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Appendix B
Stakeholder Questionnaire
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-3
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
Welcome to the Institute of Medicine's Questionnaire on Comparative Effec-
tiveness Research Priorities
BACKGROUND: The Institute of Medicine (IOM) requests your input for consideration of priori-
ties for comparative effectiveness research (CER) as called for in the American Recovery and
Reinvestment Act of 2009 (Stimulus Bill). In addition to allocating $400 million to the Secretary
of Health and Human Services for CER, the legislation mandates that the IOM produce and
submit a consensus report by June 30, 2009, that provides specific recommendations to Con-
gress and the Secretary for expenditure of these funds. The legislation also requires the IOM
committee to solicit and consider public input as it develops its recommendations.
This questionnaire is a primary vehicle by which the committee will collect information on the
priorities of all stakeholders in health care (e.g., patients, consumers, providers, state and fed-
eral agencies, employers, manufacturers, policy makers).
PUBLIC INPUT OPPORTUNITIES:
Questionnaire: This questionnaire will be active from March 6 - March 27, 2009.
All responses will be compiled into a database that will be reviewed by the commit-
tee. Please note that with the exception of individuals' email addresses and phone
numbers, all responses will also be placed in a public access file as required by fed-
eral legislation. If you choose to omit contact information, your response will still
be given full consideration.
Public Meeting: A public meeting will be convened on March 20, 2009 in Wash-
ington, DC, during which stakeholders, including members of the public, will be in-
vited to present to the committee, as time allows. Information and registration in-
structions are provided at the committee's website (www.iom.edu/cerpriorities).
SAVING AND SUBMITTING YOUR RESPONSE:
Saving: You will be able to leave the questionnaire and then return to it later;
however, you must complete an entire page and move on to the next page to save
your work.
Submitting: When you have completed the questionnaire you will see a confirma-
tion page and will then be taken back to the committee's website where you can
find additional information about the committee's work.
Printing: Please note that if you wish to print your responses, you must print each
page when you are finished with it by using your web browser’s print menu op-
tions.
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INITIAL NATIONAL PRIORITIES FOR
B-4
APPENDIX B CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
Instructions
In the next 3 pages, you will have the opportunity to submit up to 3 comparative effectiveness
studies for the committee's consideration. Please rank your suggested CER priorities as first,
second, and third by entering them in that order.
Once you have submitted your top 3 priorities, you will be asked what criteria you feel are most
important in establishing a national set of priorities for CER. You will be asked to select and
rank criteria (e.g., disease burden, disease severity, variation in care, cost, public interest, in-
formation gap) as well as identify other criteria for the committee to consider.
Lastly, you will be asked to provide recommendations to the committee regarding what new or
enhanced capacities and infrastructure are needed to sustain a national CER enterprise.
CER Scope
The committee's working definition for CER is: “The generation and synthesis of evidence that com-
pares the effectiveness of alternative methods to prevent, diagnose, treat, monitor, and improve de-
livery of care for a clinical condition. The purpose of CER is to assist patients, clinicians, purchasers,
and policy makers in making informed health decisions.”
Please consider the following as you develop your suggested priority areas for study:
Study Population: Identify a study population by disease entity, condition, susceptible
population, or population affected.
Alternative Interventions: Comparators might include systems of care as well as
specific interventions to address the prevention, diagnosis, treatment, monitoring, or
delivery of care. One comparator could be the current standard of care or usual care.
Outcome of Interest: Please identify the health related risk, side effect or harm of
greatest concern, and/or the health related benefit of greatest interest (e.g., patient-
reported outcomes, surrogate endpoints [such as change in tumor size or laboratory pa-
rameters], clinical event, death).
Study Methods: CER can include analyses of existing data, observational studies (e.g.,
Framingham study), prospective trials, and systematic reviews of published studies.
