In the last several years, there has been considerable legislative activity at the state and federal levels regarding reform of the health insurance system, especially after President Clinton made federal legislation a top priority 2 years ago. A matter that is relatively minor in the overall constellation of issues, but which is of particular importance to oncology, is insurance coverage of, and patient access to, clinical trials.
This article reviews current coverage of clinical trials, discusses how health care reform may affect insurance coverage of clinical trials, and summarizes how the issue was treated in the intensive debate on health care reform during the past 2 years. While the congressional debate failed to produce federal reform legislation in 1994, the topic is certain to be revisited in the future.
Insurance coverage of clinical trials is an aspect of broader policy on coverage of new technologies. Both private health insurance plans and such public programs as Medicare and Medicaid typically exclude coverage of "experimental" or "investigational" procedures .
A drug or device is usually considered investigational for insurance purposes if it has not been approved for marketing by the FDA. Thus, drugs and devices subject to investigational new drug or device exemptions (INDs and IDEs) are typically not covered by insurance. A procedure is usually considered investigational if it is not accepted as safe and effective by the medical community. The use of an informed consent form is often seen by insurers as evidence that a particular procedure is investigational. The result of these policies is that clinical trials are usually excluded from insurance coverage.
Although insurance plans exclude experimental or investigational treatment, they cannot necessarily enforce their views regarding whether particular procedures lack coverage. Insurance plans can be sued by beneficiaries on the ground that the plan administrators are misinterpreting the coverage rules. In the last few years, the major issue litigated in this area has been coverage of autologous bone marrow transplants for breast cancer and other conditions that many insurers regard as unproven.
The courts have often been sympathetic to plaintiffs seeking access to coverage of bone marrow transplants. Courts have often overruled coverage denials by concluding that the plan's exclusionary language was unclear, or by directly holding that the bone marrow transplant procedures were not experimental. The decided cases have split between those that have upheld insurers excluding coverage of bone marrow transplants for certain conditions and those that have sided with patients.
The tide seems generally to be turning in favor of upholding insurer decisions, as insurers have revised contract- ual language in response to early decisions holding that particular language was unclear . There is also anecdotal evidence, however, that insurers are increasingly extending coverage to what they regard as experimental procedures out of fear of punitive judicial judgments, such as the recent $89 million verdict against a health maintenance organization for denial of coverage .
Although clinical trials are ostensibly not covered by insurance, insurance plans have nevertheless unknowingly often paid many of the costs. Insurance claim forms frequently do not require information that would disclose whether the patient was enrolled in a clinical trial, and as a result, insurers often cannot tell from the face of a claim that a patient received an investigational service.
The Inspector General of the Department of Health and Human Services recently initiated an investigation that may, especially depending on its outcome, change the way health care providers bill for patient care costs associated with investigational procedures [4 ]. As part of an investigation under the False Claims Act, the Inspector General issued subpoenas to over 100 hospitals regarding claims submitted to Medicare for certain investigational cardiovascular procedures. The unstated premise of the investigation is that the submission of a claim to Medicare for costs associated with an investigational procedure may not be simply a claim that Medicare may deny, but may also constitute a false claim. The theory of the investigation seems to be that health care providers may commit a criminal offense when they submit a claim for costs associated with clinical trials, since they know Medicare does not pay for clinical trials.
Even in programs that deny coverage of clinical trials, however, there may be some intentional limited coverage. For example, in the case of in-patients, Medicare, which pays a fixed amount based on the patient's DRG, attempts, to some extent, to separate the costs related to the investigational service from costs that would have been incurred in any event. Thus, if a patient is admitted to a hospital without enrollment in a clinical trial having been decided in advance, and once admitted is enrolled in a surgical trial, Medicare will pay for the admission as if the patient had been admitted for nonsurgical treatment. On the other hand, if the admission was for the sole purpose of enrolling the patient in a clinical trial, Medicare will deny payment of the admission in its entirety.
A similar distinction is drawn with respect to treatment of adverse effects from a clinical trial. Even though Medicare will not pay for treatment costs in a clinical trial itself, if the patient subsequently develops a complication requiring treatment, Medicare will fully cover the costs of the subsequent treatment, even though the need for treatment can be traced directly to treatment in a noncovered clinical trial.
There are several types of costs that are incurred in clinical trials:
The cost of the investigational drug, device, or procedure.
The cost of collection and analysis of trial data.
The costs of patient care, including hospital, physician, laboratory, and other services.
Each of these categories is treated somewhat differently under the current system.
FDA regulations generally prohibit manufacturers from charging for investigational drugs, and manufacturers therefore supply free drugs to investigators conducting clinical trials. FDA rules are more liberal in allowing charges for investigational devices, however, with the result that patients (or some other party) may incur the cost of the device used in a clinical trial.
Data collection and analysis are ordinarily paid for by the sponsor of the clinical trial. For example, funds may come from a pharmaceutical or medical device manufacturer or from the National Institutes of Health.
Patient care costs are generally the responsibility of the patient, or are borne by the institution at which the clinical trial is conducted. Because these costs are the outgrowth of an investigational service, insurers typically will not cover them. This is the case even though the patient might have incurred substantially similar costs if the patient had undergone standard therapy instead of being enrolled in the clinical trial.
Although public debate about coverage of clinical trials sometimes suggests that the focus of interest is coverage of the investigational procedure itself, in reality the principal source of costs is frequently patient care, most or all of which might have been necessary even if the patient had undergone standard therapy. High-cost experimental procedures, such as bone marrow transplants dominate the discussion, but almost certainly do not accurately reflect the added costs of clinical trials in general.