Comparative Effectiveness Research: Comparative Effectiveness Research: Relevance and Applications to Relevance and Applications to

PharmacyPharmacy

This activity was developed by the American Pharmacists Association and the Agency for Healthcare Research and Quality.

SpeakersSpeakers

Speaker TwoSpeaker TwoGlen T. Schumock, Pharm.D., Glen T. Schumock, Pharm.D.,

M.B.A.M.B.A.Director of the Center for Director of the Center for

Pharmacoeconomic Pharmacoeconomic ResearchResearch

Professor of Pharmacy Practice, Professor of Pharmacy Practice, the University of Illinois at the University of Illinois at ChicagoChicago

Speaker ThreeSpeaker ThreeA. Simon Pickard, B.Sc.Pharm., A. Simon Pickard, B.Sc.Pharm.,

Ph.D. Ph.D. Assistant Director of the Center Assistant Director of the Center

for Pharmacoeconomic for Pharmacoeconomic ResearchResearch

Associate Professor of Pharmacy Associate Professor of Pharmacy Practice, the University of Practice, the University of Illinois at ChicagoIllinois at Chicago

ModeratorModeratorCarmen Kelly, Pharm.D., Carmen Kelly, Pharm.D.,

R.Ph.R.Ph. Pharmacist OfficerPharmacist OfficerAgency for Healthcare Agency for Healthcare

Research and QualityResearch and QualityEffective Health CareEffective Health Care

Speaker OneSpeaker OneScott Smith, R.Ph., M.S.P.H., Scott Smith, R.Ph., M.S.P.H.,

Ph.D.Ph.D. Director of Pharmaceutical Director of Pharmaceutical

Outcomes Research Outcomes Research Agency for Healthcare Agency for Healthcare

Research and QualityResearch and QualityEffective Health Care Effective Health Care

AccreditationAccreditation

APhA is accredited by the Accreditation APhA is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. The provider of continuing pharmacy education. The ACPE Universal Activity Number assigned to the ACPE Universal Activity Number assigned to the

activity by the accredited provider is: activity by the accredited provider is: 202-999-09-224-H04-202-999-09-224-H04-PP. This activity is approved for 1 contact hour of continuing . This activity is approved for 1 contact hour of continuing pharmacy education credit (0.1 CEU). pharmacy education credit (0.1 CEU).

To obtain continuing pharmacy education credit for this To obtain continuing pharmacy education credit for this activity, participants must participate in the entire activity, activity, participants must participate in the entire activity, and complete the online activity evaluation form located on and complete the online activity evaluation form located on www.pharmacist.com/educationwww.pharmacist.com/education by September 23, 2009. A by September 23, 2009. A Statement of Credit will be automatically generated upon Statement of Credit will be automatically generated upon achieving these requirements. There is no fee to achieving these requirements. There is no fee to participate in this activity. participate in this activity.

ACPE Activity Type: Knowledge-Based ACPE Activity Type: Knowledge-Based Initial Release Date: September 9, 2009Initial Release Date: September 9, 2009

DisclosuresDisclosures

Glen T. Schumock, Pharm.D., M.B.A. Glen T. Schumock, Pharm.D., M.B.A. has no has no financial interests or relationships to disclose.financial interests or relationships to disclose.

A. Simon Pickard, B.Sc.Pharm., Ph.D. A. Simon Pickard, B.Sc.Pharm., Ph.D. has no has no financial interests or relationships to disclose.financial interests or relationships to disclose.

Scott Smith, R.Ph., M.S.P.H., Ph.D., and Carmen Scott Smith, R.Ph., M.S.P.H., Ph.D., and Carmen Kelly, Pharm.D, R.Ph.,Kelly, Pharm.D, R.Ph., have no financial interests or have no financial interests or relationships to disclose.relationships to disclose.

AHRQ’s Office of Communications and Knowledge AHRQ’s Office of Communications and Knowledge Transfer staff, The Lewin Group project staff, and Transfer staff, The Lewin Group project staff, and APhA’s editorial staff APhA’s editorial staff declare no conflicts of interest declare no conflicts of interest or financial interests in any product or service or financial interests in any product or service mentioned in this program, including grants, mentioned in this program, including grants, employment, gifts, stock holdings, and honoraria.employment, gifts, stock holdings, and honoraria.

