University Hospitals Leuven, Herestraat, B 3000, Leuven, Belgium.
PCCA, Houston, TX, USA.
Orphanet J Rare Dis. 2018 Jan 4;13(1):1. doi: 10.1186/s13023-017-0741-y.
When there is no authorized on- or in absence even no off-label treatment for patients with rare diseases, pharmacists have to compound medicinal products to meet the patients special needs. However it is important that there is evidence in the medical and/or pharmaceutical literature for such compounded medications.
Pharmaceutical compounding must be performed in the best possible circumstances by certified practitioners (pharmacists) using validated standard operating procedures (standardized formulations) in order to obtain medicinal products of the highest quality to assure patient safety. More than 60 compounding procedures were identified in 17 on-line pharmaceutical compounding reference sources worldwide but more operating procedures still need to be validated. All ingredients used in the preparation of the compounded medication must be accompanied by a certificate of analysis and full records of the pharmaceutical production process need to be kept for full traceability and accountability.
当没有针对罕见病患者的授权的或甚至没有标签外治疗方法时，药剂师必须配制药物以满足患者的特殊需求。然而，重要的是，在医学和/或药物文献中有此类配制药物的证据。
药物配制必须由经过认证的从业者（药剂师）在尽可能最好的条件下进行，使用经过验证的标准操作程序（标准化配方），以获得最高质量的药物，确保患者安全。在全球 17 个在线药物配制参考来源中，确定了 60 多种配制程序，但仍需要更多的操作程序进行验证。配制药物中使用的所有成分都必须附有分析证书，并且需要保留完整的药物生产过程记录，以实现完全的可追溯性和问责制。
