State of the Science: Methods to Collect and Use Patient Preference - - PowerPoint PPT Presentation

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State of the Science: Methods to Collect and Use Patient Preference - - PowerPoint PPT Presentation

State of the Science: Methods to Collect and Use Patient Preference Data Overview of methods to collect patient preference information and active research regarding patient collecting patient preferences. For the online webcast: Please submit your


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SLIDE 1

State of the Science: Methods to Collect and Use Patient Preference Data

Overview of methods to collect patient preference information and active research regarding patient collecting patient preferences.

For the online webcast: Please submit your questions to the panel via the chat box. The

  • nline hosts will be collecting the questions during the session

to be brought to the panel moderator during the panel discussion.

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SLIDE 2

Moderator: Telba Irony, Ph.D. Chief, General Surgical Devices Branch CDRH/Office of Surveillance and Biometrics Panel:

  • A. Brett Hauber, Ph.D.

Research Triangle Institute (RTI‐Health Solutions) Bennett Levitan, M.D., Ph.D. Janssen Research & Development (Johnson and Johnson) Sonal Singh, M.D., M.P.H. Johns Hopkins University Scott Braithwaite, M.D. Society of Medical Decision Making (SMDM)

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SLIDE 3

Methods for Eliciting Benefit-Risk Preference Data

Patient Preference Initiative Symposium

  • A. Brett Hauber, Ph.D.

Senior Economics and Vice President Health Preference Assessment

September 18 September 18-

  • 19, 2013

19, 2013

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SLIDE 4

4 4

4

Benefit-Risk Tradeoff Metrics

Risk Effectiveness

Δ Effectiveness Δ Risk

Net Effectiveness Benefit Maximum Acceptable Risk Minimum Acceptable Benefit Net Safety Benefit

Patient Benefit-Risk Threshold TREATMENT A

Source: Hauber et al., Appl Health Econ Source: Hauber et al., Appl Health Econ Health Policy (2013) Health Policy (2013)

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SLIDE 5

5 5

Benefit-Risk Preference Elicitation Methods

  • Weighting methods for single decisions

Standard Gamble

Threshold Technique

  • Generalized weighting methods

Best-Worst Scaling

Discrete-Choice Experiments (DCE)

  • Decision support methods

Analytic Hierarchy Process (AHP)

Multi-Criteria Decision Analysis (MCDA)

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SLIDE 6

6 6

Benefit-Risk Preference Elicitation Methods

  • Weighting methods for single decisions

Standard Gamble

Threshold Technique

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SLIDE 7

7 7

? ?

Standard Gamble

U(Asthma) = U(Asthma) = p p· ·U(Perfect Health) + (1 U(Perfect Health) + (1-

  • p

p) ) · ·U(Death) U(Death) U (Asthma) = U (Asthma) = p p· ·(1) + (1 (1) + (1-

  • p

p) )· ·(0) (0) MAR (Death) = 1 MAR (Death) = 1-

  • p

p

Probability = p Probability = p Probability = (1 Probability = (1-

  • p)

p) A A B B Perfect Perfect Health Health Death Death Asthma Asthma

Source: Bernie O Source: Bernie O’ ’Brian, personal Brian, personal communication communication

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SLIDE 8

8 8

Threshold Technique

Source: Kopec JA et al., Source: Kopec JA et al.,

  • J. Clin Epidemiol (2007)
  • J. Clin Epidemiol (2007)

Less pain Less pain Greater Greater hypertension hypertension risk risk

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SLIDE 9

9 9

Benefit-Risk Preference Elicitation Methods

  • Generalized weighting methods

Best-Worst Scaling

Discrete-Choice Experiments (DCE)

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SLIDE 10

10 10

Best-Worst Scaling (Object Case)

MOST Important

(Please click ONE)

TREATMENT SIDE EFFECT LEAST Important

(Please click ONE)

Non-fatal stroke Serious infection requiring hospitalization Nausea and vomiting Hand and foot syndrome Bad liver test

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SLIDE 11

11 11

Discrete-Choice Experiment (DCE)

Greater Greater weight loss weight loss Longer duration Longer duration

  • f side effects
  • f side effects

Lower Risk Lower Risk

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SLIDE 12

12 12

DCE Question: Renal-Cell Carcinoma

Efficacy Mild-to Moderate Side Effects Serious Adverse- Event Risks

Source: Mohamed et al., Pharmacoeconomics, 2011 Source: Mohamed et al., Pharmacoeconomics, 2011