DisclaimerDisclaimer

This presentation and slide set do not represent the This presentation and slide set do not represent the policy of either the Agency for Healthcare Research policy of either the Agency for Healthcare Research and Quality (AHRQ) or the U.S. Department of and Quality (AHRQ) or the U.S. Department of Health and Human Services (DHHS). Health and Human Services (DHHS).

The views expressed herein are those of the The views expressed herein are those of the presenter, and no official endorsement by AHRQ or presenter, and no official endorsement by AHRQ or DHHS is intended or should be inferred. DHHS is intended or should be inferred.

Current information about the Effective Health Care Current information about the Effective Health Care Program should be obtained from AHRQ, and not Program should be obtained from AHRQ, and not from these slides.from these slides.

Learning ObjectivesLearning Objectives

Describe AHRQ’s Comparative Effectiveness Program Describe AHRQ’s Comparative Effectiveness Program and provide an overview of the factors and events and provide an overview of the factors and events leading to the current national interest in comparative leading to the current national interest in comparative effectivenesseffectiveness

Identify scientific evidence-based resources, including Identify scientific evidence-based resources, including clinician and patient guides, produced through AHRQ’s clinician and patient guides, produced through AHRQ’s Comparative Effectiveness Program that are relevant Comparative Effectiveness Program that are relevant for use in their practicefor use in their practice

Understand the process for submitting topics to AHRQ Understand the process for submitting topics to AHRQ for future comparative effectiveness researchfor future comparative effectiveness research

Compare the differences between comparative Compare the differences between comparative effectiveness studies and traditional efficacy studieseffectiveness studies and traditional efficacy studies

Explain the relevance of comparative effectiveness Explain the relevance of comparative effectiveness research to pharmacy practiceresearch to pharmacy practice

Comparative Effectiveness Research Comparative Effectiveness Research and AHRQ’s Effective Health Care and AHRQ’s Effective Health Care

ProgramProgram

Scott Smith, R.Ph., M.S.P.H., Ph.D. Agency for Healthcare Research and Quality

Effective Health Care

Comparative Comparative EffectivenessEffectiveness

“…“…a rigorous evaluation a rigorous evaluation of the impact of different of the impact of different options that are available options that are available

for treating a given for treating a given medical condition for a medical condition for a

particular set of patients.”particular set of patients.”Congressional Budget Office, 2007Congressional Budget Office, 2007

CBO Comparative CBO Comparative Effectiveness DefinitionEffectiveness Definition

Effective Health Care (EHC) Effective Health Care (EHC) Program, 2Program, 2003 – Present

Authorized in 2003 by Section 1013 of the Authorized in 2003 by Section 1013 of the Medicare Prescription Drug, Improvement, and Medicare Prescription Drug, Improvement, and Modernization Act Modernization Act

Conducts objective comparisons of the Conducts objective comparisons of the effectiveness of different health care interventionseffectiveness of different health care interventions

Goal: To support informed health care decisions Goal: To support informed health care decisions by patients, clinicians, and policymakers and by patients, clinicians, and policymakers and improve the quality, effectiveness, and efficiency improve the quality, effectiveness, and efficiency of health care to support evidence-based practiceof health care to support evidence-based practice

Effective Health Care ProgramEffective Health Care Program

To improve the quality, effectiveness, and To improve the quality, effectiveness, and efficiency of health care delivered through efficiency of health care delivered through Medicare, Medicaid, and CHIP programsMedicare, Medicaid, and CHIP programs

– Focus is on what is known Focus is on what is known nownow: ensuring : ensuring programs benefit from programs benefit from pastpast investments in investments in research and what research research and what research gapsgaps are are critical to fillcritical to fill

– Focus is on Focus is on clinical effectivenessclinical effectiveness

Approaches to ResearchApproaches to Research

Synthesizes Synthesizes existing scientific evidence existing scientific evidence

