联邦医疗保险和医疗补助服务中心强制进行药品价格谈判的《降低通货膨胀法》条款被批评为对药品创新的威胁。本研究基于大型制药公司和小型生物技术公司对临床试验和资本可用性的不同贡献,对《降低通货膨胀法案》对药品批准的潜在影响进行了建模。
■这项研究检查了研发费用,收入,以及公共生物制药公司的新投资(出售普通股和优先股),并赞助ClinicalTrials.gov.财务数据被纳入一个模型,该模型从报告的特定阶段成本和过渡率估计每个阶段的药物数量和批准,由不同规模的公司按比例赞助试验,基于公司规模的研发支出预计减少,以及大公司可以在临床阶段之间分配研发支出减少的三种情况:(1)研发按比例减少各个阶段;(2)研发在第2-3阶段不成比例地减少;(3)研发在第1-2阶段不成比例地减少。
■对1378家上市生物制药公司(2000-2018年)的财务数据进行了检查。研究和开发费用与79家市值≥70亿美元的大公司的收入相关,收入减少10%,研究和开发费用减少8.4%。对于1299家市值<70亿美元的小公司,研究和开发与新的投资有关,但不是收入。在2013-2018年的ClinicalTrials.gov中,较小的公司赞助了第一阶段的55.2%,第二阶段的55.6%和第三阶段的49.8%。在大公司和小公司之间分配临床试验并根据研发资源确定试验数量的临床开发模式中,进入开发的400种药物获得了47.3项批准(11.83%的批准率)。收入减少10%,反映了2000-2018年观察到的变化的上限,(1)在第1-3阶段的比例减少产生了45.1批准(减少了4.61%);(2)第2-3阶段的比例减少了42.8批准(减少了9.55%);(3)第1-2阶段的比例减少了46.9批准(减少了0.95%)。
■这项工作表明,《降低通货膨胀法》的药品价格谈判条款可能对药品批准的数量几乎没有影响。虽然大型制药公司可能会减少研发支出,小公司的持续研究和开发以及大公司对研究和开发资源的战略分配可能会减轻《降低通货膨胀法》的任何负面影响。
UNASSIGNED: Provisions of the Inflation Reduction Act mandating drug price negotiation by the Centers for Medicare & Medicaid Services have been criticized as a threat to pharmaceutical innovation. This study models potential impacts of the Inflation Reduction Act on drug approvals based on the differential contributions of large pharmaceutical companies and smaller biotechnology firms to clinical trials and the availability of capital.
UNASSIGNED: This study examined research and development expense, revenue, and new investment (sale of common and preferred stock) by public
biopharmaceutical companies and sponsorship of phased clinical trials in ClinicalTrials.gov. Financial data were incorporated in a model that estimates the number of drugs in each phase and approvals from reported phase-specific costs and transition rates, proportional sponsorship of trials by companies of different size, projected reductions in research and development spending based on company size, and three scenarios by which large companies may allocate reductions in research and development spending among clinical phases: (1) research and development proportionally reduced across phases; (2) research and development disproportionally reduced in phases 2-3; and (3) research and development disproportionately reduced in phases 1-2.
UNASSIGNED: Financial data were examined for 1378 public
biopharmaceutical companies (2000-2018). Research and development expense was associated with revenue for 79 large companies with market capitalization ≥$7 billion with a 10% reduction in revenue reducing research and development expense by 8.4%. For 1299 smaller companies with market capitalization <$7 billion, research and development was associated with new investment, but not revenue. Smaller companies sponsored 55.2% of phase 1, 55.6% of phase 2, and 49.8% of phase 3 trials in ClinicalTrials.gov 2013-2018. In a model of clinical development that apportions clinical trials between large and smaller companies and determines the number of trials based on research and development resources, 400 drugs entering development produced 47.3 approvals (11.83% rate). A 10% reduction in revenue, reflecting the upper boundary of observed changes 2000-2018, with (1) proportional reduction across phases 1-3 produced 45.1 approvals (4.61% reduction); (2) disproportional reduction of phases 2-3 produced 42.8 approvals (9.55% reduction); and (3) disproportional reduction of phases 1-2 produced 46.9 approvals (0.95% reduction).
UNASSIGNED: This work suggests that the drug price negotiation provisions of the Inflation Reduction Act could have little or no impact on the number of drug approvals. While large pharmaceutical companies may reduce research and development spending, continued research and development by smaller companies and strategic allocation of research and development resources by large companies may mitigate any negative effects of the Inflation Reduction Act.