The new therapy, which replaces an old immediate-release formulation,can cost up to $321,000
Following reactions from affected households and concerned physicians, Canada’s Patented Medicine Prices Review Board (PMPRB) is investigating the price of Procysbi, a genetic disease treatment that costs up to $321,000 annually.
“At this time, whether that price complies with the law remains to be determined,” Marie-Eve St-Hilaire, communications manager at the PMPRB, told Regulatory Focus.
The new treatment, called Procysbi, is manufactured by Horizons Pharma. It has the same active ingredient as Cystagon, a drug that’s been long used by families with children affected by a genetic disorder called cystinosis. The only difference is that Procysbi is designed to be released more slowly into the body.
Many families affected by the disease reacted negatively to Procysbi’s cost, which can be anywhere from $136,000 for a 2-year-old child to $321,000 for an adult per year. Because of a Health Canada policy, the old drug Cystagon — which was covered under a special access program — became unavailable, forcing doctors to prescribe the new high-priced product.
St-Hilaire told Focus that PMPRB staff conduct investigations independently and confidentially. Depending on the findings, an investigation could result in the closure of the file, a Voluntary Compliance Undertaking whereby the patentee agrees to reduce the price and offset excess revenues through a payment and/or additional price reduction, or a public hearing to determine whether the price is excessive.
If a drug’s price is found to be disproportionately high through a public hearing, the PMPRB can order price reductions and/or the offset of excess revenues.
In November, the Canadian Association of Paediatric Nephrologists wrote to Health Canada, saying that losing Cystagon as an option would “create undue financial hardships for our private insurance companies, provincial medicine formularies, and most importantly, for our patients.
“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,” the association continued. The European Union has approved Procysbi, health regulators in France, Scotland, and Ireland — countries where Cystagon is approved and available — have all decided against approving it because of that lack of evidence.
Last month, the Canadian Agency for Drugs and Technologies in Health (CADTH) issued a report on Procysbi, saying that “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].”
