Centre for Research on Health and Social Care Management, SDA Bocconi School of Management, CERGAS, Bocconi University, Via Sarfatti 25, 20136, Milano, MI, Italy.
ROCHE Spa, Viale GB Stucchi 110, MB, 20900, Monza, Italy.
BMC Health Serv Res. 2021 Dec 4;21(1):1303. doi: 10.1186/s12913-021-07255-w.
Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economic benefit of CUP exists. This research aims to address this information gap by estimating the economic consequences of 11 CUP in Italy conducted between March 2015 and December 2020 from the perspective of public health care system in Italy (National Health Service). Eight programs concern cancer treatments, two refer to spinal muscular atrophy, and one is indicated for multiple sclerosis.
Since CUP medicines are covered by the industry, the net economic benefit includes: (i) avoided costs of the Standard of Care (SoC) the patients would have received had they not joined the CUP, (ii) costs not covered by the pharmaceutical industry sponsor, but instead sustained by payers, such as those associated with adverse events (only severe side effects resulting in hospitalisation and attributable to CUP medicines), and (iii) costs for combination therapies and diagnostic procedures not used with the SoC. The SoC costing relied on publicly available data. Information on adverse events and diagnostic procedures was retrieved from the CUP and monetized using the relevant fee for episode or service. One CUP was excluded since a SoC was not identified.
2,713 patients were treated in the 11 CUP where a SoC was identified. The SoC mean cost per patient ranged from €11,415 to €20,299. The total cost of the SoC ranged between €31.0 and €55.1 million. The mean cost per patient covered by hospitals hosting CUP was equal to €1,646, with a total cost of €4.5 million. The net economic benefit ranged €26.5 million - €50.6 million.
Despite research limitations, this paper illustrates for the first time the net economic impact of CUP from a public payer perspective. It is important to integrate these estimates with the prospective effects of CUP implementation, i.e., the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including effects from a societal perspective.
药品的同情用药计划（CUP）旨在满足在药品获得营销批准前为未参与试验的患者提供药品的伦理要求。先前已经研究了临床试验的经济影响。没有关于 CUP 的净经济效益的证据。本研究旨在通过从意大利公共医疗保健系统（国家卫生服务）的角度估计 2015 年 3 月至 2020 年 12 月期间在意大利进行的 11 个 CUP 的经济后果来填补这一信息空白。其中 8 个计划涉及癌症治疗，2 个与脊髓性肌萎缩症有关，1 个用于多发性硬化症。
由于 CUP 药品由行业覆盖，净经济效益包括：（i）患者如果不参加 CUP 将接受的标准治疗（SoC）的避免成本，（ii）制药行业赞助商不涵盖但由支付者承担的成本，例如与不良事件相关的成本（仅因 CUP 药物导致住院和归因于 CUP 药物的严重副作用），以及（iii）与 SoC 不一起使用的联合治疗和诊断程序的成本。SoC 成本依赖于公开可用的数据。不良事件和诊断程序的信息是从 CUP 中检索出来的，并使用相关的服务或事件费用进行货币化。由于没有确定 SoC，因此排除了一个 CUP。
在确定了 SoC 的 11 个 CUP 中，有 2713 名患者接受了治疗。每个患者的 SoC 平均成本范围为€11415 至€20299。SoC 的总成本范围为€3100 至€551000。主办 CUP 的医院覆盖的每位患者的平均成本为€1646，总成本为€450 万。净经济效益范围为€2650 万至€5060 万。
尽管存在研究限制，但本文首次从公共支付者的角度说明了 CUP 的净经济影响。从比较效益的角度来看，将这些估计与 CUP 实施的预期效果（即 CUP 中使用的药品与 SoC 的经济价值）结合起来很重要，包括从社会角度的影响。
