Investigational: what's in a name?

Investigational: What's in a Name The Health Care Financing Administration stipulates that a technology must be "noninvestigational" as one of four criteria that are applied in determining whether or not a technology is covered under Medicare. The national Blue Cross Blue Shield Association (BCBSA) relies on five specific coverage criteria that must be satisfied in order to render an affirmative coverage decision. BCBSA, in the past, labeled technologies that did not meet all five criteria as "investigational" and ineligible for coverage. Although BCBSA has moved away from categorizing technologies as "investigational," vestiges of this terminology persist in its presentations. Additionally, to varying degrees, the 70 Blue Cross Blue Shield plans, commercial insurers, HMOs, etc. continue to use "investigational" status as a major criterion for exclusion of drugs, devices, and procedures from coverage. Notwithstanding the critical importance of the term "investigational," it remains difficult for payers to articulate a generalizable definition that, even within the same company, applies consistently as the basis for all coverage decisions.

The answer (or copout) to this conundrum is that there is no specific definition or set of criteria that can be applied across different subpopulations of patients. In coverage, as in clinical, decision making, one is using measures from evolving continuous scale. First, one is making a risk/benefit determination in that one is judging what the appropriate balance is between the expected benefit to be derived and the potential for harm to patients. Second, this judgment occurs along a continuum of accumulating evidence about the safety and effectiveness of the technology when applied for a specific indication.

The concept of this continuum is embodied in two recent court cases. In 1988, in Reilly vs. Blue Cross and Blue Shield United of Wisconsin, a case involving coverage for in vitro fertilization (IVF), an expert witness stated, "I think that like all of medicine, it (IVF) is always investigational and experimental in the sense that we have a potential for doing better and should investigate that potential."

The court correctly interpreted the statement to mean that ongoing compilation and analysis of data improve our understanding and ability to use a technology. A second case, Rollo vs. Blue Cross Blue Shield of New Jersey, involved a suit over denial of coverage of autologous bone marrow transplantation. The court discussed the issue of investigational as follows: "Certainly, every treatment that does not have a hundred percent cure rate is open for improvement and, thus, has somewhat of an investigative or experimental component. Certainly, too, data from the treatment (the patient) will undergo will be accumulated and analyzed to improve yet once again what is known and, thus, in this respect as well, the treatment has an investigative or experimental component, as does any treatment as to which data is being compiled."

The continuous or sliding scale of evidentiary standards for affirmative coverage are clearly illustrated in the statement by HCFA in its discussion and recognition (Federal Register, Jan. 30, 1989) of the criteria and procedures for coverage decisions. HCFA stated that "when evaluating breakthrough medical or surgical procedures, the standards for safety and effectiveness are less stringent. The more severe and life-threatening the illness or injury process for which a particular treatment is applied, the more acceptable a relatively less safe technology may be when no safer or more effective technologies are available." Indeed, this philosophical orientation of risk/benefit analysis was the basis for recent FDA regulations establishing the treatment IND and the expedited drug approval processes.

Risk/benefit analysis is the foundation of any physician's clinical decision making. The distinction between risk/benefit analyses performed by physicians and payers is crucial, however. The treating physician is dealing with the absolute of the hard data regarding an individual patient. The payer is dealing in the aggregate of averages of data regarding a population of patients.

The need to make risk/benefit analyses, the evolutionary nature of the available evidence, the uncertainty of dealing in the aggregate, and the primacy of individual needs obviate the possibility of developing a nice clean definition or set of criteria that will eliminate the difficult and often contentious nature of health care decision making. Rather, these factors and today's health care environment demand that all constituents of the health care community return to the much-maligned descriptor of HCFA; that we be "reasonable." Obviously, this reaffirmation to exercise sound judgment will be facilitated by the availability of cold hard data on health outcomes. The health care community must continue its efforts to organize the collection, the analysis, and the dissemination of these data.

However, according to Miriam Webster, to be reasonable also means to be moderate and fair, not extreme and excessive. We must be flexible in our support for new advances in technology, especially new technologies offering promise to those with serious and/or life-threatening illnesses and, at the same time, resolute in our efforts to eliminate obsolete technologies. Such reasonable balance in decision making will be facilitated by open and frank discussion of both general philosophical issues (i.e., coverage criteria, established vs. investigational) and specific clinical and coverage issues. Such discussions should involve patients, physicians, and payers.

The American Medical Association has recently established one such mechanism for dialogue, the DATTA Forum. The objective of the DATTA Forum is to bring together interested parties to examine the available clinical data and discuss controversial issues in clinical decision making. The inaugural DATTA Forum meeting convened representatives of more than 80 major payers, HMOs, and medical centers to discuss the issue of autologous bone marrow transplantation for breast cancer. During the discussions, the question of what is "investigational" recurred again and again. As in this article, a universally accepted definition of the term was not produced. However, the examination and discussion of data and concerns from all sides did serve to enhance understanding and communication. This last statement is not as trite as it may seem. For discussions such as this can translate the polarization of "investigational vs. established" into a reasoned balance between attending to the needs of individual patients and enhancing the efficiency of the nation's health care system.