costs and outcomes/decision analysis

Question 1

costs beyond drugs

Answer 1

cost to administer (nurse, pump)
testing/labs
drug-related complications
treatment failure

Question 2

treatment effects beyond efficacy

Answer 2

increase survival
work/social functionality, QoL
patient satisfaction

Question 3

efficacy vs effectiveness

Answer 3

efficacy: expected outcome when a drug is used under ideal conditions (phase 1-3, clinical trial)

effectiveness: expected outcome when a drug is used in a naturalistic setting (actual practice, phase IV)

Question 4

problems with RCTs

Answer 4

unrepresentative population, short duration of study, protocol-induced elements, intermediate endpoints, inappropriate comparators, artificial environment

Question 5

confounding factors in the naturalistic setting

Answer 5

differences in disease or patient severity, comorbidities, prescribing preferences & biases

Question 6

what is decision analysis

Answer 6

a visual road map to help us measure costs and consequences (under conditions of uncertainty). by combining the probabilities that events will occur with the value of each possible outcome, DA prescribes which option to select to maximize the outcome of the decision

Question 7

advantages of decision analysis

Answer 7

-can use multiple sources of evidence
-time horizon can be extended (beyond a clinical trial)
-can assess the uncertainty surrounding outcomes

Question 8

why do we have to do a decision analysis?

Answer 8

addresses limitations of cost analysis conducted as part of RCTs such as:
-RCT won’t compare all available options
-RCT won’t look at all potential outcomes
-RCT won’t last long enough to model long term cost effectiveness
-RCTs ignore efficacy/outcomes from other trials
essentially, DAs are conducted alongside RCTs to estimate cost effectiveness.

Question 9

8 steps in decision analysis

Answer 9

1 identify the problem & decision options
2 identify perspective
3 identify timeframe
4 structure decision & consequences of each decision over time
5 assess probability that each consequence will occur
6 determine value of each outcome
7 select decision option with the best outcome/value
8 conduct a sensitivity analysis

Question 11

perspective in a decision analysis

Answer 11

the POV from which the analysis is conducted (society, payer, provider, patient)
impacts what costs/consequences are measured & how they’re valued

Question 12

DA from patient perspective

Answer 12

not common; costs measured would be those only the patient incurs (co-pays, etc)

Question 13

DA from provider perspective

Answer 13

usually considers inpatient costs (direct medical) or other costs to the provider (drugs, labs, bed, OR time, inpatient procedures, personnel time)

Question 14

DA from payer perspective

Answer 14

includes insurance companies, government, employers. would consider all inpatient & outpatient charges covered by the payer program. Costs from provider, outpatient rx’s, outpatient MD visits, home health care, etc

Question 15

DA from societal perspective

Answer 15

broadest perspective
recommended
considers costs borne by the patient, provider, payer, and also considers indirect & intangible costs (loss of income, productivity, pain/suffering due to health)

Question 16

a ______ is a graphic representation of how all the possible choices relate to the possible outcomes

Answer 16

decision tree

Question 17

a ______ is defined as a possible choice among all options

Answer 17

decision option

Question 18

the first point of choice in the decision tree is represented by a ______, usually drawn as a square box

Answer 18

decision node

Question 19

an event whose outcomes are not under control of the decision maker is denoted by a _______, symbolized by a circle

Answer 19

chance node

Question 20

for each decision alternative, a __________ is assigned that represents the chance/likelihood of an event/outcome occurring

Answer 20

numerical probability

Question 21

for probabilities in a decision tree: what do you do if a range of reasonable values for the probability exists

Answer 21

conduct a sensitivity analysis on the lowest and highest values of the range

Question 22

probability estimates for decision trees can come from

Answer 22

published literature
internal data sources
expert opinion, surveys

Question 23

what is the last step to a decision tree

Answer 23

assign values to all outcomes (can be costs, utilities, life years, QALYs). determine and add all the costs that would be incurred along each tree path. the total cost of a tree path is placed at the end of the tree

Question 24

are there assumptions when making a decision tree?

Answer 24

yes when developing your tree, there are frequently items whether either no info is available, conflicting info is available, or a range of values/outcomes exist. so yes you make assumptions about your model

Question 25

the costs you include in your decision tree will depend on the ________

Answer 25

PERSPECTIVE

Question 26

what do you do after you enter in the probabilities and path “pay-offs” for your decision tree

Answer 26

average out and fold back (roll back analysis)

calculate each tree path from right to left

Question 27

important things to know for decision trees

Answer 27

time flows left to right
don’t have to be symmetric
perspective should be clearly described
list assumptions
represent all important outcomes

Question 28

why is there uncertainty in PE analysis and what do we do for that

Answer 28

there are assumptions (some may be inaccurate) and the results may not be generalizable–> for all of these reasons, it is essential to perform a sensitivity analysis

Question 29

sensitivity analysis

Answer 29

method to handle model uncertainty; also known as a “what if” analysis
-assesses whether the use of alternative estimates would change the results

Question 30

limitations of DA

Answer 30

-may oversimplify medical problems to a point where they do not reflect reality (don’t include all potential outcomes)
-inadequate availability of data
-may not reflect true concerns to the patient
-inappropriate assumptions
-traditional statistical analyses cannot be applied to the outcomes

Question 31

what is a markov model used for

Answer 31

-more complex events occurring over time or for a greater number of event repetitions
-useful when the risk of an outcome changes over time (risk of GI bleed w/ NSAID increases w/ age)
-used for transitions between various stages of health where movement can go back and forth between health states (remission & recurrence of cancer)

Question 32

discounting

Answer 32

used in PE analyses to express costs & benefits that occur in the future to “present values”

usually only discount for costs/benefits that occur >1 year from present

can range from 3-10%