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13 13

Benefit-Risk Thresholds

Lung Damage Liver Damage

Source: Johnson et al., Chapter 4. Quantifying Patient Source: Johnson et al., Chapter 4. Quantifying Patient Preferences to Inform Benefit Preferences to Inform Benefit-

  • Risk Evaluations (

Risk Evaluations (In Press In Press) )

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SLIDE 14

14 14

Benefit-Risk Preference Elicitation Methods

  • Decision support methods

Analytic Hierarchy Process (AHP)

Multi-Criteria Decision Analysis (MCDA)

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SLIDE 15

15 15

Criterion 1 Criterion 2 Criterion 3 Criterion 4 Criterion 5

Alternative 1

a1 c1 a1 c2 a1 c3 a1 c4 a1 c5

Alternative 2

a2 c1 a2 c2 a2 c3 a2 c4 a2 c5

Alternative 3

a3 c1 a3 c2 a3 c3 a3 c4 a3 c5

Alternative 4

a4 c1 a4 c2 a4 c3 a4 c4 a4 c5

Alternative 5

a5 c1 a5 c2 a5 c3 a5 c4 a5 c5

Weights

W1 W2 W3 W4 W5 where a where ai

i

c cj

j

is the criteria score (performance) of alternative is the criteria score (performance) of alternative i i on criterion

  • n criterion j

j

W Wj

j

a ai

i

c cj

j

Value for Value for alternative i alternative i = = Σ

Σ

j=1 j=1-

  • n

n

Multi-Criteria Decision Analysis (MCDA)

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SLIDE 16

16 16

Analytic Hierarchy Process (AHP)

Efficacy Efficacy Risk Risk Tolerabilit y Tolerability

Criteria Weighting Criteria Weighting: : Pairwise comparisons Pairwise comparisons

  • n a 9
  • n a 9-
  • point scale

point scale AHP Scoring AHP Scoring: : Applying weights to Applying weights to criteria values criteria values

Source: Dolan JG. The Patient 2012 Source: Dolan JG. The Patient 2012

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Multi-criteria Decision Analysis for Patient Preference Assessm ent Multi-criteria Decision Analysis for Patient Preference Assessm ent

Bennett Levitan, MD-PhD Departm ent of Epidem iology

Janssen Research and Developm ent, Titusville, NJ FDA CDRH Patient Preference I nitiative W orkshop Septem ber 1 8 , 2 0 1 3

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18

Topics

  • Multi-criteria Decision Analysis for Patient

Preferences

  • I ndustry Considerations for Eliciting Patient

Preferences

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19

Multi-criteria Decision Analysis ( MCDA)

  • A generic approach to aid decision-m aking by
  • Decomposing a complex problem into pieces
  • Applying judgment to the pieces
  • Reassembling them into a coherent whole
  • Generally conducted by a facilitator w ith a sm all

group of experts

  • Long and successful history in Decision Analysis
  • Lim ited application to benefit-risk or patient

considerations until recently

  • Dodgson JS, Spackman M, Pearman A, Phillips LD. Multi-Criteria Analysis: A Manual. London: Department for Communities

and Local Government; 2009

  • European Medicines Agency Benefit-Risk Methodology Project, EMA/213482/2010
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20

Steps in Multi-criteria Decision Analysis ( MCDA)

20

PrOACT URL Decision-making framework

  • Hammond, Keeney, Raiffa. Smart Choices: A Practical Guide to making Better Decisions. Harvard Bus Sch Press; 1999
  • Hunink, Glasziou, et al. Decision Making in Health and Medicine.

Cambridge University Press; 2001

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions Data Uncertainty Risk Tolerance Linked Decisions

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21

Steps in Multi-criteria Decision Analysis ( MCDA)

21

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

  • What is the disease and who has it?
  • What is it like to have the disease?
  • How well do existing treatments work?
  • What treatments are we considering?
  • Over what time period am I considering treatment?
  • Who is seeing this analysis?
  • What is the disease and who has it?
  • What is it like to have the disease?
  • How well do existing treatments work?
  • What treatments are we considering?
  • Over what time period am I considering treatment?
  • Who is seeing this analysis?

What is the problem I am trying to solve?

Data

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22

Steps in Multi-criteria Decision Analysis ( MCDA)

22

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

How do I want to characterize treatment performance?

