A recent analysis in the Cancer Journal outlines the various effects of last year's landmark health care reform bill on cancer research, with big questions regarding comparative effectiveness research (CER). Many changes in the pipeline will certainly be positive, but there may be potentially negative "unanticipated consequences."
Starting with the 2009 stimulus package, CER was supported with $1 billion, and with the Patient Protection and Affordable Care Act, the program has been expanded significantly.
The analysis notes that "CER provides the promise of using large data sets to define effectiveness of treatment options that have been differentially implemented in varied practice settings, especially as they relate to off-label use of drugs and devices."
Off-label use has been controversial for years and has led to denial of insurance in some cases, but "with systematic data collection, CER studies might provide convincing evidence of efficacy—or lack there of—in settings where prospective trials might never be practical."
Further, the authors mention the possibility of improving treatments for cancer types with low rates of incidence where again conducting randomized trials may not be possible. A CER study in the journal Blood which used lymphoma subtypes from the National Comprehensive Cancer Network non-Hodgkin lymphoma database is a relevant example.
Several questions in relation to cost are raised: Will cost of a drug be taken into account when comparing different treatment approaches? And will "CER be utilized to gain FDA approval for newer therapies that may be more effective (not a current requirement for new drugs) or less expensive than currently approved agents?"
Currently, the FDA only requires that drugs be safe and effective—not cheaper or better. And if a new treatment is proved to be more effective, what will happen to previously used regimens?
The authors make several points regarding the shortcomings of CER that the health reform bill fails to account for. In its current conception, CER will not be able to account for differences in similar disease type. The article cites hormone-receptor positive breast cancer as an example, a disease which, like certain other cancer types, "differs dramatically in the likely responsiveness to treatment and may have genetic differences in metabolism of drugs that impact effectiveness."
There is also the worry that resources for CER might be drawn from other types of resources, particularly NCI.
Click here to read the full article: The Anticipated and Unanticipated Consequences of the Patient Protection and Affordable Care Act on Cancer Research