MEDICAL TREATMENT EFFECTIVENESS RESEARCH -- PORT-II NIH GUIDE, Volume 23, Number 18, May 13, 1994 PA NUMBER: PA-94-066 P.T. 34 Keywords: Health Services Delivery Agency for Health Care Policy and Research PURPOSE The Agency for Health Care Policy and Research (AHCPR) invites applications for innovative and timely research that will provide convincing evidence for or against the effectiveness and/or cost effectiveness of alternative clinical interventions used to prevent, diagnose, treat, and manage common clinical conditions. "PORT-IIs" will extend the work of AHCPR's original Patient Outcomes Research Teams (PORTs) into more clinical areas and will make substantial new contributions to improved patient outcomes, clinical practice, and health care policy. Awards will be part of the new generation of research developed by AHCPR for the Medical Treatment Effectiveness Program (MEDTEP), as introduced in Request for Applications (RFA) HS-94-002. HEALTHY PEOPLE 2000 The Public Health Service (PHS) is committed to achieving the health promotion and disease prevention objectives of "Healthy People 2000," a PHS-led national activity for setting priority areas. The AHCPR urges applicants to submit grant applications with relevance to the specific objectives of this initiative. Potential applicants may obtain a copy of "Healthy People 2000" (Full Report: Stock No. 017-001-00474-0 or Summary Report: Stock No. 017-001-004374-1) through the Superintendent of Documents, Government Printing Office, Washington, DC 20402-9325 (telephone 202-783-3238). ELIGIBILITY REQUIREMENTS Applications may be submitted by domestic and foreign non-profit organizations, public and private, including universities, clinics, units of State and local governments, non-profit firms, and non-profit foundations. Applications from minority and women investigators are encouraged. MECHANISM OF SUPPORT This program announcement (PA) uses the research project grant (R01) mechanism. Responsibility for the planning, direction, and execution of the proposed project will be solely that of the applicant. The total requested project period may not exceed five years. Annual progress reviews by AHCPR and the availability of funds will determine the continuation of grants up to the five year limit. RESEARCH OBJECTIVES Background and Conceptual Framework Since 1989, AHCPR has made a major investment and major advances in medical effectiveness research, especially through the set of special projects known as PORTs. This PA describes a new generation of PORT research, introduced in July 1993 with RFA HS-94-002. Like the original PORTs, PORT-IIs are pragmatic, methodologically sophisticated, multidisciplinary projects that focus on patient outcomes for common clinical problems and emphasize the policy significance of understanding what health care services and procedures are most effective and for whom. PORT-IIs are expected to start with careful formulations of important research questions about the effectiveness and relative effectiveness of different clinical approaches to common conditions. The proposed research strategy must be tailored to the research question(s) and the population at risk; unlike the original PORT projects, PORT-IIs do not have a common research plan. PORT-IIs focus on the establishment of direct linkages between clinical practice and outcomes, and on research methods and data that facilitate direct comparisons of different clinical strategies available for use in routine practice. PORT-IIs do not test or evaluate the application of clinical practice guidelines or "appropriateness criteria;" rather, they are designed to obtain empirical evidence useful for constructing, specifying, and updating such guidelines. PORT-IIs incorporate fundamental concepts of all MEDTEP research, as defined below. "Effectiveness" refers to the outcomes experienced by, or observed in, typical patients receiving care in typical clinical situations. The intent of this emphasis is to ensure that the findings of all PORT-IIs can be widely generalized. "Patient outcomes" are the terms in which effectiveness and cost effectiveness are assessed. Outcomes of interest emphasize the patient's perspective. They include survival, symptom relief, patient-reported quality of life, functional status, satisfaction with care, and costs. In PORT-IIs, analysis of a broad set of outcomes is generally desirable. It is essential that the analysis include the most significant outcomes for the particular condition under study, both long- and short-term. "Cost effectiveness" is the comparison of the direct monetary costs of health care plus the indirect costs (e.g., lost work) associated with the outcomes of the intervention. PORT-IIs should address questions of cost effectiveness if or when the interventions to be compared are likely to be associated with significant differences in cost or outcome. In analyzing effectiveness and cost effectiveness, PORT-IIs take into account the many important clinical and non-clinical variables that influence practice and outcomes. This includes pertinent characteristics of patients (e.g., comorbidities; medical history; demographic, socioeconomic, and cultural characteristics; preferences and utilities); providers (e.g., training, skill, practice style); diseases (e.g., severity, course); and the health care system (e.g., setting, type of practice, liability issues). Topic Selection PORT-II studies will be condition-specific or technology-specific, and will conduct comparative analyses of the most important alternatives for prevention, diagnosis, treatment, and/or management. A well-defined disease (e.g., breast cancer, pediatric asthma) or a symptom or condition (e.g., headache, fatigue, obesity) may be selected. The topic must meet all of the following MEDTEP criteria: o high incidence or prevalence in the general population or in major population subgroups, as defined by age, gender, or ethnicity; o controversy or open questions over the effectiveness and relative effectiveness of available clinical strategies; and o high cost whether due to the number of people needing care, high unit cost of care, or high indirect cost. Applicants are expected to present a strong case for their selected topic in a critical literature review that: addresses the clinical, policy, and research significance of the topic; provides evidence of controversy or information gaps regarding