Lecture 8: Patient Centricity

Question 1

Concept, Outcome, Endpoint

Answer 1

Concept: The experience to be measured such as a feeling or function (pain)
Outcome: A variable that reflects how a patient feels, functions or survives (value on an 11 point NRS or 101 point VAS)
Endpoint: A variable (i.e., number) will be statistically compared among treatment groups to assess the effect of treatment

Question 2

Understanding the disease or the condition

Answer 2

1) Natural history of the disease or condition
2) Patient subpopulations
3) Health care environment
4) Patient/caregiver perspectives

Question 3

Conceptualizing treatment benefit

Answer 3

1) Identify concepts of interest (COI) for meaningful treatment benefit
2) Define context of use (COU) for clinical trial
3) Select clinical outcome assessment (COA) type

Question 4

Selecting/Developing the outcome measure

Answer 4

1) Search for existing COA measuring COI in COU
2) Begin COA development
3) Complete COA development

Question 5

Clinical outcome assessments

Answer 5

1) identify context of use and concept of interest
2) draft instrument and evaluate content validity
3) Cross sectional evaluation of other measurement properties
4) Longitudinal evaluation of measurement properties/interpretation methods
5) modify instrument

Question 6

Considerations

Answer 6

• Participants (know who your participants are so you can build a strategy around their unique needs and the context of use)
• Endpoints (consider specific endpoints that support the study objectives and timing needed to capture the right data
• Burden on participants (length of instrument and how difficult the questions are in the instrument)
• Mode and location of administration and selection of technology solution

Question 7

Developing a neW Patient reported outcome (PRO)

Answer 7

• Start with published literature
• speak with physicians and the health workers that treat the condition
• speak to patients
• develop PRO