Department of Applied Health Research, University College London, 1-19 Torrington Place, London, WC1E 7HB, UK.
University of Oxford, Oxford, UK.
Patient. 2020 Oct;13(5):599-610. doi: 10.1007/s40271-020-00430-x.
Uncertainty about the benefits new cancer medicines will deliver in clinical practice risks delaying patient access to new treatment options in countries such as England, where the cost effectiveness of new medicines affects reimbursement decisions. Outcome-based payment (OBP) schemes, whereby the price paid for the drug is linked to patients' real-world treatment outcome(s) has been put forward as a mechanism to accelerate access. Although OBP schemes have generally focused on clinical outcomes to determine reimbursement, the degree to which these represent the outcomes that are important to patients is unclear.
To advance the application of OBP we ask, what outcomes do patients with cancer value (most) that might form a practical basis for OBP?
A review of the literature on outcomes in cancer produced a long list of candidates. These were evaluated in a focus group with patients with cancer and were then, in a second focus group, distilled to a shortlist of ten outcomes using a card sort method. The ten outcomes were included in an online survey of patients with cancer and carers, who were asked to rank the importance of each outcome.
The focus groups identified a range of both clinical and functional outcomes that are important to patients. Analyses of the 164 survey responses suggested that the four most important outcomes to patients and carers are survival; progression, relapse or recurrence; post-treatment side effects; and return to normal activities of daily life.
Commissioners of cancer services wishing to instigate an OBP scheme should prioritise collecting data on these outcomes as they are important to patients. Of these, only mortality data are routinely collected within the national health service (NHS). Progression and some morbidity data exist but are not currently linked, creating a challenge for OBP.
由于不确定新癌症药物在临床实践中会带来哪些益处，这可能会延迟患者在英国等国家获得新治疗方案的机会，因为新药的成本效益会影响报销决策。基于结果的支付（OBP）方案已被提出作为一种加速获取的机制，根据该方案，药物的支付价格与患者的实际治疗结果相关联。尽管 OBP 方案通常侧重于临床结果来确定报销，但这些结果在多大程度上代表了患者重要的结果尚不清楚。
为了推进 OBP 的应用，我们提出这样一个问题，即癌症患者看重哪些结果（最重要），这些结果可能为 OBP 提供实际依据？
对癌症相关结果的文献进行综述，产生了一长串候选结果。这些结果在癌症患者的焦点小组中进行了评估，然后使用卡片分类法在第二次焦点小组中将其提炼为十个结果的短名单。这十个结果被纳入癌症患者和护理人员的在线调查中，他们被要求对每个结果的重要性进行排名。
焦点小组确定了一系列对患者重要的临床和功能结果。对 164 份调查回复的分析表明，患者和护理人员最重要的四个结果是生存；进展、复发或转移；治疗后副作用；以及恢复正常的日常生活活动。
希望启动 OBP 计划的癌症服务专员应优先收集这些对患者重要的结果数据。在这些结果中，只有死亡率数据在国民保健制度（NHS）中常规收集。存在进展和一些发病率数据，但目前未相关联，这对 OBP 构成挑战。
