Full Text HS-96-001

RESEARCH ON THE OUTCOMES OF PHARMACEUTICAL THERAPY

NIH GUIDE, Volume 24, Number 1, January 13, 1995

RFA:  HS-96-001

P.T. 34

Keywords: 
  Chemotherapy 
  0730021 


Agency for Health Care Policy and Research

Letter of Intent Receipt Date:  March 1, 1995
Application Receipt Date:  April 12, 1995

PURPOSE

The Agency for Health Care Policy and Research (AHCPR) invites
applications to conduct research on the outcomes of pharmaceutical
therapy.  This request for applications (RFA) focuses on the effects
on patient outcomes of various proposed or existing mechanisms for
managing selection, utilization, and cost of pharmaceutical therapies
and services.  Current restructuring within the health care system
provides an important opportunity to enhance understanding of the
interrelationships of pharmaceutical treatments and services with
patient outcomes.  Of particular interest are studies involving the
comparative effectiveness and/or cost effectiveness of pharmaceutical
therapies within the changing health care system.

This is an activity of the research component of AHCPR's Medical
Treatment Effectiveness Program (MEDTEP).  Section 1142 of the Social
Security Act, a part of AHCPR's authorizing legislation, refers
specifically to the need to study the outcomes of prescription drug
therapy.  Awards are a part of the Outcomes of Pharmaceutical Therapy
(OPT) program, as introduced in RFA HS-92-03.

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.  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. 017-001-00473-1)
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.  Racial/ethnic minority individuals, women, and
persons with disabilities are encouraged to apply as Principal
Investigators.

MECHANISM OF SUPPORT

This request for applications will use the research project grant
(R01) mechanism.  Responsibility for the planning, direction, and
execution of the proposed project will be solely that of the
applicant.  This is a one-time solicitation.  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.

FUNDS AVAILABLE

The AHCPR expects to award up to $2 million for first year support of
four to six awards.

RESEARCH OBJECTIVES

Background

The enabling legislation for AHCPR authorizes studies of the
effectiveness and appropriateness of health care services and
procedures.  Section 1142 of the Social Security Act recognizes drug
therapy as an important form of treatment and refers to the need to
study effectiveness and appropriateness.  As a component of MEDTEP, a
program was established in March 1992 with RFA HS-92-03, focusing on
the patient outcomes of pharmaceutical therapy.  As a result of that
RFA, AHCPR has awarded sixteen grants focusing on outcomes of
pharmaceutical therapy in ambulatory care settings.  This RFA
initiates an extension of that work, but is not limited to studies in
any particular care setting.  It focuses on the effects on patient
outcomes of various programs, proposed or existing, for managing
selection, utilization, and cost of pharmaceutical therapies and
services within the changing health care environment.

MEDTEP Research Themes

This RFA incorporates the themes and concepts common to all MEDTEP
research (MEDTEP-Summary PA-94-074).  "Effectiveness" refers to
outcomes experienced by, or observed in, patients in routine clinical
practice.  This is distinct from "efficacy" which refers to the
potential benefit of clinical interventions provided under ideal
circumstances to patients who meet specific criteria.  Cost
effectiveness summarizes the cost and effect of treatment in terms of
specified outcomes measured in nonmonetary units; it indicates value
obtained for resources expended.

MEDTEP studies focus on clinical conditions that affect large numbers
of people (or a large proportion of a major subpopulation); that
result in substantial expense; that are significant in Medicaid or
Medicare programs; and for which there are large, unexplained
variations in treatment.  The outcomes to be studied, in addition to
traditional measures such as survival and morbidity, include patient-
reported outcomes such as perceived health status, functioning, and
quality of life.  MEDTEP studies typically involve a
multidisciplinary team of researchers with clinical and
methodological expertise, plus understanding of the perspectives of
patients, providers, and policymakers.

Methods

MEDTEP studies draw on a wide range of research methods that include,
but are not limited to:  experimental and quasi-experimental designs,
case-control and cohort studies, effectiveness trials, meta-analysis,
cost effectiveness analysis, decision modeling, and combinations of
these methods.

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 efficient means to
address the research question(s).  Data sources should allow for the
detection, measurement, and/or control of:  drug exposure, indication
for drug use, severity of illness, comorbidities, relevant
confounders, and costs of care when appropriate.  Measures of short-
and long-term outcomes, as well as pre-treatment health status
information, are critical data elements.

