OBJECTIVES: CADTH (recently renamed Canada’s Drugs Agency) is the HTA body for Canada (excluding Quebec). CADTH is not a decision-maker, but provides expert advice on clinical- and cost-effectiveness to provincial payers / pCPA in published recommendations. These may include clinical restrictions, and typically a recommended percentage discount. The objective of this research was to review CADTH assessments of orphan drugs for the last 7 years and identify key lessons for manufacturers.

METHODS: A list of orphan drugs launched between 2017 and 2023 (inclusive, extracted from EMA data, as Health Canada does not provide a top-down list) was populated with data from published CADTH HTA assessments, with entries missing key data excluded. Data collected included overall recommendation, any restrictions, list price, recommended percentage-discount, and resulting recommended net price.

RESULTS: 55 orphan therapies were identified for analysis. The median recommended discount to achieve cost-effectiveness was 85% (IQR: 60%-95%); 84% (46/55) of products were associated with a discount >50%, and 31% (17/55) with a discount ≥95%. Oncology products fared slightly better (median discount: 76%, IQR: 56%-87%), while products for ultra-orphan conditions were associated with higher discounts (median discount: 92%, IQR: 85%-95%). Five CAR-Ts were included, and had a median recommended discount of 80% (IQR: 76%-89%), while the two gene therapies, Luxturna and Zolgensma, had recommended discounts of 74% and 95%-99% respectively.

CONCLUSIONS:

CONCLUSION: Our analysis provides a valuable insight into health-economic value perception for new medicines in Canada, as well as a bellwether for other CEA markets, and highlights stringent net price pressure. It is worth nothing that high recommended discounts may partially reflect artificially high maximum prices, and that Canadian payers are not bound to CADTH recommendations and may consider less substantial discounts at the point of negotiation and reimbursement.