Overview of requested information
We anticipate a high response rate to this questionnaire. Therefore, to facilitate the committee's re-
view of each response, we ask that you characterize each of your priorities by specifying the follow-
ing:
A single sentence that frames your research question (include condition, compari-
son, and outcome of interest)
Justification for why the question should be a national priority
Area to be studied (e.g., cardiovascular and peripheral vascular disease, racial and
ethnic disparities in health care, nutrition)
Comparators (i.e., include a comparison of two or more alternatives for the preven-
tion, diagnosis, treatment, monitoring, or delivery of care for a clinical condition)
Study design (e.g., analyses of existing data [systematic review], observational stud-
ies [such as the Framingham study], and prospective trials)
Study population (e.g., children/adolescents, elderly, special populations)
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-5
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
CER Priority (1 of 3)
Describe your first priority by answering the following questions.
*1. Please submit a single sentence that frames your research question. We request
that your response specify a condition; a comparison of 2 or more alternatives for the
prevention, diagnosis, treatment, monitoring, or delivery of care of the condition; and
the study outcome(s) to be assessed (e.g., patient-reported outcomes, surrogate
endpoints [such as change in tumor size or laboratory parameters], clinical event,
death).
For example: Compare the effectiveness of identifying pre-malignant lesions and early colon
cancer by either virtual or actual colonoscopy in individuals at low to moderate risk of colon
cancer.
*2. Please provide justification for why this study should be a priority. Please
consider the following criteria in your justification:
disease burden
increasing prevalence
morbidity and mortality
variability in care
cost
information gap (e.g., little is know about this topic)
funding gap (e.g., minimal research is being done on this topic)
public interest
controversy
disproportionate impact by subpopulation
potential to act on the information once generated
utility of the answer for decision making
Please limit your response to 100 words. To verify that your text is within the limits,
use this word count link.
For example: Screening for colon cancer by colonoscopy identifies pre-malignant lesions and
early cancers allowing for both prevention and cure. Many people needlessly delay this
procedure because of perceived inconveniences or pain, and thus miss the potential benefits.
Virtual colonoscopy may increase the number of people undergoing screening compared to the
current number having colonoscopy, increase the number of pre-malignant and early cancers
found, and reduce the death rate from colon cancer.
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APPENDIX B
B-6 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
3. Please specify the primary area to be studied in the left hand column. If your study
involves multiple conditions, please mark the associated conditions in the right hand
column. If you are unsure of how to characterize the condition described in questions
1 and 2 above, please indicate the specific condition/disease in the "other" box.
Comorbidity
Primary Condition
(associated conditions—you
(please select only one)
may select more than one)
Sexual Function and Reproductive Disorders
Skin Disorders
Cardiovascular and Peripheral Vascular Disease
Eyes, Ears, Nose, and Throat Disorders
Gastrointestinal System Disorders
Immune System, Connective Tissue, and Joint Disorders
Kidney and Urinary Tract Disorders
Pancreatic Disorders
Endocrinology and Metabolism Disorders (includes Dia-
betes)
Infectious Diseases (including HIV/AIDS)
Liver and Biliary Tract Disease
Musculoskeletal Disorders
Neurologic Disorders
Oncology and Hematology
Psychiatric Disorders
Respiratory Disease
Trauma, Emergency Medicine, Critical Care Medicine
Medical Aspects of Bioterrorism
Nutrition (includes Obesity)
Complementary and Alternative Medicine
Alcoholism, Drug Dependency, and Overdosage
Functional Limitation and Disabilities
Birth and Developmental Disorders
Genetics and Disease
Regenerative Medicine (e.g., stem cell research)
Safety and Quality of Health Care (e.g., delivery and
organization of care)
Racial and Ethnic Disparities
Women's Health (including disorders during pregnancy)
Pediatrics
Geriatric Medicine
Palliative and End-of-Life Care
Other (please specify)
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-7
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
4. For this priority, please select the type(s) of interventions to be compared. You
may select more than one.
Prevention Procedures – Surgery
Testing, monitoring, and evaluation (e.g., lab, Devices (e.g., artificial joint, spinal cage,
imaging, psychosocial and functional assess- stent, pacemaker, breast implants)
ments)
Systems of Care (e.g., organization, man-
Standard of care/usual care (please elaborate agement, delivery of healthcare services)
on the specific elements of standard of care in
Provider/Patient Relationships (e.g., counsel-
the "other" box)
ing, education)
Treatment – Behavioral
Treatment pathways (e.g., strategy for early
Treatment – Alternative
stage prostate cancer, team approach to dia-
betes care vs. general care)
Treatment – Pharmacological
Other (please specify)
5. For the topic you suggested, which of the following types of research would be
most effective in providing the needed evidence?
