Leadiant is involved simultaneously in administrative proceedings and in a public relations struggle to justify its pricing for chenodeoxycholic acid Leadiant (“CDCA”), a medicine indicated for the treatment of patients afflicted with cerebrotendinous xanthomatosis, a rare metabolic disorder (“CTX”). In Europe, the number of these patients is thought to be in a range between 200 and 250. Leadiant secured orphan medicine status for CDCA in December 2014 after it succeeded in demonstrating the significant benefit of CDCA over existing treatments of CTX. However, by Leadiant’s own acknowledgement, the list price of CDCA “appears to be high” especially when that price is compared with that of an old medicine with the same active substance indicated for the treatment of cholesterol gallstones. As a result, Leadiant has come under fire from various political leaders, healthcare regulators and competition authorities on account of the allegedly excessive prices which it charges for CDCA (see, Van Bael & Bellis Life Sciences Newsflashes of 9 September 2019, 9 April 2019, and 12 February 2019). Leadiant has now taken the unusual step of making publicly available a letter which it sent to the Dutch Minister of Medical Care, Bruno Bruins, following talks over the price of CDCA (the “Letter” – see attached Dutch-language original and English translation). The Letter sets forth Leadiant’s arguments in favour of the current price of CDCA in The Netherlands, but also highlights Leadiant’s willingness to continue the discussions in hopes of finding an “acceptable price” and thus securing what Leadiant describes as “access to an innovative medicine”. In the Letter, Leadiant makes the following points: • CDCA is a new medicine that should be distinguished from the old medicine with the same active substance for a different therapeutic indication. It faced much more exacting hurdles to secure a marketing authorisation than did the marketing authorisation holder of the old medicine many years ago. • Leadiant was able to show the significant benefit of CDCA over other forms of treatment of CTX. • Leadiant invested significant amounts in retrospective studies that demonstrated the safety and efficacy of CDCA (The European Medicines Agency actually indicates that the marketing authorisation for CDCA results from a hybrid application which relied in part on the results of pre-clinical tests and clinical trials for a reference product and in part on new data. Additionally, the marketing authorisation was granted “under exceptional circumstances” which implies that Leadiant was unable to provide comprehensive data on the efficacy and safety under normal conditions of use). • Leadiant was required to modernise the active substance master file for the active pharmaceutical ingredient at the heart of CDCA. • Leadiant created a European patient registry which just became operational and is designed to monitor the benefits and safety of the medicine and thus make up for the insufficient data available at the time the marketing registration was granted. • Leadiant committed to developing a paediatric formulation which will specifically cater to the needs of 30 to 60 patients in the EU. • Lastly, Leadiant strongly disputes the public health benefits of a pharmaceutical preparation that was touted in The Netherlands as a possible alternative to CDCA. The Letter should be understood as an attempt by Leadiant to demonstrate its bona fides and operate as a reliable partner of the health authorities in the interest of patients. Time will tell whether that attempt is successful.