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Summary
bACKGROuND
Today, when a patient and physician, perhaps with other clinicians
and family caregivers, are discussing the best course of treatment for the
patient’s medical condition, they often do not have the scientific evidence
they need to make a determination. Although there may be studies that in-
dicate that a treatment is efficacious relative to a placebo, there frequently
are no studies that directly compare the different available alternatives or
that have examined their impacts in populations of the same age, sex, and
ethnicity or with the same comorbidities as the patient. Comparative effec-
tiveness research (CER) is designed to fill this knowledge gap. CER focuses
attention on the evidence base to assist patients and health care providers
across diverse health settings in making more informed decisions. They will
need useful, practical information concerning the most effective interven-
tions and health care services for their particular situation.
To help identify which health care services work best, Congress, in the
American Recovery and Reinvestment Act (ARRA) of 2009 (P.L. 111-5),
appropriated $1.1 billion as a down-payment to provide strong federal sup-
port of CER. This provision in the law reflected the legislators’ belief that
better decisions about the use of health care resources could improve the
public’s health and reduce the costs of care. According to the legislation,
CER covers “research that compares the clinical outcomes, effectiveness,
and appropriateness of items, services, and procedures that are used to
prevent, diagnose, or treat diseases, disorders, and other health conditions.”
The law appropriated $400 million to the National Institutes of Health
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INITIAL NATIONAL PRIORITIES FOR CER
(NIH), $300 million to the Agency for Healthcare Research and Quality
(AHRQ), and the remaining $400 million to the Secretary of Health and
Human Services (HHS). According to the language of the law, the purposes
of the appropriations were
• “to evaluate the relative effectiveness of different health care ser-
vices and treatment options” and
• “to encourage the development and use of clinical registries, clini-
cal data networks, and other forms of electronic data to generate
outcomes data.”
The law also charged the Institute of Medicine (IOM) to form a con-
sensus committee and solicit stakeholder input to recommend national
priorities for spending the $400 million designated for the Secretary. The
legislation imposed a short time frame on this study—the IOM report
deadline of June 30, 2009, was 19 weeks after the president signed the
legislation into law.
The National Academies President’s Fund generously supported the
study process until the study’s sponsor, AHRQ, could contract with the
IOM; IOM funds entirely paid for the public questionnaire and its analysis.
The Robert Wood Johnson Foundation also contributed significantly to
this study. This support permitted the IOM to rapidly establish a commit-
tee and to commence work. The committee encompassed a broad range of
expertise, perspectives, and experience, including members who work with
consumers and patients, in clinical care and research, or in health care and
government administration.
The committee’s principal task was to prepare a list of priorities for
CER funding; most of its time was spent developing a process for priority
setting, eliciting a wide array of input from the public, and deliberating
over a list of nominated research topics. Then, as the complexities of prior-
ity setting for CER became apparent, the committee began to outline the
development of an infrastructure that would sustain a long-term, national
CER effort. The committee provided recommendations to implement that
infrastructure required for a sustained CER effort. The main justification
for including economic considerations is that the overall value of a strategy
can be understood best by considering costs and benefits together. In such
a circumstance, value may be judged from the perspective of the patient,
provider, or payer. Many stakeholders thought CER might persuade payers
to support or improve reimbursement for particular services, but the com-
mittee did not discuss leveraging research findings to payment policy.
The committee presents its recommended list of 100 top priority CER
topics in Table S-1. The individual topics are grouped into quartiles ac-
cording to the number of votes each received during the committee’s voting
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SUMMARY
process. Topics within the First Quartile were considered higher priority
than those in the Fourth Quartile, but the order within quartiles does not
signify rank. Following Table S-1 is a brief discussion of how the commit-
tee created the priority list, a section on what the committee learned from
the process, and implications and recommendations for establishing a solid
foundation for CER in the future.
LIST OF PRIORITy CER TOPICS
TAbLE S-1 Final List of Priority Topics, by Quartile Ratings
*display within quartile does not indicate priority rank—topics are listed
alphabetically by primary research area
First Quartile
(listed alphabetically by primary research area)
CAD Compare the effectiveness of treatment strategies for atrial fibrillation including
surgery, catheter ablation, and pharmacologic treatment.
DIS Compare the effectiveness of the different treatments (e.g., assistive
listening devices, cochlear implants, electric-acoustic devices, habilitation
and rehabilitation methods [auditory/oral, sign language, and total
communication]) for hearing loss in children and adults, especially individuals
with diverse cultural, language, medical, and developmental backgrounds.
ENDO Compare the effectiveness of primary prevention methods, such as exercise and
balance training, versus clinical treatments in preventing falls in older adults at
varying degrees of risk.
GI Compare the effectiveness of upper endoscopy utilization and frequency for
patients with gastroesophageal reflux disease on morbidity, quality of life, and
diagnosis of esophageal adenocarcinoma.
HCDS Compare the effectiveness of dissemination and translation techniques to
facilitate the use of CER by patients, clinicians, payers, and others.
