Introduction to Pharmacoeconomics Part 2

Question 1

What is the main aim of clinical research?

Answer 1

Determine efficacy

Question 2

What is the main aim of outcomes research?

Answer 2

Determine effectiveness

Question 3

What is the main aim of pharmacoeconomics?

Answer 3

Determine efficiency

Question 4

What is the study design of clinical research?

Answer 4

Randomized clinical trials

Question 5

What is the study design of outcomes research?

Answer 5

Observational studies and randomized clinical trials

Question 6

What is the study design of pharmacoeconomics?

Answer 6

Economic analyses based on outcomes and clinical trials

Question 7

What is the main measure of clinical research?

Answer 7

Efficacy and safety intermediate end points

Question 8

What is the main measure of outcomes research?

Answer 8

Patient-related outcomes

Question 9

What is the main measure of phamacoeconomics?

Answer 9

Cost and outcomes

Question 10

What is the time frame of clinical research?

Answer 10

Short term
Ideal (not actual) clinical practice

Question 11

What is the time frame of outcomes research?

Answer 11

Long term
Actual clinical practice

Question 12

What is the time frame of pharmacoeconomics?

Answer 12

Long term
Actual clinical practice

Question 13

What are some ADVANTAGES of prospective observational analysis?

Answer 13

Flexible
Provider-specific data
Reflects usual care
Offers comparative data
Less $ than RCTs
Prospective (a benefit and drawback!)

Question 14

What are some DISADVANTAGES of prospective observational analysis?

Answer 14

Expensive
Difficult to control and randomize
Potential for patient selection bias
Small sample size
Difficulty generalizing results to other providers
Longer timeframe

Question 15

What are some ADVANTAGES of retrospective database analyses?

Answer 15

Has potential for large sample size
Can provide data quickly
Customer specific
Reflects ‘actual care’ or effectiveness
Relatively inexpensive
Shorter timeframe
Data collection is unobtrusive

Question 16

What are some DISADVANTAGES of retrospective database analyses?

Answer 16

Retrospective
Inconsistent coding
Variations in database quality among managed care plans
Inconsistent access to pharmacy versus medical claims
Inability to randomize patients to treatment
(At the whims of the system)

Question 17

_______, _______ and ________ of resources is necessary to predict the pharmacoeconomic value?

Answer 17

Identification - identify the resource and any variables
Measurement - count resource in units service required to produce benefit. How are resources measured?
Valuation - market value of goods and services (theoretically the opportunity costs)

Question 18

What are CLINICAL outcomes in pharmacoeconomics?

Answer 18

Therapeutic outcomes, controllable by a RPh. Use clinical indicators to measure. Death is a clinical outcome too!

Question 19

What are ECONOMIC outcomes of pharmacoeconomics?

Answer 19

Direct medical resources
Health-related QoL
Patient satisfaction
Reduced hospital stay

Question 20

When identifying outcomes of interest (economic, clinical, humanistic) we must account for the __________ of all these outcomes

Answer 20

Multidimensionality

Question 21

What are three possible study designs?

Answer 21

1. Experimental
   - piggyback RCTs
   - Economic clinical trials
2. Observational designs
   - Claims analysis
3. Simulation designs
   - machine learning, computer simulated

+ Combo designs

Question 22

What may become barriers to the VALIDITY of a study?

Answer 22

Biases!
Internal
Patient selection bias
Crossover bias
Errors in measurement of outcomes
Errors in ascertainment of exposure to technology

External
Differences among people studied versus people with whom the results will be applied

Question 23

How might we resolve possible ethical issues?

Answer 23

Prospective:
- BEFORE start, ensure all pts are not at risk and have full consent

Retrospective:
- Institutions should be allowed to review study protocol, contact IRB members for clarification

Question 24

What is involved in conducting a pharmacoeconomic evaluation? Why might one be done?

