Schmidt L, Sehic O, Wild C
Austrian Institute for Health Technology Assessment GmbH, Garnisongasse 7/20, 1090, Vienna, Austria.
J Pharm Policy Pract. 2022 Aug 16;15(1):47. doi: 10.1186/s40545-022-00445-9.
Lack of transparency around manufacturing costs, who bears the bulk of research and development costs and how total costs relate to the pricing of products, continue to fuel debates. This paper considers the case of olaparib (Lynparza®), recently indicated for use among BRCA-mutant breast cancer patients, and estimates the extent of public and philanthropic R&D funding.
We know from previous work that attempting to ascertain the amount of public and philanthropic funding using purely bibliographic sources (i.e., authors' declarations of funding sources and amounts traced through funders) is limited. Since we knew that a publically funded research unit was pivotal in developing olaparib, we decided to supplement bibliographic data with a Freedom of Information request for administrative records on research funding data from this research centre.
In terms of stages of product development, work conducted in the pre-clinical research stage was the most likely to report non-industry funding (> 90% of pre-clinical projects received public or philanthropic funding). Clinical trials were least likely to be funded through non-industry sources-although even here, contrary to the popular assertion that this is wholly industry-financed, we found public or philanthropic funding declared by 23% of clinical trials. Using information reported in the publications, we identified approximately £128 million of public and philanthropic funding that may have contributed to the development of olaparib. However, this amount was less than one-third of the total amount received by one research institute playing a pivotal role in product discovery. The Institute of Cancer Research reported receiving 38 funding awards to support olaparib work for BRCA-mutant breast cancer totalling over £400 million.
Government or charitable funding of pharmaceutical product development is difficult to trace using publicly available sources, due to incomplete information provided by authors and/or a lack of consistency in funding information made available by funders. This study has shown that a Freedom of Information request, in countries where such requests are supported, can provide information to help build the picture of financial support. In the example of olaparib, the funding amounts directly reported considerably exceeded amounts that could be ascertained using publically available bibliographic sources.
生产成本缺乏透明度、谁承担大部分研发成本以及总成本与产品定价之间的关系,继续引发争论。本文以奥拉帕利(Lynparza®)为例,该药最近被批准用于携带BRCA突变的乳腺癌患者,并估算了公共和慈善研发资金的规模。
我们从之前的工作中了解到,试图仅通过文献来源(即作者对资金来源和通过资助者追踪到的金额的声明)来确定公共和慈善资金的数额是有限的。由于我们知道一个由公共资金资助的研究单位在奥拉帕利的研发中起关键作用,我们决定通过信息公开请求,补充该研究中心关于研究资金数据的行政记录,以此补充文献数据。
就产品开发阶段而言,临床前研究阶段开展的工作最有可能报告非行业资金(超过90%的临床前项目获得了公共或慈善资金)。临床试验最不可能通过非行业来源获得资金——尽管即便在此,与普遍认为的临床试验完全由行业资助相反,我们发现23%的临床试验声明获得了公共或慈善资金。利用出版物中报告的信息,我们确定了约1.28亿英镑的公共和慈善资金,这些资金可能为奥拉帕利的研发做出了贡献。然而,这一数额不到在产品发现中起关键作用的一家研究机构所获资金总额的三分之一。癌症研究所报告称,其获得了38项资助奖励,以支持针对携带BRCA突变的乳腺癌开展的奥拉帕利相关研究,总计超过4亿英镑。
由于作者提供的信息不完整和/或资助者提供的资金信息缺乏一致性,利用公开可用来源很难追踪政府或慈善机构对药品开发的资助情况。本研究表明,在支持此类请求的国家,通过信息公开请求可以获取有助于构建资金支持情况图景的信息。以奥拉帕利为例,直接报告的资助金额大大超过了利用公开可用文献来源所能确定的金额。