week 4 - clinical effectiveness

Question 1

What are the 4 underlying principles of good clinical effectiveness assessment?

Answer 1

1. seeking information
2. asking relevant questions
3. understanding differences
4. valuing differences

Question 2

What is “seeking information” in clinical effectiveness assessment?

Answer 2

use clinical info to estimate the outcomes patients might experience when given a new health tech

Question 3

How can they seek info for clinical effectivness assessment 3 different ways?

Answer 3

1. review existing evidence
   2 . conduct a new study to gather and evaluate info in real world setting
2. Ask clinicians and patients (“experts”) their expectations

Question 4

What 3 things do they evaluate for “asking relevant questions” in clinical effectiveness assessment?

Answer 4

1. Cause and effect
2. Health improvements and harms identified
3. Uncertainties

Question 5

What are the two gold standard for every circumstance of strong evidence?

Answer 5

1. RCT
2. blinded trial

Question 6

What is “safety” in clinical effectiveness consist of?

Answer 6

• condition of being protected against consequences of failure, error, or anything non-desirable
• it is the MEDICAL RISK to the patient
• assess in clinical trial

Question 7

Can unitended consequences arise when trying to enhance a patient’s safety in a new health tech?

Answer 7

YES!

Question 8

Are all unintended consequences negative?

Answer 8

NO!
some can be positive
example: bariatric surgery for weight loss in obese patients –> lead to curing type 2 diabetes

Question 9

What are the 3 most common ways we can measure health outcomes?

Answer 9

1. mortality (death rate)
2. Morbidity (disease rate)
3. Adverse Health events (harmful side effects)

Question 10

What is a “clinical endpoint”? provide an example

Answer 10

used to measure the impact of a treatment on how a patient feels, fxns or survives

could improve or worsen health status

ex: survival, death

Question 11

What is a humane (clinical) endpoint?

Answer 11

any disease or sign that strongly motivates withdrawl of the patient from the trial

Question 12

What is an intermediate endpoint?

Answer 12

measure of a function or of a symptom but is NOT the ultimate endpoint of disease

may be of value to the patient!

ex: rate of irreversible morbid events

Question 13

What is a surrogate endpoint?

Answer 13

where there is good evidence that it PREDICTS the clinical endpoint

• used as a substitute for a clinical endpoint of interest
• used in clinical trials when it is impractical to measure the primary endpoint during course of trial

ex: using hypertension as a surrogate for clinical end point of a stroke

Question 14

What is a biomarker?

Answer 14

measured variable or trait that is used as an indicator of a normal biological process, disease state, or effect of treatment

Question 15

can a biomarker or an intermediate endpoint be used as surrogate endpoints?

Answer 15

YES!

Question 16

What are measures of treatment effect and provide examples?

Answer 16

compare the probability of a given health outcome in the treatment group with the probability of the same outcome in a control group

ex: odds ratio, effect size, number needed to treat, absolute risk reduction

Question 17

What is “understanding differences” in clinical effectiveness assessment?

Answer 17

• where you compare the outcomes of new tech with the standard of care that already exists for a health condition
• can be challenging because there outcomes can be measured different ways

Question 18

What is “valuing differences” in clinical effectiveness assessment?

Answer 18

• understanding how to perceive and value the differences between the outcomes of new tech and already existing care
• qualitative (interviews) or quantitative (surveys)

Question 19

make sure to study regulatory framework!

Answer 19

• understand the regulatory framework
• what is considered a device under the regulatory framework
• devices are regulated based on ____________
• know the difference between the manufacturer and the qualified investigator
• what are the 3 pillars of the regulatory framework for medical devices
• which agency is responsible for each pillar
• what are the pathways to authorization?
• what distinguishes each pathway?