Benefits Benefits Harms Harms Benefit 1 Benefit 1 Benefit 2 Benefit 2 Benefit 3 Benefit 3 Harm 1 Harm 1 Harm 2 Harm 2 Harm 3 Harm 3 Harm 4 Harm 4 B-R Balance B-R Balance Benefits Benefits Harms Harms B-R Balance B-R Balance

Identify and pare down the key endpoints (attributes)

Data

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23

Steps in Multi-criteria Decision Analysis ( MCDA)

23

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

  • Critical step for benefit-risk
  • Organizes endpoints into an interpretable tree
  • Distinguishes between critical and less important endpoints
  • Can find differences between what patients consider

important and what is measured in clinical studies

  • Enables identifying symptoms or functional outcomes that

can be used for PRO development

  • Critical step for benefit-risk
  • Organizes endpoints into an interpretable tree
  • Distinguishes between critical and less important endpoints
  • Can find differences between what patients consider

important and what is measured in clinical studies

  • Enables identifying symptoms or functional outcomes that

can be used for PRO development How do I want to characterize treatment performance?

Data

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24

Exam ple Value Tree: Statins for Coronary Heart Disease

Benefit- Risk Balance Benefit- Risk Balance

Benefits Benefits Risks Risks

Cardiovascular Issues Cardiovascular Issues Ischemic Stroke Ischemic Stroke Liver Damage Liver Damage Muscle Damage Muscle Damage

Fatal ischemic stroke Fatal ischemic stroke Coronary heart disease death Coronary heart disease death Lipid levels meet target Lipid levels meet target Angina requiring CABG Angina requiring CABG Nonfatal myocardial infarction Nonfatal myocardial infarction Nonfatal ischemic stroke Nonfatal ischemic stroke Hepatitis with hospitalization Hepatitis with hospitalization Hepatitis without hospitalization Hepatitis without hospitalization Liver failure Liver failure Persistently elevated liver enzymes Persistently elevated liver enzymes Myopathy (weakness) Myopathy (weakness) Severe rhabdomyolysis leading to kidney failure Severe rhabdomyolysis leading to kidney failure Rhabdomyolysis (breakdown) Rhabdomyolysis (breakdown)

Levitan BS, Andrews EB, Gilsenan A, et al. Application of the BRAT Framework to Case Studies: Observations and

  • Insights. Clin Pharmacol Ther. Feb 2011;89(2):217-224.
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25

Steps in Multi-criteria Decision Analysis ( MCDA)

25

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

  • Two considerations:
  • What changes are important?
  • Which endpoints make the biggest difference?
  • Example:
  • Does 40% vs. 50% chance hepatitis really matter?
  • Which is worse:
  • Heart attack vs. stroke?
  • Muscle weakness vs. hospitalization for hepatitis?
  • Two considerations:
  • What changes are important?
  • Which endpoints make the biggest difference?
  • Example:
  • Does 40% vs. 50% chance hepatitis really matter?
  • Which is worse:
  • Heart attack vs. stroke?
  • Muscle weakness vs. hospitalization for hepatitis?

Which endpoints are important and by how much?

Data

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26

Steps in Multi-criteria Decision Analysis ( MCDA)

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

What changes are important?

0% 100%

How much it matters to me (Utility) Chance I have a non-fatal stroke ?

10%

All I care about are changes from 0% to 10%: The lowest and high values relevant to the decision

Data

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27

Steps in Multi-criteria Decision Analysis ( MCDA)

Clinical Context Treatments Endpoints Data Tradeoffs Uncertainty Risk Tolerance Linked Decisions

Which endpoints make the biggest difference (weighing!)?

  • Several methods to elicit weights
  • Common approach is “swing weighting”
  • Facilitated process that can take a few hours
  • Results in weights that reflect the relative impact for each

endpoint “swinging” from its lowest to highest value

  • Several methods to elicit weights
  • Common approach is “swing weighting”
  • Facilitated process that can take a few hours
  • Results in weights that reflect the relative impact for each

endpoint “swinging” from its lowest to highest value

Data

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28

Multi-criteria Decision Analysis: Putting it all together

  • Most of the value from MCDA com es from the

collective discussion during the facilitated m eetings

CV Stroke Liver Muscle CV Stroke Liv Muscle

Treatments

Study Drug Comparator

Example of MCDA Model Results Higher scores reflect better performance

  • Modeling results can inform decision-making by handling all

the assumptions and data

  • The endpoints, data, utility functions and weights result in

scores and graphics that show how each endpoint contributes to the value of a treatment

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29

Agenda

  • Multi-criteria Decision Analysis for Patient

Preferences

  • I ndustry Considerations for Eliciting Patient

Preferences

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30

Som e Sponsor Considerations for Preference Studies

  • I s a preference study needed for benefit-risk?
  • W hen to com m it to a preference study?
  • W hat type of study?
  • W hose preferences?
  • Challenges w ith m ultiple regions
  • Patients and physicians from a trial or a panel?
  • How w ill the study be view ed by health authorities?
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SLIDE 31

State of the Science: Methods to Collect and Use Patient Preference Data

Overview of methods to collect patient preference information and active research regarding patient collecting patient preferences.