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
proposed research question(s).

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).

Topic Selection

Current restructuring within the health care system provides an
opportunity to enhance understanding of the interrelationships of
pharmaceutical treatments and services, with patient outcomes.  The
changing mix of payment mechanisms, benefit plans, and cost-
containment strategies has widespread implications for patient care.
Of particular interest are studies involving the comparative
effectiveness and/or cost effectiveness of pharmaceutical therapies
within the changing health care system.

Studies are expected to compare different clinical approaches to the
prevention, diagnosis, treatment, and/or management of common
clinical conditions.  While most of these studies will involve
comparisons among drug therapies and/or related services, studies of
drug versus non-drug treatment options also are responsive to the
RFA.  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 selections and
exclusions.

Studies should focus on one or both of the following topic areas:

1.  Pharmaceutical Economic Analyses

Evaluations of the economic impact of drug therapy are of particular
policy relevance in the changing health care environment.  Economic
information is needed by large-volume purchasers such as managed care
organizations to guide formulary decisions, price negotiations, and
clinical decision making.  Cost-effectiveness, cost-benefit, and
cost-utility analyses are responsive to this RFA provided patient
outcomes are the central focus.  Studies that evaluate monetary cost
alone are not responsive.  Of special interest are studies that
develop tools or analytic models that will be useful in other
research.  Examples of suitable topics include the following:

o  Comparative cost effectiveness of alternative drug therapies for
treatment of a selected common condition.  Choice of condition and
treatment options should be justified in terms of clinical and policy
relevance.

o  Cost-utility studies that include user-friendly (to patients and
providers) methods or measures for assessing patient preferences,
values, and utilities.

2.  Effects of Changes in the Health Care Environment

There is a trend in health care toward systems of managed care,
including health maintenance organizations, individual practice
organizations, preferred provider organizations, and coordinated care
networks.  Often, these systems include some form of case management
or pharmaceutical benefit management, for coordination of services
and patient monitoring.  They also include mechanisms for managing
selection, utilization, and cost for pharmaceutical therapies and
services.

Previous studies have explored the effects of policy and educational
interventions on the appropriateness of drug prescribing and use.
Many questions remain, however, about the long-term effects of
programs that monitor and intervene in the drug management process.
Innovations that affect patient outcomes and cost, and that warrant
evaluation include:

o  Interventions designed to change prescribing practices.  These
range from face-to-face academic detailing, to computer-generated
reminders, to adoption of clinical practice guidelines and treatment
algorithms that address drug selection and use.

o  Interventions such as those implemented by large-volume drug
purchasers including HMOs, Medicaid programs, and pharmaceutical
benefit managers, to optimize the clinical and/or cost effectiveness
of drug utilization review (DUR).

o  Interventions designed to encourage the provision or alter the
content of pharmaceutical care.  These include reimbursement programs
for pharmacist cognitive services within managed care organizations.

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 28, 1994 (FR 59 14508-14513) 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.

LETTER OF INTENT

Prospective applicants are asked to submit, by March 1, 1995, a
letter of intent that includes a descriptive title of the proposed
research; the name, address, and telephone number of the Principal
Investigator, co-investigators and other key personnel; the applicant
institution and other participating organizations or institutions;
and the number and title of this RFA.  Although a letter of intent is
not required, is not binding, and does not enter into the
consideration of any subsequent application, the information allows
AHCPR staff to estimate the potential review workload and avoid
conflicts of interest in the review.  The letter of intent is to be
sent to:

Joanne S. Book
Center for Medical Effectiveness Research
Agency for Health Care Policy and Research
East Jefferson Street, Suite 605
Rockville, MD  20852-4908

APPLICATION PROCEDURES

Applications are to be submitted on the research grant application
form PHS 398 (rev. 9/91).  State and local government agencies may
use form PHS 5161 and follow those requirements for copy submission.
These forms are available at most institutional offices of sponsored
research; 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 Avenue, Suite
400, Bethesda, MD 20814-3015, telephone (301) 656-3100 (FAX 301
652-5264).