HCDS Compare the effectiveness of comprehensive care coordination programs, such
as the medical home, and usual care in managing children and adults with
severe chronic disease, especially in populations with known health disparities.
IMUN Compare the effectiveness of different strategies of introducing biologics into
the treatment algorithm for inflammatory diseases, including Crohn’s disease,
ulcerative colitis, rheumatoid arthritis, and psoriatic arthritis.
INFD Compare the effectiveness of various screening, prophylaxis, and treatment
interventions in eradicating methicillin resistant Staphylococcus aureus (MRSA)
in communities, institutions, and hospitals.
continued
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INITIAL NATIONAL PRIORITIES FOR CER
TAbLE S-1 Continued
INFD Compare the effectiveness of strategies (e.g., bio-patches, reducing central
line entry, chlorhexidine for all line entries, antibiotic impregnated catheters,
treating all line entries via a sterile field) for reducing health care associated
infections (HAI), including catheter-associated bloodstream infection, ventilator
associated pneumonia, and surgical site infections in children and adults.
KUT Compare the effectiveness of management strategies for localized prostate
cancer (e.g., active surveillance, radical prostatectomy [conventional, robotic,
and laparoscopic], and radiotherapy [conformal, brachytherapy, proton-beam,
and intensity-modulated radiotherapy]) on survival, recurrence, side effects,
quality of life, and costs.
MS Establish a prospective registry to compare the effectiveness of treatment
strategies for low back pain without neurological deficit or spinal deformity.
NEURO Compare the effectiveness and costs of alternative detection and management
strategies (e.g., pharmacologic treatment, social/family support, combined
pharmacologic and social/family support) for dementia in community-dwelling
individuals and their caregivers.
NEURO Compare the effectiveness of pharmacologic and non-pharmacologic treatments
in managing behavioral disorders in people with Alzheimer’s disease and other
dementias in home and institutional settings.
NUTR Compare the effectiveness of school-based interventions involving meal
programs, vending machines, and physical education, at different levels of
intensity, in preventing and treating overweight and obesity in children and
adolescents.
NUTR Compare the effectiveness of various strategies (e.g., clinical interventions,
selected social interventions [such as improving the built environment in
communities and making healthy foods more available], combined clinical
and social interventions) to prevent obesity, hypertension, diabetes, and heart
disease in at-risk populations such as the urban poor and American Indians.
ONC Compare the effectiveness of management strategies for ductal carcinoma in
situ (DCIS).
ONC Compare the effectiveness of imaging technologies in diagnosing, staging,
and monitoring patients with cancer including positron emission tomography
(PET), magnetic resonance imaging (MRI), and computed tomography (CT).
ONC Compare the effectiveness of genetic and biomarker testing and usual care in
preventing and treating breast, colorectal, prostate, lung, and ovarian cancer,
and possibly other clinical conditions for which promising biomarkers exist.
ORAL Compare the effectiveness of the various delivery models (e.g., primary care,
dental offices, schools, mobile vans) in preventing dental caries in children.
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SUMMARY
TAbLE S-1 Continued
PEDS Compare the effectiveness of various primary care treatment strategies (e.g.,
symptom management, cognitive behavior therapy, biofeedback, social skills,
educator/teacher training, parent training, pharmacologic treatment) for
attention deficit hyperactivity disorder (ADHD) in children.
PSYCH Compare the effectiveness of wraparound home and community-based services
and residential treatment in managing serious emotional disorders in children
and adults.
RED Compare the effectiveness of interventions (e.g., community-based multi-level
interventions, simple health education, usual care) to reduce health disparities
in cardiovascular disease, diabetes, cancer, musculoskeletal diseases, and birth
outcomes.
RED Compare the effectiveness of literacy-sensitive disease management programs
and usual care in reducing disparities in children and adults with low literacy
and chronic disease (e.g., heart disease).
WH Compare the effectiveness of clinical interventions (e.g., prenatal care,
nutritional counseling, smoking cessation, substance abuse treatment,
combinations of these interventions) to reduce incidences of infant mortality,
pre-term births, and low birth weights, especially among African American
women.
WH Compare the effectiveness of innovative strategies for preventing unintended
pregnancies (e.g., over-the-counter access to oral contraceptives or other
hormonal methods, expanding access to long-acting methods for young
women, providing free contraceptive methods at public clinics, pharmacies, or
other locations).
Second Quartile
(listed alphabetically by primary research area)
BDEV Compare the effectiveness of therapeutic strategies (e.g., behavioral or
pharmacologic interventions, the combination of the two) for different
autism spectrum disorders (ASD) at different levels of severity and stages of
intervention.
BDEV Compare the effectiveness of the co-location model (psychological and primary
care practitioners practicing together) and usual care (identification by primary
care practitioner and referral to community-based mental health services)
in identifying and treating social-emotional and developmental disorders in
children ages 0-3.
BDEV Compare the effectiveness of diverse models of comprehensive support services
for infants and their families following discharge from a neonatal intensive care
unit.
continued
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INITIAL NATIONAL PRIORITIES FOR CER
TAbLE S-1 Continued
CAD Compare the effectiveness of treatment strategies for vascular claudication
(e.g., medical optimization, smoking cessation, exercise, catheter-based
treatment, open surgical bypass).
