[Federal Register Volume 61, Number 103 (Tuesday, May 28, 1996)]
[Notices]
[Pages 26519-26520]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 96-13195]



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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Agency for Health Care Policy and Research


Nominations of Outcomes and Effectiveness Research Priority 
Topics

    The Agency for Health Care Policy and Research (AHCPR) is inviting 
suggestions for priority topics for research related to prevention, 
diagnosis, treatment and/or management of common diseases and clinical 
conditions. These suggestions will be considered in AHCPR's plans for 
future research on the outcomes and effectiveness of health care 
services. The process AHCPR will employ in establishing priorities and 
selecting topics for outcomes/effectiveness research is described 
below.

Background

    AHCPR is charged under Title IX of the Public Health Service Act 
(42 U.S.C. 299-299c-6) with enhancing the quality, appropriateness, and 
effectiveness of health care services and access to such services. 
AHCPR accomplishes these goals through scientific research that 
promotes improvements in clinical practice (including the prevention of 
diseases and other health conditions) and improvements in the 
organization, financing, and delivery of health care services. Section 
1142 of the Social Security Act (42 U.S.C. 1320b-12) enhances and 
elaborates on AHCPR's program of outcomes and effectiveness research. 
Outcomes of care and effectiveness research constitutes a major portion 
of AHCPR's health services research agenda.

Outcomes and Effectiveness Research Program

    The outcomes and effectiveness research program grew out of 
awareness of significant unexplained variations in clinical (medical, 
nursing, and allied health) practice and the inadequacy of scientific 
evidence to support many common treatments and procedures. Outcomes and 
effectiveness research encompasses three main areas of emphasis for the 
prevention, diagnosis, treatment, and management of illness: (1) 
Determination of the clinical interventions that are most effective, 
cost effective, and appropriate; (2) development of methods and data to 
advance effectiveness research; and (3) dissemination and evaluation of 
the impact of research findings on clinical practice and outcomes. 
Other distinctive characteristics of outcomes and effectiveness 
research include its multidisciplinary nature; use of a variety of 
research designs (e.g., observational studies, prospective trails, 
database studies) and analytical methods (e.g., decision analysis, 
utility analysis, and cost-effectiveness analysis); incorporation of 
both objective and subjective measures of outcomes; and emphasis on 
policy relevance.
    To date, AHCPR's outcomes and effectiveness research has focused on 
conditions that meet the following criteria:
     High incidence or prevalence in the general population or 
in major population subgroups, as defined by age, gender, or ethnicity;
     Controversy or uncertainty about the effectiveness and 
relative effectiveness of available clinical strategies;
     High cost, whether due to the number of people needing 
care, high unit cost of care, or high indirect costs;
     Needs, of the Medicare and Medicaid programs; and
     Data available, or readily developed.
    In addition, all outcomes and effectiveness research is expected to 
be:
     Generalizable: Outcomes and effectiveness research is 
concerned with the outcomes that can be expected in typical patients, 
receiving care in typical clinical situations, not with outcomes that 
can be achieved only in selected patients and in controlled clinical 
situations. Thus, critical features of all outcomes and effectiveness 
research projects are that the questions have broad applicability and 
the research designs support wide generalizability of findings.
     Pragmatic: Outcomes and effectiveness research projects 
address questions that have high clinical and policy significance and 
are designed with attention to the eventual implementation of findings. 
They strengthen the science base in ways that can directly contribute 
to improved patient outcomes and decisionmanking processes (including 
practice guidelines), and to a more equitable and cost-effective health 
care system. The usefulness of outcomes and effectiveness research 
stems, in part, from the requirement that the clinical problems and 
practices addressed are common and costly, and from attention to the 
realities of clinical practice.
     Patient-Centered: Outcomes and effectiveness research 
evaluates health care in terms of outcomes that emphasize the patient's 
experience and perspective. In addition to survival, morbidity, and 
complications, outcomes and effectiveness studies consider patient-
reported symptom relief, functional capacity, quality of life, 
satisfaction with care, and economic burden. Demographic, social and 
cultural characteristics, as well as personal preferences, are 
important independent variables.
     Multidisciplinary: Outcomes and effectiveness research 
requires theoretical and practical understanding of a wide range of 
clinical and non-clinical variables that determine the structure, 
processes, and outcomes of health care. Studies typically involve teams 
of researchers who bring together the knowledge and methodological 
expertise of both the clinical and social sciences, plus understanding 
of the perspectives of patients, providers, and policymakers.
    Since 1989, AHCPR has supported significant advances in medical 
effectiveness research, especially through the set of special projects 
known as Patient Outcomes Research Teams (PORTs). PORTs are large-
scale, 5-year studies designed to determine ``what works best'' in 
clinical treatment for common diseases and conditions. PORTs have 
succeeded in (1) documenting the scientific basis for many common 
clinical practices, (2) demonstrating the relative benefits of 
different interventions, and (3) identifying areas for further 
research. The following clinical conditions addressed by the AHCPR PORT 
program meet the criteria of being common, costly, and feasible to 
study:

