Outcomes of Pharmaceutical Outcomes Research: Impact of Studies Funded
In a report entitled The Outcome of Outcomes Research at AHCPR, Tunis and Stryer1 defined the types of impact that outcomes research can have on further research, on policymaking, on clinical practice, and on patient outcomes. While recognizing that it is important to fund research that is likely to have an impact in these areas, the authors also stressed the significant challenges faced in connecting specific research efforts with their effects. The process of change in program, policy, and clinical practice is complex and subject to many external influences. Thus, it is difficult to trace changes in health care practice and outcomes to a particular study or set of studies that may have contributed to them.
The present report is part of a project that provides research and editorial support to assess the impact of 18 pharmaceutical research studies funded during the 1990s by the Center for Outcomes and Effectiveness Research (COER) at the Agency for Healthcare Research and Quality (AHRQ). These research studies varied in scope but addressed the following key areas of interest:
- Data and analytic methods involved in the study of drug therapy effectiveness.
- Factors affecting the appropriateness of drug prescribing.
- The role of the patient in drug therapy effectiveness.
- Economic analysis and the effects of changes in the health care environment.
The first part of the Outcomes of Pharmaceutical Outcomes Research project involved development of an updated list of publications generated by these research projects, entitled Outcomes of Pharmaceutical Therapy (OPT) Program Update.2 A subsequent phase of work involved analysis of these publications in terms of their contribution to knowledge about health care outcomes, their policy relevance, and their levels of impact as defined by Tunis and Stryer1. In addition, some Principal Investigators (PIs) were contacted for the purpose of learning about examples of project impacts that may not be reflected in published literature; 10 interviews were conducted with Principal Investigators and with users of research results. Analysis for several projects is still underway, and additional publications will be forthcoming, along with findings and related impacts on other research, policy, practice and outcomes.
In reviewing the report it is important to understand how Tunis and Stryer1 define impact levels. Their definition reflects the process by which basic findings in outcomes and effectiveness research are linked over time in increasingly concrete impacts on the health of patients as illustrated below:
Levels of Impact Resulting from Outcomes Studies1
Level I—Impact on Further Research. This level includes effects of research studies that do not represent a direct change in policy or practice. This includes new tools and methods for research, instruments and techniques to assist clinical decisionmaking and studies that identify areas in which scientific knowledge is absent but needed. Level I impacts are also provided when studies describe findings that are inconsistent with current clinical paradigms and stimulate rethinking and questioning within a clinical specialty.
Level II—Impact on Policies. A policy or program is created as a direct result of the research, e.g. the information is used by health plans, public programs such as Medicaid, professional organizations, legislative bodies, regulators, and/or accrediting organizations.
Level III—Impact on Clinical Practice. The research results in a change in what clinicians or patients do, or in changes in a pattern of care. These changes may be demonstrated in a limited study population as a result of a specific intervention, or they may be trends identified outside a formal research context.
Level IV—Impact on Health Outcomes. This includes actual impact on health outcomes including those that are clinical, economic, related to quality of life, or related to satisfaction. They may be demonstrated in a limited study population as a result of a specific intervention, or beyond and outside of a formal research context.
The following section of this report summarizes the types of impact demonstrated by the 18 COER-funded studies. In addition, this section illustrates the degree to which these studies address:
- Current important policy issues.
- Key target populations of the Department of Health and Human Services (HHS).
- Diseases and conditions that are important to the Nation's health.
In addition to an overview of each project, specific findings are presented from one or more studies or research publications within the project that are relevant to further research or that have had an impact on policies, clinical care, or outcomes.
Summary of Studies' Impact and Relevance in Today's Policy Environment
Impact on Research
The research projects funded by COER reflect the breadth and depth of pharmaceutical-related issues faced in health care delivery in the United States today. Some focus on the role of the pharmacist, some on the use of information technology, and others on prescribing patterns among physicians. Yet others look at the role of patients in managing drug regimens and how to effectively elicit information from patients to improve treatment and outcomes.
Taken together, these projects have generated a significant impact on the research base that is necessary to address the complex and evolving range of questions related to drug use and drug policy. As Table 1 shows, these projects have contributed important primary research (Level I) in a number of key areas, including:
- Treatment effectiveness.
- Cost and economics of health care.
- Tools for patient management.
- Research tools and translating research for clinical care.
- Special needs of target populations.
- Public health and prevention, and chronic and persistent disease.
Impact on Policies, Clinical Practice, and Health Care Outcomes
Eight of the projects have demonstrated direct influence on the policies (Level II) of one or more organizations, including managed care organizations, State Medicaid programs, clinical associations, quality accreditation commissions, private insurance programs, integrated delivery systems and/or Federal agencies. Further, despite the challenges that Tunis and Stryer1 cite about linking research to impact Levels III (clinical practice) and IV (health care outcomes), at least four funded studies have demonstrated effects at one or both of these levels. These projects are identified in Table 2.
Focus on Target Populations and Key Diseases and Conditions
It is also important to note that taken together these projects address a range of population groups that are important because they represent:
- Key public financing programs (e.g., the elderly for Medicare, the poor for Medicaid, and children for SCHIP [State Children's Health Insurance Program]).
- Racial or ethnic subgroups of the population that are of significance because of health care and other disparities (e.g., African Americans, Hispanics, Native Americans).
Eleven projects address issues related to key populations, as presented in Table 3.
Most of the projects also focus on diseases and/or conditions of significant public health interest, including those that are highly prevalent, chronic and/or costly (Table 4), such as HIV/AIDS, asthma, and heart disease.
Relevance to Current Policy Issues
Another illustration of the relevance of this body of work can be seen by comparing the issues it addresses to topics of current health policy interest. The rise in health care premiums and costs, and particularly pharmacy costs, and the current debate about a Medicare prescription drug benefit are among the top issues in U.S. domestic policy today.
Representative of this policy focus was a high-profile meeting convened by the Office of the Assistant Secretary for Planning and Evaluation (OASPE) on Pharmaceutical Pricing Practices, Utilization and Costs on August 8-9, 2000, in Washington, DC. Conference background materials and presentations addressed topics ranging from drug effectiveness and outcomes, to drug use by key target populations, to pricing and cost. Comparing the themes and issues raised in the conference with the research funded by COER reveals the ongoing relevance of much of the research to critical questions that we continue to face or that have become increasingly important. Table 5 illustrates key issues raised during the conference and identifies the COER-funded projects that address those issues.