C110
Efficient trial design for new cancer therapies
Peter Hall1, Julia Brown1, David Cameron2, Christopher McCabe1
1University of Leeds, UK, 2National Cancer Research Network, Leeds, UK
How can multinational trials of new targeted therapies for cancer be efficiently designed to inform national reimbursement decision makers in the UK?
The decision to adopt new expensive drug therapies for cancer is increasingly based on the results of multinational trials. These provide information allowing licensing authorities to assess suitability for drug registration.
This information is rarely sufficient for the health technology assessment required by decision makers in the UK. Recently global phase III trials have been followed by rejection by the National Institute of Clinical Excellence (NICE) but widespread adoption outside the UK. These include lapatinib for advanced breast cancer, bevacizumab for advanced kidney cancer and advanced breast cancer and tyrosine kinase inhibitors for advanced kidney cancer. Adjustments to the acceptance threshold for funding end-of-life drugs may provide a limited short term fix, but the problem is likely to grow as similar drugs prove efficacy in the adjuvant setting.
Further research to aid an adoption decision is often requested by NICE, but is generally too late to provide a meaningful contribution. The NHS and NICE require trials to inform technology appraisal at the time of licensing. Decision analytic methods have been developed by health economists which facilitate optimisation of trial design based on the expected value of information they will provide. Application has been demonstrated in a variety of clinical settings in the UK such as TNF-α for rheumatoid arthritis. We demonstrate their potential to objectively aid development of oncology trial portfolios and enable prioritisation of trial design elements.
