MEDICAL TREATMENT EFFECTIVENESS RESEARCH - SUMMARY

NIH GUIDE, Volume 23, Number 22, June 10, 1994



PA NUMBER:  PA-94-074



P.T. 34



Keywords:

  0730021 

  Health Care Economics 



Agency for Health Care Policy and Research



PURPOSE



The Agency for Health Care Policy and Research (AHCPR) has ongoing

interest in research under the Medical Treatment Effectiveness

Program (MEDTEP).  This program announcement (PA) outlines the common

themes inherent in all MEDTEP projects and identifies major ongoing

areas of research.  MEDTEP research encompasses three main areas of

emphasis:  (1) determining what clinical interventions are most

effective, cost effective, and appropriate; (2) methods and data to

advance effectiveness research; and (3) dissemination and evaluation

of the impact of research findings on clinical practice and outcomes.

This PA serves as a general reference for other publications and

contacts regarding current MEDTEP research interests and award

mechanisms.



This Program Announcement (PA) supersedes the "Medical Treatment

Effectiveness Research" announcement published in the Federal

Register of August 14, 1990 (FR 55 33170-33172).



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.  Foreign applicants are advised to

contact the AHCPR Grants Management Officer regarding limitations and

special requirements (see INQUIRIES).



MECHANISM OF SUPPORT



The research project grant (R01) mechanism, which may provide support

for up to five years, is the principal mechanism of support for

MEDTEP research.  The small grant (R03) mechanism is available for

projects that do not exceed two years and $50,000 in direct costs for

the entire project period.  Responsibility for planning, direction,

and execution of the proposed project is solely that of the

applicant.



In addition, AHCPR may issue requests for applications (RFAs) and PAs

that announce new MEDTEP program interests and/or the availability of

other mechanisms of support for MEDTEP research.  For further

information on specific areas of MEDTEP research, contact Dr. Richard

Greene (see INQUIRIES); or Dr. Norman Weissman or Ms. Zucker as

indicated below.



RESEARCH OBJECTIVES



MEDTEP Research Themes



Medical effectiveness research is a major component of the health

services research agenda of AHCPR.  MEDTEP 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 practices and procedures.  MEDTEP projects assess the

relative effectiveness, cost effectiveness, and appropriateness of

available strategies for the prevention, diagnosis, treatment, and

management of illness, in terms of patient outcomes.  While MEDTEP

research projects vary in focus, size, scope, methods, and

complexity, all are expected to be:



Generalizable:  "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 only be

achieved in selected patients and in controlled clinical situations.

Thus, a critical feature of all MEDTEP projects is that the questions

have broad applicability and the research design supports wide

generalization of the findings.



Pragmatic:  MEDTEP projects address questions that have high clinical

and policy significance and are designed with attention to the

eventual implementation of findings.  They obtain empirical evidence

or strengthen the science base in ways that can directly contribute

to improved patient outcomes and decisionmaking processes (including

practice guidelines), and to a more equitable and cost-effective

health care system.  The usefulness of MEDTEP research stems, in

part, from MEDTEP's requirement that the clinical problems and

practices addressed are common and costly, and from attention to the

realities of clinical practice.



Patient-Centered:  MEDTEP research evaluates health care in terms of

outcomes that emphasize the patient's experience and perspectives.

In addition to survival, morbidity, and complications, MEDTEP 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:  MEDTEP 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 a team of researchers who

bring the knowledge and methodological expertise of both the clinical

and social sciences, plus understanding of the perspectives of

patients, providers, and policymakers.



Types of Studies



MEDTEP research encompasses three main areas of emphasis:  (1)

determining what clinical interventions are most effective, cost

effective, and appropriate; (2) methods and data to advance

effectiveness research; and (3) dissemination and evaluation of the

impact of research findings on clinical practice and outcomes.