CAM Compare the effectiveness of mindfulness-based interventions (e.g., yoga,
meditation, deep breathing training) and usual care in treating anxiety and
depression, pain, cardiovascular risk factors, and chronic diseases.
ENDO Compare the long-term effectiveness of weight-bearing exercise and
bisphosphonates in preventing hip and vertebral fractures in older women with
osteopenia and/or osteoporosis.
HCDS Compare the effectiveness of shared decision making and usual care on
decision outcomes (treatment choice, knowledge, treatment-preference
concordance, and decisional conflict) in children and adults with chronic
disease such as stable angina and asthma.
HCDS Compare the effectiveness of strategies for enhancing patients’ adherence to
medication regimens.
HCDS Compare the effectiveness of patient decision support tools on informing
diagnostic and treatment decisions (e.g., treatment choice, knowledge
acquisition, treatment-preference concordance, decisional conflict) for elective
surgical and nonsurgical procedures—especially in patients with limited
English-language proficiency, limited education, hearing or visual impairments,
or mental health problems.
HCDS Compare the effectiveness of robotic assistance surgery and conventional
surgery for common operations, such as prostatectomies.
HCDS Compare the effectiveness (including resource utilization, workforce needs, net
health care expenditures, and requirements for large-scale deployment) of new
remote patient monitoring and management technologies (e.g., telemedicine,
Internet, remote sensing) and usual care in managing chronic disease, especially
in rural settings.
HCDS Compare the effectiveness of diverse models of transition support services for
adults with complex health care needs (e.g., the elderly, homeless, mentally
challenged) after hospital discharge.
HCDS Compare the effectiveness of accountable care systems and usual care on costs,
processes of care, and outcomes for geographically defined populations of
patients with one or more chronic diseases.
HCDS Compare the effectiveness of different residential settings (e.g., home care,
nursing home, group home) in caring for elderly patients with functional
impairments.
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SUMMARY
TAbLE S-1 Continued
KUT Compare the effectiveness (including survival, hospitalization, quality of life,
and costs) of renal replacement therapies (e.g., daily home hemodialysis,
intermittent home hemodialysis, conventional in-center dialysis, continuous
ambulatory peritoneal dialysis, renal transplantation) for patients of different
ages, races, and ethnicities.
MS Compare the effectiveness of treatment strategies (e.g., artificial cervical discs,
spinal fusion, pharmacologic treatment with physical therapy) for cervical disc
and neck pain.
ONC Compare the effectiveness of film-screen or digital mammography alone and
mammography plus magnetic resonance imaging (MRI) in community practice-
based screening for breast cancer in high-risk women of different ages, risk
factors, and race or ethnicity.
ONC Compare the effectiveness of new screening technologies (such as fecal
immunochemical tests and computed tomography [CT] colonography) and
usual care (fecal occult blood tests and colonoscopy) in preventing colorectal
cancer.
PELC Compare the effectiveness of coordinated care (supported by reimbursement
innovations) and usual care in long-term and end-of-life care of the elderly.
PSYCH Compare the effectiveness of pharmacologic treatment and behavioral
interventions in managing major depressive disorders in adolescents and adults
in diverse treatment settings.
RD Compare the effectiveness of an integrated approach (combining counseling,
environmental mitigation, chronic disease management, and legal assistance)
with a non-integrated episodic care model in managing asthma in children.
SKIN Compare the effectiveness (including effects on quality of life) of treatment
strategies (e.g., topical steroids, ultraviolet light, methotrexate, biologic
response modifiers) for psoriasis.
TEMC Compare the effectiveness of treatment strategies (e.g., cognitive behavioral
individual therapy, generic individual therapy, comprehensive and intensive
treatment) for Post-traumatic Stress Disorder stemming from diverse sources of
trauma.
WH Compare the effectiveness and outcomes of care with obstetric ultrasound
studies and care without the use of ultrasound in normal pregnancies.
WH Compare the effectiveness of birthing care in freestanding birth centers and
usual care of childbearing women at low and moderate risk.
continued
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INITIAL NATIONAL PRIORITIES FOR CER
TAbLE S-1 Continued
Third Quartile
(listed alphabetically by primary research area)
ADDO Compare the effectiveness of different opioid and non-opioid pain relievers,
in different doses and durations, in avoiding unintentional overdose and
substance dependence among subjects with acute and non-cancer chronic pain.
CAD Compare the effectiveness of aggressive medical management and percutaneous
coronary interventions in treating stable coronary disease for patients of
different ages and with different comorbidities.
CAD Compare the effectiveness of innovative treatment strategies (e.g., cardiac
resynchronization, remote physiologic monitoring, pharmacologic treatment,
novel agents such as CRF-2 receptors) for congestive heart failure.
CAD Compare the effectiveness of traditional risk stratification for coronary heart
disease (CHD) and noninvasive imaging (using coronary artery calcium, carotid
intima media thickness, and other approaches) on CHD outcomes.