Synthesis of existing evidence (e.g., qualitative review, meta-analysis)
Primary research using existing health care databases (might include electronic health record data or
other clinical data)
Primary research using prospective data collection without randomization (e.g., observational study,
registry)
Primary research through a prospective randomized trial
Other (please specify)
6. Please describe the study population(s). You may select more than one.
Population at Large Elderly
Men Long-term care (institutionalized and home
care)
Women
Ethnic sub-populations only
Children/Adolescents
Rare Diseases
Adults (excluding elderly)
Special Populations (e.g., pregnant women,
Adults (including elderly)
prisoners, low income, persons with disability)—
please elaborate in the "other" box
Other (please specify)
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APPENDIX B
B-8 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
*7. Would you like to submit another priority?
Yes
No
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-9
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
CER Priority (2 of 3)
Describe your second priority by answering the following questions.
*8. Please submit a single sentence that frames your research question. We request
that your response specify a condition; a comparison of 2 or more alternatives for the
prevention, diagnosis, treatment, monitoring, or delivery of care of the condition; and
the study outcome(s) to be assessed (e.g., patient-reported outcomes, surrogate
endpoints [such as change in tumor size or laboratory parameters], clinical event,
death).
*9. Please provide justification for why this study should be a priority. Please
consider the following criteria in your justification:
disease burden
increasing prevalence
morbidity and mortality
variability in care
cost
information gap (e.g., little is know about this topic)
funding gap (e.g., minimal research is being done on this topic)
public interest
controversy
disproportionate impact by subpopulation
potential to act on the information once generated
utility of the answer for decision making
Please limit your response to 100 words. To verify that your text is within the limits,
use this word count link.
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APPENDIX B
B-10 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
10. Please specify the primary area to be studied in the left hand column. If your
study involves multiple conditions, please mark the associated conditions in the right
hand column. If you are unsure of how to characterize the condition described in
questions 8 and 9 above, please indicate the specific condition/disease in the "other"
box.
Comorbidity
Primary Condition
(associated conditions—you
(please select only one)
may select more than one)
Sexual Function and Reproductive Disorders
Skin Disorders
Cardiovascular and Peripheral Vascular Disease
Eyes, Ears, Nose, and Throat Disorders
Gastrointestinal System Disorders
Immune System, Connective Tissue, and Joint Disorders
Kidney and Urinary Tract Disorders
Pancreatic Disorders
Endocrinology and Metabolism Disorders (includes Dia-
betes)
Infectious Diseases (including HIV/AIDS)
Liver and Biliary Tract Disease
Musculoskeletal Disorders
Neurologic Disorders
Oncology and Hematology
Psychiatric Disorders
Respiratory Disease
Trauma, Emergency Medicine, Critical Care Medicine
Medical Aspects of Bioterrorism
Nutrition (includes Obesity)
Complementary and Alternative Medicine
Alcoholism, Drug Dependency, and Overdosage
Functional Limitation and Disabilities
Birth and Developmental Disorders
Genetics and Disease
Regenerative Medicine (e.g., stem cell research)
Safety and Quality of Health Care (e.g., delivery and
organization of care)
Racial and Ethnic Disparities
Women's Health (including disorders during pregnancy)
Pediatrics
Geriatric Medicine
Palliative and End-of-Life Care
Other (please specify)
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0 INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-11
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
11. For this priority, please select the type(s) of interventions to be compared. You
may select more than one.
