Developing a patient-directed policy framework for managing orphan and ultra-orphan drugs throughout their lifecycle.
Developing a patient-directed policy framework for managing orphan and ultra-orphan drugs throughout their lifecycle.
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DOI：
10.1007/s40271-014-0108-6
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发表时间：
2015-02
影响因子：
3.6
通讯作者：
Wong-Rieger, Durhane
中科院分区：
文献类型：
--
作者：
Menon, Devidas;Stafinski, Tania;Dunn, Andrea;Wong-Rieger, Durhane
与孤儿药和超孤儿药有关的政策决定挑战了传统的决策过程，往往使受影响的人感到沮丧。一般来说，这些药物与临床获益、“物有所值”、可负担性和“采用/扩散”的重大不确定性有关，所有这些都是由于缺乏可用的高质量证据而引起的。越来越多的罕见病患者及其家属正在寻找机会，为旨在减少这些不确定性的举措做出贡献。因此，需要有一个指导他们参与的政策框架，以优化所产生的任何证据的影响。本研究的目的是（1）从加拿大罕见病社区患者的角度探索患者参与减少孤儿药和超孤儿药整个生命周期中决策不确定性的机会;（2）制定患者意见的政策框架，最大限度地提高患者参与孤儿药和超孤儿药决策不确定性的影响。举办了两次为期一天的会议和四次研讨会，与会者包括来自加拿大罕见病社区的患者和/或家庭，讨论了孤儿和超孤儿药物开发、获取和覆盖范围的问题，并确定了患者参与的机会，以减少相关决策的不确定性。他们的反馈和最近的一项关于罕见疾病患者参与的文献综述的结果被结合到一个基于Kingdon多流决策模型的政策框架草案中。该框架被提交给一组患者和其他利益相关者，包括供应商，药物计划经理和行业代表，然后进行相应的修订。患者和家庭成员/护理人员确定了在药物生命周期的所有阶段促进信息生成的切实方法。然而，这些信息与减少具体决策不确定性的接近程度各不相同。虽然与文献综述的结果相比，提到的可能方法的范围不那么广泛，但重点是相似的-捕获孤儿药或超孤儿药的临床获益。制定了一个包括三个阶段的政策框架，每个阶段都有一个关键问题和一组相应的子问题供患者提问。三个主要的连续问题如下。(1)需要解决哪些不确定性？(2)患者应该扮演什么角色？(3)每个角色都可行吗？减少孤儿药和超孤儿药的决策不确定性需要一个政策框架，明确何时以及需要生成何种类型的信息，并认识到患者在这些药物的整个生命周期中作为此类信息的重要来源的作用。
Policy decisions related to orphan and ultra-orphan drugs challenge traditional decision-making processes and often frustrate those affected by them. In general, these drugs are associated with significant uncertainties around clinical benefit, ‘value for money’, affordability, and ‘adoption/diffusion’, all of which arise from a lack of available high-quality evidence. Increasingly, patients with rare diseases and their families are looking for opportunities to contribute to initiatives aimed at reducing these uncertainties. Therefore, a policy framework for guiding their involvement is needed to optimize the impact of any evidence generated. The aims of this study were (1) to explore opportunities for patient involvement in reducing decision uncertainties throughout the lifecycle of orphan and ultra-orphan drugs from the perspectives of patients within the Canadian rare disease community; and (2) to develop a policy framework for patient input that maximizes the impact of their involvement on decision uncertainties around orphan and ultra-orphan drugs. Two one-day conferences and four workshops involving patients and/or families from rare disease communities in Canada were held to discuss issues around orphan and ultra-orphan drug development, access, and coverage, and identify opportunities for patient input to reduce related decision uncertainties. Their feedback and the findings from a recent literature review on patient involvement in rare diseases were combined into a draft policy framework based upon Kingdon’s multiple streams model of decision making. The framework was presented to a group of patients and other stakeholders, including providers, pharmaceutical drug plan managers, and industry representatives, and then revised accordingly. Patients and family members/caregivers identified tangible ways of contributing to the generation of information at all stages of the drug lifecycle. However, the proximity of that information to the reduction of a specific decision uncertainty varied. While the scope of possible ways mentioned was less broad when compared with the findings of the literature review, the focus was similar—capturing the clinical benefit of an orphan or ultra-orphan drug. A policy framework comprising three stages, each with a key question and corresponding set of sub-questions to be asked by patients, was developed. The three main sequential questions were as follows. (1) What uncertainties need to be addressed? (2) What roles should patients play? (3) Is each role feasible? Reducing decision uncertainties around orphan and ultra-orphan drugs requires a policy framework that explicates when and what type of information needs to be generated, and recognizes the role of patients as important sources of such information throughout the lifecycle of these drugs.