INTERNATIONAL AVAILABILITY AND PRICE OF ULTRA-EXPENSIVE DRUGS IN MEDICARE PART D: ASSESSING THE FEASIBILITY OF EXTERNAL REFERENCE PRICING
Author(s)
Allison Kim, BS, So-Yeon Kang, PhD, MBA, MPH.
Department of Health Care Management and Policy, Georgetown University School of Health, Washington, DC, USA.
Department of Health Care Management and Policy, Georgetown University School of Health, Washington, DC, USA.
OBJECTIVES: The increasing prevalence of high-cost specialty drugs poses key concerns for policymakers and patients. Particularly, ultra-expensive drugs (UEDs) compose a growing share of Medicare Part D spending, prompting interest in external reference pricing. However, limited evidence on their international availability and pricing constrains feasibility assessments. This study characterizes UEDs in Medicare Part D and examines their availability and relative prices across industrialized countries.
METHODS: We conducted a retrospective cohort analysis of UEDs, defined as therapies with annual per-beneficiary spending above U.S. per-capita GDP. Data sources included Medicare Part D Drug Spending (2019-2023) and OECD countries’ wholesaler acquisition prices (November 2025) through Eversana® Navlin’s pricing database. We characterized UEDs by spending, years since launch, therapeutic area, orphan designation, and modality. Outcomes were international availability across 36 OECD nations and U.S.-to-foreign price ratios. Subgroup analyses examined 22 OECD countries with GDP per capita ≥60% of the U.S. Logistic regression assessed associations between drug characteristics and availability.
RESULTS: UEDs in Medicare Part D increased from 123 to 189 between 2019-2023, with spending rising from $28.9B to $45.5B (35.4% of Part D spending). Of 2023 UEDs, 31.9% were biologics, 77.8% had orphan indications, and 31.3% were antineoplastic therapies. Across OECD countries, availability ranged from 9 to 125 UEDs (median 102). Internationally available UEDs were more likely antineoplastic (44.3%), small-molecule (65.1%), orphan-indicated (74.9%), and had higher per-beneficiary spending (p<0.001). Biologics were associated with increased availability (OR 1.3, p=0.03). The median U.S.-to-foreign price ratio was 3.62 (IQR 2.10-5.96).
CONCLUSIONS: Despite rapid expansion of UEDs in Medicare Part D, international availability in comparable economies remains limited, concentrated in small-molecule antineoplastic agents with orphan indications, with US prices 3.6-fold higher than reference countries. The absence of international price benchmarks limits feasibility of external reference pricing and indicates policy challenges for drugs reliant on the U.S. market.
METHODS: We conducted a retrospective cohort analysis of UEDs, defined as therapies with annual per-beneficiary spending above U.S. per-capita GDP. Data sources included Medicare Part D Drug Spending (2019-2023) and OECD countries’ wholesaler acquisition prices (November 2025) through Eversana® Navlin’s pricing database. We characterized UEDs by spending, years since launch, therapeutic area, orphan designation, and modality. Outcomes were international availability across 36 OECD nations and U.S.-to-foreign price ratios. Subgroup analyses examined 22 OECD countries with GDP per capita ≥60% of the U.S. Logistic regression assessed associations between drug characteristics and availability.
RESULTS: UEDs in Medicare Part D increased from 123 to 189 between 2019-2023, with spending rising from $28.9B to $45.5B (35.4% of Part D spending). Of 2023 UEDs, 31.9% were biologics, 77.8% had orphan indications, and 31.3% were antineoplastic therapies. Across OECD countries, availability ranged from 9 to 125 UEDs (median 102). Internationally available UEDs were more likely antineoplastic (44.3%), small-molecule (65.1%), orphan-indicated (74.9%), and had higher per-beneficiary spending (p<0.001). Biologics were associated with increased availability (OR 1.3, p=0.03). The median U.S.-to-foreign price ratio was 3.62 (IQR 2.10-5.96).
CONCLUSIONS: Despite rapid expansion of UEDs in Medicare Part D, international availability in comparable economies remains limited, concentrated in small-molecule antineoplastic agents with orphan indications, with US prices 3.6-fold higher than reference countries. The absence of international price benchmarks limits feasibility of external reference pricing and indicates policy challenges for drugs reliant on the U.S. market.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
P60
Topic
Health Policy & Regulatory
Topic Subcategory
Insurance Systems & National Health Care, Pricing Policy & Schemes, Public Spending & National Health Expenditures, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas