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Onakpoya IJ, Spencer EA, Thompson MJ, et al. Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open 2015;24;5:e007199. There have been corrections to the online supplement as follows:

In Appendix 1, Orphacol has been added to the list, and the appropriate references used in the quality and level of the evidence for Orphacol and Cholic Acid FGK are:

In Appendix 2, table 2, the authors have revised the indications for Orphacol, and added ‘deficiency’ to the indication for Cholic Acid FGK:

  • Cholic Acid FGK: IEM in primary bile acid synthesis due to Sterol 27-hydroxylase deficiency.

  • Orphacol: IEM in primary bile acid synthesis due to 3beta-Hydroxy-delta5-C27-steroid oxidoreductase deficiency or delta4-3-Oxosteroid-5beta-reductase.

In Appendix 2, table 2, and in Appendix 3, the authors have revised the annual cost of Litak to £835 (1 cycle) based on correspondence with the manufacturers.

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