CMTP Releases Two Effectiveness Guidance Documents

September 14, 2010

The Center for Medical Technology Policy (CMTP) announces today the release of two Effectiveness Guidance Documents on Pragmatic Phase 3 Pharmaceutical Trials and New Indications for Oncology Drugs. 

Effectiveness Guidance Documents (EGDs) provide recommendations to drug and device developers about how to design trials that are more useful for patients and their clinicians.  Although more than a trillion dollars is spent on health care each year in the United States, less than half of treatment decisions are guided by scientific evidence about what works.  This is partly because the right kinds of clinical studies are not being done.  The Pragmatic Phase 3 Pharmaceutical Trials EGD recommends ways to make drug trials designed for regulatory approval by the Food and Drug Administration (FDA) more pragmatic.  A major conclusion of this work is that small steps, such as enrolling the types of patients who will most likely use a drug once it is approved, can markedly improve the utility of information obtained from regulatory trials. 

Oncology drugs are often used outside the indications approved by the FDA and the well-recognized lack of evidence for these uses prevents physicians from knowing and providing the best treatments for their cancer patients.  The New Indications for Oncology Drugs EGD attempts to close these evidence gaps by providing recommendations to researchers on the design and implementation of clinical trials that have a greater potential to answer these crucial treatment questions than historically found in current research. 

The principles and recommendations made in these EGD’s were developed using a unique process that solicits input various stakeholders spanning the spectrum of those with considerable interest in the outcomes of these studies, including representatives from patient advocacy groups, public and private payers, academics, pharmaceutical companies, and government agency representatives.  By engaging stakeholders with perspectives that commonly differ from researchers, recommendations for clinical research can be developed with the potential to significantly improve medical research.

Click here for additional information and the Pragmatic Phase 3 Pharmaceutical Trials EGD and here for the New Indications for Oncology Drugs EGD.  As a vital part of our process, we welcome your feedback on both documents at info@cmtpnet.org.

CMTP is a private, non-profit organization which serves as a neutral forum to promote discussion and develop strategies that improve the quality of clinical research for healthcare decision making.   CMTP is uniquely qualified to help develop the methods and strategies that will ensure that comparative effectiveness research fulfills its intended goal of helping patients, clinicians and payers make more informed healthcare decision.