GeneratesGenerates new scientific evidence to new scientific evidence to address gapsaddress gaps

TranslatesTranslates research into plain-language research into plain-language guides guides

Effective Health Care ProgramEffective Health Care Program

Available EHC ProductsAvailable EHC Products

Clinician ResourcesClinician Resources

ConciseConcise ActionableActionable Paired with Paired with

consumer guidesconsumer guides Convey level of Convey level of

uncertainty/certainty uncertainty/certainty of findingsof findings

Priority Conditions for the Priority Conditions for the Effective Health Care ProgramEffective Health Care Program

Arthritis and non-Arthritis and non-traumatic joint disorderstraumatic joint disorders

CancerCancer Cardiovascular disease, Cardiovascular disease,

including stroke and including stroke and hypertensionhypertension

Dementia, including Dementia, including Alzheimer’s DiseaseAlzheimer’s Disease

Depression and other Depression and other mental health disordersmental health disorders

Developmental delays, Developmental delays, attention-deficit attention-deficit hyperactivity disorder, hyperactivity disorder, and autism and autism

Diabetes mellitusDiabetes mellitus Functional limitations Functional limitations

and disabilityand disability Infectious diseases, Infectious diseases,

including HIV/AIDSincluding HIV/AIDS ObesityObesity Peptic ulcer disease Peptic ulcer disease

and dyspepsiaand dyspepsia Pregnancy, including Pregnancy, including

pre-term birthpre-term birth Pulmonary Pulmonary

disease/asthmadisease/asthma Substance abuseSubstance abuse

Comparative Effectiveness Reviews: Comparative Effectiveness Reviews: New and Coming Soon…New and Coming Soon…

Comparative Effectiveness ReviewsComparative Effectiveness Reviews – Stable Ischemic Heart Disease (draft report)Stable Ischemic Heart Disease (draft report)– Particle Beam Therapies for CancerParticle Beam Therapies for Cancer (technical (technical

briefbrief) ) – Stereotactic Radiosurgery (technical briefStereotactic Radiosurgery (technical brief) ) – Heart Valve Replacement (technical briefHeart Valve Replacement (technical brief) ) – Core Needle Breast BiopsyCore Needle Breast Biopsy– Lipid-Modifying AgentsLipid-Modifying Agents– Radiofrequency Catheter Ablation for Atrial Radiofrequency Catheter Ablation for Atrial

FibrillationFibrillation– Chemotherapy Agents in the Prevention of Breast Chemotherapy Agents in the Prevention of Breast

CancerCancer– 9 Comparative Effectiveness Review Updates9 Comparative Effectiveness Review Updates

Summary Guides: New and Summary Guides: New and Coming Soon…Coming Soon…

Summary guidesSummary guides - Osteoarthritis of the Knee (consumer and clinician)- Osteoarthritis of the Knee (consumer and clinician)

- Gestational Diabetes (consumer and clinician)- Gestational Diabetes (consumer and clinician)

- Induction of Labor (consumer and clinician)- Induction of Labor (consumer and clinician)

- Particle Beam (policymaker)- Particle Beam (policymaker)

Spanish translations are available for 9 Spanish translations are available for 9 summary guidessummary guides

Coming Soon… Coming Soon… (cont.)(cont.)

Interactive Decision AidsInteractive Decision Aids

Important Role of Outside Important Role of Outside InputInput

Nomination of research topicsNomination of research topics Input on research questionsInput on research questions Comment on draft reportsComment on draft reports Focus test translation productsFocus test translation products Comments on overall program direction and Comments on overall program direction and

quality improvementquality improvement

Topic Nomination FormTopic Nomination Form

4. Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

5. Describe any health-related risks, side effects, or harms that you are concerned

about.

