Gray Cori, Kenney James T
Health Economics & Outcomes Research Fellow, Sanofi, Bridgewater, NJ.
Founder, JTKENNEY, Waltham, MA, and former Manager, Specialty and Pharmacy Contracts, Harvard Pilgrim Health Care.
Am Health Drug Benefits. 2019 Dec;12(8):390-398.
In therapeutic areas with uncertainty regarding clinical outcomes that are dependent on high-cost specialty medications, outcomes-based contracts can be a tool to reduce financial risk for payers and for drug manufacturers. With a high treatment cost, large number of therapy choices, and variability of responses to therapy across patients, multiple sclerosis is a compelling therapeutic area to support outcomes-based contracts.
To identify the necessary conditions to support the widespread adoption of outcomes-based contracts for high-cost drug therapy, with a focus on disease-modifying therapies for multiple sclerosis.
We conducted a series of in-depth, semi-structured phone interviews during fall 2018 with 17 healthcare stakeholders representing payers, manufacturers, and industry consultants, all of whom had some involvement in outcomes-based contract development or evaluation. The qualitative data management program from QSR International, N-VIVO 11, was used to store, organize, categorize, analyze, and produce visualization tools to explore, map ideas, and understand themes from the data.
Overall, payers and manufacturers agreed that outcomes-based contracts are an effective vehicle to mitigate financial risk and deliver value for disease-modifying therapies for multiple sclerosis, but they noted that the widespread adoption of outcomes-based contracts was tempered by 5 broad categories of challenges, including data-related issues, outcome measurement and confounding factors, regulatory barriers, levels of risk mitigation, and patient adherence. The majority of participants were receptive to using blood-based clinical biomarkers as outcomes-based contract end points, as long as the biomarkers are validated, accurately predict clinical outcomes, are well-established in the therapeutic area, and are readily accessible to various stakeholders.
Our findings indicate there is general support from payers and drug manufacturers to adopt outcomes-based contracts for disease-modifying therapies for multiple sclerosis. However, some conditions need to be met to allow their widespread adoption, including resolving data issues, ensuring patient adherence to therapy, having a level of risk mitigation that is significant for both parties to make the endeavor economically worthwhile, and fostering a supportive regulatory environment. Blood-based clinical biomarkers that meet certain criteria could be viable end points in outcomes-based contract for disease-modifying therapies for multiple sclerosis and can address many of the necessary conditions regarding data issues, including timeliness.
在临床结果存在不确定性且依赖高成本专科药物的治疗领域，基于结果的合同可以成为降低付款方和药品制造商财务风险的一种工具。由于治疗成本高、治疗选择多以及患者对治疗反应的变异性，多发性硬化症是支持基于结果的合同的一个极具吸引力的治疗领域。
确定支持广泛采用针对高成本药物治疗的基于结果的合同的必要条件，重点关注多发性硬化症的疾病修正治疗。
2018年秋季，我们对17位医疗保健利益相关者进行了一系列深入的半结构化电话访谈，这些利益相关者代表付款方、制造商和行业顾问，他们都参与了基于结果的合同开发或评估。使用QSR International公司的定性数据管理程序N-VIVO 11来存储、组织、分类、分析并制作可视化工具，以探索、梳理思路并理解数据中的主题。
总体而言，付款方和制造商一致认为，基于结果的合同是减轻财务风险并为多发性硬化症的疾病修正治疗提供价值的有效手段，但他们指出，基于结果的合同的广泛采用受到5大类挑战的限制，包括数据相关问题、结果测量和混杂因素、监管障碍、风险减轻水平以及患者依从性。只要生物标志物经过验证、能准确预测临床结果、在治疗领域已确立且各利益相关者都易于获取，大多数参与者都愿意将基于血液的临床生物标志物用作基于结果的合同终点。
我们的研究结果表明，付款方和药品制造商普遍支持采用针对多发性硬化症疾病修正治疗的基于结果的合同。然而，要使其广泛采用需要满足一些条件，包括解决数据问题、确保患者坚持治疗、具备对双方而言使该努力在经济上值得的风险减轻水平，以及营造支持性的监管环境。符合某些标准的基于血液的临床生物标志物可以成为多发性硬化症疾病修正治疗基于结果的合同中的可行终点，并可解决许多与数据问题相关的必要条件，包括及时性。
