USC Week 9 Outcomes Measurement and Pharmacoepidemiologic Methods Lecture Summary

University of Southern California

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Outcomes Measurement and Pharmacoepidemiologic Methods • • The economic, clinical, and humanistic outcomes (ECHO) model Characteristic differences between randomized control trials and observational studies Rascati (Chapter 11) 1 Learning objectives 1. Outline the differences/similarities between outcomes research and PE 2. Describe the components of clinical decision making using the economic, clinical, and humanistic outcomes (ECHO) model 3. Describe how economic, clinical, and humanistic outcomes are measured 4. Compare and contrast randomized controlled trials (RCTs) and observational studies 2 Outcomes Measurement 3 Differences between PE and Outcomes Research •Pharmacoeconomics is the identification, measurement, and comparison of the costs and outcomes of pharmacy products (e.g., drugs) or services ―Bootman, 2005 • PE is a type of “outcomes research” ―However, not all outcomes research are PE research 4 What is outcomes research? • Outcomes research seeks to understand the end results of health care practices and interventions • Outcomes research is more broadly defined as “studies that attempt to identify, measure, and evaluate the end results of health care services” (Bungay, 2013) ―End results = Outcomes ―Not only clinical outcomes are measured, but also economic and humanistic outcomes • End results include effects that people experience and care about such as improved ability to function (e.g., activities of daily living) or an increased quality of life Source: Agency for Healthcare Research and Quality (AHRQ) 5 Examples of health outcomes • Health outcomes include ―Physiologic measure (e.g., blood pressure, A1C) ―Clinical events (e.g., stroke, myocardial infarction) ―Mortality (e.g., infant mortality rate) ―Symptoms (e.g., difficulty breathing) ―Quality of life (using health surveys such as SF-36) ―Patient’s experiences with care (evaluated using a health survey such as Consumer Assessment of Health Plans) 6 The ECHO Model Economic Clinical Humanistic Outcome 7 The ECHO model • It has been proposed that the evaluation of drug therapy and related services should include an assessment of the economic, clinical, and humanistic outcomes (ECHO) (Kozma, 1993) ―For example, cost-utility analysis encompasses a measurement of the economic (costs), clinical (e.g., effectiveness, side-effects, complications), and humanistic (quality of life, QALY) outcomes 8 Components of clinical decision making -The ECHO model Source: Bungay, 2013 9 The ECHO model Disease Humanistic Intermediaries Effects of disease and treatment on humanistic outcomes (e.g., side effects, satisfaction with therapy) Medical events that occur as a result of disease or treatment (e.g., asthma attacks, ER visits, hospitalization Costs Treatment Modifiers Factors that alter outcomes associated with treatment alternatives (e.g., compliance with therapy, dosage forms) Direct medical, direct non-medical, indirect, and intangible costs External Controls Source: Kozma, 1993 Non-clinical factors that affect availability or use of treatment alternatives (e.g., formularies, prior authorization) Functional status, QOL (e.g., physical function, social function, pain, vitality) Clinical Outcomes Clinical Indicators Measurements of a patient’s physical and biological status used to infer the severity or risk of disease (e.g., FEV, wheezing, breathlessness) Humanistic Outcomes Economic Outcomes Treatment Alternatives (e.g., betaagonist vs. theophylline) Total costs of medical care associated with treatment alternatives balanced against clinical or humanistic outcomes(e.g., cost/QALY, cost/successful treatment) The ECHO model Outcomes resulting from medical care can be classified along three dimensions: 1. Economic outcomes ― Direct, indirect, and intangible costs resulting from clinical or humanistic outcomes 2. Clinical outcomes ― Medical events that occur as a result of disease or treatment 3. Humanistic outcomes ― Consequences of disease or treatment on patients’ functional status or quality of life 11 Measurement of economic outcomes 12 Measurement of economic outcomes • Economic outcomes are usually of primary interest to the payer • Cost component of economic outcomes include direct costs (medical and non-medical), indirect costs, and intangible costs • Economic outcomes also include measures of health resource utilization such as ―number of inpatient or outpatient visits ―total days of hospitalization in a given year ―number of days a patients received a medication 13 Measurement of clinical outcomes 14 Measurement of clinical outcomes • Clinical outcomes are usually of primary interest to the health care professional • Clinical outcomes include coronary events, osteoporosis, death, rheumatoid arthritis, cancer, diabetes, psoriasis, Parkinson’s disease, depression, surgery, etc. 15 Categories