OBJECTIVES:

• To compare payer/ regulator assessments for 5 AstraZeneca cancer medicines in the context of clinical uncertainty
• To quantify time to patient access
• To identify potential improvements for the medicine access pathway, during long-term clinical benefit uncertainty

METHODS: The transition from regulatory approval to reimbursement for the first launches of 5 AstraZeneca cancer medicines in Canada, England, France, Germany, Italy and Spain was assessed. Initial and follow-up clinical survival benefit data were compared. Time to access was calculated as the time between regulatory approval and reimbursement listing. Lost clinical survival benefit was calculated as the product of the number of patients diagnosed with disease per year, time to access, and the added clinical survival benefit.

RESULTS: The average time to access was over 15 months for the first commercial launches, leaving an estimated 2425 patients with lost clinical benefit, and more than 2285 life-years lost. The implication is that many patients do not have access to new therapies resulting in the loss of significant additional progression free survival, disease free survival and overall survival time.

CONCLUSIONS: The root causes for observed delays to medicine access entail uncertain long-term clinical benefit, divergent perspectives and goals between manufacturers, regulators and payers, and inefficient dual assessment processes. To mitigate the influence of any bias on decision making, it is critical to decouple the clinical survival benefit assessment from negotiation during the reimbursement process. Effective collaboration, with increased transparency and harmonisation, is required between regulators, payers and manufacturers to uncover shared interests and reduce uncertainty. Performance-based access agreements between manufacturers and payers provide a balanced solution to accelerate medicine access to patients during the period where long-term clinical benefit is uncertain. Reimbursement systems must continue to evolve and align with scientific advances to unlock the shared value of innovative medicines for manufacturers, regulators, payers and, most importantly, patients.