Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2018 Jan 4;13(1):1.
doi: 10.1186/s13023-017-0741-y.

Compounded medication for patients with rare diseases

Marc Dooms  1 , Maria Carvalho  2
Affiliations
Review

Compounded medication for patients with rare diseases

Marc Dooms et al. Orphanet J Rare Dis. .

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.

Keywords: Compounded medications; Extemporaneous preparations; Magistral preparations; Official preparations; Orphan drugs; Pharmaceutical compounding; Rare diseases; Unlicensed medicines.

PubMed Disclaimer

Conflict of interest statement

Ethics approval and consent to participate

Not applicable.

Consent for publication

Not applicable.

Competing interests

M D declares that he has no competing interests.

References

    1. Koch J, Mayr JA, Alhaddad B, Rauscher C, Bierau J, Kovacs-Nagy R, Coene KL, Bader I, Holzhacker M, Prokisch H, Venselaar H, Wevers RA, Distelmaier F, Polster T, Leiz S, Betzler C, Strom TM, Sperl W, Meitinger T, Wortmann SB, Haack TB. CAD mutations and uridine-responsive epileptic encephalopathy. Brain. 2017;140(Pt 2):279–286. doi: 10.1093/brain/aww300. - DOI - PubMed
    1. Carvalho, M. Extemporaneously Compounded Oral Medicines in European Hospital Pharmacies. Doctoral thesis, UCL (University College London); 2013.
    1. Aprofarm, IX Symposium: Unification of criteria for paediatric formulations – Programme leaflet. Available at: http://www.formulistasdeandalucia.es/ficheros/9472%20triptico%20Pediatri... Accessed: 21 May 2017.
    1. European Parliament and the Council Directive 2001/83/EC of 6 November on the community code relating to medicinal products for human use. Off J Eur Communities. 2001;L311:67–128.
    1. European Parliament and the Council Directive 2004/27/EC of 31 march amending directive 2001/83/EC on the community code relating to medicinal products for human use. Off J Eur Union. 2004;L136:34–57.