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Compounded medication for patients with rare diseases.

Marc Dooms¹, Maria Carvalho²

¹University Hospitals Leuven, Herestraat, B 3000, Leuven, Belgium. dooms@evonet.be.

Orphanet Journal of Rare Diseases

|January 6, 2018

Summary

For rare diseases lacking treatments, pharmacists compound medications. Evidence-based compounding ensures patient safety and medication quality through validated procedures and traceable production.

Keywords:

Compounded medications Extemporaneous preparations Magistral preparations Official preparations Orphan drugs Pharmaceutical compounding Rare diseases Unlicensed medicines

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Area of Science:

Pharmaceutical Sciences
Medicinal Chemistry

Background:

Compounding medications is essential for rare diseases when no authorized or off-label treatments exist.
Evidence in medical and pharmaceutical literature is crucial for validating compounded medications.

Purpose of the Study:

To highlight the importance of evidence-based pharmaceutical compounding for rare disease patients.
To emphasize the necessity of quality control and safety in compounding practices.

Main Methods:

Literature review of on-line pharmaceutical compounding reference sources.
Identification and validation of standard operating procedures (SOPs) for compounding.
Ensuring ingredient quality through certificates of analysis and maintaining production records.

Main Results:

Over 60 compounding procedures were identified across 17 global online sources.
Further validation of compounding procedures is required.
Emphasis on the use of certified ingredients and comprehensive record-keeping for traceability.

Conclusions:

Pharmaceutical compounding by certified pharmacists using validated SOPs is critical for patient safety.
Adherence to quality standards, including ingredient verification and detailed record-keeping, ensures the highest quality compounded medications.
Continued validation of compounding procedures is necessary to expand evidence-based options for rare disease treatment.