APPROPRI ATENESS FOR EHC PROGRAM 6. Does your question include a health care drug, intervention, device, or

technology available (or likely to be available) in the U.S.? ? Yes ? No ? Unsure

7. Which priority area(s) and population(s) does this topic apply to? (check all that

apply) EHC Priority Conditions (updated in 2008)

? Arthritis and nontraumatic joint disorders

? Cancer ? Cardiovascular disease, including

stroke and hypertension ? Dementia, including Alzheimer’s

disease ? Depression and other mental health

disorders ? Developmental delays, attention

deficit hyperactivity disorder, and autism

? Diabetes mellitus ? Functional limitations and disability ? Infectious diseases, including

HIV/AIDS ? Obesity ? Peptic ulcer disease and dyspepsia ? Pregnancy, including preterm birth

AHRQ Priority Populations ? Low income groups ? Minority groups ? Women ? Children ? Elderly ? Individuals with

special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care

Federal Health Care Program

? Medicaid ? Medicare ? State Children’s

Health Insurance Program (SCHIP)

TOPI C NOMI NATION

1. Your Nomination –Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

Examples: For patients with low bone density, what is the comparative effectiveness of exercise

versus drug therapy to treat or prevent osteoporosis? For adult patients with a major depressive disorder (MDD), what are the comparative

risks and benefits of older versus newer antidepressants? In patients with cystic fibrosis, what is the effectiveness of recombinant human growth

hormone (rhGH) in improving intermediate health outcomes, such as pulmonary function and nutritional status?

2. Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.) ? No ? Yes If yes, explain the specific technologies, devices, drugs, or

interventions you would like to see compared:

Examples: Calcium versus biphosphonates for the prevention of vertebral fractures Core needle biopsy versus open surgical biopsy for diagnosing breast lesions Antireflux medication versus diet and exercise for the control of acid reflux symptoms

3. What patients or group(s) of patients does your question apply to? (Please

include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

TOPIC NOMINATION FORM

Suggesting a Topic for Effective Health Care Research

The Effective Health Care Program (EHC) researches available health care tests and treatments to determine whether there are significant advantages or disadvantages with different approaches. The results of this comparative effectiveness research can help people make better decisions about what health care they want to have, and can help clinicians and health care purchasers to focus on the best tests and treatments. SEE A LIST OF EXISTI NG EHC PRODUCTS The process of identifying, selecting, and developing important research topics for research reviews or new research is a key aspect of the EHC program. The process is enhanced by strong involvement of stakeholders, maintaining transparency and public accountability, and striving for continuous self-evaluation and improvement. The program uses an established set of criteria to guide the process of topic selection. This form is organized by these selection criteria.

SEE HOW YOUR TOPI C WILL BE EVALUATED

We would like to understand important aspects of the health care service you are interested in, including to whom it applies, what benefits or harms are of greatest interest, and with what other health care services or tests you think it should be compared. Your answers to these questions will help us phrase your suggestion as one or more research questions that could be answered through AHRQ EHC comparative effectiveness or effectiveness products. LEARN MORE ABOUT EHC RESEARCH REVIEWS LEARN MORE ABOUT EHC NEW RESEARCH To nominate a topic for research in this program, please fill in the form below as completely as possible and click on “submit” at the end. If you prefer to submit a paper copy, you may print the form and mail it. If you have any supporting documents you would like to include with your nomination, please click here to upload the document(s). All topic nominations, including those submitted on paper, will appear in the public reading room. Click here to view an example of a completed form.

Thank you for participating in the program!

http://effectivehealthcare.ahrq.gov/aboutUs.cfm?abouttype=program#Topic

Selection CriteriaSelection Criteria

ImportanceImportance

DuplicationDuplication

FeasibilityFeasibility

PotentialPotential ImpactImpact

AppropriatenessAppropriateness 3 criteria3 criteria

7 criteria7 criteria

1 criterion1 criterion

1 criterion1 criterion

6 criteria6 criteria

TransparencyTransparency

Nominations are posted Nominations are posted on the Web siteon the Web site

Research selection Research selection process including process including selection criteria are selection criteria are posted on the Web siteposted on the Web site

Moving toward posting Moving toward posting all of the final decisions all of the final decisions on the Web siteon the Web site

Results of Topic Selection Process & Next Steps

Sleep apnea treatment will go forward for refinement as a comparative effectiveness or effectiveness review. The scope of this topic, including populations, interventions, comparators, and outcomes, will be further developed in the refinement phase.

