2015-2024 Pricing Advantage of First-in-Indication Non-Oncology Orphan Products Versus Late Entrants and Recent Trends
Moderator
Daniel Krieger, Red Nucleus, Yardley, PA, United States
Speakers
Brian Privett, Red Nucleus, Brookline, MA, United States; Asenath Marchena
OBJECTIVES: Rare disease-indicated therapeutics represent a unique pricing environment for pharmaceutical products. This research aims to evaluate the relationship between disease prevalence, launch wholesale acquisition cost (WAC), and launch sequence within indications to determine the extent of upwards pricing pressure for first-in-indication orphan disease products with the inclusion of more recently approved orphan drugs.
METHODS: All FDA CDER new molecular entity approval lists from 2015-2024 were documented, then non-orphan and oncology products were removed (CDER excludes cellular and gene therapy products, blood products, plasma derivatives, allergenic products, and vaccines). US prevalence and WAC at launch were calculated for each therapy. First-in-indication therapies at launch were classified. Statistical comparisons were performed using multivariable linear regressions.
RESULTS: A total of n=108 orphan drug launches were assessed, of which 51 were first-in-indication and 57 launched with at least 1 competitor. There was an increase in expected price for first-in-indication orphan drug launches as the historical data suggests an expected price $254,280 over second-or-later to market products. Removing ultra-rare products from the dataset (<1,000 US patients) results in an expected price $82,813 over second-or-later to market products.
CONCLUSIONS: Analysis shows that first-to-market rare disease drugs have a significant pricing advantage over subsequent market entrants, and that this gap is widening with recent launches. However, removing ultra-rare products from the analysis results in a lower expected price advantage for first-in-indication products, suggesting in many cases the advantage is reduced. Continued analysis of orphan drug products will further elaborate the advantage to better understand the true expected pricing advantage for first-in-indication orphan products.
METHODS: All FDA CDER new molecular entity approval lists from 2015-2024 were documented, then non-orphan and oncology products were removed (CDER excludes cellular and gene therapy products, blood products, plasma derivatives, allergenic products, and vaccines). US prevalence and WAC at launch were calculated for each therapy. First-in-indication therapies at launch were classified. Statistical comparisons were performed using multivariable linear regressions.
RESULTS: A total of n=108 orphan drug launches were assessed, of which 51 were first-in-indication and 57 launched with at least 1 competitor. There was an increase in expected price for first-in-indication orphan drug launches as the historical data suggests an expected price $254,280 over second-or-later to market products. Removing ultra-rare products from the dataset (<1,000 US patients) results in an expected price $82,813 over second-or-later to market products.
CONCLUSIONS: Analysis shows that first-to-market rare disease drugs have a significant pricing advantage over subsequent market entrants, and that this gap is widening with recent launches. However, removing ultra-rare products from the analysis results in a lower expected price advantage for first-in-indication products, suggesting in many cases the advantage is reduced. Continued analysis of orphan drug products will further elaborate the advantage to better understand the true expected pricing advantage for first-in-indication orphan products.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE119
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases