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Orphanet Journal of Rare Diseases

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Open Access 01-12-2018 | Position statement

Compounded medication for patients with rare diseases

Authors: Marc Dooms, Maria Carvalho

Published in: Orphanet Journal of Rare Diseases | Issue 1/2018

Abstract

Background

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.

Position statement

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.

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Title: Compounded medication for patients with rare diseases
Authors: Marc Dooms
Maria Carvalho
Publication date: 01-12-2018
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2018
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-017-0741-y

At a glance: The STEP trials

Springer Medicine

Compounded medication for patients with rare diseases

Abstract

Background

Position statement

At a glance: The STEP trials

Springer Medicine

Abstract

Background

Position statement

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