CAD Compare the effectiveness of different treatment strategies (e.g., modifying
target levels for glucose, lipid, or blood pressure) in reducing cardiovascular
complications in newly diagnosed adolescents and adults with type 2 diabetes.
CAM Compare the effectiveness of acupuncture for various indications using a
cluster randomized trial.
CAM Compare the effectiveness of dietary supplements (nutriceuticals) and usual
care in the treatment of selected high-prevalence conditions.
EENT Compare the effectiveness of different treatment options (e.g., laser therapy,
intravitreal steroids, anti-vascular endothelial growth factor [anti-VEGF]) for
diabetic retinopathy, macular degeneration, and retinal vein occlusion.
EENT Compare the effectiveness of treatment strategies for primary open-angle
glaucoma (e.g., initial laser surgery, new surgical techniques, new medical
treatments) particularly in minority populations to assess clinical and patient-
reported outcomes.
ENDO Compare the effectiveness and cost-effectiveness of conventional medical
management of type 2 diabetes in adolescents and adults, versus conventional
therapy plus intensive educational programs or programs incorporating
support groups and educational resources.
HCDS Compare the effectiveness of alternative redesign strategies—using decision
support capabilities, electronic health records, and personal health records—for
increasing health professionals’ compliance with evidence-based guidelines and
patients’ adherence to guideline-based regimens for chronic disease care.
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SUMMARY
TAbLE S-1 Continued
HCDS Compare the effectiveness of adding information about new biomarkers
(including genetic information) with standard care in motivating behavior
change and improving clinical outcomes.
HCDS Compare the effectiveness of different quality improvement strategies in disease
prevention, acute care, chronic disease care, and rehabilitation services for
diverse populations of children and adults.
HCDS Compare the effectiveness of formulary management practices and usual
practices in controlling hospital expenditures for products other than drugs
including medical devices (surgical hemostatic products, radiocontrast,
interventional cardiology devices, and others).
HCDS Compare the effectiveness of different benefit design, utilization management,
and cost-sharing strategies in improving health care access and quality in
patients with chronic diseases (e.g., cancer, diabetes, heart disease).
INFD Compare the effectiveness of HIV screening strategies based on recent Centers
for Disease Control and Prevention recommendations and traditional screening
in primary care settings with significant prevention counseling.
MS Establish a prospective registry to compare the effectiveness of surgical and
nonsurgical strategies for treating cervical spondylotic myelopathy (CSM)
in patients with different characteristics to delineate predictors of improved
outcomes.
NEURO Compare the effectiveness of traditional and newer imaging modalities (e.g.,
routine imaging, magnetic resonance imaging [MRI], computed tomography
[CT], positron emission tomography [PET]) when ordered for neurological and
orthopedic indications by primary care practitioners, emergency department
physicians, and specialists.
NEURO Compare the effectiveness of comprehensive, coordinated care and usual care
on objective measures of clinical status, patient-reported outcomes, and costs
of care for people with multiple sclerosis.
NUTR Compare the effectiveness of treatment strategies for obesity (e.g., bariatric
surgery, behavioral interventions, pharmacologic treatment) on the resolution
of obesity-related outcomes such as diabetes, hypertension, and musculoskeletal
disorders.
ORAL Compare the clinical and cost-effectiveness of surgical care and a medical
model of prevention and care in managing periodontal disease to increase tooth
longevity and reduce systemic secondary effects in other organ systems.
continued
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0 INITIAL NATIONAL PRIORITIES FOR CER
TAbLE S-1 Continued
PSYCH Compare the effectiveness of atypical antipsychotic drug therapy and
conventional pharmacologic treatment for Food and Drug Administration-
approved indications and compendia-referenced off-label indications using
large datasets.
PSYCH Compare the effectiveness of management strategies (e.g., inpatient psychiatric
hospitalization, extended observation, partial hospitalization, intensive
outpatient care) for adolescents and adults following a suicide attempt.
RED Compare the effectiveness of different strategies to engage and retain patients
in care and to delineate barriers to care, especially for members of populations
that experience health disparities.
SKIN Compare the effectiveness of topical treatments (e.g., antibiotics, platelet-
derived growth factor) and systemic therapies (e.g., negative pressure wound
therapy, hyperbaric oxygen) in managing chronic lower extremity wounds.
Fourth Quartile
(listed alphabetically by primary research area)
ADDO Compare the effectiveness of smoking cessation strategies (e.g., medication,
individual or quitline counseling, combinations of these) in smokers from
understudied populations such as minorities, individuals with mental illness,
and adolescents.
CAD Compare the effectiveness of computed tomography (CT) angiography
and conventional angiography in assessing coronary stenosis in patients at
moderate pretest risk of coronary artery disease.
CAD Compare the effectiveness of anticoagulant therapies (e.g., low-intensity
warfarin, aspirin, injectable anticoagulants) for patients undergoing hip or knee
arthroplasty surgery.
DIS Compare the effectiveness of focused intense periodic therapy and usual weekly
therapy in managing cerebral palsy in children.
