Presentation (CTI)

OBJECTIVES: As drug for rare diseases (DRDs) have become a growing focus of pharmaceutical development, their high costs pose significant challenges to healthcare budgets. This research aims to characterize key drivers influencing the feasibility and timing of adoption for DRDs in Canadian public reimbursement. We seek to determine whether rarity of conditions, competition, and potential benefits influences acceptability of DRDs’ substantial price tags, ultimately informing policy decisions that balance innovation, affordability, and equitable access.
METHODS: We extracted non-oncology DRDs designated as ‘orphan’ per the Orphanet portal with Canadian Drug Agency (CDA) recommendations between 2021 and 2024. DRDs were categorized as (1) rare or ultra-rare based on disease prevalence of 0.001%, and (2) first or subsequent entrant for the condition in Canada. Our internal market intelligence tool was used to gather CDA, pan-Canadian negotiation data, estimated three-year budget impact analyses (BIAs), quality-adjusted life years (QALYs), eligible patient numbers, and annual list price per-patient cost. We examined trends by disease prevalence and entry order and assessed timelines using the Kaplan-Meier method.
RESULTS: CDA reviewed 153 non-oncology drugs between 2021 and 2024, with 57 (37%) targeting DRDs, including 13 for ultra-rare indications and 21 as first entrants. The majority (51/57) received positive recommendations. Three-year projected BIAs at list prices ranged from cost savings of $49M to an added impact of $2.6B. Rare DRDs showed a higher median BIA ($52M) than ultra-rare DRDs ($25M). First entrant DRDs had a higher median BIA ($65M) than subsequent entrants ($29M), and required longer median time to complete negotiations (245 vs 147 days, p=0.0024).
CONCLUSIONS: While most DRDs have received positive reimbursement recommendations and completed pan-Canadian price negotiations, significant differences in their characteristics affect feasibility and timing of adoption. Novel, first entrant DRDs with high prices pose additional challenges for implementation and overall affordability within the healthcare system.