Compounded medication for patients with rare diseases

Orphanet Journal of Rare Diseases
Marc Dooms, Maria Carvalho

Abstract

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.

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Citations

Mar 23, 2019·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Shoko KomatsuzakiMarkus Ries
Feb 28, 2019·Expert Review of Neurotherapeutics·Hiide Yoshino
Jul 4, 2020·Drug Development and Industrial Pharmacy·Ivana Cotabarren, Loreana Gallo
Apr 8, 2020·Clinical & Experimental Optometry : Journal of the Australian Optometrical Association·Lynn Weekes, Iqbal Ramzan
Sep 8, 2018·World Journal of Clinical Pediatrics·Alberto TommasiniIrene Bruno
May 15, 2019·Journal of the American Association of Nurse Practitioners·Darlene Deters, Darpan I Patel
Jul 23, 2020·Journal of Pharmaceutical Policy and Practice·A B BayoumyC J J Mulder
Nov 11, 2019·BMC Health Services Research·Hatim S AlKhatibSuha Al Muhaissen
Jan 8, 2021·Orphanet Journal of Rare Diseases·K P ThompsonUNKNOWN Brain Vascular Malformation Consortium HHT Investigator Group
Apr 4, 2021·Pharmacy : Journal of Pharmacy, Education and Practice·Janosch Klinger, Rolf Daniels
Apr 10, 2021·Scientific Reports·Thomas Planté-BordeneuveSophie Gohy
Apr 14, 2021·Orphanet Journal of Rare Diseases·Eva M M Hoytema van KonijnenburgClara D M van Karnebeek
May 30, 2021·The European Respiratory Journal·Antoni Noguera-JulianNicole Ritz

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Software Mentioned

OpenPHACTS
Aprofarm
PubChem
ChEMBL
FagronFormulas
ChemSpider

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