Effectiveness Science
Marita G. Titler, PhD, RN, FAAN
Professor and Associate Dean for
Clinical Scholarship & Practice
Rhetaugh G. Dumas Endowed Chair
Division III Chair
University of Michigan School of
Nursing
Ann Arbor, Michigan
Objectives
 Overview of effectiveness science (ES).
 Contributions and impact of nurse
investigators to ES
 Future Directions
Efficacy Studies
 Investigate the benefits of an intervention under
ideal and highly controlled conditions (RCTs).
 Substantial differences from clinical practice
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Study protocol - minimize treatment preferences &
multimodal treatment programs,
control of the skill levels of the person(s)
delivering the intervention, and
 restrictive control over the study sample.
 High internal validity. Limitations in external validity
(generalizability to practice)
 Example: A phase III double blind trial of duloxetine
to treat painful chemotherapy-induced peripheral
neuropathy (Ellen Smith; ASCO meeting, 2012)
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Effectiveness Studies
 Examine outcomes of interventions under
circumstances that approximate the real world.
 Employ more heterogeneous samples,
clinicians delivery of treatment (rather than a
trained RA), and settings are routine clinical
practice.
 High external validity; more applicable to
everyday practice. Sacrifice some degree of
internal validity.
 Examples:
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Impact of nursing surveillance on failure to
rescue (Shever, 2011).
Dissemination of the Tobacco Tactics
Intervention In Community Hospitals (Duffy et al,
2012)
Efficacy and Effectiveness
 Distinction between efficacy and
effectiveness studies viewed as a
spectrum along a continuum.
 Study guided by
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State of the science on the topic.
Research questions or hypotheses.
Effectiveness Science
 Testing healthcare interventions in real
world settings, advancing use of
research findings by diverse populations,
training and career development of the
next generation of investigators.
 Uses comparisons, subgroups and
context to understand what healthcare
treatments work for whom and in what
settings.
(Simpson et al, 2010; special issue of Medical Care, June 2012)
Why Effectiveness Science
 Help consumers, clinicians, purchasers,
& policy makers to make informed
decisions that will improve healthcare at
the individual & population level.
 Knowing efficacy does not necessarily
guarantee effectiveness in practice.
Effectiveness Science includes
 PCOR
 Comparative effectiveness research
 Implementation and dissemination
science
 Interface with electronic healthcare data
repositories, and informatics
Terminology
 CER – generation and synthesis of evidence that
compares alternative methods to prevent,
diagnose, treat and monitor a clinical condition or
improve the delivery of care.
 Translation/Implementation science – testing
effectiveness of implementation strategies to
promote adoption of evidence in healthcare
delivery.
 Dissemination – targeted distribution of
intervention materials to a specific public health
or clinical practice audience.
(Titler & Everett, 2002; Wensing et al 2006; NIH; IOM, 2009; Simpson, 2010)
Examples of Top Priorities
CER
 Primary prevention of falls in older adults
 Dissemination & translation strategies to
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promote use of research findings
Strategies to reduce HAI
Comprehensive care coordination for chronic
illness
School-based interventions – prevention and
treatment of over-weight and obesity
Strategies to prevent obesity, hypertension,
diabetes, & heart disease in at risk populations.
Strategies to reduce health disparities in
cardiovascular disease, diabetes, cancer,
musculoskeletal diseases, and birth outcomes
(IOM, 2009; pg 3-5)
Patient Centered Outcomes
Research Institute (PCORI)
 Nonprofit institute established by health
care reform legislation
 Assist policy makers, purchasers,
consumers, and clinicians in making
informed health decisions by conducting
research that provides quality and relevant
evidence ….
 Board of governors – approved research
priorities May 21, 2012.
