Stanford Health Care, Stanford, CA.
Stanford University School of Medicine, Stanford, CA.
Am J Health Syst Pharm. 2019 Feb 21;76(6):381-386. doi: 10.1093/ajhp/zxy010.
A novel value-based approach to evaluate costly specialty drugs for formulary addition was developed.
In February 2016, Stanford Health Care launched the specialty drug subcommittee (SDSC), a subcommittee of the pharmacy and therapeutics committee, responsible for the formulary review of specialty pharmaceuticals. A process was developed for value-based review that includes not only consideration of clinical trial data and institutional acquisition costs but also internal patient outcomes and a cost-effectiveness model using internal financial data. A Markov model was developed to assess the value of trabectedin, which was approved for formulary addition in April 2016, relative to the addition of dacarbazine. The economic model and internal patient outcome analysis were presented to the prescribing oncologist and the SDSC for review. Internal data revealed that fewer patients than had been estimated received trabectedin, with outcomes significantly worse than those observed in the clinical trial leading to Food and Drug Administration approval. In the cost-effectiveness model, trabectedin had higher costs and poorer outcomes compared with dacarbazine. Based on the economic model, low utilization, and real-world outcomes, trabectedin was removed from formulary and a restrictive treatment pathway for nonformulary use, developed by the primary prescriber, was implemented. This process has since been applied to 11 more specialty drugs.
Internal cost-effectiveness models in combination with real-world patient outcomes data can be effective formulary management tools. Engagement and collaboration with the requesting provider are key to developing thoughtful treatment pathways.
开发了一种新颖的基于价值的方法来评估昂贵的专科药物以纳入处方集。
2016 年 2 月，斯坦福健康医疗中心（Stanford Health Care）成立了专科药物小组委员会（SDSC），这是药剂和治疗委员会的一个小组委员会，负责审查专科药品的处方集。制定了一个基于价值的审查流程，不仅考虑临床试验数据和机构收购成本，还考虑内部患者结果和使用内部财务数据的成本效益模型。开发了一个马尔可夫模型来评估在 2016 年 4 月获得处方集添加批准的 trabectedin 的价值，与添加达卡巴嗪进行比较。经济模型和内部患者结果分析被提交给处方肿瘤学家和 SDSC 进行审查。内部数据显示，接受 trabectedin 的患者人数比估计的要少，结果明显比导致食品和药物管理局批准的临床试验中观察到的结果更差。在成本效益模型中，与达卡巴嗪相比，trabectedin 的成本更高，结果更差。基于经济模型、低利用率和真实世界的结果，trabectedin 已从处方集删除，并由主要处方医生制定了非处方使用的限制性治疗途径。此后，该流程已应用于另外 11 种专科药物。
内部成本效益模型与真实世界患者结果数据相结合可以成为有效的处方集管理工具。与请求提供者的参与和合作是制定深思熟虑的治疗途径的关键。