ENDO Compare the effectiveness of different disease management strategies in
improving the adherence to and value of pharmacologic treatments for the
elderly.
HCDS Compare the effectiveness of care coordination with and without clinical
decision supports (e.g., electronic health records) in producing good health
outcomes in chronically ill patients, including children with special health care
needs.
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SUMMARY
TAbLE S-1 Continued
HCDS Compare the effectiveness of coordinated, physician-led, interdisciplinary
care provided in the patient’s residence and usual care in managing advanced
chronic disease in community-dwelling patients with significant functional
impairments.
HCDS Compare the effectiveness of minimally invasive abdominal surgery and open
surgical procedures on post-operative infections, pain management, and
recuperative requirements.
HCDS Compare the effectiveness of traditional behavioral interventions versus
economic incentives in motivating behavior changes (e.g., weight loss, smoking
cessation, avoiding alcohol and substance abuse) in children and adults.
HCDS Compare the effectiveness of diagnostic imaging performed by non-radiologists
and radiologists.
HCDS Compare the effectiveness of different techniques (e.g., audio, visual, written)
for informing patients about proposed treatments during the process of
informed consent.
HCDS Compare the effectiveness of different disease management strategies for
activating patients with chronic disease.
HCDS Compare the effectiveness of different delivery models (e.g., home blood
pressure monitors, utilization of pharmacists or other allied health providers)
for controlling hypertension, especially in racial minorities.
INFD Compare the effectiveness of alternative clinical management strategies for
hepatitis C, including alternative duration of therapy for patients based on viral
genomic profile and patient risk factors (e.g., behavior-related risk factors).
MS Compare the effectiveness of different treatment strategies in the prevention of
progression and disability from osteoarthritis.
MS Compare the effectiveness (e.g., pain relief, functional outcomes) of different
surgical strategies for symptomatic cervical disc herniation in patients for
whom appropriate nonsurgical care has failed.
NEURO Compare the effectiveness of different treatment strategies on the frequency
and lost productivity in people with chronic, frequent migraine headaches.
NEURO Compare the effectiveness of monotherapy and polytherapy (i.e., use of two
or more drugs) on seizure frequency, adverse events, quality of life, and cost in
patients with intractable epilepsy.
ONC Compare the effectiveness of surgical resection, observation, or ablative
techniques on disease-free and overall survival, tumor recurrence, quality of
life, and toxicity in patients with liver metastases.
continued
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INITIAL NATIONAL PRIORITIES FOR CER
TAbLE S-1 Continued
PELC Compare the effectiveness of hospital-based palliative care and usual care on
patient-reported outcomes and cost.
PSYCH Compare the effectiveness of different treatment approaches (e.g., integrating
mental health care and primary care, improving consumer self-care, a
combination of integration and self-care) in avoiding early mortality and
comorbidity among people with serious and persistent mental illness.
PSYCH Compare the effectiveness of traditional training of primary care physicians in
primary care mental health and co-location systems of primary care and mental
health care on outcomes including depression, anxiety, physical symptoms,
physical disability, prescription substance use, mental and physical function,
satisfaction with the provider, and cost.
PSYCH Compare the effectiveness of different treatment strategies (e.g., psychotherapy,
antidepressants, combination treatment with case management) for depression
after myocardial infarction on medication adherence, cardiovascular events,
hospitalization, and death.
SKIN Compare the effectiveness of different long-term treatments for acne.
WH Compare the effectiveness of different strategies for promoting breastfeeding
among low-income African American women.
NOTE: ADDO = Alcoholism, Drug Dependency, and Overdose; BDEV = Birth and Develop-
mental Disorders; CAD = Cardiovascular and Peripheral Vascular Disease; CAM = Comple-
mentary and Alternative Medicine; DIS = Functional Limitations and Disabilities; EENT =
Eyes, Ears, Nose, and Throat Disorders; ENDO = Endocrinology and Metabolism Disorders
and Geriatrics; GI = Gastrointestinal System Disorders; HCDS = Health Care Delivery Sys-
tems; IMUN = Immune System, Connective Tissue, and Joint Disorders; INFD = Infectious
Diseases Liver and Biliary Tract Disorders; KUT = Kidney and Urinary Tract Disorders; MS
= Musculoskeletal Disorders; NEURO = Neurologic Disorders; NUTR = Nutrition (including
obesity); ONC = Oncology and Hematology; ORAL = Oral Health; PEDS = Pediatrics; PELC
= Palliative and End-of-Life Care; PSYCH = Psychiatric Disorders; RD = Respiratory Disease;
RED = Racial and Ethnic Disparities; SKIN = Skin Disorders; TEMC = Trauma, Emergency
Medicine, and Critical Care Medicine; WH = Women’s Health.
