Canadian Government is Investigating Price of Genetic Disease Treatment

Regulatory NewsRegulatory News | 27 March 2018 |  By 

Canada’s Patented Medicine Prices Review Board (PMPRB) is actively investigating the price of Horizon Pharma’s Procysbi (cysteamine bitartrate), a treatment for an inherited medical condition called nephropathic cystinosis.
Marie-Eve St-Hilaire, communications manager at Canada’s PMPRB, confirmed to Focus that the investigation is ongoing. “At this time, whether that price complies with the law remains to be determined,” she said.
Investigations, she noted, are conducted independently and confidentially by PMPRB staff. An investigation could result in:
  • The closure of the file;
  • A Voluntary Compliance Undertaking (VCU), whereby the patentee agrees to reduce the price and to offset excess revenues through a payment and/or additional price reduction; or
  • A public hearing to determine whether the price is excessive. If, after a public hearing, a price is found to be excessive, the PMPRB can order price reductions and/or the offset of excess revenues. 
The investigation follows last month’s publishing of a report on Procysbi from the Canadian Agency for Drugs and Technologies in Health (CADTH) noting, “A price reduction of more than 95% is required for the ICUR [incremental cost-utility ratio] to be less than $100,000 per QALY [quality-adjusted life-year].”
The report noted that the delayed-release drug’s cost can run patients anywhere from $136,000 for a 2-year-old child to $321,000 for an adult annually, and that an immediate-release formulation was previously available in Canada through a Health Canada Special Access Programme but was ended by Health Canada in late October 2017, making the immediate-release formulation, known as Cystagon (cysteamine), no longer available.
In November 2017, the Canadian Association of Paediatric Nephrologists wrote to Health Canada’s Special Access Programme to say that losing the option of prescribing Cystagon “will create undue financial hardships for our private insurance companies, provincial medicine formularies, and most importantly, for our patients, including those who are currently well-controlled on Cystagon.
“We expect many provincial formularies will have difficulty justifying such a costly treatment since there is no outcome-based evidence suggesting that Procysbi is better than Cystagon. While it is approved for use in the European Union, the Scottish Medicines Consortium, Ireland’s Centre for Pharmacoeconomics and France’s Hautes Autorité de Santé all recently ruled against approving Procysbi for this reason. Cystagon is approved, available and affordable in these countries,” the association wrote.
Horizon said it did not have any comment.


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