Pharmaceutical compounding of orphan active ingredients in Belgium: how community and hospital pharmacists can address the needs of patients with rare diseases

V. Vanhoorne, E. Peeters, I. Van Tongelen, K. Boussery, E. Wynendaele, B. De Spiegeleer, J. P. Remon & C. Vervaet ; Orphanet Journal of Rare Diseasesvolume 14, Article number: 186 (2019) DOI 10.1186/s13023-019-1154-x

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. 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.
Compounded preparations of OAIs can improve patients’ access to treatment for rare diseases. However, 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. In current study, such standardized formulas and compounding procedures were developed for seven OAIs 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 OAIs whose clinical efficacy is well-known but are not available yet to patients due to lack of interest from the pharmaceutical industry to apply for market authorization as OMP. Additionally, a legal framework at EU level is required to enable the potential of pharmaceutical compounding for OAIs.