Skip to main content

Table 2 Assumptions for predicting the future costs of pipeline DRDs

From: Historical and projected public spending on drugs for rare diseases in Canada between 2010 and 2025

Assumption

Parameters

Only a subset of DRDs will be approved by the FDA or EMA after completing a phase 3 trial [35]

66.3% of DRDs with a completed phase 3 trial are approved, 33.7% are not approved

HC often delays/does not grant approval to DRDs that are approved by the FDA or EMA [11, 36]

HC approval rates were estimated as 16% after 1 year, 30% after 2 years, 40% after 3 years, 50% after 4 years, and 60% after 5 years

Some DRDs are not recommended for reimbursement despite HC approval [24]

69.15% of DRDs would receive a positive HTA recommendation, 30.85% would receive a negative recommendation

Canadian market penetration of reimbursed DRDs will increase over time [20]

DRDs capture 10% of the market in the first year of reimbursement, 12% in year 2, 15% in year 3, 20% in year 4, and 25% in year 5

Public and private payers pay for DRDs [assumption]

60% of costs borne by public payers, 40% borne by private payers

A half-cycle correction is applied during the launch year [assumption]

DRDs will capture 50% of their eligible market during the launch year

  1. Assumptions for predicting future costs of a pipeline DRD were applied based on the DRD’s status in Table 1 (e.g., the FDA/EMA/HC and HTA assumptions would not be applied to a product in pCPA negotiations, as this product has already received regulatory approval and a positive HTA recommendation)
  2. DRDs drugs for rare diseases, EMA European Medicines Agency, FDA US Food and Drug Administration, HC Health Canada, HTA health technology assessment, pCPA pan-Canadian Pharmaceutical Alliance