For the online webcast: Please submit your questions to the panel via the chat box. The

  • nline hosts will be collecting the questions during the session

to be brought to the panel moderator during the panel discussion.

slide-32
SLIDE 32

Moderator: Telba Irony, Ph.D. Chief, General Surgical Devices Branch CDRH/Office of Surveillance and Biometrics Panel:

  • A. Brett Hauber, Ph.D.

Research Triangle Institute (RTI‐Health Solutions) Bennett Levitan, M.D., Ph.D. Janssen Research & Development (Johnson and Johnson) Sonal Singh, M.D., M.P.H. Johns Hopkins University Scott Braithwaite, M.D. Society of Medical Decision Making (SMDM)

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SLIDE 33

Methods for Eliciting Benefit-Risk Preference Data

Patient Preference Initiative Symposium

  • A. Brett Hauber, Ph.D.

Senior Economics and Vice President Health Preference Assessment

September 18 September 18-

  • 19, 2013

19, 2013

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SLIDE 34

34 34

34

Benefit-Risk Tradeoff Metrics

Risk Effectiveness

Δ Effectiveness Δ Risk

Net Effectiveness Benefit Maximum Acceptable Risk Minimum Acceptable Benefit Net Safety Benefit

Patient Benefit-Risk Threshold TREATMENT A

Source: Hauber et al., Appl Health Econ Source: Hauber et al., Appl Health Econ Health Policy (2013) Health Policy (2013)

slide-35
SLIDE 35

35 35

Benefit-Risk Preference Elicitation Methods

  • Weighting methods for single decisions

Standard Gamble

Threshold Technique

  • Generalized weighting methods

Best-Worst Scaling

Discrete-Choice Experiments (DCE)

  • Decision support methods

Analytic Hierarchy Process (AHP)

Multi-Criteria Decision Analysis (MCDA)

slide-36
SLIDE 36

36 36

Benefit-Risk Preference Elicitation Methods

  • Weighting methods for single decisions

Standard Gamble

Threshold Technique

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SLIDE 37

37 37

? ?

Standard Gamble

U(Asthma) = U(Asthma) = p p· ·U(Perfect Health) + (1 U(Perfect Health) + (1-

  • p

p) ) · ·U(Death) U(Death) U (Asthma) = U (Asthma) = p p· ·(1) + (1 (1) + (1-

  • p

p) )· ·(0) (0) MAR (Death) = 1 MAR (Death) = 1-

  • p

p

Probability = p Probability = p Probability = (1 Probability = (1-

  • p)

p) A A B B Perfect Perfect Health Health Death Death Asthma Asthma

Source: Bernie O Source: Bernie O’ ’Brian, personal Brian, personal communication communication

slide-38
SLIDE 38

38 38

Threshold Technique

Source: Kopec JA et al., Source: Kopec JA et al.,

  • J. Clin Epidemiol (2007)
  • J. Clin Epidemiol (2007)

Less pain Less pain Greater Greater hypertension hypertension risk risk

slide-39
SLIDE 39

39 39

Benefit-Risk Preference Elicitation Methods

  • Generalized weighting methods

Best-Worst Scaling

Discrete-Choice Experiments (DCE)

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SLIDE 40

40 40

Best-Worst Scaling (Object Case)

MOST Important

(Please click ONE)

TREATMENT SIDE EFFECT LEAST Important

(Please click ONE)

Non-fatal stroke Serious infection requiring hospitalization Nausea and vomiting Hand and foot syndrome Bad liver test

slide-41
SLIDE 41

41 41

Discrete-Choice Experiment (DCE)

Greater Greater weight loss weight loss Longer duration Longer duration

  • f side effects
  • f side effects

Lower Risk Lower Risk

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SLIDE 42

42 42

DCE Question: Renal-Cell Carcinoma

Efficacy Mild-to Moderate Side Effects Serious Adverse- Event Risks

Source: Mohamed et al., Pharmacoeconomics, 2011 Source: Mohamed et al., Pharmacoeconomics, 2011