Answer 24

May be done if:
- Insufficient literature
- Published results cannot be extrapolated to clinical practice,
- Building a model is not appropriate

COSTLY! Save this for only if there’s significant impact
(Retrospective database studies are far more common now

Question 25

What are 6 steps to conducting pharmacoeconomic research?

Answer 25

1. Define the problem 2. Define objectives 3. Identify alternatives 4. Formulate research hypothesis 5. Establish a framework 6. Conducting pharmacoeconomic research

Question 26

When creating a research framework for pharmacoeconomics, what considerations should be taken?

Answer 26

- What can be done within the problem/research question identified? - consider both the processes and outcomes of care - Consider dependent and independent variables

Question 27

What four perspectives should the problem/objectives/outcomes of pharmacoeconomic studies be viewed?

Answer 27

Patient Payer Provider Society

Question 28

When coming up with possible solutions to a pharmacoeconomic problem, why is it important to identify possible alternatives?

Answer 28

Compares options Would this add new therapy to standard of care? Do guidelines or systematic reviews exist comparing alternatives?

Question 29

When establishing a framework, the relationship of information is input -> output. A _____ must be developed based in understanding of care provision, and ______ _______ must always be documented based in care pathway.

Answer 29

A MODEL must be developed for the framework! Must always describe resource utilization

Question 30

What is a defined intervention in pharmacoecon research?

Answer 30

Determine parameters specifically! - what data is being collected? - Who is collecting this data? - what model will be used? Make a timeline!

Question 31

When collecting data, can we use existing published data or collected data?

Answer 31

Both

Question 32

What are some resources to be identified when assessing resource utilization?

Answer 32

Salaries/wages $ savings/losses Time used

Question 33

What is a sensitivity analysis?

Answer 33

May replace statistical analysis Often data is obtained from other studies - must establish reasonable high and low values for variables

Question 34

How is pharmacoeconomics used today?

Answer 34

To evaluate the value of pharmacy services - justify novel services to higher-ups, expand scope of practice To establish contracts with payers/PCMHs/ACOs - offer clinical services aimed at improving quality/reducing cost for sellers Determine cost-effective therapy recommendations for patients or partners New jobs or opportunities

Question 35

What is applied pharmacoeconomics?

Answer 35

The practice of pharmacoeconomic principles/methods/theories to assess the value of pharmaceutical products/services in **”the real world”**

Question 36

What are some specific applications of applied pharmacoeconomics?

Answer 36

Formulary Management Clinical Guidelines Drug Use Policies Service or Program Evaluation Individual Patient Treatment Decisions

Question 37

What is Formulary Management?

Answer 37

A cost-containment tool used by hospitals/managed care organizations (MCO) that does **NOT** always represent a list of the least expensive alternatives. Optimizes therapeutic outcomes balanced with controlling costs!

Question 38

What are Clinical Guidelines in terms of pharmacoeconomics?

Answer 38

Developing *drug use guidelines/policies/protocols* in hospitals, influencing/prescribing/promoting the most cost-effective and desirable outcomes.

Question 39

What are Drug Use Policies?

Answer 39

*Policies* implemented at institutional/organizational/government level to promote the optimal use of health care products and services (Pharmacoeconomics should plan a major role in the evaluation of this policy.)

Question 40

What is a Service (Program) Evaluation?

Answer 40

Principles & methods of pharmacoeconomics are useful in determining the VALUE of an EXISTING pharmacy or medical service, or estimating the POTENTIAL WORTH of implementing a NEW services Why do this? -> increased competition for resources. Make yourself a necessity!

Question 41

What are Individual Patient Treatment Decisions?

Answer 41

Pharmacoeconomic studies should evaluate diverse patient groups and include both **economic and humanistic outcomes** so it is easier to apply to many different individuals

Question 42

WHOSE approval is needed for human research subjects?

Answer 42

IRB

Question 43

Study design affects WHAT aspect of the research?

Answer 43

• **Cost** of the evaluation • **Time** required to conduct the evaluation • **Accuracy** of the information gained • Complexity of **conducting** the evaluation • Ease of **defending** the subsequent decisions