The mandate for investigational cancer therapies.

HEALTH CARE TECHNOLOGY

The heated national health care reform debate of the summer of 1993 has now simmered down, but pieces of the debate are still percolating along at the state level. Within the past year, numerous states have introduced bills that would mandate insurance coverage for investigational cancer therapies - in particular high-dose chemotherapy and bone marrow transplant for breast cancer. The problem with these initiatives, well-intended though they may be, is that they threaten to spread unproven technologies at a rapid rate and at the same time miss the opportunity to collect data that would prove the safety and effectiveness of the methods. The author explores these issues and suggest how managed care companies can play a more aggressive role in parrying the threat.

Ever since its inception some seven years ago, high-dose therapy for breast cancer has served as a lightning rod for debate. It touches on all aspects of the health care controversy - the high cost of new medical technologies and patients' access to them, the critical need for outcome data to validate new therapies, add the issue of who should subsidize medical research - the federal government, policyholders, patients, or manufacturers. Current initiatives at the state level reflect the frustration over the use of "investigational" as an exclusionary criterion for coverage and place the burden of clinical research costs squarely on the shoulders of policyholders. Although payers and their policyholders may feel that subsidizing medical research is not their responsibility, patients and physicians have argued that the investigational coverage exclusion is used primarily as an arbitrary cost control mechanism. The situation becomes particularly emotional (and litigious) when it involves a patient with cancer who may have exhausted all other treatment options and a promising but unproved therapy - for example, high-dose chemotherapy - is offered as the only hope of cure.

The list of states mandating coverage for investigational cancer therapies is growing. So far, the list includes, but is not limited to, Florida, Illinois, Louisiana, Maryland, Massachusetts, Missouri, New York, Pennsylvania, Rhode Island, and Virginia. Some bills, such as those of Virginia and Massachusetts, mandate coverage only for high-dose chemotherapy for breast cancer, but others propose sweeping mandates to cover a wide variety of investigational therapies. In the proposed New York bill, for example, coverage would be mandated for patients enrolled in clinical trials if, among other things, the trials have been approved by the Food and Drug Administration as part of investigational new drug exemptions. This mandate would clearly relieve drug manufacturers of their legitimate responsibility to pay for their own research and development costs for drugs that have not even received full FDA approval. While most of the state initiatives limit their mandates to cancer therapies, New York goes one step farther and includes patients with "disabling or degenerative diseases."

Florida has taken a slightly different approach. While the majority of the bills do not deny that the proposed mandated benefits involve investigational therapies, Florida has set up a process by which the state will determine when a therapy is no longer considered investigational/experimental and thus should be eligible for coverage. For example, the bill states, "An insurer may not exclude coverage for bone marrow transplant procedures recommended by the referring physician and the treating physician under a policy exclusion ... if the particular use of the bone marrow transplant procedure is determined to be accepted within the appropriate oncological specialty and not experimental." A nine-member panel (four payer representatives, four local oncologists, and one consumer representative) determines which applications of bone marrow transplant are or are not experimental. The initial proposed list of indications eligible for coverage includes brain tumors, melanoma, lung cancer, and "miscellaneous solid tumors" - all indications for which there are clearly very few data.

Aside from the issue of who should pay for investigational research, the New York and other similar bills have the possible advantage that at least patients will be funneled into clinical trials so that they are treated in some systematic fashion and outcome data can be analyzed. In contrast, any incentive on the part of the patient to participate in a clinical trial is destroyed when a therapy is simply labeled "nonexperimental," as in Florida's proposed bill. One has only to look at the issue of bone marrow transplantation for breast cancer to appreciate the havoc this type of policy causes. Although payers were initially reluctant to provide coverage for this treatment because of the lack of clinical outcomes, the resultant negative publicity and adverse court decisions prompted early coverage for this unproved therapy. The consequences were perhaps predictable. Bone marrow transplant for breast cancer has diffused rapidly, patients are treated under a variety of protocols (if under any established protocol at all), patient selection criteria have broadened to include patients earlier in the course of their disease, results are not collected in a coordinated fashion, and the National Cancer Institute is having difficulty recruiting patients into its studies. Furthermore, in some institutions, the technique has now evolved to include multiple rounds of high-dose therapy and transplant, while we still do not know the long-term outcomes of a single round of high-dose therapy.

In summary, state-mandated benefits and broad definitions of what is considered an approved clinical trial create a disastrous recipe for the rapid and uncoordinated diffusion of unproved technologies, and for missed opportunities to collect the outcome data needed to determine safety and effectiveness of treatments.

How can a managed care company use its coverage leverage more constructively to address the issue of support for clinical research without creating an environment of uncontrolled diffusion? In a reversal of existing policies, managed care companies and insurers could provide coverage only to those patients who agreed to participate in clinical trials at a limited number of institutions, thus limiting premature diffusion and creating the opportunity to develop the needed outcomes research. A national advisory board could be established to oversee this process, not only to prioritize which technologies should be studied but also to analyze the collected data. Coverage would only be broadened when outcomes data had validated the safety and effectiveness of the technology. Assurance of coverage would permit earlier access to investigational treatment for patients in need and would eliminate the "hassle" factor. Finally, this proposal would permit efficient collection of desperately needed outcomes data.

Clearly, the days are gone when the government, through generous NHI funding, subsidized a larger share of the costs of clinical research. And recent court cases and negative publicity have clearly illustrated that patients and physicians are increasingly frustrated with the use of the concept of investigational therapy as an exclusion for coverage. However, the proposed state initiatives unreasonably shift this burden entirely to policy holders, even to the extent of subsidizing the research and development costs of drugs unapproved by FDA. Furthermore, as illustrated by the experience of bone marrow transplant for breast cancer, there is no question that managed care companies and other payers are already subsidizing all sorts of investigational therapies, but the tragedy is that rapid and uncoordinated diffusion outside of clinical trials makes analysis of outcomes problematic. Managed care companies may be more willing to support clinical research if there is some guarantee that diffusion will be limited until the potential health benefits of a new technology are more fully known.

Elizabeth Brown, MD is Director, Technology Assessment and Clinical Guidelines, Aetna Health Plans, Aetna Life and Casualty, Chicago, Ill. The opinions expressed in this article are those of the author and not necessarily those of Aetna Health Plans.