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SLIDE 43

43 43

Benefit-Risk Thresholds

Lung Damage Liver Damage

Source: Johnson et al., Chapter 4. Quantifying Patient Source: Johnson et al., Chapter 4. Quantifying Patient Preferences to Inform Benefit Preferences to Inform Benefit-

  • Risk Evaluations (

Risk Evaluations (In Press In Press) )

slide-44
SLIDE 44

44 44

Benefit-Risk Preference Elicitation Methods

  • Decision support methods

Analytic Hierarchy Process (AHP)

Multi-Criteria Decision Analysis (MCDA)

slide-45
SLIDE 45

45 45

Criterion 1 Criterion 2 Criterion 3 Criterion 4 Criterion 5

Alternative 1

a1 c1 a1 c2 a1 c3 a1 c4 a1 c5

Alternative 2

a2 c1 a2 c2 a2 c3 a2 c4 a2 c5

Alternative 3

a3 c1 a3 c2 a3 c3 a3 c4 a3 c5

Alternative 4

a4 c1 a4 c2 a4 c3 a4 c4 a4 c5

Alternative 5

a5 c1 a5 c2 a5 c3 a5 c4 a5 c5

Weights

W1 W2 W3 W4 W5 where a where ai

i

c cj

j

is the criteria score (performance) of alternative is the criteria score (performance) of alternative i i on criterion

  • n criterion j

j

W Wj

j

a ai

i

c cj

j

Value for Value for alternative i alternative i = = Σ

Σ

j=1 j=1-

  • n

n

Multi-Criteria Decision Analysis (MCDA)

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SLIDE 46

46 46

Analytic Hierarchy Process (AHP)

Efficacy Efficacy Risk Risk Tolerabilit y Tolerability

Criteria Weighting Criteria Weighting: : Pairwise comparisons Pairwise comparisons

  • n a 9
  • n a 9-
  • point scale

point scale AHP Scoring AHP Scoring: : Applying weights to Applying weights to criteria values criteria values

Source: Dolan JG. The Patient 2012 Source: Dolan JG. The Patient 2012

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47

Role of the analytic hierarchy process in measuring patient preferences

Sonal Singh MD MPH Johns Hopkins University

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48

When is AHP useful as tool

  • Stakeholder engagement and incorporation of experts’

and patient preferences at any stage of health care decision-making

  • Uncertainities about benefit and risk decisions

48

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49

Overview of Analytic Hierarchy Process

49

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50

Step 1. Decision Context

  • Goals, alternatives, criterion by which

alternatives are judged to meet the goal

  • Stated goal: Best (safe and effective)

treatment for type 2 diabetes

  • Comparators: Product A vs B (standard)
  • Criteria: Maximize benefits via glucose

reduction and minimize adverse effects (hypoglycemia, CV effects, fractures, lactic acidosis)

  • Refined after stakeholder input

50

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51

AHP Model for Type 2 Diabetes

51

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52

Step 2. Data Inputs : Assembling & Organizing Outcomes in an Evidence Matrix

  • Determine the most reliable source of evidence on each
  • utcomes
  • Regulatory context may require critical attention to data

sources- Evidentiary standards of the harm? Single clinical trial, meta-analysis of trials, observational studies, Adverse Event Reports

52

Singh S, et al Drug safety assessment in clinical trials: methodological challenges &opportunities. Trials 2012 ;13(1):138

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Step 3. Comparison among alternative criteria using AHP pairwise comparisons

  • Comparisons among criterion : AHP Pairwise comparisons

conducted to determine the priorities of the criteria relative to decision goal.

  • Baseline analysis assume all criteria equally important
  • Pair wise comparisons are conducted among the alternative drugs

with regard to fulfilling each criterion

53

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54 Verbal judgment of preferences Numerical rating Extremely preferred 9 Very strongly to extremely 8 Very strongly 7 Strongly to very strongly 6 Strongly preferred 5 Moderately to strongly 4 Moderately preferred 3 Equally to moderately 2 Equally preferred 1 AHP Pairwise comparison scale

54

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55

Comparison among

  • ptions/criteria
  • Compare the ability of alternative options to achieve

the decision goal by making comparison among alternatives for each criterion

  • Standard AHP pairwise comparisons among

alternatives for each of the criterion

  • Same pairwise comparison to determine the priorities
  • f the each of the criteria relative to decision goal.

