CMTP Launches Initiative to Improve Evidence for New Indications in Oncology

October 5, 2009

The Center for Medical Technology Policy (CMTP) has launched a six-month initiative with the overarching goal of developing recommendations on methods and strategies to improve clinical research designed to assess the comparative effectiveness and value of oncology drugs used for new indications. 

The need for this initiative emerges from the current approaches to research on off-label uses of cancer drugs that frequently do not incorporate the type and/or quality of evidence requested by post-regulatory decision-makers.  As a result, there are large evidence gaps that impair decision making at the individual and population levels, resulting in inconsistency in how off-label prescribing occurs across prescribers and patient groups.  

As part of this initiative, CMTP will convene a one-day multi-stakeholder Oncology New Indications Think Tank on November 12, 2009, to improve the systematic generation of evidence about the effectiveness of off-label uses of oncology drugs, providing a neutral ground to establish the steps necessary for improved evidence for drug therapy in cancer. 

Confirmed participants include eminent clinical researchers and representatives from the insurance and pharmaceutical industries, the U.S. Food and Drug Administration, Medicare, and patient advocacy organizations, as well as international regulatory agencies including the National Institute for Health and Clinical Excellence (NICE) in the United Kingdom and the Pharmaceutical Benefits Advisory Committee (PBAC) within the Australian Department of Health and Aging.

Through informed discussions, the New Indications Think Tank will produce a framework of the issues to address in a methodological guidance surrounding post-approval study design that will support an evidence-based approach to determining the value of off-label use of oncology drugs.

In the succeeding months, CMTP will work with invited members of an expert working group to complete a draft methodological guidance document.  The broader stakeholder group will then reconvene to provide input and comments on a second draft.  The project will culminate in the public release of a guidance document for off-label oncology clinical trial design.

More information

• Project overview (PDF)