COMPARATIVE ANALYSIS OF REGULATORY APPROVAL AND HTA EVALUATION FOR FDA-APPROVED ONCOLOGY THERAPIES ACROSS MFN-BASKET COUNTRIES
Author(s)
James Chambers, MSc, PhD1, Sophie Eileen Knox, BS, BSc, MSc1, Fariel LaMountain, BA1, Bengt Liljas, PhD2, Molly Beinfeld, MPH1, Antony Martin, BSc, MSc, PhD1.
1Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA, 2AstraZeneca, Gaithersburg, MD, USA.
1Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA, 2AstraZeneca, Gaithersburg, MD, USA.
OBJECTIVES: Regulatory approval and reimbursed access to oncology therapies vary across MFN comparator countries in both timing and eligible patient populations. This study compared regulatory approval timing and eligible patient populations in MFN comparator countries relative to FDA approvals and assessed the role of HTA evaluations in reimbursed access.
METHODS: We identified oncology drugs approved by the FDA in 2022-2023 and extracted the first approved indication, with the drug-indication as the unit of analysis. Regulatory approval and HTA evaluation data were obtained from the NAVLIN database (Eversana) and supplemented with regulatory and HTA agency websites (May 2026). Outcomes included regulatory approval status, concordance between local and FDA-approved indications, HTA recommendations, and time from FDA approval to local regulatory approval.
RESULTS: We identified 30 drug-indications; 57% were biologics and 87% received at least one FDA expedited review designation. Overall, 75.8% (182/240) of country-drug-indication assessments resulted in regulatory approval, with approval rates ranging from 53% in Japan to 80% in European Medicines Agency (EMA)-linked jurisdictions. Mean time from FDA approval to local regulatory approval ranged from 6.5 months in EMA-linked jurisdictions to 16.4 months in Canada. Eligible patient populations often differed from FDA approvals. Among regulatory approvals in comparator countries (n=182), 44.5% were concordant with the FDA-approved indication, 21.4% were broader, and 34.1% were more restrictive. Japan most frequently approved broader patient populations than the FDA (75%), whereas Switzerland most frequently approved narrower populations (50%). Overall, HTA agencies assessed 122 of 182 (67.0%) regulatory approvals, of which 14.8% (18/122) resulted in a negative recommendation. Among positive recommendations, 21.2% (22/104) were restricted, and half of these required a price reduction.
CONCLUSIONS: Regulatory approval and reimbursed access to oncology therapies varied considerably across comparator countries. Regulatory delays, differences in eligible patient populations, and HTA recommendations contributed to differences in approved and reimbursed patient populations.
METHODS: We identified oncology drugs approved by the FDA in 2022-2023 and extracted the first approved indication, with the drug-indication as the unit of analysis. Regulatory approval and HTA evaluation data were obtained from the NAVLIN database (Eversana) and supplemented with regulatory and HTA agency websites (May 2026). Outcomes included regulatory approval status, concordance between local and FDA-approved indications, HTA recommendations, and time from FDA approval to local regulatory approval.
RESULTS: We identified 30 drug-indications; 57% were biologics and 87% received at least one FDA expedited review designation. Overall, 75.8% (182/240) of country-drug-indication assessments resulted in regulatory approval, with approval rates ranging from 53% in Japan to 80% in European Medicines Agency (EMA)-linked jurisdictions. Mean time from FDA approval to local regulatory approval ranged from 6.5 months in EMA-linked jurisdictions to 16.4 months in Canada. Eligible patient populations often differed from FDA approvals. Among regulatory approvals in comparator countries (n=182), 44.5% were concordant with the FDA-approved indication, 21.4% were broader, and 34.1% were more restrictive. Japan most frequently approved broader patient populations than the FDA (75%), whereas Switzerland most frequently approved narrower populations (50%). Overall, HTA agencies assessed 122 of 182 (67.0%) regulatory approvals, of which 14.8% (18/122) resulted in a negative recommendation. Among positive recommendations, 21.2% (22/104) were restricted, and half of these required a price reduction.
CONCLUSIONS: Regulatory approval and reimbursed access to oncology therapies varied considerably across comparator countries. Regulatory delays, differences in eligible patient populations, and HTA recommendations contributed to differences in approved and reimbursed patient populations.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR183
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Topic Subcategory
Approval & Labeling, Coverage with Evidence Development & Adaptive Pathways
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Rare & Orphan Diseases