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血浆源性药品面临的挑战。

Challenges for Plasma-Derived Medicinal Products.

作者信息

Strengers Paul F W

机构信息

Consultant Blood and Plasma Strategies, Amsterdam, The Netherlands.

出版信息

Transfus Med Hemother. 2023 Jan 18;50(2):116-122. doi: 10.1159/000528959. eCollection 2023 Apr.

DOI:10.1159/000528959
PMID:37066053
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10091012/
Abstract

BACKGROUND

Plasma-derived medicinal products (PDMPs) are medicinal products derived from human plasma, and a number of PDMPs are listed on the WHO Model List of Essential Medicines. These and other PDMPs are crucial for the prophylaxis and treatment of patients with immune deficiencies, autoimmune and inflammatory diseases, bleeding disorders, and a variety of congenital deficiency disorders. The majority of plasma supplies for manufacturing of PDMPs is coming from the USA.

SUMMARY

The future of treatments with PDMPs for PDMP-dependent patients depends on the supply of plasma. An imbalance in the global collection of plasma has resulted in regional and global shortages of essential PDMPs. The challenges at different level are mainly related on a balanced and sufficient supply in order to help the patients in need and should be addressed in order to safeguard the treatment with these essential lifesaving and disease mitigating medicines.

KEY MESSAGES

It is advocated to consider plasma as a strategic resource comparable to energy and other rare resources and to investigate whether for the treatment of patients with rare diseases, a free market of PDMPs has its limitations and special protection measures should be developed. At the same time, plasma collections should be increased outside the USA, including in low- and middle-income countries.

摘要

背景

血浆衍生药物(PDMPs)是源自人血浆的药物,世界卫生组织基本药物示范清单中列出了多种PDMPs。这些以及其他PDMPs对于免疫缺陷、自身免疫和炎症性疾病、出血性疾病以及各种先天性缺陷疾病患者的预防和治疗至关重要。用于制造PDMPs的大部分血浆供应来自美国。

总结

对于依赖PDMPs治疗的患者而言,PDMPs治疗的未来取决于血浆供应。全球血浆采集的不平衡导致了基本PDMPs的区域和全球短缺。不同层面的挑战主要与平衡充足的供应有关,以便帮助有需要的患者,应加以应对以保障这些基本的救命和缓解疾病药物的治疗。

关键信息

提倡将血浆视为与能源和其他稀有资源相当的战略资源,并研究对于罕见病患者的治疗,PDMPs自由市场是否存在局限性以及是否应制定特殊保护措施。同时,应在美国以外地区增加血浆采集,包括在低收入和中等收入国家。

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