Schmidt L, Sehic O, Wild C
HTA Austria- Austrian Institute for Health Technology Assessment GmbH, Garnisongasse 7/20, 1090, Vienna, Austria.
J Pharm Policy Pract. 2021 Apr 28;14(1):37. doi: 10.1186/s40545-021-00317-8.
We considered the extent of the contribution of publicly funded research to the late-stage clinical development of pharmaceuticals and medicinal products, based on the European Commission (EC) FP7 research funding programme. Using two EC FP7-HEALTH case study examples-representing two types of outcomes-we then estimated wider public and charitable research funding contributions.
Using the publicly available database of FP7-HEALTH funded projects, we identified awards relating to late-stage clinical development according to the systematic application of inclusion and exclusion criteria, classified them according to product type and clinical indication, and calculated total EC funding amounts. We then identified two case studies representing extreme outcomes: failure to proceed with the product (hepatitis C vaccine) and successful market authorisation (Orfadin® for alkaptonuria). Total public and philanthropic research funding contributions to these products were then estimated using publicly available information on funding.
12.3% (120/977) of all EC FP7-HEALTH awards related to the funding of late-stage clinical research, totalling € 686,871,399. Pharmaceutical products and vaccines together accounted for 84% of these late-stage clinical development research awards and 70% of its funding. The hepatitis C vaccine received total European Community (FP7 and its predecessor, EC Framework VI) funding of €13,183,813; total public and charitable research funding for this product development was estimated at € 77,060,102. The industry sponsor does not consider further development of this product viable; this now represents public risk investment. FP7 funding for the late-stage development of Orfadin® for alkaptonuria was so important that the trials it funded formed the basis for market authorisation, but it is not clear whether the price of the treatment (over €20,000 per patient per year) adequately reflects the substantial public funding contribution.
Public and charitable research funding plays an essential role, not just in early stage basic research, but also in the late-stage clinical development of products prior to market authorisation. In addition, it provides risk capital for failed products. Within this context, we consider further discussions about a public return on investment and its reflection in pricing policies and decisions justified.
基于欧盟委员会（EC）的第七框架计划（FP7）研究资助项目，我们考量了公共资助研究对药品和医疗产品后期临床开发的贡献程度。通过两个代表两种不同结果的EC FP7-HEALTH案例研究示例，我们进而估算了更广泛的公共和慈善研究资助贡献。
利用公开可用的FP7-HEALTH资助项目数据库，我们根据纳入和排除标准的系统应用，识别与后期临床开发相关的奖项，根据产品类型和临床适应症对其进行分类，并计算欧盟委员会的总资助金额。然后我们确定了两个代表极端结果的案例研究：产品研发终止（丙型肝炎疫苗）和成功获得市场授权（用于治疗黑尿症的奥法丁®）。随后利用公开可用的资助信息估算了对这些产品的公共和慈善研究总资助贡献。
所有EC FP7-HEALTH奖项中有12.3%（120/977）与后期临床研究资助相关，总计686,871,399欧元。药品和疫苗共同占这些后期临床开发研究奖项的84%及其资助的70%。丙型肝炎疫苗获得了欧盟（FP7及其前身欧盟第六框架计划）总计13,183,813欧元的资助；该产品开发的公共和慈善研究总资助估计为77,060,102欧元。行业赞助商认为该产品进一步开发不可行；这现在代表了公共风险投资。用于治疗黑尿症的奥法丁®后期开发的FP7资助非常重要，其资助的试验构成了市场授权的基础，但尚不清楚该治疗的价格（每位患者每年超过20,000欧元）是否充分反映了大量的公共资助贡献。
公共和慈善研究资助不仅在早期基础研究中发挥着重要作用，而且在产品上市前的后期临床开发中也发挥着重要作用。此外，它为失败产品提供了风险资本。在此背景下，我们认为有必要进一步讨论公共投资回报及其在定价政策和决策中的体现。