of clinical outcomes • Four categories of clinical outcomes are identified: 1. Objective outcomes 2. Subjective outcomes 3. Composite endpoints/outcomes 4. Intermediate endpoints/outcomes Source: Agency for Healthcare Research and Quality 16 Categories of clinical outcomes Objective outcomes • Objective outcomes are defined as measures that are not subject to a large degree of individual interpretation, and are likely to be reliably measured across patients in a study, by different health care providers, and over time ― For example, blood and urine test results for glucose, blood test results for cholesterol, blood pressure measurement, etc. Source: Agency for Healthcare Research and Quality 17 Categories of clinical outcomes Examples of objective clinical outcomes Clinical outcome Objective measure 1. Diabetes A1C, FPG 2. Hypertension Blood pressure (mmHg) 3. Myocardial infarction ↑Troponin levels, ST elevation/depression 4. Asthma Spirometry – Forced Expiratory Volume 1 (FEV1) Source: Agency for Healthcare Research and Quality 18 Categories of clinical outcomes Subjective outcomes • These are outcome measures that are subject to interpretation by individual health care providers and may not be reliably measured across patients and by different health care providers over time ―Examples include assessment of depression, pain, anxiety • However, instruments have been developed to help standardize the assessment of some conditions for which a subjective clinical assessment might introduce unwanted variability ―This help produce a quantifiable measure that should be reproducible across patients and raters Source: Agency for Healthcare Research and Quality 19 Categories of clinical outcomes Subjective outcomes • For example, treatment of psoriasis • Subjective outcome assessment of psoriasis: ―Has the patient’s condition improved a lot, a little, or not at all? • Objective outcome assessment using a reliable and validated instrument: ―Instrument=Psoriasis Area Severity Index (PASI) ―Rating of the severity of target symptoms: erythema, infiltration, and desquamation (0-4 scale; with 0=none and 4=very severe) ―Rating of the area of psoriatic involvement for each of four main body areas - head, trunk, upper extremities, and lower extremities (0-6 scale; with 0=0% and 6=90-100%) ―The final calculated score ranges from 0 (no disease) to 72 (severe disease) 20 Categories of clinical outcomes Composite endpoints/outcomes • These are series of individual clinical outcomes which are grouped together into one outcome measure ―This makes the composite endpoint occur more frequently than any of the individual components • Composite endpoints are necessitated when the individual events included in the score are rare, and/or when it makes clinical sense to group them 21 Categories of clinical outcomes Composite endpoints/outcomes • For example, a composite endpoint called major adverse cardiac events (MACE) ―MACE may be defined differently from study to study and hence interpretation or meaning may not be consistent from study to study ―MACE as an outcome may be compared among patients receiving different types of antiplatelet drugs (e.g., occurrence of MACE between patients prescribed ticagrelor or prasugrel) ―A study may define MACE as occurrence of any of: (i) all-cause mortality, (ii) myocardial infarction, and (iii) coronary revascularization ―Another study may define MACE as occurrence of any of: (i) cardiacrelated mortality, (ii) stent thrombosis, and (iii) coronary revascularization 22 Categories of clinical outcomes Intermediate endpoints/outcomes • Intermediate endpoints/outcomes are biological markers for the condition of interest • Their use is common in clinical trials • They are also called surrogate markers (used in lieu of the main endpoint) • They may be used to reduce the follow-up period required to obtain results from a study of treatment effectiveness in RCTs ―For example, RCT of statin efficacy →Main clinical outcome of interest = reduction in coronary heart disease incidence; reduction in cardiac mortality • may take many years of data collection to manifest these outcomes →Intermediate endpoint = serum lipids (LDL-cholesterol) • A predetermined percentage reduction of LDL-C can serve as evidence of treatment effectiveness 23 Measurement of humanistic outcomes 24 Measurement of humanistic outcomes • Humanistic outcomes are of primary interest to the patients ( e.g., pain level, degree of functioning, etc.) • Humanistic outcomes can be measured using patient-reported outcomes (PRO) instruments ―PRO instrument may include the use of any health-related quality of life (HRQoL) instruments measured from the patient’s perspective alone →Generic HRQoL (e.g., SF-36, QWB scale, Dartmouth COOP) →Disease-specific HRQoL (e.g., Arthritis Impact Measure Scale [AIMS] →Preference-based measures (EQ-5D, SF-6D) 25 PROs • The FDA defines a PRO as “a measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else” • A PRO can be measured by self-report or by interview provided that the interviewer records only the patient’s response Source: Agency for Healthcare Research and Quality 26 Outcomes measured by PROs PROs refer to patient ratings and reports about any of several outcomes, including: 1. 