies. Possible explanations are lack of expertise in pharmacoeconomics and/or scepticism regarding the importance of guidelines and submission quality in decision making. As corroborated by the strong associations between type of recommendation and compliance, and between type of recommendation and type of analysis, these 2 characteristics have a significant impact on decision making.
根据不列颠哥伦比亚省（BC）省级药品计划，申请纳入药品目录的药品制造商必须按照已发布的指南提交药物经济学分析报告。这些报告由BC省药物经济学倡议组织（PI）进行审查。
评估提交的研究报告是否符合指南中规定的特定标准，评估各报告的方法学质量，并证明提交符合指南的药物经济学分析的重要性。
纳入了1996年1月至1999年4月间BC省PI评估的所有报告。根据一份清单对报告进行审查，以确定在对照药物选择、研究视角、敏感性分析、分析期限和贴现方面是否符合要求。审查报告中分析技术与作者之间以及提交来源与合规性之间的关联。还审查了合规性与批准建议之间的关联。
共审查了95份申请。7份未提交任何分析报告。有25份成本比较/结果分析、14份成本效益分析、11份成本最小化分析、9份成本效用/效益分析和29份预算影响分析。这88份报告中有65份不符合指南要求。其中，45%使用了不恰当的对照药物，61%缺乏敏感性分析，73%采用第三方支付方视角且未纳入社会视角，66%未进行长期评估，25%未明确任何时间期限。80%的不符合要求的研究是成本比较/结果分析或预算影响分析（p<0.001，Fisher精确检验）。在25份成本比较/结果分析和29份预算影响分析中，分别有19份（76%）和24份（83%）是由行业开展的，而成本效益分析（14份中的11份）和成本效用/效益分析（9份中的6份）大多分包给了私人顾问或学者（p<0.001，Fisher精确检验）。PI不建议将所有报告（包括符合和不符合要求的）中的74%列入省级药品计划福利清单，16%获得有限福利批准，9%被建议作为全额福利。80%的不符合要求的报告未获建议（p = 0.06，Fisher精确检验）。此外，发现分析类型与建议类型之间存在强烈关联（p = 0.03，Fisher精确检验）。成本比较/结果分析和预算影响分析获得建议的可能性较小。研究结果的启示：我们的研究结果表明对指南的遵守情况较差，尤其是在行业开展的研究中。可能的原因是缺乏药物经济学专业知识和/或对指南及提交质量在决策中的重要性持怀疑态度。建议类型与合规性之间以及建议类型与分析类型之间的强烈关联证实，这两个特征对决策有重大影响。
