NIH Clinical Trials

Nearly all the 26 private health insurers, the large indemnity plans, and the several health maintenance organizations that GAO interviewed approve case-by-case exceptions to the general exclusion of coverage for patients' participation in experimental clinical trials for the sake of finding effective treatments. Insurers generally agree in approved cases to pay for standard, nonexperimental care costs associated with a trial, but payments vary from insurer to insurer. Uncertainty about approval and payment levels results in patients and physicians being discouraged from seeking prior approval for trial enrollment, although insurers may pay for medical care that they are unaware is being provided in a research context. Cancer research centers reported no serious difficulties in enrolling adequate numbers of patients for trials sponsored by the National Cancer Institute but described enrollment as challenging because of administrative burdens, payment issues, and paperwork requirements. Other difficulties are community physicians' being unaware of trials and patients' being ineligible or uncertain about the benefits and risks of experimental treatments. Available data do not document the concern expressed anecdotally by the National Institutes of Health that trial enrollment is declining.

GAO noted that: (1) despite policies that generally exclude coverage for clinical trials, nearly all the insurers that GAO interviewed allow for exceptions following case-by-case reviews by the insurers' medical personnel; (2) if coverage is approved, insurers generally agree to pay the standard, nonexperimental care costs associated with a trial; but because there is little agreement on which trial services constitute standard care, payments can vary from insurer to insurer; (3) given the uncertainty about approval and payment levels, patients and physicians can be discouraged from seeking prior approval from insurers; (4) most cancer research centers GAO contacted said that they did not experience what they considered to be serious difficulties enrolling adequate numbers of patients for NCI-sponsored clinical trials; (5) but all the centers described clinical trials enrollment as challenging because of the significant administrative burden incurred in dealing with health insurers about trial coverage and payment issues; (6) paperwork requirements can be labor-intensive and time-consuming when staff physicians and nurses must document the necessity of enrolling each patient and negotiate the specific services and amounts to be paid as standard care; (7) center representatives also cited an array of physican- and patient-related factors that affect the availability of patients for NIH-sponsored clinical trials; (8) NIH has expressed concern that trial enrollment is declining, but the data provided to GAO by several of the largest institutes did not document the basis for NIH's concerns; (9) patient enrollment in the NIH-sponsored clinical trials for which GAO could obtain data appeared to be meeting the goals of those trials; (10) in 1998, NIH officials reported to the Office of Management and Budget that patient participation in trials was a substantial problem, particularly for cancer trials; (11) they cited 1996 testimony from clinical investigators that managed care seemed to have affected patient participation in cancer clinical trials; (12) beyond such anecdotal information, however, NIH does not have quantitative data that indicate that patient enrollment has slowed or that trials have been delayed or prematurely closed because of patient enrollment problems; and (13) NCI is in the process of converting its existing clinical trial reporting systems to a new consolidated electronic system, which should support improved trial monitoring.