2. 3. 4. 5. 6. 7. 8. Health status Health related quality of life (HRQoL) Quality of life (QoL), defined more broadly Symptoms Functioning Satisfaction with care/treatment Health behaviors, including adherence and health habits Health care utilization and out-of-pocket costs 27 Summary-Outcomes Measurement 1. PE is a type of outcomes research; not all outcomes research are PE research 2. Evaluation of drug therapy should include an assessment of the economic, clinical, and humanistic outcomes (ECHO) 3. Economic outcomes include direct, indirect, and intangible costs resulting from clinical and humanistic outcomes 4. Clinical outcomes refer to medical events that occur as a result of disease or treatment 5. Humanistic outcomes refer to consequences of disease or treatment on patients’ functional status or quality of life 6. Categories of clinical outcomes include objective outcomes, subjective outcomes, composite endpoints/outcomes, and intermediate endpoints/outcomes 28 Pharmacoepidemiologic Methods RCTs Observational studies 29 Pharmacoepidemiologic methods • The types of research methods that can help answer the question of whether a drug ‘works’ include: 1. Meta-analyses of RCTs ― Synthesis of results from several RCTs ― Highest level of evidence ― Helps minimize uncertainty when results differ between two or more RCTs 2. Individual randomized controlled trials (RCTs) ― Answers efficacy question 3. Observational studies (cohort, case-control, surveys) ― Answers effectiveness question 4. Expert opinion ― lowest level of evidence 30 Randomized Control Trials (RCTs) 31 RCTs • RCTs are the gold standard for proving that a drug ‘works’ ―they provide evidence of efficacy • Proof of efficacy through RCTs are required by the US Food and Drug Administration (FDA) before a drug can be approved for use in the general population 32 RCT-defined • Randomized = Randomization ―Randomization is allocation (through random procedure) of subjects into groups who will receive the treatment of interest (the treatment or intervention group), or to another group who will not receive the treatment of interest (the control group) ―Randomization is used to reduce selection bias (imbalance in baseline characteristics between treatment and control groups) ―For example, in observational studies where randomization does not occur, patients with more severe form of a disease may be ‘selected’ to receive the newer medication →The researcher may not know this ‘selection’ has occurred in the data and may draw incorrect conclusions about the new drug • Patients taking the new drug may appear as though they have worse outcomes 33 RCT-defined • Controlled = controlled or ‘supervised’ environment, and/or use of a ‘control’ group (standard treatment or placebo) ―subjects must meet study inclusion criteria and are monitored or supervised over a period of time • Trial = study or experiment ―RCTs can be used to infer causality whereas causality cannot be inferred in observational studies 34 RCT - Process Source: Schulz, 2010 35 Advantages of RCTs 1. The randomization process ensures that groups being compared have similar baseline characteristics ―Thus, efficacy observed is valid and attributable to the drug effects only 2. RCTs have good internal validity ―Minimization of known or unknown biases through randomization ―Causality can be inferred 3. RCTs are more scientifically rigorous and needed for FDA drug approval 36 Disadvantages of RCTs 1. Time frame for follow-up may be too short ― There is a limitation of extrapolating RCT findings for economic evaluation of chronic diseases such as diabetes and cardiovascular diseases (which occurs over the lifetime of the patient) 2. High cost of recruitment and follow-up 3. High adherence rates among patients which is unrealistic in everyday practice ―Due to monitoring and ‘controlled’ or supervised environment 4. Small sample size compared to observational studies ― Reduced power may lead to non-significant results 37 Disadvantages of RCTs 5. Due to inclusion and exclusion criteria, RCT patients may not be representative of the general population ―Subjects cannot have other diseases (comorbidities) ―Subjects cannot take other drugs except those under investigation ―Other exclusion criteria based on age, race, sex, education ―Higher medication adherence rates than obtained normally due to higher monitoring ―Thus, RCTs have poor external validity but good internal validity 38 Observational Studies Prospective Retrospective 39 Observational studies • In contrast to RCTs, observational studies are not conducted in controlled or supervised environments ―Subjects are ‘observed’ either directly (prospective methods) or indirectly (retrospective