 Check out the PCORI web-site
(www.PCORI.org)
Methods and Approaches
 Systematic reviews
 Meta-analysis
 Practice guidelines – based on scientific
evidence
 Experimental designs
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Pragmatic clinical trials
Clustered Randomized trials
 Observational Approaches
 Qualitative methods
 Mixed methods
(Conn et al, 2012; IOM, 2009; Tunis et al, 2009; Pressler, 2011)
Clustered Randomized Trials
 Evidence-Based Practice: From Book to Bedside
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(PI: Titler; R01 HS10482). Book to Bedside:
Sustaining Evidence-Based Practices in Elders (PI:
Titler; R01 HS10482)
Implementation of Practice Standards for ECG
Monitoring (PI: Funk; R01HL081642)
An intervention to improve outcomes in patients with
advanced cancer (PI: McCorkle; R01NR011872)
RWJ foundation INQRI program – upcoming issue
of Medical Care (falls; delirium; pain; substance
abuse).
Incontinence Prevention: The TULIP Project (PI:
Sampselle; R01NR012011; RCT)
Observational Designs
 Nursing Contributions to Patient Outcomes for 3
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Older Populations (PI: Titler; R01 NR05331)
Prevention of Nosocomial Infections & Costeffectiveness Refined (P-NICER) (PI: Stone;
R01NR010107).
Prevention of Nosocomial Infections & Costeffectiveness in Nursing Homes (PI: Stone;
R01NR013687)
Implementing Evidence to Prevent Urinary
Infection & Enhance Patient Safety (PI: Krein;
R01NR010700).
Nursing Impact on Care Outcomes For
Chronically Ill & Minority Patients (PI: Aiken;
R01NR004513)
Qualitative and Mixed Methods
 Used in many studies
 Focus groups
 Interviews of participants
 Key informants
 Used to understand context of care delivery
 Necessary to understand users’/clinicians’
perceptions about the implementation
intervention, usefulness of each component,
and to uncover the underlying mechanisms
of the intervention.
Examples of Areas of Impact
 Patient Safety and Quality
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ECG Monitoring (Funk et al)
Fall Prevention (Titler; Anderson; Dyke)
HAI (Stone, Krein, Lake)
 Healthcare System
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Acute care (Aiken; Keenan; Titler; Lake)
Long-term care (Anderson; Stone, Rantz)
Ambulatory care (Friese et al)
 Health Promotion (Duffy; Sampselle)
Examples of Areas of Impact
 Symptom management, QOL, Palliative
Care, EOL care (e.g. Daly; Northouse;
Doorenbos; Dunbar; Beck; Happ;
Resnick)
 Cost (e.g. Stone; Titler; Berry; Dunbar;
Happ)
 Models and frameworks to guide the
research (complexity science;
implementation models; outcomes
effectiveness models)
RWJ INQRI Program
 Examine nurses’ practices, processes
and work environments and determine
the impact nurses have on the quality of
patient care.
 40 interdisciplinary research teams
 www.INQRI.org
 Demonstrates nursing’s unique
contributions to patients, families and
communities across a diverse range of
settings
(Mary Naylor and Mark Pauly)
The FOCUS Program
Designed to facilitate communication and other
outcomes cancer patients and spouse/partners
 Breast Cancer Clinical Trial (1996-2000)
ACS
 Prostate Cancer Clinical Trial (2000-2005)
NCl
 Advanced Cancer Clinical Trial (2005-2010)
NCI
Laurel Northouse PhD, RN, FAAN
Translation, Implementation,
Dissemination
 Three major areas
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Translation: to the web
Implementation: in community agencies
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Rosalyn Carter Institute/Cancer Support Community
Dissemination: program materials widely
 National, state, local
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National Cancer Institute
Michigan Dept of Community Health
UM Comprehensive Cancer Center
 Activities are occurring simultaneously
Laurel Northouse PhD, RN, FAAN
FOCUS Prostate Program Materials
Laurel Northouse PhD, RN, FAAN
Sponsored by NCI and SAMHSA
Evidence-Based Practice: From Book
to Bedside
 Improved pain assessment and pain
management.
 Less pain intensity.
 For each one-unit increase in the
Summative Index, total costs decreased
by $1,598.75 (p = 0.002)
 A net savings to the hospital of more
than $131,000 per 100 patients, even
after implementation costs are taken into
account.
