Will Comparative Effectiveness Research lead to Rationing?

Senators Ron Kyl (R-AZ) and Mitch McConnell (R-KY) recently introduced the ''Preserving Access to Targeted, Individualized, and Effective New Treatments and Services Act of 2009" (S1259), a legislative proposal that Sen. Kyl's office said was designed to prevent comparative effectiveness research (CER) from being used to ration healthcare. The economic stimulus package passed earlier this year contained $1.1 Billion for such research; the House Democrats' draft health reform plan now being debated provides an additional $100 Million per year for the next three years for CER; the research is a fundamental building block of the President's approach to health reform. Are we inviting rationing of health care when we support the reform effort?

A few Republican Senators aren't the only ones nervous about CER. AdvaMed, the leading medical device industry trade association, is cautiously supportive, so long as CER is not "used by Medicare, insurance companies, or other public or private payers to deny coverage". PhRMA is worried that CER could take treatment decisionmaking out of the hands of physicians and patients. BIO "is concerned that comparative effectiveness information may be used strictly as a means to contain costs, rather than deliver health care value by improving patient health outcomes". None of these associations uses the dreaded R word, but their concern is clear - in the wrong hands (i.e. the government's hands), CER could be used to justify insurers' decisions not to pay for services and technologies doctors want to prescribe and companies want to sell. And isn't that rationing?

Well ... No, not by any rational use of the term. Formally, rationing is the limitation of the amount of some scarce commodity that is available to an individual, family or community - think about food or gas rationing during WW II. Rationing can be overt and explicit policy in response to unavoidable supply limitations, as in those examples, or it can be indirect, through the purposeful limitation of supply - think about planned limitations on kidney dialysis treatment capacity in Britain until just a few years ago, or waiting lists for artificial hip procedures in Canada on account of conscious budget allocation decisions. Any purposeful management of a supply deficit for a good or service that people want and need can fairly be characterized as rationing. We've typically not done anything like that in healthcare in the United States, and CER doesn't open the door to it. CER won't artificially limit the supply of technologies or services; CER won't limit the amount of healthcare, either segmentally or in toto, available to individuals; CER won't arbitrarily impose fixed budget ceilings that translate into service shortages.

What CER does promise to do is to gather reliable information about the relative merits of different treatment options for particular classes of patients under defined circumstances. The information would be used in different ways by different health system stakeholders. It is pretty universally agreed that:

doctors would and should use CER findings to make better therapy choices or recommendations to patients; and
patients would and should use CER findings to more effectively evaluate physicians' advice and to be better informed participants in decisionmaking about their healthcare.

What seems to be in dispute is whether health insurers should be allowed to use CER findings to define the circumstances under which they will or will not pay for specified technologies or services. Let's look at the language of S1259:

Notwithstanding any other provision of law, the Secretary of Health and Human Services--
(1) shall not use data obtained from the conduct of comparative effectiveness research, including such research that is conducted or supported using funds appropriated under the American Recovery and Reinvestment Act of 2009 (Public Law 111-5), to deny coverage of an item or service under a Federal health care program (as defined in section 1128B(f) of the Social Security Act (42 U.S.C. 1320a-7b(f))).

This is pretty remarkable. Medicare and Medicaid, faced with data from a CER study indicating that Treatment A is clearly superior to Treatment B for a particular clinical problem for all patient groups under all conditions studied, would be prohibited from denying coverage to the clearly inferior treatment. I'll say it again differently: Medicare would be required by law to cover and pay for a treatment option known to be inferior because the data establishing inferiority were collected in a CER study.

This is nonsense. We want insurers, including Medicare and Medicaid, to use the best available data to cover what works and not cover what doesn't. And that is exactly what Medicare and every private insurer has been doing for years. The coverage analyses and decisions, often with detailed documentation of the determinants of the decision, are available online for anyone to review: when and where Medicare will pay for an implantable left ventricular assist device; under what circumstances Aetna will or will not pay for continuous mobile cardiac monitoring to help diagnose cardiac rhythm disorders; etc. Making these decisions is what insurers do, and they would be irresponsible - with our well-being as well as with our tax monies or premium payments - if they didn't use the best available information to make them.