Prevention Procedures – Surgery
Testing, monitoring, and evaluation (e.g., lab, Devices (e.g., artificial joint, spinal cage,
imaging, psychosocial and functional assess- stent, pacemaker, breast implants)
ments)
Systems of Care (e.g., organization, man-
Standard of care/usual care (please elaborate agement, delivery of healthcare services)
on the specific elements of standard of care in
Provider/Patient Relationships (e.g., counsel-
the "other" box)
ing, education)
Treatment – Behavioral
Treatment pathways (e.g., strategy for early
Treatment – Alternative
stage prostate cancer, team approach to dia-
betes care vs. general care)
Treatment – Pharmacological
Other (please specify)
12. For the topic you suggested, which of the following types of research would be
most effective in providing the needed evidence?
Synthesis of existing evidence (e.g., qualitative review, meta-analysis)
Primary research using existing health care databases (might include electronic health record data or
other clinical data)
Primary research using prospective data collection without randomization (e.g., observational study,
registry)
Primary research through a prospective randomized trial
Other (please specify)
13. Please describe the study population(s). You may select more than one.
Population at Large Elderly
Men Long-term care (institutionalized and home
care)
Women
Ethnic sub-populations only
Children/Adolescents
Rare Diseases
Adults (excluding elderly)
Special Populations (e.g., pregnant women,
Adults (including elderly)
prisoners, low income, persons with disability)—
please elaborate in the "other" box
Other (please specify)
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APPENDIX B
B-12 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
*14. Would you like to submit another priority?
Yes
No
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-13
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
CER Priority (3 of 3)
Describe your third priority by answering the following questions.
*15. Please submit a single sentence that frames your research question. We request
that your response specify a condition; a comparison of 2 or more alternatives for the
prevention, diagnosis, treatment, monitoring, or delivery of care of the condition; and
the study outcome(s) to be assessed (e.g., patient-reported outcomes, surrogate
endpoints [such as change in tumor size or laboratory parameters], clinical event,
death).
*16. Please provide justification for why this study should be a priority. Please
consider the following criteria in your justification:
disease burden
increasing prevalence
morbidity and mortality
variability in care
cost
information gap (e.g., little is know about this topic)
funding gap (e.g., minimal research is being done on this topic)
public interest
controversy
disproportionate impact by subpopulation
potential to act on the information once generated
utility of the answer for decision making
Please limit your response to 100 words. To verify that your text is within the limits,
use this word count link.
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APPENDIX B
B-14 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
17. Please specify the primary area to be studied in the left hand column. If your
study involves multiple conditions, please mark the associated conditions in the right
hand column. If you are unsure of how to characterize the condition described in
questions 15 and 16 above, please indicate the specific condition/disease in the
"other" box.
Comorbidity
Primary Condition
(associated conditions—you
(please select only one)
may select more than one)
Sexual Function and Reproductive Disorders
Skin Disorders
Cardiovascular and Peripheral Vascular Disease
Eyes, Ears, Nose, and Throat Disorders
Gastrointestinal System Disorders
Immune System, Connective Tissue, and Joint Disorders
Kidney and Urinary Tract Disorders
Pancreatic Disorders
Endocrinology and Metabolism Disorders (includes Dia-
betes)
Infectious Diseases (including HIV/AIDS)
Liver and Biliary Tract Disease
Musculoskeletal Disorders
Neurologic Disorders
Oncology and Hematology
Psychiatric Disorders
Respiratory Disease
Trauma, Emergency Medicine, Critical Care Medicine
Medical Aspects of Bioterrorism
Nutrition (includes Obesity)
Complementary and Alternative Medicine
Alcoholism, Drug Dependency, and Overdosage
Functional Limitation and Disabilities
Birth and Developmental Disorders
Genetics and Disease
Regenerative Medicine (e.g., stem cell research)
Safety and Quality of Health Care (e.g., delivery and
organization of care)
Racial and Ethnic Disparities
Women's Health (including disorders during pregnancy)
Pediatrics
Geriatric Medicine
Palliative and End-of-Life Care
Other (please specify)
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-15
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
18. For this priority, please select the type(s) of interventions to be compared. You
may select more than one.