methods) ―Subjects are already taking the treatment or intervention in the ‘real world’ and are just being observed for outcomes ―Subjects are not under supervised or “controlled” conditions ―There is no randomization of subjects into groups ―The outcome of interest is effectiveness rather than efficacy →Effectiveness evaluate if the drug ‘does work’ in the real-world environment 40 Types of observational studies There are two main types of observational studies: 1. Prospective observational studies ― These record treatments and outcomes as they occur ― Exposed and unexposed groups are identified (exposure occurs before outcome), then subject are followed up over time for manifestation of the outcome ― For example, prospective cohort study 2. Retrospective observational studies ― These analyze treatments and outcomes that have already occurred in the past ― Treatment was given in the past, outcomes had occurred in the past, data and results are then analyzed in the present ― For example, retrospective database analysis (retrospective cohort study, retrospective case-control study) 41 Advantages of observational retrospective database analysis 1. Less expensive to conduct than RCTs 2. Less time-consuming to conduct than RCTs ―No need to enroll patients into study 3. Many years’ worth of patient data can be examined in a short period of time at low cost ― Larger sample size 4. Data from observational studies are more representative of the ‘real’ clinical practice world (hence, they have good external validity) ― More realistic adherence rates and treatment patterns 42 Advantages of observational retrospective database analysis 5. Data is more inclusive and include patients from different socio-demographic background ―Age, sex, race, ethnicity, religion, income level 6. Easier to recalculate with different criteria (sensitivity analyses) ―Age range, diagnoses, medication adherence rates can be changed to determine if results are robust (i.e., insensitive) to initial study results 43 Disadvantages of observational retrospective database analysis 1. Information may be incomplete ― Most retrospective databases are not primarily collected for research purpose but for reimbursement (payments for services) ―Information may be incomplete if: →Patient switches insurance company →Patient pays out-of-pocket for some health expenses rather than through insurance →Patient have more than one insurance type (Medicare + Medicaid; insurance from employer and another from spouse) 44 Disadvantages of observational retrospective database analysis 2. Data may be inaccurate or incomplete ―ICD-9-CM diagnosis and procedure codes and cost of medication may be entered wrongly into the electronic health record ―Data may not be measured, recorded, or available →Smoking information →Family history of disease →Past rates of medication adherence →Cholesterol information 45 Disadvantages of observational retrospective database analysis 3. Selection bias ― This occurs when patients with certain characteristics are more likely to receive one treatment compared to another treatment → Due to lack of randomization of patients to treatment groups ― For example, patients with a more severe form of a disease who have been resistant to standard therapy may be receiving (or be ‘selected’ to receive) a newer, more effective medication → Thus, the risk of an undesirable outcome of interest may be higher among patients receiving the newer medication because they are sicker from the beginning → Hence, higher risk of the undesirable outcome may be wrongly attributed to the newer medication 46 Disadvantages of observational retrospective database analysis • Example: • Channeling bias (this is a type of selection bias) ―Channeling occurs when drug therapies with similar indications are preferentially prescribed to groups of patients with varying baseline prognoses →For example, Medications A and B are used for the treatment of diabetes →However, Medication A is found to cause weight gain more often than Medication B →A doctor might prescribe more of Medication B for his/her overweight patients →Therefore, comparing health outcomes among patients using Medications A and B may be problematic due to this intrinsic channeling bias which may be unknown to the researcher using the retrospective database 47 Methods to minimize biases in observational retrospective database analysis 48 I ...
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Week 9 Outcomes Measurement and Pharmacoepidemiologic Methods
Institution Affiliation

Outcomes Measurement and Pharmacoepidemiologic Methods
Societal, ethical, and medical concerns about access, costs, and quality of care are
making health care practitioners opt for a more comprehensive model for medical decision
making. The economic, clinical, and humanistic outcomes (ECHO) model is a way of modeling
outcomes from pharmaceutical services or tr...

Teodorof (5767)
University of Virginia

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