When key questions have been drafted, they will be posted on the AHRQ web site and open for public comment. To sign up for notification when this and other Effective Health Care Program topics are up for public comment, please go to http://effectivehealthcare.ahrq.gov/getInvolved.cfm?involvetype=subscribe.

Topic Description Nominators: 2 public payers, 1 health care professional association

Nomination Summary:

This topic represents nominations, respectively, by three nominating groups. The treatments for sleep apnea covered in these nominations include continuous positive airway pressure (CPAP), oral appliances, uvulopalatopharyngoplasty (UPPP), radiofrequency ablation (RFA), bariatric surgery, counseling, and lifestyle modifications. In addition, one nominator expressed interest in patients with comorbidities (including pulmonary hypertension and heart failure). Outcome measures include mortality reduction, quality of life (objectively measured), functional improvement, and reduction in comorbid conditions or resolution of these conditions.

Key Questions from Nominators:

Nominator 1 (public payer): For each of the treatment options for obstructive sleep apnea (CPAP, oral appliances, UPPP, and RFA), we ask the following questions:

1. Does the technology improve quality of life and function, and if so, to what extent and at what cost?

2. For which presenting symptoms of obstructive sleep apnea (OSA) does the technology result in an improvement, and if so, to what extent and at what cost?

3. Does the technology prevent or reduce comorbidities associated with OSA, and if so, to what extent and at what cost?

4. Does the technology prevent or reduce mortality, and if so, to what extent and at what cost?

Nominator 2 (public payer):

1. What other modalities of treatment, including weight loss, surgery, and oral appliances, would be better tolerated and more efficacious than CPAP?

Treatment of Sleep Apnea Nomination Summary Document

How To Stay InformedHow To Stay Informed

EHC Web SiteEHC Web Site E-Mail Notices Through ListservE-Mail Notices Through Listserv

– Select to receive information on a range of topics:Select to receive information on a range of topics:- Arthritis and nontraumatic joint disordersArthritis and nontraumatic joint disorders- Cancer Cancer - Cardiovascular disease, including stroke and hypertensionCardiovascular disease, including stroke and hypertension- Dementia, including Alzheimer's DiseaseDementia, including Alzheimer's Disease- Depression and other mental health disordersDepression and other mental health disorders- Developmental delays, attention-deficit hyperactivity disorder, and Developmental delays, attention-deficit hyperactivity disorder, and

autismautism- Diabetes mellitus Diabetes mellitus - Functional limitations and disability Functional limitations and disability - Infectious diseases, including HIV/AIDS Infectious diseases, including HIV/AIDS - Obesity Obesity - Peptic ulcer disease and dyspepsia Peptic ulcer disease and dyspepsia - Pregnancy, including preterm birth Pregnancy, including preterm birth - Pulmonary disease/asthma Pulmonary disease/asthma - Substance abuseSubstance abuse

Polling QuestionPolling Question

Are you currently using, or planning to Are you currently using, or planning to use, AHRQ's Comparative Effectiveness use, AHRQ's Comparative Effectiveness reports in your setting?reports in your setting?

a.a. Yes, currently Yes, currently

b.b. Yes, within the next 12 monthsYes, within the next 12 months

c.c. Yes, after 12 months or laterYes, after 12 months or later

d.d. NoNo

Comparative Effectiveness Research: Comparative Effectiveness Research: Relevance and Applications to Relevance and Applications to

PharmacyPharmacy

Glen T. Schumock, Pharm.D., M.B.A.Glen T. Schumock, Pharm.D., M.B.A.A. Simon Pickard, B.Sc.Pharm., Ph.D.A. Simon Pickard, B.Sc.Pharm., Ph.D. University of Illinois at Chicago University of Illinois at Chicago

Health and Health Care Health and Health Care in the United Statesin the United States

The U.S. continues to spend more on The U.S. continues to spend more on health care, including prescriptions, than health care, including prescriptions, than other countriesother countries

Outcomes of the health care system are Outcomes of the health care system are not better in the U.S. compared to other not better in the U.S. compared to other developed countriesdeveloped countries

There is much variation in the provision There is much variation in the provision and cost of care between regions of the and cost of care between regions of the USUS

Health Care Spending Health Care Spending Per CapitaPer Capita

Source: Congressional Research Service. Washington, DC. Pub No. RL34175.

