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Polak Y, Jacobs BAW, Kemper EM. Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid. Front Pharmacol. 2021;12:758210doi: 10.3389/fphar.2021.758210.
Polak, Y., Jacobs, B. A. W., & Kemper, E. M. (2021). Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid. Frontiers in pharmacology, 12758210. https://doi.org/10.3389/fphar.2021.758210
Polak, Yasmin, et al. "Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid." Frontiers in pharmacology vol. 12 (2021): 758210. doi: https://doi.org/10.3389/fphar.2021.758210
Polak Y, Jacobs BAW, Kemper EM. Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid. Front Pharmacol. 2021 Sep 28;12:758210. doi: 10.3389/fphar.2021.758210. eCollection 2021. PMID: 34650439; PMCID: PMC8505773.
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Front Pharmacol. 2021 Sep 28;12:758210. doi: 10.3389/fphar.2021.758210. eCollection 2021.

Pharmacy Compounded Medicines for Patients With Rare Diseases: Lessons Learned From Chenodeoxycholic Acid and Cholic Acid.

Frontiers in pharmacology

Yasmin Polak, Bart A W Jacobs, E Marleen Kemper

Affiliations

  1. Department of Pharmacy and Clinical Pharmacology, University of Amsterdam, Amsterdam, Netherlands.
  2. Platform Medicine for Society, Amsterdam, Netherlands.

PMID: 34650439 PMCID: PMC8505773 DOI: 10.3389/fphar.2021.758210

Abstract

Patients with rare diseases are often confronted with the fact that effective medicines are unavailable or simply not being developed. This situation jeopardizes the health of a large population of vulnerable patients with rare diseases. Pharmacy compounded formulations can provide a safe alternative when authorized treatments are unavailable or unsuitable. Practical guidelines on how to develop and implement pharmacy compounded formulations for patients with rare diseases are limited. The aim of this article is to provide guidance for when and how to apply pharmacy compounded formulations for patients with rare diseases. This is illustrated with two challenging examples: the development and implementation of pharmacy compounding of 1) chenodeoxycholic acid (CDCA) capsules for patients with cerebrotendinous xanthomatosis (CTX) and 2) cholic acid (CA) capsules for patients with rare bile acid synthesis defects (BASD). All critical steps of the development of CDCA and CA capsules are explained and summarized in a practical guideline.

Copyright © 2021 Polak, Jacobs and Kemper.

Keywords: bile acid synthesis defects; cerebrotendinous xanthomathosis; chenodeoxycholic acid; cholic acid; orphan medicines; pharmacy compounding; rare diseases

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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