Mahlich Jörg, Bartol Arne, Dheban Srirangan
Health Economics and Outcomes Research, Janssen, Pharmaceutical Companies of Johnson & Johnson, Neuss, Germany.
Düsseldorf Institute of Competition Economics (DICE), University of Düsseldorf, Düsseldorf, Germany.
Health Econ Rev. 2021 Jan 16;11(1):4. doi: 10.1186/s13561-021-00302-6.
The productivity of pharmaceutical research and development (R&D) investments is declining due to high failure rates in clinical research. Recently, the US Food and Drug Administration (FDA) acknowledged that adaptive designs can make drug development more efficient and less costly. Our objective is to simulate cost-saving effects and estimate the impact on global R&D expenditures as well as possible outcomes measured in life-years gained.
Based on published drug-development cost data we calculate potential cost savings derived from variations in clinical success rates that result from employing adaptive trial designs. In a subsequent step we estimate how those cost changes affect global R&D expenditures and outcomes.
Our calculations indicate that an adaptive trial design with the potential to increase success rates of clinical trials by 4 percentage points could lower development costs for a new drug from 2.6 to 2.2bn USD. On a global scale, this cost reduction would free up an additional 4.2bn USD for investment into pharmaceutical R&D to bring about drug innovations that in turn would be capable of generating up to 3.5 million life-years.
New clinical trial designs are crucial to improving productivity within the pharmaceutical industry and to fostering a sustainable health-care system.
由于临床研究的高失败率，制药研发（R&D）投资的生产率正在下降。最近，美国食品药品监督管理局（FDA）承认适应性设计可以使药物开发更高效、成本更低。我们的目标是模拟成本节约效果，并估计对全球研发支出的影响以及以获得的生命年数衡量的可能结果。
基于已发表的药物开发成本数据，我们计算采用适应性试验设计导致临床成功率变化所带来的潜在成本节约。在后续步骤中，我们估计这些成本变化如何影响全球研发支出和结果。
我们的计算表明，一种有可能将临床试验成功率提高4个百分点的适应性试验设计，可以将一种新药的开发成本从26亿美元降至22亿美元。在全球范围内，这种成本降低将为制药研发释放额外的42亿美元用于投资，从而带来药物创新，进而能够产生多达350万个生命年。
新的临床试验设计对于提高制药行业的生产率和促进可持续的医疗保健系统至关重要。
