Comparative Clinical Effectiveness - An Issue or of Value?

There has been a flap over the inclusion in the HR 1 Senate version of a Section 802 mandating the development of methods of Comparative Clinical Effectiveness (CCE) . The Section of the Bill is devoid of definition but one need look no further than a CBO report in December 2007 by Orszag which details these efforts. The Section states:

"SEC. 802. FEDERAL COORDINATING COUNCIL FOR COMPARATIVE CLINICAL EFFECTIVENESS RESEARCH. (a) ESTABLISHMENT.—There is hereby established a Federal Coordinating Council for Comparative Clinical Effectiveness Research (in this section referred to as the ‘‘Council’’). (b) PURPOSE; DUTIES.—The Council shall— (1) assist the offices and agencies of the Federal Government, including the Departments of Health and Human Services, Veterans Affairs, and Defense, and other Federal departments or agencies, to coordinate the conduct or support of comparative clinical effectiveness and related health services research; and (2) advise the President and Congress on— (A) strategies with respect to the infrastructure needs of comparative clinical effectiveness research within the Federal Government; (B) appropriate organizational expenditures for comparative clinical effectiveness research by relevant Federal departments and agencies; and (C) opportunities to assure optimum coordination of comparative clinical effectiveness and related health services research conducted or supported by relevant Federal departments and agencies, with the goal of reducing duplicative efforts and encouraging coordinated and complementary use of resources."

It is not something new and frankly it is akin to the work done in the area of Evidenced Based Medicine (EBM). EBM espouses the practice of medical procedures based upon established clinical trials and using those trials one would incorporate a set of best practices. The CCE as defined by the CBO states:

"A variety of evidence suggests that opportunities exist to constrain health care costs both in the public programs and in the rest of the health system without adverse health consequences. Perhaps the most compelling evidence of those opportunities involves the substantial geographic differences in spending on health care—both among countries and within the United States—which do not translate into higher life expectancy or measured improvements in other health statistics in the higher spending regions. For example, Medicare’s costs per beneficiary vary significantly among different regions of the country, but much of the variation cannot be explained by differences in the population, and the higher-spending regions perform no better on available measures of average health outcomes than the lower-spending regions do.

Furthermore, hard evidence is often unavailable about which treatments work best for which patients and whether the added benefits of more-effective but more expensive services are sufficient to warrant their added costs—yet the current health system tends to adopt more expensive treatments even in the absence of rigorous assessments of their impact. Indeed, the extent of the variation in treatments may be greatest when evidence about their relative effectiveness is lacking. Together, those findings suggest that better information about the
costs, risks, and benefits of different treatment options..."

Regrettably Senator Specter was questioned on this Section 802 as if it were a rationing provision. If done properly it is a quality improving position. As we have argued we must reduce costs and we must reduce demand. However, on the other hand, if one desires to pay personally for services which exceed those clinically proven to be effective, say rhinoplasty for a stuffy nose, then one should have the right to seek that out and pay accordingly. However Medicare or Medicaid, or even a private plan should have no obligation to pay.

The shaky ground occurs when dealing with catastrophic diseases and new protocols! Take the simple case of imatinib and CML. When it first came out, it was and frankly still is quite expensive. Does it prolong life, slightly, does it improve the quality of life, greatly. Thus the problem will be one of determining quality effects and their values and thus seeking the CCE solution. This will be especially the problem in Medicare, and with an aging population what treatments are proper, and which exceed the bounds. Marrow transplants for CML in patients over 80 is most likely not reasonable.

Then what of prostatectomies in men over 75, must they have only the watchful waiting option. For many men that is acceptable, but frankly we do not know enough of the genetic elements of PC to determine for what patients the protocol is best.

Thus the major problem of CCE is that it makes measurements which may fail to reflect the underlying differentiator, namely the genetic class of the patient. A woman with a BRCA gene defect will most likely best be served with aggressive treatment. A man with an indolent PC can watch and wait. In contrast a man with an aggressive PC, one which we cannot yet determine since we do not have the proven CCE approved test, will suffer bone mets and in in agony! The balance is a challenge to the Hippocratic Oath and to the bean counters in DC. Perhaps Orszag would think differently if he ever had to assist and dying patient with bone mets who had poorly treated PC!