current clinical strategies; and supports the formulation of the proposed research question(s). The formulation of the problem must reflect understanding of the issues regarding clinical decisionmaking and the translation of study findings into clinical practice. Further, the research questions must be answerable within the proposed grant period. PORT-IIs are expected to compare distinctly different clinical approaches to the prevention, diagnosis, treatment, or management (including rehabilitation) of common clinical conditions. Examples of responsive studies include comparisons of: medical vs. surgical treatment; treatment vs. watchful waiting; psychotherapy vs. pharmacotherapy; or invasive vs. non-invasive screening technologies. Other possible comparisons include care prescribed or provided by different kinds of health care professionals, or in different care settings. If it is not feasible to address all important treatment options in a single study, applicants must identify the specific interventions the study will address and justify the selections and exclusions. In general, the most comprehensive assessments of pertinent clinical strategies will be of greatest interest for PORT-IIs. Methods Investigators are encouraged to design new research strategies, use new combinations of methods, or tailor existing methods to their research questions so that convincing evidence will be obtained for or against the effectiveness of alternative clinical interventions. PORT-IIs may employ experimental, quasi-experimental, or observational designs; methods include, but are not limited to, case-control studies, cohort studies, clinical trials, meta-analyses, cost effectiveness analyses, decision modeling, and combinations of these methods. MEDTEP's emphasis on the generalizability of results precludes traditional randomized controlled trials (RCTs) whose findings of "efficacy" apply only to narrowly defined patient groups and circumstances. PORT-IIs do, however, include "effectiveness trials," designed to answer questions about the likely outcomes of health care in the "real world." Thus, randomized studies that include a broad range of patients and practitioners are encouraged. Types and sources of data may include clinical, patient-reported, and administrative data. The data may be obtained prospectively or retrospectively from registries or records of health care providers, or via new, established, or adapted surveys of patients and health care providers. Primary data will generally be required; however, these may be combined with, and occasionally replaced by, secondary data when the latter will provide adequate information and an efficient means to address the research questions. For example, administrative data, although they generally lack clinical detail, may be useful in identifying cases and controls, estimating costs, or measuring some outcomes. Applicants who propose to use Medicare or Medicaid data must specify the required data files and explore the availability and cost of obtaining these data with the Health Care Financing Administration (HCFA). The estimated cost must be presented along with documentation from HCFA, as part of the grant application. This cost should not be included in the total budget request for the project. For more information about data budgets, contact Ralph L. Sloat, AHCPR Grants Management Officer; at the address listed under INQUIRIES. The application must be explicit and detailed in justifying the proposed methods and data in terms of their potential for answering the research questions under study and the generalizability of results. Descriptions of the data collection and analysis plans, including strategies for case-finding, measuring outcomes, and comparing alternative treatments, must be specific. Adequate attention must be paid to relevant characteristics of: o the population at risk for the condition, o the condition, o the clinical interventions, o the outcomes, o the providers, o available data and measures, and o the sociocultural context of illness and health care. Project Organization To address the clinical and non-clinical dimensions of effectiveness research, PORT-IIs will require multidisciplinary research teams. The composition of the team and relative time commitments of each member should be well justified in terms of substantive knowledge, methodological expertise, and experience in conducting or managing related research projects. Each team should include at least one individual who is actively involved in patient care central to the study, and who contributes understanding of how and why clinical decisions are made in routine clinical practice. Applicants are encouraged to take full advantage of opportunities for efficient enhancements of available expertise, data, and other resources. This might include collaboration with researchers and practitioners outside applicants'/ grantees' own institutions, creative use of existing data, or "piggybacking" on other research activities. In addition, existing practice variations that are known to exist across health care settings, systems, or international borders can provide special opportunities for comparative analysis of outcomes. STUDY POPULATIONS INCLUSION OF WOMEN AND MINORITIES IN RESEARCH INVOLVING HUMAN SUBJECTS It is the policy of AHCPR that women and members of minority groups must be included in all AHCPR supported health services research projects involving human subjects, unless a clear and compelling rationale and justification are provided that inclusion is inappropriate with respect to the health of the subjects or the purpose of the research. A new NIH policy resulting from the NIH Revitalization Act of 1993 (Section 492B of Public Law 103-43) supersedes and strengthens NIH's previous policies (Concerning the Inclusion of Women in Study Populations, and Concerning the Inclusion of Minorities in Study Populations), which were in effect since 1990 and which AHCPR had adopted. The new NIH policy contains some provisions that are substantially different from the 1990 policies. AHCPR plans to publish guidelines specific to AHCPR. In the interim, AHCPR will follow the NIH guidelines, as applicable. All investigators proposing research involving human subjects should read the "NIH Guidelines for Inclusion of Women and Minorities as Subjects in Clinical Research," published in the Federal Register of March 9, 1994 (FR 