Bethesda, MD—Greater adoption of personalized medicine and comparative effectiveness research (CER) in healthcare will depend on coverage of these approaches by public and private payers. But payer evaluation of these approaches is hampered by legislative roadblocks and a lack of research in CER and slowness in adopting CER into clinical practice, according to speakers at a session of the ECRI Institute’s Comparative Effectiveness and Personalized Medicine: An Essential Interface conference.
For Lee Newcomer, MD, Senior Vice President of Oncology at United Healthcare, CER is a positive development, one that might weed out outmoded therapies, focus innovation, reduce toxicities, and allow rational pricing for competing therapies. Ultimately, it should accelerate the better use of healthcare resources. Personalized medicine will need to be evidence based, and for Dr Newcomer, CER is the way to supply that evidence.
But in discussing UnitedHealthcare’s experience with physicians’ use of bevacizumab (Avastin), Dr Newcomer recounted how data showed that physicians were using this drug in the adjuvant setting, be fore any trials in that setting had been done. The danger, he fears, is that personalized medicine may be thought of as giving physicians “carte blanche” to try treatments that are outside the evidence.
In addition, before wading into the complexities of genomics, CER, and coverage issues, Dr Newcomer also cautioned that more prosaic comparative effectiveness matters need to remain in the forefront of consciousness. For example, 2 studies in the same issue of the New England Journal of Medicine looked at adjuvant chemotherapy with standard chemotherapy versus adjuvant chemotherapy with trastuzumab in breast cancer, and the first one indicated more complications with what has become the standard treatment approach in the United States (52 weeks of trastuzumab therapy [Romond EH, et al. Trastuzumab plus adjuvant chemotherapy for operable HER2-positive breast cancer. N Engl J Med. 2005;353:1673-1684]). The second study indicated 12 weeks of therapy was efficacious and did not cause the cardiac side effects seen in the first study (Joensuu H, et al. Adjuvant docetaxel or vinorelbine with or without trastuzumab for breast cancer. N Engl J Med. 2006; 354:809-820); nevertheless, there has been no outcry in the advocacy community for a comparative effectiveness study in this area. Research downstream from the original investigations has much potential value, he suggested. “That part of comparative effectiveness would benefit patients dramatically.”
A second example offered by Dr Newcomer discussed the role of CER in evaluating various therapies for prostate cancer. Newer, increasingly expensive radiation therapies, including intensity-modulated radiation therapy, stereotactic body radiosurgery, and proton beam therapy, have all gained wide acceptance, despite a lack of comparative effectiveness studies on them. CER would help inform individual patients on which approach might be the best fit for them.
Right now, we have “lots of technologies, and no idea how to use them [effectively],” he lamented. Evidence informs good decisions, Dr Newcomer stressed, and although some therapies may go away through the use of CER, they may disappear for the right reasons.
As a public payer responsible for more than 114 million American lives, the Centers for Medicare & Medicaid Services (CMS) is also dealing with covering personalized medicine and CER, albeit at a much greater scope. As noted by Barry M. Straube, MD, Chief Medical Officer and Director at the Office of Clinical Standards and Quality at CMS, those deliberations are shaped by the legislation that details what is covered and how CER information can and cannot be used. In some ways, Dr Straube acknowledged, CMS is still struggling to understand the intent of coverage language in bills crafted 40 years ago.
Medicare coverage outlined under section 1862(a)(1) of the Social Security Act specified coverage is required for items and services deemed “reasonable and necessary for the diagnosis or treatment of illness or injury or to improve the functioning of a malformed body member.” According to Dr Straube, CMS has defined “reasonable and necessary” as demonstrating adequate evidence to conclude that the item or service or test improves clinically meaningful health outcomes for the Medicare population. And in evaluating therapies, he emphasized, CMS examines the new therapy compared to usual care, not another device or therapy. So a shift to using CER will require a fundamental change.
But the healthcare reform law states explicitly that cost should not be a factor in coverage determinations, and that coverage cannot be denied “solely on the basis” of CER. Further, the Patient Centered Outcomes Research Institute, an independent advisory committee being set up as part of healthcare reform, is also proscribed from developing or employing a dollars/ quality-adjusted life-year threshold. Although Congress has legislated that cost-effectiveness is not allowed to be a factor in coverage determination, it is allowed under the Medicare Improvements for Patients and Pro viders Act of 2008 (Section 101), which covers preventive services. Whether personalized medicine is defined as a preventive service remains to be seen. So “although there’s been the mandate to do more comparative effectiveness research, clearly we have limitations,” Dr Straube acknowledged.
There are difficulties in drafting coverage for CER, Dr Straube pointed out. If CER determines that a test or technology is far better than others, that single item may be the only one covered. But if something then prevents that technology from being used (ie, a manufacturing problem), there may be no reimbursable treatment. Patient outliers to approved treatments may also be left out if CER is adhered to as a strict doctrine.
Legislation also must be carefully drafted so that unintended consequences do not crop up. A question from the audience, for example, pointed out that genetic counselors are not considered providers and are therefore not reimbursed under Medicare. Although CMS agrees that genetic counseling is a necessary service, the law does not allow this reimbursement, and the ultimate solution will require further legislation, said Dr Straube.
Finally, Dr Straube mentioned recommendations regarding CMS coverage for genetic testing arising from 2 Medicare Evidence Development & Coverage Advisory Committee meetings. These committees recommended the evidence standards for genetic and genomic testing need to be high.
As Dr Straube noted at the outset of his presentation, when patients start coming in to medical offices, requesting access to the latest genetic tests and the treatments that result from them, the enormity of costs to society are frightening.
