Novartis Pharma AG, Basel, Switzerland.
GE2P2 Global Foundation, Philadelphia, Pennsylvania.
JAMA Health Forum. 2022 Apr 15;3(4):e220475. doi: 10.1001/jamahealthforum.2022.0475. eCollection 2022 Apr.
Compassionate use (CU) is a treatment option for patients with serious or life-threatening medical conditions that provides access to locally unlicensed medications (generally free of charge) when all available treatment options have been exhausted and enrollment in a clinical trial is not possible.
To examine the disparity in CU access observed across countries and explore the key driving factors.
This study analyzed all Novartis CU requests (for individual/named patients and cohort programs) received between January 1, 2018, and December 31, 2020, and investigated selected country-specific factors for association with request activity. Data analysis was performed from February 2021 to February 2022.
Country-specific request activity was quantified using request counts and rates per million population and examined in stratified and multivariable analyses (negative-binomial regression) for association with the following covariates: existence of local CU regulations and their public availability, clinical trial activity, population size, and gross domestic product.
During the 36-month observation period, 31 711 CU requests were received from 110 countries, 23 194 (73%) of which came from only 10 high-income countries. All high-income countries combined accounted for 27 612 (87%) of all requests, while lower-middle-income and low-income countries contributed only 1021 (3%). Of all requests, 29 870 (94%) were from countries with CU regulations made publicly available on the internet, and higher request activity was demonstrated in countries conducting more clinical trials. Presence and public availability of CU regulations, population size, gross domestic product, and clinical trial activity were independently associated with the CU request activity in multivariable analysis.
In this cohort study analyzing Novartis CU requests over a 3-year period, existence and public availability of CU regulations and local clinical trial activity were positively associated with higher CU request rates. The analysis also identified an association between macroeconomic factors and CU request activity, despite the generally free provision of unlicensed therapeutic products. Similar analyses of other comparable experiences are needed to supplement these initial observations. Ultimately, better understanding of factors associated with CU request activity would translate into improved early access to novel lifesaving products for patients with unmet medical needs around the world.
同情用药（CU）是一种治疗选择，适用于患有严重或危及生命的医疗状况的患者，当所有可用的治疗选择都已用尽且无法参加临床试验时，为其提供当地未许可药物（通常免费）的途径。
本研究旨在考察各国之间观察到的 CU 可及性差异，并探讨关键驱动因素。
设计、设置和参与者：本研究分析了诺华公司在 2018 年 1 月 1 日至 2020 年 12 月 31 日期间收到的所有 CU 请求（针对个体/指定患者和队列计划），并对与请求活动相关的选定特定国家因素进行了调查。数据分析于 2021 年 2 月至 2022 年 2 月进行。
使用请求计数和每百万人口的请求率来量化特定国家的请求活动，并对分层和多变量分析（负二项回归）进行分析，以确定以下协变量与请求活动的关联：是否存在当地 CU 法规及其公开可用性、临床试验活动、人口规模和国内生产总值。
在 36 个月的观察期内，从 110 个国家收到了 31711 份 CU 请求，其中 23194 份（73%）来自仅 10 个高收入国家。所有高收入国家的总和占所有请求的 27612 份（87%），而中低收入和低收入国家仅贡献了 1021 份（3%）。所有请求中，29870 份（94%）来自于在互联网上公开提供 CU 法规的国家，且在开展更多临床试验的国家中，请求活动更为活跃。在多变量分析中，CU 法规的存在和公开可用性、人口规模、国内生产总值和临床试验活动与 CU 请求活动呈独立相关。
在这项为期 3 年的诺华 CU 请求队列研究中，CU 法规的存在和公开可用性以及当地临床试验活动与更高的 CU 请求率呈正相关。该分析还确定了宏观经济因素与 CU 请求活动之间的关联，尽管通常免费提供未许可的治疗产品。需要对其他类似经验进行类似的分析，以补充这些初步观察。最终，更好地了解与 CU 请求活动相关的因素将转化为改善全球有未满足医疗需求的患者对新型救生产品的早期获得。
