Novel cancer therapies improve the length and quality of life for patients worldwide. However, adoption of such promising therapies is often delayed due to the collection of evidence required to make regulatory and reimbursement decisions. Access with Evidence Development schemes (AEDs) are a potential mechanism to support the timely adoption of novel therapies. They allow patient access whilst mandating that additional data be collected and analysed to support the revaluation of the therapy and potential reversal of the approval decision. Thus, AEDs have been used in several jurisdictions to provide access to innovative therapies while mitigating the harm associated with approving ineffective therapies; either to the patients directly or by diverting funds from alternative therapies.
There is limited guidance on how to design AEDs; regulators must determine what evidence should be collected and when the approval decision should be reassessed. Value of Information (VoI) is a method that has been suggested for AED design as it balances the consequences of making an incorrect decision due to immature data with the risks of preventing access to therapies by postponing the decision. AEDs that request the analysis of routinely collected health data are attractive to regulators and industry as they incur a relatively small financial burden. However, VoI has not been used to design studies that analyse routinely collected data, methods to optimise the time to reassessment are computationally challenging and there is a lack of expertise in using VoI for AED design.
The overall aim of this project is to develop an AED framework that requests the analysis of routinely collected data. We will achieve this by:
- Performing a VoI analysis to assess the value of using routinely collected data in AEDs.
- Adapting recent VoI calculation methods to efficiently optimise time until reassessment.
- Developing guidance for the use of VoI when developing AEDs.
The first aim will reuse a cohort of patients with metastatic colorectal cancer (mCRC) to develop an AED. We will develop a decision model to compare two treatments for mCRC using these data. We will then use a VoI analysis to compute the value of analysing routinely collected data. For Aim 2, we will adapt an efficient VoI calculation method so it can be used to efficiently optimise the time to reassessment for an AED. Finally, in Aim 3, we will collaborate with Canadian policymakers to create an online resource hub for VoI with videos, educational materials and worked examples and develop a bespoke software package. This will allow us to understand and surmount the barriers to implementing VoI in AED design.
Our team comprises of experts in VoI methods, the use of routinely collected data in healthcare evaluations and decision modelling and policymakers from the Canadian Agency for Drugs and Technologies in Health and provincial cancer funding agencies. We are also linking two collaborative research networks focusing on the use of VoI and routinely collected data.
This project will reduce the financial and administrative burden of designing AEDs to ensure that these agreements are respected and implemented. This contrasts with their current use by Health Canada. Our methods will optimise the design of AEDs, thereby improving the speed at which innovative therapies in oncology are made available to Canadian patients.