试图量化药物开发和财务回报之间的关系的模型缺少一个关键元素:对批准后研究的影响
Dan Crippen1, Kirsten Axelsen1
1Board Advisor and Former Congressional Budget Office Director, Senior Policy Advisor DLA Piper, New York, NY 10029, United States.
Health affairs scholar
|October 23, 2025
概括
通过改变财务回报,通货膨胀减缓法案 (IRA) 可能会减少对关键的批准后药物研究的投资. 政策制定者需要模型来评估对整个药物开发生命周期的影响,以避免低估后果.
科学领域:
- 生物制药政策 生物制药政策
- 卫生经济学 卫生经济学
- 药物开发生命周期 药物开发生命周期
背景情况:
- "通货膨胀减缓法案" (IRA) 在美国引入了药品价格的行政设定.
- 政策评估的现有经济模型往往忽视了批准后的研究和开发.
- 批准后的研究对于扩大药物指示和验证新患者群体的疗效至关重要.
研究的目的:
- 突出IRA的定价机制对药物研究投资的后审核影响.
- 强调需要全面的经济模型,包括整个药物开发生命周期.
- 通知决策者关于当前政策评估的潜在意外后果.
主要方法:
- 本评论分析了行政药品定价对制药投资的经济影响.
- 它审查了现有的政策影响模型,并确定了它们在市场后研究方面的局限性.
- 该分析侧重于在价格控制下开发二次指示的财务激励.
主要成果:
- 在IRA的价格控制,在批准后的研究时有效,减少从二次指示预期的财务回报.
- 这种金融激励措施的减少可能会阻止对必要的市场后研究的投资.
- 目前的政策模式风险低估了对药物开发的负面影响,因为排除了批准后的研究.
结论:
- 评估生物制药政策的模型必须纳入对新药和批准后药物开发的影响.
- 从经济评估中排除批准后的研究是一个重大差距,可能会损害健康结果,特别是对于慢性疾病.
- 需要进一步的研究来开发全面的模型,以支持整个药物开发生命周期的投资.
更多相关视频
06:40Author Spotlight: Cost-Effective Transcriptomic Drug Screening - Unlocking New Targets
Published on: February 23, 2024
1.7K
08:47Experimental Quantification of Interactions Between Drug Delivery Systems and Cells In Vitro: A Guide for Preclinical Nanomedicine Evaluation
Published on: September 28, 2022
2.3K
相关概念视频
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
404
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
404
01:11Clinical Trials: Overview
4.6K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
4.6K
01:20Measurement of Bioavailability: Pharmacodynamic Methods
230
Pharmacodynamic methods provide insights into a drug's effects on physiological processes over time and play a crucial role in understanding bioavailability and therapeutic efficacy. These methods can be broadly classified into acute pharmacological and therapeutic response approaches, each with distinct mechanisms and applications.The acute pharmacological response method directly correlates a drug's physiological effects, such as ECG or pupil diameter changes, to its time course in the body.
230
01:20Drug Product Performance: In Vitro–In Vivo Correlation
228
In pharmaceutical development, it's crucial to establish a predictive in vitro–in vivo correlation (IVIVC) for two or more formulations to gain a comprehensive understanding of release properties. IVIVC reduces the need for costly in vivo studies and facilitates the establishment of meaningful dissolution specifications with significant cost savings and decreased regulatory burden. Furthermore, a meaningful IVIVC should predict Cmax and AUC within 20%, aligning with FDA guidance while...
228
01:28Preclinical Development: Overview
5.8K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
5.8K
01:26Drug Discovery: Overview
11.0K
Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
11.0K