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SUMMARY
DEFINING COMPARATIvE EFFECTIvENESS RESEARCH
An agreed-upon definition of CER is an essential first step for setting
priorities and developing a sustainable national CER Program. It informs
the public of the focus of this research and its importance in their lives, and
it informs investigators of the characteristics of the research to be supported
by CER funds. It provides a basis for judging research proposals to perform
CER and for evaluating the impact of that research and the success of a
national CER Program. In formulating its definition, this committee drew
upon definitions by several government agencies and other IOM commit-
tees (see Chapter 2):
Comparative effectiveness research (CER) is the generation and synthesis
of evidence that compares the benefits and harms of alternative methods
to prevent, diagnose, treat, and monitor a clinical condition or to improve
the delivery of care. The purpose of CER is to assist consumers, clinicians,
purchasers, and policy makers to make informed decisions that will im-
prove health care at both the individual and population levels.
CREATING THE PRIORITy LIST OF CER STuDIES
The committee received several broad directives. The legislative lan-
guage directed the IOM to solicit the opinions of stakeholders. The IOM’s
charge from the contracting agency, AHRQ, stipulated that the committee
provide a well-balanced portfolio of research topics for the list of priorities.
The committee’s approach to priority setting included the following:
• Extensive consultation with and input from stakeholders. The
committee widely solicited input through three mechanisms: (1) an
invitation to the public and key stakeholders to testify at a 1-day
public meeting in Washington, DC, at which the committee heard
54 speakers and received additional written testimony (available on
the report’s website at www.iom.edu/cerpriorities); (2) a web-based
nomination process through which 1,758 respondents, mostly phy-
sicians and representatives of professional organizations, but also
many members of the general public nominated a total of 1,268
unique research topics (see questionnaire in Appendix B); and
(3) the project’s website, which received emails and letters (see
Chapter 3).
• Development and consideration of written priority-setting criteria.
To guide judgments about each nominated topic, the committee
formulated priority-setting criteria to identify high priority target
conditions, such as their prevalence, mortality, aggregate costs,
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INITIAL NATIONAL PRIORITIES FOR CER
gaps in knowledge, and small area variation in rates of tests and
treatments of top conditions as well as criteria focused on specific
research topics (see Chapter 4).
• Commitment to developing a broad-based portfolio of high priority
topics. The committee’s criteria for creating a balanced portfolio
considered four dimensions: (1) clinical category (e.g., cardio-
vascular and peripheral vascular disease), (2) study population,
(3) categories of interventions, and (4) research methodology (e.g.,
randomized trial, cohort study) suggested by the nominator (see
Chapter 5).
• A three-round voting process to narrow the nominated CER topics
to a final list of 00. Members voted independently based on the
committee-specified criteria and their own values; votes were tallied
to rank each nominated topic (see Chapter 4).
• Committee discussion of the highest-scored topics. After the second
round of voting, the committee had a detailed discussion of the
highest-scored topics. The objective of this discussion was to see if
the committee agreed on the nominator’s intent and also to reframe
some of the nominations to adhere to a common format. The com-
mittee also reached consensus on topics to fill or eliminate gaps in
the portfolio representation. A total of 26 topics were nominated
by the committee. These topics were incorporated into the 129
remaining submitted topics without distinguishing them, providing
a total of 155 unique nominated research topics for consideration
in the third round of voting.
PORTFOLIO DISTRIbuTION OF THE PRIORITy TOPICS
The committee’s goal in examining the list of priority research topics
as a portfolio was to include balance across the four dimensions previously
mentioned. A balanced CER portfolio not only studies those diseases and
conditions with the greatest effects on the health of the U.S. population,
but also includes rare diseases and conditions that disproportionately and
seriously affect subgroups of the population (such as women, minorities,
and different groups across the age continuum). The committee sees great
value in extending the concept of drug-to-drug comparisons to a variety of
interventions including tests to screen for or monitor disease (e.g., imaging
for cancer or during normal pregnancy), surgical techniques (e.g., closed
vs. open procedures), and therapeutic alternatives (e.g., medical therapy
vs. surgery vs. radiotherapy for prostate cancer). Additionally, CER that
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SUMMARY
examines different means of delivering health care was considered to be an
important determinant of quality and was incorporated into the options
for intervention.
Finally, CER priorities should be balanced in the primary method-
ologies employed to conduct them: systematic reviews, database research,
observational studies, and randomized trials. There are some studies that
can be completed in the short term with relatively minimal resources, but
other studies will require a longer time frame and a substantial investment
of resources. The committee was charged with developing a portfolio of
topics that would lead to an appropriate expenditure of the $400 million
for CER under the ARRA time frame. Determination of the specific design,
questions to be answered by each individual research project, and meth-
odology, as defined by the potential researcher, will determine the research
costs; however, this task is well beyond the scope of this committee. The
committee sought balance in the methodologies proposed by the nomina-
tors for all 100 priority topics and determined that they were reasonably
well balanced across the four major study methodologies.
Systematic review of existing literature is a relatively inexpensive and
rapidly performed methodology when compared with other methods. It
can identify both information gaps requiring new data generation as well
as areas in which sufficient data exist to establish best practices. Research
using established databases and registries can be undertaken in a reasonable
time frame, inexpensively, and can generate new hypotheses and identify
major health care gaps. The generation of new information, either through
initiation of new databases or prospective observational studies or through
prospective, randomized controlled trials is far more expensive and time
consuming, but is often necessary to provide sufficient evidence of what
works best and for whom. Thus, the committee balanced the types of study
designs so that many studies could be conducted within the time period
identified in ARRA.