1.  Clinical Studies



All MEDTEP clinical studies address the basic MEDTEP themes described

above to obtain evidence for or against the effectiveness, cost

effectiveness, and appropriateness of available interventions.  Most

focus on a particular disease or clinical condition and assess the

outcomes associated with different interventions that are available

for its prevention, diagnosis, treatment, and/or management.  Some

MEDTEP clinical studies focus on established technologies or

procedures.  Of interest are conditions or procedures that are common

and costly, either in the general population or in a major

subpopulation.  Major categories of MEDTEP clinical studies are

described below.



Patient Outcomes Research Teams (PORTs) and PORT-IIs. Between 1989

and 1992, AHCPR awarded fourteen special MEDTEP projects known as

Patient Outcomes Research Teams (PORTs). PORTs are distinguished from

other MEDTEP clinical studies by their broad scope, multi-method

approach to patient outcomes questions, and by the standard five year

model they follow.  AHCPR does not anticipate award of additional

projects that use the PORT model.



In July 1993, AHCPR issued an RFA initiating a new generation of

MEDTEP research, called "PORT-II," with the first awards to be made

this year.  An ongoing PA, "Medical Treatment Effectiveness Research

-- PORT-II," was published in the NIH Guide for Grants and Contracts,

Volume 23, Number 18, May 13, 1994.  PORT-IIs continue the PORT

tradition by addressing important clinical questions and breaking new

methodological ground.  They are distinguished from the original

PORTs by their individualized research strategies and from other

MEDTEP clinical projects by the expected direct impact of the

empirical evidence they obtain, on clinical practice, patient

outcomes, and health care policy.



PORT-IIs are not feasible or desirable in all clinical areas.  There

must be sufficient existing information to permit the formulation of

effectiveness questions and design of a research strategy tailored to

the clinical problem and the population at risk, so that convincing

evidence of optimal patient care can be realistically expected within

the project period.  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, e.g., medical vs. surgical treatment.  For information on

MEDTEP clinical studies, including PORT-IIs, contact the Center for

Medical Effectiveness Research (CMER), see INQUIRIES.



Other MEDTEP Clinical Studies.  The majority of MEDTEP clinical

studies are designed to build the science base in areas where a

PORT-II is not currently feasible or desirable.  This includes

research designed to 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's program of studies on pharmaceutical therapy, established in

1992, focuses on the effectiveness and cost effectiveness of

available pharmaceutical interventions, especially the relationships

among drug therapy, other pharmaceutical services, and patient

outcomes.  Studies address preventive, acute, or chronic treatment in

inpatient, ambulatory, or long-term care settings.  For further

guidance, contact program staff listed under INQUIRIES.



In 1991, AHCPR established a research program focused on the

effectiveness of current clinical practice for health conditions of

special significance among racial and ethnic minorities.  This

program is highlighted by the eleven MEDTEP Research Centers on

Minority Populations, which train minority investigators who are new

to outcomes research to develop and conduct effectiveness research.

Although additional MEDTEP Minority Centers are not anticipated,

AHCPR has continuing interest in studies of the effectiveness of care

related to special problems in minority populations.  For

information, contact CMER program staff or the Associate

Administrator for Minority Health, Dr. Morgan Jackson, telephone

301-594-6665.



AHCPR's program of primary care research includes, but is not limited

to, effectiveness research topics.  MEDTEP primary care studies focus

on the effectiveness and cost effectiveness of care for conditions

that are often undifferentiated, as they present in unselected or

nonreferred populations, in primary care settings, and the role of

primary care physicians in enhancing the effectiveness and cost

effectiveness of care.  For information, contact the Center for

General Health Services Extramural Research (CGHSER), Dr. Norman

Weissman, Director, telephone 301-594-1349, ext. 109.; or CGHSER,

Division of Primary Care, Dr. Carolyn Clancy, Director, telephone

301-594-1357, ext. 137.



