Pathways to early access in oncology
August 23, 2023

The pressure on payers to offer patients timely access to life-saving oncology medicines has increased with the rapid development of innovative treatments. Balancing the urgency to provide access with the need for evidence-based decision-making from phase III trials presents a significant challenge to publicly funded systems worldwide. As a result, timelines to market access vary by country.

A recent study conducted in collaboration with AbbVie, 20Sens Corp., and Medlior took a closer look at various types of early access schemes for innovative oncology medicines, to facilitate adoption in countries without an expedited process. The published study “Early access for innovative oncology medicines: A different story in each nation” in the Journal of Medical Economics describes the findings.1

An international comparison

Across six OECD countries with established health technology assessment (HTA) frameworks and publicly funded healthcare systems, a variety of mechanisms were identified for accelerating public funding for patient access.

Established early access pathways were identified for England and Wales, France, Germany, Italy, and Australia. Some HTA organizations (NICE in England and Wales, AIFA in Italy, and HAS in France) were actively engaged in coverage-with-evidence agreements while others (G-BA in Germany and CADTH/INESSS in Canada) had no formal established process in place linked to HTAs. 1

Access through an RWE lens

Real-world evidence (RWE) enables funding decision-makers to utilize data gathered from a variety of sources (e.g., patient registries, administrative data, and claims data), to complement clinical trial results. RWE provides a more comprehensive understanding of treatment effectiveness, long-term safety, and real-world patient outcomes, to potentially accelerate early access to new medicines.

In coverage-with-evidence agreements as in England/Wales, Italy, and France, patient access is granted while ongoing data such as RWE are still being collected to support the final recommendation or listing decision. Comprehensive guidance documents are emerging for planning and reporting RWE studies as part of the regulatory and reimbursement processes.

The shared goal

Early access schemes can potentially transform patients’ lives. In the example of Tagrisso (osimertinib) in England, where the early trial results were promising and there was a high unmet need for treatment, an early access agreement was reached that provided patients with early access on a budget-neutral basis while the HTA was being conducted.

Around 100 patients had access to osimertinib in 2021, after the regulatory approval and early access scheme were in place. The time from the early access agreement to Cancer Drug Fund (CDF) listing was approximately 208 days; therefore, patients had access to therapy 208 days earlier than if it had gone through the traditional CDF process.2

Moving forward

Utilizing RWE to facilitate earlier access via a coverage-with-evidence development pathway is a common approach across many countries. Early access schemes that leverage RWE present unique opportunities for decision-makers and industry to collaborate on delivering better outcomes for patients and health systems.


  1. Cowling T, Nayakarathna R, Wills AL, Tankala D, Paul Roc N, Barakat S. Early access for innovative oncology medicines: a different story in each nation. J Med Econ. 2023 Jan-Dec;26(1):944-953.
  2. NHS UK, NHS to offer new drug that halves the risk of cancer returning., 2021 (accessed Sep 18, 2022).