 Acute Myocardial Infarction
 Ischemic Heart Disease
 Low Back Pain
 Total Knee Replacement
 Hip Fracture and Osteoarthritis
 Low Birth Weight Prevention
 Cataract
 Community-Acquired Pneumonia
 Childbirth
 Schizophrenia
 Stroke Prevention
 Type II Diabetes
 Biliary Tract Disease
 Prostrate Disease

    In July 1993, AHCPR introduced a new generation of PORT research, 
known as PORT II. A program announcement inviting applications for PORT 
IIs was published in the May 13, 1994 ``NIH Guide for Grant and

[[Page 26520]]

Contracts,'' Vol. 23, No. 18. Like the original PORTs, PORT IIs are 
pragmatic, methodologically sophisticated, multidisciplinary projects 
that focus on patient outcomes for common clinical problems. They 
differ from the original PORTs by their individualized research 
strategies and are also distinguished by their expected impact on 
clinical practice, patient outcomes, and health care policy. PORT IIs 
focus on the establishment of direct linkages between practice and 
outcomes and on research methods that facilitate direct comparisons of 
two or more distinct clinical strategies. Clinical conditions addressed 
to date by the AHCPR PORT II program include:

 Localized Breast Cancer
 Cardiac Arrhythmia
 End-stage Renal Disease
 Depression
 Prostate Disease
 Infant Dehydration
 Cataract: Preoperative Testing
 Pelvic Inflammatory Disease

    In addition to PORTs and PORT IIs, AHCPR has funded approximately 
130 other outcomes and effectiveness research clinical studies. For 
clinical subjects as diverse as AIDS, dental disease, emergency 
medicine, and cancer, these studies document patterns of practice, 
describe the natural history of diseases, synthesize the evidence for 
various clinical strategies, or answer relatively discrete 
effectiveness questions. Major ongoing program areas focus on 
pharmaceutical therapy, minority health, and primary care.

AHCPR Process for Determining Priority Topics

    Topic selection for the original PORT projects was guided by work 
of the Institute of Medicine (IOM) which was described in the 1990 IOM 
publication entitled ``National Priorities for the Assessment of 
Clinical Conditions and Medical Technologies.'' A new process to 
identify priorities for future outcomes research was discussed at a 
November, 1995 expert panel meeting. During this meeting, the AHCPR 
conferred with health services and effectiveness experts, representing 
multiple disciplines, specialties, and institutions. Alternative 
approaches for prioritizing topic areas and identification of 
populations whose major health conditions have not yet been adequately 
addressed (e.g., young children, the very elderly, women, and ethnic 
minorities) were considered.
    Based on the IOM work and expert discussions, AHCPR has initiated a 
three stage process for identifying topics:
    1. Develop a preliminary list of priority topics and reasons for 
importance, representing the views of health care providers, insurers, 
medical and health specialty societies, consumers, and the general 
public;
    2. Convene an expert panel to review and assess the preliminary 
research priorities and suggested criteria; and
    3. Identify which topic areas can be most appropriately addressed 
using outcomes and effectiveness research methods.
    This Notice initiates the first step, that is, a solicitation of 
topics from health care providers, insurers, health-related societies, 
consumers, and the public. Written suggestions for research topics that 
fit within the parameters of AHCPR's outcomes and effectiveness 
research program are invited.
    For each suggestion, the nominee should provide a clear rationale 
and supporting evidence for the topic's importance and clinical 
relevance. Responses should be submitted by July 29, 1996 to: Carolyn 
Clancy, M.D., Acting Director, Center for Outcomes and Effectiveness 
Research, Agency for Health Care Policy and Research, Suite 605, 2101 
East Jefferson Street, Rockville, Maryland 20852. All responses will be 
available for public inspection at the Center for Outcomes and 
Effectiveness Research, Telephone (301) 594-1485, weekdays between 8:30 
a.m. and 5 p.m. The AHCPR will not reply to individual responses, but 
will consider all submissions in developing the research priorities.
    For further information on the outcomes and effectiveness research 
program, contact: Carolyn Clancy, M.D., Acting Director, Center for 
Outcomes and Effectiveness Research, Agency for Health Care Policy and 
Research, Suite 605, 2101 East Jefferson Street, Rockville, Maryland 
20852; Telephone (301) 594-1485.

    Dated: May 16, 1996.
Clifton R. Gaus,
Administrator.
[FR Doc. 96-13195 Filed 5-24-96; 8:45 am]
BILLING CODE 4160-90-M