Guest editors' introduction: what can large pragmatic clinical trials do for public mental health care?

Persons in positions of responsibility often need to make important decisions with inadequate information. Those involved in public mental health care are no exception. Promising new treatments often come with high price tags, and decision makers must use whatever information they have to weigh benefits against costs. Of late, this tension has become more acute, threatening to reach crisis proportions. With the costs for health care once again rising at a rate that exceeds inflation and Federal and State governments facing budget shortfalls, increasing pressure is being placed on Medicare, Medicaid, the Veterans Health Administration, and private third party payers to limit expenditures (Abelson 2002; Freudenheim 2002; Pear 2002fc, 2002c). A growing number of people are uninsured, and the insured are expected to have fewer options and to face higher out-of-pocket expenditures (Strunk et al. 2001; Abelson 2002; Pear 2002a). The revolution in biomedical research technology and the burgeoning knowledge base that this has produced, in concert with the prospect of large profits, have accelerated the pharmaceutical industry's rate of drug development (Berndt 2001). Consequently, an increasing number of drugs are approved by the U.S. Food and Drug Administration (FDA) and introduced into clinical use each year (Berndt 2001). Antipsychotic drugs are a case in point. After the first antipsychotic drug chlorpromazine was introduced in the United States in 1953, 11 agents were approved by the FDA and made available for clinical use through 1989. Chlorpromazine was the prototype of a class of drugs that were initially termed neuroleptics but have since become known by a variety of names, including typical, conventional, or first generation antipsychotic drugs. In 1989, clozapine, considered the prototype of a second generation of antipsychotic drugs called atypical antipsychotic drugs, was approved by the FDA. In the 12 years prior to the introduction of clozapine, there were no new drugs approved as antipsychotics in the United States, while in the 12 years since clozapine, there have been six though only five are currently marketed for clinical use The newer drugs, still under patent protection, are priced many times higher than the older drugs, for which there are often generic versions. Because the new medications are promoted as having superior efficacy and/or safety, they are often used preferentially, thus increasing the costs of drug acquisition. Prescription drugs are the fastest-growing component of recent rises in health care costs (Levit et al. 2003). A result of this phenomenon is the proliferation of cost containment mechanisms—including preferred drug lists, treatment algorithms, prior approval procedures, and higher copayments— employed by third party payers (Bemdt 2001). Despite their justification on economic grounds, such measures create concerns and conflicts among various stakeholders. Consumers and advocates want unrestricted access to the best available treatments, and they want to make sure that consumers of mental health care are not unfairly targeted by cost control efforts. Pharmaceutical companies want to be able to sell their products at market-based prices that will enable them to return a profit to their shareholders and fund their research and development programs. Mental health care clinicians want to be able to use the best treatments for their patients, and mental health care administrators want to provide high-quality care in a cost-effective manner. How is one to effect a mental health care policy that satisfies all of these competing interests? If new treatments are indeed more effective, then the difference in their cost may be partially offset by reductions in the need for other services that patients receiving the treatments will require, as well as increased productivity. Indeed, this is the claim of the pharmaceutical companies and proponents of the new medications. However, the evidence for such claims comes largely from studies conducted as part of the phase HI and IV development of the medications, along with optimistic extrapolations of these results. Objective and dispassionate analyses of the existing data provide neither a clear