Stakeholders potentially adversely (financially) affected by insurance coverage decisions have historically had recourse to two arguments: that the insurers are "interfering with the practice of medicine" (sometimes transposed into "interfering with the sanctity of the physician-patient relationship"); or that they are rationing care. As we've come to understand that docors are as human, fallible and self-interested as the rest of us, the first argument has lost its bite. Now, those who see wide dissemination of better information as a threat are left to play the rationing card. Get below the surface, and it is absurd - but we'll see whether it retains any power.

Will Healthcare Reform Stifle Technology Innovation?

At CIMIT's Innovation Grand Rounds yesterday, an audience member posed an intriguing question (paraphraphed as follows): "Since almost all the real health technology innovation we see is generated in the U.S., will our move toward a more European-style, single-payer system destroy our ability to innovate?".

Let's ignore, for the moment, that the question greatly overstates domestic hegemony in the innovation department; even as we ought to be aware of centers of health technology innovation developing and growing elsewhere around the world, it remains true that the U.S. continues to produce a disproportionate share of new diagnostics, therapeutics and healthcare management technologies. And let's not get sidetracked by the observation that we are today no closer to a single-payer system than we ever have been (you need only refer to congressional resistance to a publicly administered health insurance plan that would compete with private plans), or the fact that most European health care systems are moving toward diversification of payers, not unification. A real question remains: Will the reforms currently in the works weaken the ability of U.S. life sciences companies to develop and commercialize innovative technologies?

The CIMIT panelists (moderator Michael Greeley of Flybridge Capital Partners, Dr. Marsha Moses of Predictive Biosciences and Children's Hospital Boston, Dr. Elazar Edelman of the Harvard - MIT Health Sciences and Technology Program and Harvard Medical School, Dr. Joe Smith of Johnson & Johnson's Corporate Office of Science and Technology, and Zen Chu of Accelerated Medical Ventures), all viscerally entrepreneurial, thought not. The consensus: We'll continue to innovate - it's cutural, part of our national DNA; but there will be a turn toward innovations that generate cost-savings rather than enhanced capabilities regardless of cost. One panelist even opined that he wished that Medicare would just come out and formally adopt cost-effectiveness standards so that we could unambiguously get on with the job. And no one even flinched at the heresy.

I don't think our desire or our ability to innovate in the life sciences are at risk from any reforms that are likely to receive serious consideration in the current administration - and certainly not fom any current proposals. And I agree that there is likely to be a turn toward innovations that can lower the cost of high quality care - Christensen's disruptive innovations. But I also think it would be foolish to believe that our innovation rate cannot be affected by our healthcare reimbursement system - most particularly, by the ability of that system to allow returns to life sciences capital investments commensurate with that available to investments in other sectors. The entrepreneurial spirit needs to be fed.

I'm also impressed by the disjunction between the CIMIT panel's easy acceptance of the possibility of formal cost-effectiveness standards and the continued industry doubts about relatively benign initiatives toward Comparative Effectiveness Research. The ECRI Institute provides a rich compendium of background materials on CER, including a comprehensive catalogue of trade association and professional society position papers, for those who might be interested in digging deeply into the subject. An April 14 Wall Street journal report is characteristic: "A $1.1 billion infusion for research that compares the effectiveness of differing treatments raises thorny issues on how that research might be used, including the possibility of denying patient access to treatment options, according to some members of Congress and drug and medical-device companies." This despite the fact that it is very clear from multiple sources that current law forbids such use of CER, that current legislative proposals are all designed to reinforce rather than undermine the prohibition, and that testimony from administration officials, including a letter to concerned Senators from DHHS Secretary-designate Sibelius as reported in the Pink Sheet Daily on April 13, is consistent: no intent or desire to use CER to make national coverage policy.