 Improvements sustained
(Titler et al, 2008 HSR; Brooks et al, 2008)
Nursing Contributions to Patient
Outcomes for 3 Older Patient
Populations
Purpose: to conduct outcomes effectiveness
research using data in electronic
repositories from one medical center.
Unique contributions of nursing treatments
to patient outcomes. (Exploratory study)
Sample: 4 years of data from 3 pt. populations:
 heart failure (1435 hosp; 1035 patients)
 hip fracture and procedures (569 hosp; 524
patients)
 those who receive Fall Prevention
intervention (10,187 hosp; 7,851 patients)
(Titler et al; Funded by NINR; R01 NR05331)
Studies and Publications
 15 studies reported in peer reviewed
publications
 Two doctoral dissertations
 Papers published by students during their PhD
program.
 Propensity scoring papers:
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Qin, R., Titler, M., Shever, L., & Kim, T. (2008).
Estimating causal effects of nursing intervention via
propensity score analysis. Nursing Research, 57(6),
444-452.
Shever, L. L. (2011). The Impact of Nursing
Surveillance on Failure to Rescue. Research and
Theory for Nursing Practice, 25 (2), 127-151.
Shever, L. L., Titler, M., Kerr, P., Qin, R., Kim, T., &
Picone, D. M. (2008). Effects of nursing surveillance
on hospital cost for older adults at risk for falling.
Journal of Nursing Scholarship, 40(2), 161-169.
Impact
 Documentation of
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nursing treatments to
patient outcomes and
cost.
Dose of nursing
treatments
Explicated methods for
conducting ES using
large data repositories
of clinical care delivery
Conceptual Model to
guide research
Application of model to
analytic methods (e.g.
propensity scoring)
Implementation of Risk Specific Fall Prevention
Interventions to Reduce Falls in Hospitals
 Purpose - to implement, in 3 community
hospitals (14 nursing units), fall reduction
interventions targeted to patient specific
risk factors.
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Funded by RWJ foundation INQRI program
Preliminary Findings
 Increased use of fall prevention
interventions targeted to patient specific
risk factors
 Reduction in falls
 Rounding by nurse managers to address
fall prevention practices
 Request for QRGs
 Conduct a multi-site study
Topics: VAP reduction; Enteral tube feeding; Family presence in ICU;
Depression Screening in Primary Care; Care of adults with diabetes; Facilitating
EBP during military operations.
 The NNPN promotes implementation of
evidence-based practices (EBP) through a
collaborative model of shared learning and
participation.
 Multi-site research
 Community members
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100 hospitals
Represent 32 states, Puerto Rico and
Singapore
Hawaii State Center for Nursing
Marita Titler PhD, RN, FAAN
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Resources
 Education
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Newsletters
14 Podcasts
How to start a journal
club
EBP references
Eye on Evidence
Webinars – lunch and
learn; journal clubs
 Research
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Network of sites for
research
Process for investigators
to access NNPN
organizations for
research
Organization context
measurement
instruments
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Marita Titler PhD, RN, FAAN
Culture
Climate
Interactive human
relationships
Hawaii State Center for
Nursing
 Hawaii Nurses Shaping Healthcare: A
State-Wide Evidence-Based Practice
Initiative
 Legislative mandate to for EBP and
quality outcomes
Debra D. Mark, RN, PhD
Nurse Researcher, Hawai’i State Center for Nursing
debramar@hawaii.edu
Outcomes to Date
 Increasing EBP capacity across the state
 Trained 39 teams
 8 Health care systems
 Institutionalizing practice change
 Papers and conference presentations
Future Directions
 Education and training programs in ES
 Maximize use of registries, clinical and
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administrative data repositories
Multi-site research
Terminology/nomenclature; taxonomy
Context
Measurement (e.g. self-report; MRA;
observation)
Complexity of the intervention – implementation
science.
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Core elements
Mode of delivery
Intervention dose (intensity and duration of each
component)
Future Directions
 More collaboration with CTSAs
 Nurse scientists lead interdisciplinary
teams (e.g. behavioral interventions;
symptom management; implementation
science)
 Key stakeholder partnerships at the
initiation of the study.
 Scale-up and spread what works
 Public policy messages
 Funding sources