Based on 2003 data from the Organisation for Economic Co-operation and Development ( (OECD)

Pharmaceutical Spending Pharmaceutical Spending Per CapitaPer Capita

Source: Congressional Research Service. Washington, DC. Pub No. RL34175.

Based on OECD data 2006.

Global Trends in Health Global Trends in Health ExpendituresExpenditures

From: http://www.oecd.org/document/16/0,3343,en_2649_34631_2085200_1_1_1_1,00.html

0

2

4

6

8

10

12

14

16

18

1960 1965 1970 1975 1980 1985 1990 1995 2000 2005 2006

Hea

lth E

xp a

s %

of T

ota

l GD

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Australia

Canada

France

Germany

Italy

Japan

Korea

Mexico

Poland

Spain

Sweden

Turkey

United Kingdom

United States

Health Care Spending Per Health Care Spending Per Capita and Life ExpectancyCapita and Life Expectancy

Source: Congressional Research Service. Washington, DC. Pub No. RL34175.

Based on OECD data 2006

Health Care Spending and Health Care Spending and Self-Reported Health StatusSelf-Reported Health Status

Source: Congressional Research Service. Washington, DC. Pub No. RL34175.

Based on OECD data 2006

Projected Federal SpendingProjected Federal Spending

Per Capita Medicare Spending: Per Capita Medicare Spending: Regional VariationsRegional Variations

From: Congressional Budget Office. Research on Comparative Effectiveness of Medical Treatments. 2008.

Geographic variation in practice patternsGeographic variation in practice patterns

– Poor relationship between costs and outcomesPoor relationship between costs and outcomes

– Need to establish best practicesNeed to establish best practices

Cost containment Cost containment

– Recognition of limited resources Recognition of limited resources

System managementSystem management

– Improved management, accountabilityImproved management, accountability

The Outcomes MovementThe Outcomes Movement

A. Epstein, NEJM 1990

Information GapInformation Gap

Many gaps in the literatureMany gaps in the literature– Need evidence to support rational decisions in Need evidence to support rational decisions in

health carehealth care– AppropriatenessAppropriateness

Patients of interest/indicationPatients of interest/indication Comparators/therapeutic alternativesComparators/therapeutic alternatives Study designStudy design Outcomes measuredOutcomes measured

The drug development and approval process The drug development and approval process in the U.S. does not provide evidence in the U.S. does not provide evidence necessary to clinicians or policymakers to necessary to clinicians or policymakers to choose between drugs for the same indicationchoose between drugs for the same indication

Address variations in practice patterns by adopting best practicesAddress variations in practice patterns by adopting best practices

Address evidence gaps in best practices by generating evidence Address evidence gaps in best practices by generating evidence using appropriately designed studiesusing appropriately designed studies

Secondary effect of best practices is cost containment (not Secondary effect of best practices is cost containment (not directly addressed in legislation) directly addressed in legislation)

Improve the management of health care systems and Improve the management of health care systems and accountability of providers through measuring and monitoring accountability of providers through measuring and monitoring outcomesoutcomes

Effective Health CareEffective Health Care

Definitions of Comparative Definitions of Comparative Effectiveness Research (CER)Effectiveness Research (CER)

Comparative effectiveness research is:Comparative effectiveness research is:– The conduct and synthesis of research comparing the The conduct and synthesis of research comparing the

benefits and harms of different interventions and benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor strategies to prevent, diagnose, treat, and monitor health conditions health conditions in “real world” settingsin “real world” settings. .

– To improve health outcomes by developing and To improve health outcomes by developing and disseminating evidence-based information to patients, disseminating evidence-based information to patients, clinicians, and other decisionmakers, responding to clinicians, and other decisionmakers, responding to their expressed needs, about which interventions are their expressed needs, about which interventions are most effective for which patients under specific most effective for which patients under specific circumstances. circumstances.