55

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56

Combining judgments to evaluate how alternatives meet goal

  • Standard AHP process weighting to combine results of

judgment made in Step 3 to determine relative ability of drugs to meet stated goal

  • After all the comparisons are made they are combined to

create a normalized, ratio scale that summarizes the results

  • f the direct and indirect comparisons made among the

decision elements

  • Relative differences > 1.1 considered significant

56

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57

57

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58

58

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59

Sensitivity analysis

  • Explore impact of different judgments on relative

importance of criteria varying priorities from 0 (no importance) to 1 (most important).

  • Input on relative importance from various

stakeholders.

  • Conduct additional sensitivity analysis to determine

impact on decision goals

  • Vary probability inputs
  • Vary preferences

– Input on relative importance (priorities) from various stakeholders

59

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60

Consistency

  • Measure consistency among comparisons to estimate

consistency ratio

  • Perfect consistency occurs when judgments are transitive

and numerically consistent

– i.e. if a/b = 2 and b/c = 3, a/c should = 6

  • AHP does not require transitivity or numerical consistency
  • Generally 10 ( or 15% ) inconsistency (ratio <=1) is tolerated

because it is one order of magnitude smaller than the scale (0-1)

60

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61

Validation

  • AHP has been extensively validated
  • AHP is a method of measurement
  • If the underlying assumptions met and it is

technically AHP is considered industry standard

61

Whitaker R. Validation examples of the Analytic Hierarchy Process and Analytic Network Process. Math Comput Model. 2007.46:840-59

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62

Advantages of AHP

  • Helps to improve judgement
  • Low cognitive stress
  • Hierarchial structure
  • Flexible, easy to apply
  • Possible to use us a group consensus

building tool in small groups

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63

Limitations of AHP

  • Task sometimes seen as less realistic
  • Some assumptions, such as those of

independence may be unrealistic in certain scenarios

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64

Facilitating preference-concordant decisions by patients

  • R. Scott Braithwaite, MD, MS, FACP

President, Society for Medical Decision Making Chief, Division of Comparative Effectiveness and Decision Sciences Professor of Medicine and Population Health New York University School of Medicine

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65

Disclaimer

  • Not an expert on preference elicitation though

many in SMDM are

– Methods concierge service

  • Will focus on gathering and presenting

information to facilitate patient-centered decision making

–Preference-concordance

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66

First steps towards preference- concordance

  • What do patients expect of FDA?

– “Safety” is context-dependent – May be easy place to hide but hard place for patient decision making – Does risk aversion of FDA match risk aversion of consumers? » Societal versus individual preference?

  • Establish explicit systematic quantitative criteria for

harm/benefit assessment

– Risk tolerance may vary depending on presence of alternatives and seriousness of untreated risk

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67

Does FDA try hard enough to pick up “harm” signal?

  • Add generic QOL measure (e.g. EQ 5-D) to the more

typical disease-specific measures

– May pick up “harm” signal otherwise undetected

  • Be explicit about whether studies have power

sufficient to pick up amount of harm that would

  • ffset hypothesized benefit
  • Are comparators the next best alternative?
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68

Does one size fit all?

  • Preference-concordant decisions may require

different messaging for different harm/benefit

  • Should FDA have different “labels” reflecting

– Magnitude of potential treatment harm? – Size of harm/benefit ratio – Certainty of harm/benefit ratio

»Statistical uncertainty (insufficient power to detect harms sufficient to offset benefits), »Evidence uncertainty (study design limitations or uncertainty regarding effect

  • f comparator)

»Uncharacterized heterogeneity of treatment effect

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69

What different “labels” could look like: one vision

  • “Safe”
  • “Benefits proven to exceed risks for most

patients”

  • “Benefits proven to exceed risks for some

patients”

  • “Benefits likely to exceed risks for most

patients”

  • “Benefits likely to exceed risks for some

patients”

– +/- “harms may be severe” –May complement drug “fact box”

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70

FDA as “learning institution”

  • Important to test whether information from

FDA facilitates patient-centered and preference-concordant decisions

  • Should FDA routinely conduct research using

standard instruments for decision quality?

– Decision satisfaction, – Decision regret – Decision conflict – Knowledge

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SLIDE 71

Panel Questions

  • What methods and tools can be used to collect

patient information?

  • What are the relative strengths and limitations of

these methods and tools?

  • What approaches should be used to collect patient

preference information?

  • Who should collect patient preference

information?

  • What solutions do you see going forward?

For the online webcast: Please submit your questions to the panel via the chat box. The

  • nline hosts will be collecting the questions during the session

to be brought to the panel moderator during the panel discussion.