OBJECTIVES : Prolonged time between marketing authorization and reimbursement leads to missed health gains, raising the question: what could be gained by ensuring earlier reimbursement?

METHODS : A health impact model was developed for two selected oncological drugs to calculate the impact of earlier reimbursement on the number of patients that could have been treated and accompanying health gains. Health impact outcome measures were obtained from national assessment dossiers and could differ per drug and country. Time to reimbursement was optimized in three hypothetical scenarios: 1) timing of reimbursement coinciding with EC marketing authorization (as is the case in Germany; ambitious scenario), 2) as fast as the fastest country (best practice), and 3) assessments within 180 days after the EC marketing authorization, unless faster in reality (as described in the EC Transparency Directive; base case). The number of new patients per month (uptake data) were retrieved from the respective companies marketing these drugs. Only countries with recorded uptake data for at least 1 year after reimbursement were used in the analysis.

RESULTS : Midostaurin and pertuzumab and four European countries (England, Italy, the Netherlands, and Sweden) were included in the analysis. In the ambitious scenario, 1,689 more AML patients could have been treated with midostaurin in England, Italy, the Netherlands, and Sweden, accounting for 82,920 life months gained (LMG) and 8,665 months of event-free survival. With pertuzumab, 2,180 patients could have been treated in England, Netherlands, and Sweden, accounting for 12,718 LMG and 8,842 quality-adjusted life months gained. For the countries that lacked (sufficient) uptake data, even more health gains could be expected.

CONCLUSIONS : Drugs gaining positive reimbursement decision are apparently worthwhile for patient- and population’s health, needing a quick access. Today, time to reimbursement varies greatly between countries, demonstrating the need for optimization. This analysis quantified the health gains demonstrating the urgency of rapid patient access.