Prevention Procedures – Surgery
Testing, monitoring, and evaluation (e.g., lab, Devices (e.g., artificial joint, spinal cage,
imaging, psychosocial and functional assess- stent, pacemaker, breast implants)
ments)
Systems of Care (e.g., organization, man-
Standard of care/usual care (please elaborate agement, delivery of healthcare services)
on the specific elements of standard of care in
Provider/Patient Relationships (e.g., counsel-
the "other" box)
ing, education)
Treatment – Behavioral
Treatment pathways (e.g., strategy for early
Treatment – Alternative
stage prostate cancer, team approach to dia-
betes care vs. general care)
Treatment – Pharmacological
Other (please specify)
19. For the topic you suggested, which of the following types of research would be
most effective in providing the needed evidence?
Synthesis of existing evidence (e.g., qualitative review, meta-analysis)
Primary research using existing health care databases (might include electronic health record data or
other clinical data)
Primary research using prospective data collection without randomization (e.g., observational study,
registry)
Primary research through a prospective randomized trial
Other (please specify)
20. Please describe the study population(s). You may select more than one.
Population at Large Elderly
Men Long-term care (institutionalized and home
care)
Women
Ethnic sub-populations only
Children/Adolescents
Rare Diseases
Adults (excluding elderly)
Special Populations (e.g., pregnant women,
Adults (including elderly)
prisoners, low income, persons with disability)—
please elaborate in the "other" box
Other (please specify)
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APPENDIX B
B-16 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
Criteria for Establishing Priorities
*21. Briefly describe the process used to develop your priorities. Please limit your re-
sponse to 100 words. To verify that your text is within the limits, use this word count
link.
*22. Please rank what criteria you, or your organization view as most important for
setting national priorities for CER.
Criteria in order of importance
1st
2nd
3rd
4th
5th
Other(s) (specify and include rank)
NOTE: The drop down choices for question 22 included the following:
Disease burden
Increasing prevalence
Morbidity and mortality
Variability in care
Cost
Information gap
Funding gap
Public interest
Controversy
Disproportionate impact by subpopulation
Potential to act on the information
Utility of the answer for decision making
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INITIAL NATIONAL PRIORITIES FOR CER
APPENDIX B B-17
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
Additional Input Regarding CER Infrastructure
Effective implementation of CER priorities may require investments in infrastructure. If
you have suggestions on specific investments necessary to support expanded CER re-
search, please submit your recommendations for the committee's consideration.
*23. What are the highest priorities for developing new or enhanced systems, alli-
ances, or capacities to sustain a national comparative effectiveness research enter-
prise? You may select up to four.
Increased workforce training (e.g., clinical researchers, epidemiologists, statisticians, informatics)
Clinical trials support (e.g., ad hoc collaborations)
Clinical data pooling and mining support
Novel method development for data analysis and modeling
Clinical registry development, application, and networking
Creation of robust national registries for tracking both short- and long-term performance of therapeutic
strategies, drugs, or devices
Capacity to use electronic health records for safety and effectiveness monitoring
Inclusion or creation of direct patient data entry (e.g., questionnaires, surveys, personal health records
to provide patient-reported outcomes)
Increased coordination of CER through existing Health and Human Services entities
Creation of an independent institute for CER
24. Additional suggestions for infrastructure development for committee considera-
tion. Please limit your response to 100 words. To verify that your text is within the
limits, use this word count link.
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APPENDIX B
B-18 INITIAL NATIONAL PRIORITIES FOR CER
IOM COMMITTEE ON COMPARATIVE EFFECTIVENESS RESEARCH PRIORITIZATION:
Demographic Information
The following questions will help ensure that the committee has gathered input from a
broad range of perspectives.
25. Please select a description that best describes your role or perspective:
Employer Medical Administrator
Government - Programs (e.g., Medicare, Nonprofit/Policy Institute
Medicaid)
Patient/Family (including family caregiver)
Government - Research
Professional Association
Health Care Provider
Public/Consumer
Health Plan/Insurance Carrier
Researcher
Manufacturer (Device)
Manufacturer (Drug or Biologic)
Other (please specify)
*26. Who are you representing with your response?
Self
Organization
27. Please provide your contact information (optional).
Note that your email address and phone number will not be released to the public. If you
choose to omit contact information, your response will still be given full consideration.
Name:
Organization:
Title:
Email Address:
Phone Number:
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