| 来源类型 | Working Paper |
| 规范类型 | 报告 |
| DOI | 10.3386/w27978 |
| 来源ID | Working Paper 27978 |
| Valuing Rare Pediatric Drugs: An Economics Perspective | |
| Rena M. Conti; Jonathan Gruber; Daniel A. Ollendorf; Peter J. Neumann | |
| 发表日期 | 2020-10-26 |
| 出版年 | 2020 |
| 语种 | 英语 |
| 摘要 | There is a coming wave of novel genetic therapies aiming to treat rare pediatric disease. A large literature investigates the valuation of new treatments, but the valuation of treatments for rare pediatric illness raises a host of unique issues. In this paper, we review the challenges of applying both the standard economic model and standard approaches to estimating cost-effectiveness using the quality-adjusted life year (QALY) to this case. We argue that there are a large number of special issues that have only been partially addressed by past work and we conclude that more data and the development of new methods are vital as innovators, health technology assessment practitioners and policymakers confront the launch of these new drugs. |
| 主题 | Health, Education, and Welfare ; Health |
| URL | https://www.nber.org/papers/w27978 |
| 来源智库 | National Bureau of Economic Research (United States) |
| 引用统计 | |
| 资源类型 | 智库出版物 |
| 条目标识符 | http://119.78.100.153/handle/2XGU8XDN/585651 |
| 推荐引用方式 GB/T 7714 | Rena M. Conti,Jonathan Gruber,Daniel A. Ollendorf,et al. Valuing Rare Pediatric Drugs: An Economics Perspective. 2020. |
| 条目包含的文件 | ||||||
| 文件名称/大小 | 资源类型 | 版本类型 | 开放类型 | 使用许可 | ||
| w27978.pdf（237KB） | 智库出版物 | 限制开放 | CC BY-NC-SA | 浏览 | ||
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