An interactive file of the list of priority topics is available on the report
website at www.iom.edu/cerpriorities. Using this file, readers can sort the
list of topics by various portfolio characteristics such as research area, study
population, or type of intervention.
RECOMMENDATIONS FOR A RObuST
NATIONAL CER ENTERPRISE
Based on stakeholder input and its own deliberations, the committee
concluded that the country needs a robust CER infrastructure—referred to
throughout as the “CER Program”—to sustain CER well into the future,
including carrying out the research recommended in this report and study-
ing new topics identified by future priority setting. The committee’s list of
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INITIAL NATIONAL PRIORITIES FOR CER
100 priority topics responds to the requirements of ARRA to advise the
Secretary on how to distribute CER funds from the bill. In addition, the list
could be useful beyond the $400 million appropriated to the Secretary by
influencing the distribution of funds by NIH, AHRQ, and other agencies
that fund CER. The list is not sufficient, however, to ensure the needs of a
future in which new interventions and new diseases will mandate new pri-
orities for CER. The committee’s examination of previous priority-setting
efforts and its study of the nominated research topics conveyed through
its questionnaire led it to conclude that CER must be an ongoing process.
Health care is dynamic; new diseases and health needs can arise suddenly
and other health problems might become insignificant when a treatment is
found. As new CER produces new evidence and closes gaps in evidence,
CER might need to take new directions. A continuous process is necessary
to update funding priorities as conditions change and the impact of previ-
ous CER becomes evident (see Chapter 4 for discussion of Recommenda-
tions 1 through 4).
Recommendation 1: Prioritization of CER topics should be a sustained
and continuous process, recognizing the dynamic state of disease, inter-
ventions, and public concern.
The committee acknowledges the critical role that the general public
and other stakeholders played in this current report and their potential to
enhance CER in the future. CER generates results that bear directly on deci-
sions in which individual patients play an active role. Active involvement of
consumers, patients, and caregivers is essential to identifying CER topics of
real concern to them as well as for suggesting criteria for the prioritization
process that reflect public goals and values.
Recommendation 2: Public (including consumers, patients, and care-
givers) participation in the priority-setting process is imperative to
provide transparency in the process and input to delineating research
questions.
The committee noted that more complete background information
about the suggested research topics would have substantially enhanced its
prioritization process. A national CER enterprise should, on an ongoing
basis, collate national data concerning the significance of diseases and con-
ditions as well as information about current research gaps and redundancies
related to the specific research topics under consideration. The committee
found that the descriptions of research topics were often difficult to under-
stand; an opportunity for a priority-setting body to interrogate CER topic
nominators would help to clarify the nominator’s intent.
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SUMMARY
Recommendation 3: Consideration of CER topics requires the devel-
opment of robust, consistent topic briefs providing background infor-
mation, current practice, and research status of the condition and its
interventions.
The committee concluded that a high level of transparency is essential
for setting priorities for expending public funds on research from which the
public expects so much. Given the magnitude of public investment in CER,
a rolling evaluation of the selection and prioritization processes, as well as
the return on investment of prior CER research by application throughout
the health system should be incorporated in the prioritization process to
ensure quality improvement.
Recommendation 4: Regular reporting of the activities and recommen-
dations of the prioritizing body is necessary to evaluate the portfolio’s
distribution, its impact for discovery, and its translation into clinical care
in order to provide a process for continuous quality improvement.
The committee’s work, including stakeholder input, revealed the scope
of research infrastructure needed to support CER in its goal of improving
health care decisions and their implementation. The committee does not
attempt to fill in all the details, but it concludes that the country must have
a federal organizational infrastructure with appropriate responsibility and
authority to coordinate the prioritization process, support the development
of necessary databases and registries, fund the training of needed research-
ers, conduct the research, and support a vigorous translational effort to
help bring research findings into everyday clinical practice. Without federal
support for an infrastructure to coordinate the national CER effort, all the
CER that the committee identified as high priority is unlikely to occur (see
Chapter 6 for a discussion of infrastructure issues).
Objectivity will be central to the public’s trust and confidence in the
integrity of the CER Program. CER is as vulnerable to bias and conflict of
interest as any other area of medical research. A 2009 IOM report, Con-
flict of Interest in Medical Research, Education, and Practice, recommends
principles to inform the design of policies to identify, limit, and manage
conflicts of interest in health care research. The committee urges that the
CER Program be constituted and managed in accordance with the recom-
mendations of this report.
Recommendation 5: The HHS Secretary should establish a mechanism—
such as a coordinating advisory body—with the mandate to strategize,
organize, monitor, evaluate, and report on the implementation and
impact of the CER Program.
