E-DRUG: Orphan drugs and pharma pirates: the CDCA Leadiant case
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dear colleagues,
You may have heard about the 500-fold price hike (from Eur 0.28 to Eur
140 per capsule) of the orphan medicine CDCA (chenodeoxycholic acid) by the pharmaceutical company Leadiant?
CDCA is inexpensive to produce and, from 1976 until 2008, it was available on the market in The Netherlands at the cost of Eur 0.28 per capsule for the treatment of gallstones. The medicine has also been prescribed 'off label' for the treatment of the rare metabolic illness cerebrotendinous xanthomatosis (CTX) at a cost of Eur 308 per treatment-year.
The company Sigma-Tau (later renamed Leadiant) obtained the rights to CDCA from the original manufacturer Dr Falk in 2008, and removed this and other affordable CDCA sources from the EU market, so that they obtained a de facto monopoly.
In 2017 Leadiant was granted exclusive rights to market the product in Europe for the treatment of CTX after the European Medicines Agency (EMA) approved Leadiant's CDCA for marketing and granted the medicine an orphan drug designation. Leadiant is currently charging in the Netherlands Eur 140 per capsule. This is equivalent to Eur 153.300 per treatment-year for each CTX patient, 500x the cost of 2008.
Our Foundation issued a complaint about the excessive price hike with the Dutch Competition Authority in September 2018.
Our complaint was transparently published on our website:
https://www.farmaterverantwoording.nl/wp-content/uploads/2018/11/2018.09.07_Enforcement_request_Pharmaceutical_Accountability_Foundation_Leadiant.pdf
Similar complaints have been raised in Belgium, Spain and Italy:
- Belgium (TestAankoop): https://www.test-aankoop.be/gezond/ziekten-en-geneesmiddelen/geneesmiddelen/nieuws/klacht-tegen-wanpraktijken-van-farmaceutische-firma-leadiant
- Spain (OCU): https://www.ocu.org/organizacion/prensa/notas-de-prensa/2019/denuncialeadiant240619
- Italy (AltroConsumo) https://www.agcm.it/media/dettaglio?id=5628e16a-fa1a-42b7-9d68-23f7ddd2ef8f
The Dutch Competition Authority has issued a second progress report on 29 June 2020:
https://www.acm.nl/en/publications/acm-extends-its-investigation-orphan-drug-cdca-leadiant
Our Pharmaceutical Accountability Foundation has responded with another press release:
https://www.farmaterverantwoording.nl/response-of-the-pharmaceutical-accountability-foundation-to-the-press-release-of-the-dutch-competition-authority-netherlands-authority-for-consumers-and-markets-acm-on-cdca-leadiant-29-june-2020/
We expect the Dutch Authority to issue a fine later this year. We hope this will be a warning for other so called 'pharma pirates' who buy up old products, remove affordable products from the market, then get them registered as an orphan drug, get 10 years of market exclusivity, and then raise the price excessively.
Ellen 't Hoen and undersigned recently got an article published in the Dutch Medical Journal on 'orphan drugs and pharma pirates' explaining how pharma pirates game the EU orphan drug system:
https://www.farmaterverantwoording.nl/orphan-drugs-and-drug-pirates/
Health Insurance companies have objected to the excessive price, and patients in the Netherlands are now getting a much cheaper version of CDCA that is compounded at 20% of the cost by the Amsterdam University Medical Centre:
https://www.farmaterverantwoording.nl/amsterdam-umc-resumes-supply-of-compounded-cdca/
The CDCA raw material now also adheres to the European Pharmacopeial standard, and the information about pharmacy compounding can be made available to interested pharmacists.
We welcome feedback!
Wilbert Bannenberg
Wilbert Bannenberg, MD (no longer practicing),
public health consultant
Chairperson, Pharmaceutical Accountability Foundation
Netherlands
Wilbert Bannenberg <voorzitter@farmaterverantwoording.nl>