59 11146-11151) and reprinted in the NIH Guide for Grants and Contracts, Volume 23, Number 11, March 18, 1994. Investigators also may obtain copies of the NIH policy from the AHCPR program staff listed under INQUIRIES. AHCPR program staff may also provide additional relevant information concerning this policy. APPLICATION PROCEDURES Applications are to be submitted on the grant application form PHS 398 (rev. 9/91), and will be accepted at the standard application deadlines as indicated in the application kit. State and local government agencies may use form PHS 5161 and follow those requirements for copy submission. Application kits are available at most institutional offices of sponsored research; from the Office of Grants Information, Division of Research Grants, National Institutes of Health, Westwood Building, Room 449, Bethesda, MD 20892, telephone 301-710-0267; and for AHCPR applications from Global Exchange Inc., 7910 Woodmont Ave Suite 400, Bethesda, MD 20814-3015, telephone 301-656-3100 (FAX 301-652-5264). The completed, signed, original application and five legible copies must be sent or delivered to: Division of Research Grants National Institutes of Health Westwood Building, Room 240 Bethesda, MD 20892** The Division of Research Grants (DRG) will not accept any application in response to this program announcement that is essentially the same as one currently pending initial review, unless the applicant withdraws the pending application. The DRG will not accept any application that is essentially the same as one already reviewed. This does not preclude the submission of substantial revisions of applications already reviewed, but such applications must include an introduction addressing the previous critique. REVIEW CONSIDERATIONS Upon receipt, applications will be reviewed for completeness. Incomplete applications will be returned to applicants without further consideration. General scientific review criteria are: significance and originality from a scientific and technical viewpoint; adequacy of the method(s); availability of data or adequacy of proposed plan to collect data; qualifications and experience of the principal investigator and proposed staff; adequacy of the plan for organizing and carrying out the project; reasonableness of the proposed budget; and adequacy of the facilities and resources available to the applicant. Each PORT-II application will be independently evaluated for scientific/technical merit in accordance with the general criteria stated above and the special scientific review criteria listed below, by an appropriate peer review group. Applications assigned to the AHCPR requesting direct costs, over the life of the project, exceeding $250,000 will be reviewed by AHCPR's National Advisory Council for Health Care Policy, Research, and Evaluation, as may applications requesting direct costs, over the life of the project, in excess of $50,000. Special Scientific Review Criteria The major scientific criterion for evaluating PORT-II applications assigned to the AHCPR is the potential for obtaining convincing, new evidence for the effectiveness or ineffectiveness of health care services and procedures. The topic must be compatible with the MEDTEP criteria listed under Topic Selection. Other special criteria are: o scientific importance and policy relevance of the clinical topic and the particular clinical interventions to be studied, as justified in a review of the literature; o generalizability of results; o feasibility of answering the proposed research question(s) within the project period; o attention to technical issues in case-definition, case-finding, data collection, and analysis; o quality and adequacy of the proposed data; o justification for focus on the outcomes specified; o adequacy of outcomes measures, including costs if applicable; o extent to which research design permits direct comparisons of treatment effectiveness and/or cost effectiveness; o evidence of understanding of the issues in clinical decisionmaking and the translation of research findings into clinical practice; o sensitivity to patient heterogeneity and individual preferences; o specification of useful findings or products and identification of constituency(ies) for these; o efficiency of the research plan; and o evidence of productive collaborations (e.g., with other institutions, appropriate professional groups, other sources of support). AWARD CRITERIA Applications will compete for available funds with all other investigator-initiated applications. In making funding decisions, AHCPR will consider: quality of the proposed project as determined by peer review, availability of funds, and program balance. The earliest anticipated date of award is nine months from the date of submission. INQUIRIES Those considering applying in response to this PA are strongly encouraged to discuss their project with AHCPR program administrators before formal submission. The AHCPR welcomes the opportunity to clarify any issues or questions from potential applicants. Direct inquiries regarding programmatic issues, including information on the policy of inclusion of women and minorities in study populations, to: Richard Greene, M.D., Ph.D. Center for Medical Effectiveness Research Agency for Health Care Policy and Research 2101 East Jefferson Street, Suite 605 Rockville, MD 20852 Telephone: (301) 594-1485 Direct inquiries regarding fiscal matters to: Ralph L. Sloat Grants Management Office Agency for Health Care Policy and Research 2101 East Jefferson Street, Suite 601 Rockville, MD 20852 Telephone: (301) 594-1447 AUTHORITY AND REGULATIONS This program is described in the Catalog of Federal Domestic Assistance No. 93.180. Awards are made under authorization of the Public Health Service Act, Title IX, and Section 1142 of the Social Security Act. Awards are administered under the PHS Grants Policy Statement and Federal Regulations 42 CFR Part 67, Subpart A, and 45 CFR Part 74 (45 CFR Part 92 for State and local governments). This program is not subject to the intergovernmental review requirements of Executive Order 12372. .
Return to NIH Guide Main Index
Office of Extramural Research (OER) |
National Institutes of Health (NIH) 9000 Rockville Pike Bethesda, Maryland 20892 |
Department of Health and Human Services (HHS) |
||||||||
Note: For help accessing PDF, RTF, MS Word, Excel, PowerPoint, Audio or Video files, see Help Downloading Files. |