2.  Methodological Studies



Effectiveness research frequently requires new kinds of data and

analysis, and new applications of existing tools. MEDTEP supports

projects that aim to strengthen or define the limits of existing data

and develop or test measurement and data collection instruments, and

analytic methods useful for outcomes research.  For example, MEDTEP

projects may develop new outcomes measures, methods for linking or

enhancing existing databases, methods to assess patient preferences,

or methods for cross-cultural or international comparisons of patient

outcomes.  Methodological work may be the main focus of a project or

may be embedded in a larger project.  For information on these

studies, contact CMER program staff or CGHSER, Dr. Norman Weissman

(see above).



3.  Dissemination and Evaluation Studies



Some MEDTEP projects focus on approaches or technologies for

achieving optimal dissemination and integration of new knowledge into

practice.  This includes research, demonstrations, and evaluations

that examine issues of diffusion, awareness, acceptance, and adoption

of research findings and clinical practice guidelines by health care

providers and consumers.  For example, projects may examine the role

of opinion leaders and practitioner study groups in influencing

practice, or various approaches to enhancing patient participation in

health care decisions.  MEDTEP also supports studies to develop and

evaluate clinical practice guidelines, information systems, and

clinical evaluation tools designed to help practitioners and

consumers make better health care decisions.  Contact the Center for

Research Dissemination and Liaison, Ms. Phyllis Zucker, Director,

telephone 301-594-1360, regarding dissemination studies.  Contact

CGHSER, Dr. Norman Weissman, regarding evaluation studies (see

above).



Research Methods



MEDTEP studies draw on a wide range of research methods, especially

those used in the clinical, evaluative, and social sciences.  The

research design may be experimental, quasi-experimental,

observational, or a combination of designs.  Any appropriate type(s)

of statistical analysis, modeling, or synthesis may be proposed.

Types and sources of data may include:  new, established, or adapted

surveys of patients or providers; clinical data obtained

prospectively, or from clinical registries, practice-based networks,

or other health care providers; administrative data maintained by

providers, insurers, or institutions; and published research

findings.  Laboratory-based studies are not appropriate.



Applications must be explicit and detailed in describing data,

methods, and tools for data collection and analysis.  The research

plan must be justified in terms of potential for answering the

research questions under study.



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 Mr. Ralph L. Sloat,

AHCPR Grants Management Officer (see INQUIRIES).



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 accompanying

requirements for submission.)  Application kits are available at most

institutional offices of sponsored research; from the Office of Grant

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 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 by the

referral office, DRG.  Incomplete applications will be returned to

applicants without further consideration.



General review criteria for all grant applications are:  significance

and originality from a scientific and technical viewpoint; adequacy

of the method(s); availability of data or adequacy of plan to collect

required data; qualifications and experience of the principal

investigator and proposed staff; adequacy of the plan for organizing

and managing the project; reasonableness of the proposed budget; and

adequacy of the facilities and resources available to the applicant.



An appropriate peer review group will evaluate applications for

scientific/technical merit in accordance with the general criteria

stated above, and any special review criteria applicable to an

individual mechanism or as listed in specific announcements.

Applications assigned to the AHCPR and requesting total direct costs

in excess of $250,000 will be reviewed by AHCPR's National Advisory

Council for Health Care Policy, Research, and Evaluation, as may

applications requesting total direct cost in excess of $50,000.



Special Review Criteria



Applicants are advised to refer to individual announcements and to

contact the staff offices listed below regarding special review

criteria.



AWARD CRITERIA



In making funding decisions, quality of the proposed project as

determined by peer review, program balance, and availability of funds

will be considered.



INQUIRIES



Those considering applying in response to this PA are strongly

encouraged to discuss their project with AHCPR program

administrators; 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, including budget

justification for HCFA data, to:



Ralph L. Sloat

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 and 93.226.  Awards are made under

authorization of the Public Health Service Act, Title IX (42 U.S.C.

299-299c-6 and Section 1142 of the Social Security Act (42

U.S.C.1320b-12).  Awards are administered under the PHS Grants Policy

Statement; and 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.



.

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