– Generation and synthesis of evidence that compares Generation and synthesis of evidence that compares the benefits and harms of alternative methods. the benefits and harms of alternative methods.

– To prevent, diagnose, treat, and monitor a clinical To prevent, diagnose, treat, and monitor a clinical condition condition or to or to improve the delivery of careimprove the delivery of care. .

Purpose of Comparative Purpose of Comparative Effectiveness Research (CER)Effectiveness Research (CER)

The purpose of CER is: The purpose of CER is:

– to assist consumers, clinicians, purchasers, and to assist consumers, clinicians, purchasers, and policymakers to make informed decisions that will policymakers to make informed decisions that will improve health care at both the individual and improve health care at both the individual and population level.population level.

(Federal Coordinating (Federal Coordinating Council 2009)Council 2009) (IOM 2009)(IOM 2009)

Two Essential ElementsTwo Essential Elements

Comparison of two or more agents or interventions Comparison of two or more agents or interventions that are considered true therapeutic alternatives that are considered true therapeutic alternatives

Examination of the effects/outcomes in actual practice Examination of the effects/outcomes in actual practice (i.e., effectiveness)(i.e., effectiveness)

U.S.-based initiatives have framed outcomes/effects U.S.-based initiatives have framed outcomes/effects in terms of benefits/harmsin terms of benefits/harms– More broadly, they include economic outcomes as well as More broadly, they include economic outcomes as well as

clinical and humanistic benefits/harmsclinical and humanistic benefits/harms

Types of Comparative Types of Comparative Effectiveness ResearchEffectiveness Research

Primary comparative effectivenessPrimary comparative effectiveness– Retrospective observational studies (case Retrospective observational studies (case

control or cohort studies)control or cohort studies)– Prospective observational studies (aka Prospective observational studies (aka

“large simple clinical trial,” “pragmatic “large simple clinical trial,” “pragmatic clinical trials”)clinical trials”)

– Cluster randomized studiesCluster randomized studies– Registry-based studiesRegistry-based studies

Secondary comparative effectivenessSecondary comparative effectiveness– Systematic review and meta-analysesSystematic review and meta-analyses– Modeling and decision-analysisModeling and decision-analysis

Differences Between Efficacy Differences Between Efficacy and Effectiveness Studiesand Effectiveness Studies

Source: Schumock & Pickard, AJHP 2009

Example: CER Example: CER (primary retrospective)(primary retrospective)

Patients:Patients:– Adults with heart Adults with heart

failure failure Intervention:Intervention:

– Drug therapy Drug therapy (beta-blockers)(beta-blockers)

Comparators:Comparators:– Atenolol, Atenolol,

metoprolol, metoprolol, carvedilol, otherscarvedilol, others

Endpoint/outcomeEndpoint/outcome– DeathDeath

Example: CER Example: CER (primary prospective)(primary prospective)

Clinical Clinical Antipsychotic Trials Antipsychotic Trials of Intervention of Intervention Effectiveness Effectiveness (CATIE)(CATIE)

Patients:Patients:– Schizophrenia Schizophrenia

Intervention:Intervention:– Drug treatment Drug treatment

(antipsychotics)(antipsychotics) Comparators:Comparators:

– Olanzapine, Olanzapine, perphenazine, perphenazine, quetiapine, quetiapine, risperidone, risperidone, ziprasidoneziprasidone

Endpoint/outcomeEndpoint/outcome– Treatment failure Treatment failure

(time to (time to discontinue) discontinue)

Example: CER Example: CER (secondary – meta-analysis)(secondary – meta-analysis)

Patients:Patients:– Chronic obstructive Chronic obstructive

pulmonary disease pulmonary disease (COPD) (COPD)

Intervention:Intervention:– Drug therapy Drug therapy

(anticholinergics)(anticholinergics) Comparators:Comparators:

– Ipratropium or Ipratropium or tiotropium vs. control tiotropium vs. control (placebo or active (placebo or active comparator)comparator)

Endpoint/outcomeEndpoint/outcome– DeathDeath– Myocardial infarction Myocardial infarction