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INITIAL NATIONAL PRIORITIES FOR CER
A central focus on the patient is fundamental to high-quality health
care. To meet the requirement of patient-centeredness, respect for individual
patients’ unique needs, beliefs, and values must drive the development of
the field of CER and the application of its findings to patient care. Con-
sumers, patients, and caregivers have a key role to play in informing and
framing CER. They typically have different perspectives from researchers,
and there is strong evidence that many consumers—but not all—want to
be involved in decision making about their care. Involving them in CER
will help to keep the research relevant and applicable to real-world set-
tings. Also, if consumers, patients, and caregivers are engaged and informed
about CER activities, they are more likely to trust the research findings and
insist that their own care take account of the results.
Recommendation 6: The CER Program should fully involve consum-
ers, patients, and caregivers in key aspects of CER, including strategic
planning, priority setting, research proposal development, peer review,
and dissemination.
• The CER Program should develop strategies to reach out to, engage,
support, educate, and, as necessary, prepare consumers, patients,
and caregivers for leadership roles in these activities.
• The CER Program should also encourage broad participation in
CER in order to create a representative evidence base that could help
identify health disparities and inform decisions by patients in special
population groups.
CER comprises a broad spectrum of established and emerging research
methods including clinical trials, observational studies, and systematic
reviews of existing evidence. There is a significant need for better research
methods. Current study designs—experimental and nonexperimental—must
be refined to ensure scientific rigor. Clinical trials will always be essential to
CER, but more efficient, larger, simpler, and pragmatic designs are needed. In
systematic reviews, for example, research is needed on how to identify and
use evidence from observational studies on intervention effectiveness, and
also on how to assess a heterogeneous body of evidence. New analytic tech-
niques are needed to evaluate the effects of bias due to confounding when
assessing comparative effectiveness using large observational datasets.
Recommendation 7: The CER Program should devote sufficient re-
sources to research and innovation in the methods of CER, including
the development of methodological guidance for CER study design
such as the appropriate use of observational data and more informa-
tive, practical, and efficient clinical trials.
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SUMMARY
CER should also draw from analyses of existing data, such as that held
by payers, health care delivery systems, and electronic health records. How-
ever, if the CER enterprise is to harness the rich potential of these data, it
must protect the privacy and maintain the security of patient data, develop
efficient means for linking data from multiple databases, and engage hold-
ers of large datasets such as health insurers, health care delivery systems,
and health care providers.
Recommendation 8: The CER Program should help to develop large-
scale, clinical and administrative data networks to facilitate better use
of data and more efficient ways to collect new data to inform CER.
• The CER Program should ensure that CER researchers and institu-
tions consistently adhere to best practices to protect privacy and
maintain security.
• The CER Program should support the development of methodolo-
gies for linking patient-level data from multiple sources.
• The CER Program should encourage data holders to participate in
CER and provide incentives for cooperation and maintaining data
quality.
ARRA’s infusion of federal funds into CER will stress the limited ca-
pacity of the current CER workforce. AHRQ’s CER appropriation alone
increased tenfold. Whether the current research workforce can meet the
human resource demands of the $1.1 billion ARRA appropriation for
CER is uncertain. A significant increase in CER activity will certainly cre-
ate a substantial need for experts in biostatistics, epidemiology, systematic
reviews (including meta-analysis), clinical trials (including head-to-head
effectiveness trials), statistical modeling, observational analytic methods,
use of analysis of large datasets, cost-effectiveness analysis, clinical out-
comes research, and communication of research findings. The methods
of CER must advance, which will require training and career support for
methodologists.
Recommendation 9: The CER Program should develop and support
the workforce for CER to ensure the nation’s capacity to carry out the
CER mission. Important next steps include the following:
• Development of a strategic plan for research workforce development.
• Long-term, sufficient funding for early career development including
expanding grants for graduate and postgraduate training opportuni-
ties in comparative effectiveness methods as well as career develop-
ment grants and mid-career merit awards.
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0 INITIAL NATIONAL PRIORITIES FOR CER
The substantial geographic variability in health care delivery suggests
that physicians differ in what they consider to be “best practice.” By discov-
ering what works best, for whom, and under what circumstances, CER has
the potential to narrow the spectrum of what health professionals consider
to be best practice. Health care professionals and patients should be able to
use CER results to make informed decisions based on the best available evi-
dence, the patients’ preferences, and the patient’s unique characteristics.
However, an ambitious research enterprise alone will not improve
health care in the United States without significant attention to high fidel-
ity translation of knowledge into practice. At present, the translation of
research findings into practice is slow and incomplete. Many barriers exist:
perverse reimbursement incentives, physician perceptions about patients’
expectations, and patients’ concerns about denials of care or their reluc-
tance to question clinicians. The CER Program should require researchers
to publish all federally funded CER studies and make the research avail-
able to the public. Moreover, research into knowledge translation must be
a high priority.
Recommendation 10: The CER Program should promote rapid adop-
tion of CER findings and conduct research to identify the most effective
strategies for disseminating new and existing CER findings to health
care professionals, consumers, patients, and caregivers and for helping
them to implement these results in daily clinical practice.
