Related Experiment Video
Updated: Jul 23, 2025
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Early access for innovative oncology medicines: a different story in each nation
Tara Cowling1, Ransi Nayakarathna1, Allison L Wills2
1Medlior Health Outcomes Research Ltd., Calgary, Canada.
Background:
As innovative oncology medicines are rapidly developed, there is increasing pressure on payers to offer patients timely access to life-saving therapies. The uncertainty surrounding these therapies when phase III clinical trials are pending has necessitated new, adapted pathways to market access, with timelines that greatly vary by country. Understanding differences between pathways may identify opportunities to expedite patient access universally.
Objectives:
To describe early access pathways for new oncology medicines among selected countries with established health technology assessment (HTA) frameworks and publicly funded health systems, with a special focus on real-world evidence (RWE).
Methods:
We reviewed the HTA agency websites of the selected OECD countries: National Institute for Health and Care Excellence (NICE) for England and Wales; Haute Autorité de Santé (HAS) for France; IQWiG and G-BA for Germany; Agenzia Italiana del Farmaco (AIFA) for Italy; Pharmaceutical Benefits Advisory Committee (PBAC) for Australia; and CADTH and Institut National d'Excellence en Santé et Services Sociaux (INESSS) for Canada as the primary source of evidence.
Results:
Processes for early patient access to innovative oncology therapies varied across selected countries; however, most countries have an established pathway for publicly funded early access (England and Wales, France, Germany, Italy, and Australia). The utilization of RWE to support earlier access (coverage with evidence) also varied by country, with some HTA organizations being actively engaged in these agreements (NICE, AIFA, and HAS) and others having no established processes in place (G-BA and CADTH/INESSS).
Conclusions:
This review of early access pathways for novel oncology medicines found substantial variability between countries of interest. Coverage with evidence frameworks may provide a unique opportunity for industry and payers to collaborate on earlier access to innovative cancer therapies with life-saving potential.
Insights
Early access pathways for innovative oncology medicines vary significantly by country. Utilizing real-world evidence (RWE) in coverage with evidence agreements offers a collaborative opportunity for faster patient access to life-saving cancer therapies.
Area of Science:
- Oncology
- Health Technology Assessment
- Pharmaceutical Policy
Background:
- Innovative oncology medicines require timely patient access.
- Phase III trial data is often pending for novel therapies.
- Varying international market access timelines create disparities.
Purpose of the Study:
- To describe early access pathways for new oncology medicines.
- Focus on countries with established health technology assessment (HTA) frameworks.
- Emphasis on the role of real-world evidence (RWE) in these pathways.
Main Methods:
- Review of HTA agency websites from selected OECD countries.
- Included NICE (England/Wales), HAS (France), IQWiG/G-BA (Germany), AIFA (Italy), PBAC (Australia), CADTH/INESSS (Canada).
Main Results:
- Most selected countries have established publicly funded early access pathways.
- Utilization of RWE for earlier access (coverage with evidence) varied significantly.
- Some HTA organizations actively engage in RWE agreements, others do not.
Conclusions:
- Substantial variability exists in early access pathways for novel oncology medicines.
- Coverage with evidence frameworks present opportunities for industry and payer collaboration.
- Collaborative frameworks can expedite access to life-saving cancer therapies.
More Related Videos
Related Concept Videos
01:11Clinical Trials: Overview
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:28Preclinical Development: Overview
02:50Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
01:02Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
02:57Targeted Cancer Therapies
There are several types of targeted therapies against...