Abstract
BackgroundPharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages. Additionally, it allows customized therapy for patients with rare diseases. However, standardized compounding formulas and procedures, and monographs are required to ensure the patients’ safety.ResultsStandardized formulas and compounding procedures were developed for seven orphan active ingredients (L-arginine, sodium benzoate, sodium phenylbutyrate, L-carnitine, chenodesoxycholic acid, primaquine phosphate, pyridoxal phosphate) and one non-orphan molecule (sodium perchlorate) regularly compounded by hospital pharmacists for extemporaneous use. The stability of these formulations was evaluated over 3 months at refrigerated (5 °C) and standard storage conditions (25 °C/60%RH) using HPLC-based assays and a suitable shelf life was assigned to the formulations. Additionally, suitable analytical methods for quality control of formulations of pyridoxal phosphate and sodium perchlorate were developed as monographs for these components were not available in the European Pharmacopeia or United States Pharmacopeia.ConclusionsAvailability of compounding formulas and protocols, as well as stability information, for orphan active ingredients can improve patients’ access to treatment for rare diseases. Such data were collected for seven orphan active ingredients to treat patients with rare diseases when no other treatment is available. More efforts are needed to develop standardized formulas and compounding procedures for additional orphan active ingredients whose clinical efficacy is well-known but which are not available as products with a marketing authorization. Additionally, a legal framework at EU level is required to enable the full potential of pharmaceutical compounding for orphan active ingredients.
Highlights
Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages
Standardized formulas and compounding procedures were developed for three licensed Orphan Active Ingredient (OAI) which are frequently compounded in Belgian hospital pharmacies but whose stability was not investigated up to now: L-arginine, L-carnitine and sodium benzoate
Compounded preparations of OAIs can improve patients’ access to treatment for rare diseases. They should be prepared in accordance to standardized formulas and compounding procedures and the quality of the OAI should be demonstrated by a certificate of analysis according to a specific monograph
Summary
Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages. It allows customized therapy for patients with rare diseases. It is estimated that 5000 to 8000 rare diseases exist worldwide, while approximately 250 new diseases are described annually [1,2,3]. Their prevalence is low, rare diseases affect 27 to 36 million people in the EU [1, 2].
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