The Pharmaceutical Accountability Foundation welcomes the news that the Amsterdam UMC (Amsterdam University Medical Centers) has resumed supplying Dutch patients with the rare disease Cerebrotendinous Xanthomathosis (CTX) with pharmacy compounded chenodeoxycholic acid (CDCA).
This action became necessary after Leadiant Biosciences had increased the price of one CDCA capsule from €0,28 in 2008 to €140 in 2018, and the annual treatment costs for CTX rose 500x from €308 to €153.000 per patient per year. Leadiant could do so as they had obtained a 10-year EU monopoly (market exclusivity) based on the EU orphan drug regulation, and after they had bought and removed all competing CDCA versions from the EU market.
The steep price increase was however unacceptable to the Dutch health insurers, which threatened the accessibility of treatment for patients. Since continuation of treatment is absolutely necessary, an initiative by Amsterdam UMC to start compounding the drug was welcomed.
Pharmaceutical compounding is a last resort to ensure access if drug companies ask excessive prices.
The Dutch Ministry of Health has amended the reimbursement rules, so that compounded medicines can be reimbursed when an (expensive) product, registered in the EU enters the market, pending a decision for reimbursement.
In April 2018 the Amsterdam UMC hospital pharmacy started supplying CTX patients with compounded CDCA, which cost less than 25% of the CDCA Leadiant price.
Leadiant complained about the compounding by Amsterdam UMC at the Dutch Health Inspectorate IGJ in May 2018. In August 2018, the Inspectorate informed AmsterdamUMC that they intended an enforcement to stop the compounding due to impurities in the raw material beyond the limits set in the CDCA European Pharmacopoeia (EurPh) monograph. The AmsterdamUMC recalled the compounded capsules, and patients were given the more expensive CDCA Leadiant. Health insurers agreed to pay the high price out of leniency.
Since then the search has been for affordable CDCA raw material (API) that complies with the EurPh quality standards. At the same time EurPh is also developing a new CDCA monograph using advanced methods to quantify impurities.
Dutch Health Minister Bruno Bruins recently informed the Dutch Parliament that he is still negotiating an acceptable price with Leadiant, and that he wants more transparency from Leadiant about the production and development costs. The Minister has also pleaded in Brussels to reduce the 10 years market exclusivity as part of the ongoing EU Pharmaceutical Incentives Review.
As Dutch CTX patients are now able to receive their CDCA at a more affordable price, it should also be possible for other EU countries to consider compounding CDCA. In Belgium, 11 CTX patients were using capsules compounded in Leuven University hospital. In Italy patients also received compounded CDCA, but the hospital stopped when Leadiant came to the market with its expensive brand. On epidemiological grounds, more than 1500 CTX patients could be expected in the EU, but it seems that many are undiagnosed or not on an affordable treatment. It is time they all receive effective and affordable CDCA treatment!
The Pharmaceutical Accountability Foundation (in Dutch Stichting Farma ter Verantwoording) complained in September 2018 about the 500x price increase by Leadiant to the Dutch Competition Authority ACM on the grounds of ‘abuse of a dominant position’. NGOs in Belgium, Spain and Italy have also complained about Leadiant with their national Competition Authorities.
As 4 countries have now seen competition law complaints, it is possible that the European Commission will take an interest in the CDCA Leadiant case. Leadiant is by no means the only company misusing the EU Orphan Drug Regulation 2000 but is an important case to consider in the upcoming Pharmaceutical Incentives review.