The RFA label available in the form PHS 398 (rev. 9/91) form must be
affixed to the bottom of the face page of the original application.
Failure to use this label could result in delayed processing of the
application such that it may not reach the review committee in time
for review.  In addition, type "RFA HS-96-001" in Section 2a on the
face page of the application form and mark the "YES" box.

Submit a signed, typewritten original of the application, including
the Checklist, and three signed, photocopies, in one package to:

Division of Research Grants
National Institutes of Health
Westwood Building, Room 240
Bethesda, MD  20892**

At the time of submission, two additional copies of the application
must be sent to:

Center for Medical Effectiveness Research
Agency for Health Care Policy and Research
2101 East Jefferson Street, Suite 605
Rockville, MD  20852-4908

Applications submitted under this RFA must be received in the
Division of Research Grants, NIH, by April 12, 1995.  If an
application is received after that date, it will be returned to the
applicant without review.

REVIEW CONSIDERATIONS

Upon receipt, applications will be reviewed by the Referral Office,
Division of Research Grants, NIH, for completeness, and by AHCPR
staff for responsiveness to the RFA.  Incomplete applications will be
returned to the applicant without further consideration.
Nonresponsive applications will be transferred to a standing AHCPR or
other appropriate scientific review group for review through routine
mechanisms.  The determination of any application as nonresponsive
will be the sole responsibility of AHCPR.  Applications may undergo
triage by the peer review group on the basis of relative scientific
and technical competitiveness.  The AHCPR will withdraw from further
consideration those applications judged to be non-competitive for
award and notify the applicant Principal Investigator and
institutional official.  When an application is reviewed, the peer
review committee may recommend further consideration or no further
consideration.  The committee also assigns priority scores to the
applications for which further consideration is recommended.
Recommendations of the peer review committee may be reviewed
subsequently by AHCPR's National Advisory Council for Health Care
Policy, Research, and Evaluation.  The peer review process is
rigorous, and only those applications judged to be of greatest merit
will be recommended for further consideration.

Review Criteria

The general review criteria for AHCPR grant applications are:

o  significance and originality from a scientific and technical
viewpoint;

o  adequacy of the proposed method(s);

o  availability of data or proposed plan to collect data required for
the project;

o  adequacy of the plan for organizing and carrying out the project;

o  qualifications and experience of the Principal Investigator and
proposed staff;

o  reasonableness of the proposed budget;

o  adequacy of the facilities and resources available to the
applicant; and

o  adequacy of plans to include both genders and minorities and their
subgroups, as appropriate for the scientific goals of the research.
Plans for the recruitment and retention of subjects will also be
evaluated.

The initial review group will also examine the provisions for the
protection of experimental subjects, and the safety of the research
environments.

Special Review Criteria

In addition to the general review criteria noted above, the following
special scientific and technical review criteria will apply to this
RFA:

o  scientific importance and policy relevance of the topic;

o  generalizability of results;

o  feasibility of answering 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 proposed data;

o  justification for focus on outcome(s) specified;

o  adequacy of outcome measure(s);

o  sensitivity to patient heterogeneity and individual preference;

o  specification of useful findings or products, and identification
of constituency(ies) for these; and

o  efficiency of the research plan.

AWARD CRITERIA

Applications will compete for available funds with all other
applications for this RFA.  In making funding decisions, AHCPR will
consider:  quality of the proposed project as determined by peer
review, availability of funds, and program balance.

INQUIRIES

The AHCPR welcomes the opportunity to clarify any issues or questions
from potential applicants.  Direct inquiries regarding program
matters to:

Richard J. Greene, M.D., Ph.D.
Center for Medical Effectiveness Research
Agency for Health Care Policy and Research
East Jefferson Street, Suite 605
Rockville, MD  20852-4908
Telephone:  (301) 594-1485
Email:  JBook@po4.ahcpr.gov

Direct inquiries regarding fiscal matters to:

Ralph Sloat, Grants Management Officer
Agency for Health Care Policy and Research
2101 East Jefferson Street, Suite 601
Rockville, MD  20852-4908
Telephone:  (301) 594-1447
Email:  RSloat@po7.ahcpr.gov

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.

The Public Health Service strongly encourages all grant recipients to
provide a smoke-free workplace and promote the non-use of all tobacco
products.  This is consistent with the PHS mission to protect and
advance the physical and mental health of the American people.

.

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