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

301-435-0714; 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.



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