HTA Evidence in Rare Diseases: Just Rare or Also Special?

IF 4.6 3区医学 Q1 ECONOMICS

PharmacoEconomics Pub Date : 2025-09-09 DOI:10.1007/s40273-025-01538-4

Anirban Basu, Simu K Thomas, Richard H Chapman, Jason Spangler

引用次数: 0

Abstract

Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.

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HTA在罕见疾病中的证据：是罕见还是特殊？

孤儿药制造商在满足卫生技术评估要求方面面临一些障碍，因为自然史数据的可得性差、样本量小、单组试验以及缺乏确定的疾病特异性终点。有必要在保健技术评估方面进行具体考虑和改进方法，以应对孤儿药开发方面的挑战。多方利益攸关方合作可使患者、其家庭和更广泛的社会受益，并减少罕见病患者面临的不平等。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

PharmacoEconomics 医学-药学

CiteScore

8.10

自引率

9.10%

发文量

审稿时长

6-12 weeks

期刊介绍： PharmacoEconomics is the benchmark journal for peer-reviewed, authoritative and practical articles on the application of pharmacoeconomics and quality-of-life assessment to optimum drug therapy and health outcomes. An invaluable source of applied pharmacoeconomic original research and educational material for the healthcare decision maker. PharmacoEconomics is dedicated to the clear communication of complex pharmacoeconomic issues related to patient care and drug utilization. PharmacoEconomics offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by a Key Points summary, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article.