(MI)(MI)– StrokeStroke

Applications of Comparative Applications of Comparative Effectiveness ResearchEffectiveness Research

CER data can be used to help inform:CER data can be used to help inform:– Individual patient care decisions/recommendations Individual patient care decisions/recommendations

(patient-level)(patient-level)

– Policy such as formulary decisions (system-level)Policy such as formulary decisions (system-level)

CER data can add to body of evidence on:CER data can add to body of evidence on:– Effectiveness of one drug compared to anotherEffectiveness of one drug compared to another

– Safety of one drug compared to anotherSafety of one drug compared to another

Outcomes from CER studies provide inputs for Outcomes from CER studies provide inputs for cost-effectiveness analyses and decisionscost-effectiveness analyses and decisions

The 3T’s Road MapThe 3T’s Road Map

Dougherty D, Conway PH. High-Quality Care: The "3T’s" Road Map to Transform US Health Care: the "how" of high-quality care. JAMA. 2008;299(19):2319-2321

CER and the Role of the CER and the Role of the Pharmacist Pharmacist

Understand study design and methods used in Understand study design and methods used in comparative effectiveness researchcomparative effectiveness research

Understand differences between efficacy and Understand differences between efficacy and effectiveness studies and their strengths and effectiveness studies and their strengths and weaknessesweaknesses

Monitor literature for results of comparative Monitor literature for results of comparative effectiveness studieseffectiveness studies

Integrate evidence from these studies with Integrate evidence from these studies with existing knowledge base relevant to patient existing knowledge base relevant to patient and system-level decisionmakingand system-level decisionmaking

Will Comparative Effectiveness Will Comparative Effectiveness (CE) Affect Pharmacy Practice?(CE) Affect Pharmacy Practice?

Scientific considerations issues:Scientific considerations issues:– Timeliness of evidenceTimeliness of evidence– Rigor and quality of methodsRigor and quality of methods– Ability to widely disseminate results of studiesAbility to widely disseminate results of studies

Policy-based initiatives issues:Policy-based initiatives issues:– Uncertainty about health care reform Uncertainty about health care reform – Extent to which medications are a priority area for CERExtent to which medications are a priority area for CER– Whether CE is eventually required for regulatory approvalWhether CE is eventually required for regulatory approval– Cost as an explicit criteria by which to evaluate alternativesCost as an explicit criteria by which to evaluate alternatives

If costs are not considered, how is value for money to be assessed? If costs are not considered, how is value for money to be assessed? Clinical practitioners and professional societies’ issues:Clinical practitioners and professional societies’ issues:

– How well CE evidence is understood and disseminatedHow well CE evidence is understood and disseminated– Extent to which CE evidence supports or does not support current practiceExtent to which CE evidence supports or does not support current practice– Willingness and ability of clinicians to change practiceWillingness and ability of clinicians to change practice

Reimbursement structure may not incentivize providers to act upon evidenceReimbursement structure may not incentivize providers to act upon evidence

Final Thoughts on RelevanceFinal Thoughts on Relevance

CE-based evidence is relevant to all stakeholders in health CE-based evidence is relevant to all stakeholders in health carecare– Physicians, pharmacists, health care provider Physicians, pharmacists, health care provider

organizations, pharmaceutical manufacturers, organizations, pharmaceutical manufacturers, employers, insurers, government agenciesemployers, insurers, government agencies

CE is not the only solution needed to address excessive CE is not the only solution needed to address excessive health care costs in the US, but it can be an important part health care costs in the US, but it can be an important part of the solutionof the solution

While CE is not limited to pharmaceuticals, these are often While CE is not limited to pharmaceuticals, these are often cited as the quintessential example of where CE is neededcited as the quintessential example of where CE is needed

See: Am J Health-System Pharm 2009;66:1278-1286.

For More InformationFor More Information

Questions and Answers

To submit any followup questions, please e-mail us at:

AHRQScience2Practice@ahrq.hhs.gov

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Activity Number: 202-999-09-224-H04-P).4. Log in. APhA members, enter your user name

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5. Once you have completed the online posttest and evaluation form, your Statement of Credit will be accessible immediately.