Abstract

Dr Zozulya-Weidenfeller responds on behalf of Lupin, the marketing authorization holder of NaMuscla®, to our article “Cost-Based Price Calculation of Mexiletine for Non-dystrophic Myotonia” published online on April 9, 2021.1 She emphasizes the importance of registering medicinal products especially for treating rare diseases. Furthermore, the case is made that repurposing existing or previously authorized medicines for new indications may have a substantial value for patients and warrants sustainable access. We fully agree with this, as well as with the need to incentivize commercial parties to bring old products to the market. Nevertheless, we believe that a healthy business case, with acceptable profits and at the same time avoidance of unnecessary high pricing, requires transparency. A cost-based pricing approach is a transparent element in such a business case, which in our view helps to create a framework for sustainable drug repurposing for rare diseases.