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Patient Preferences in Selecting Treatment Endpoints F. Reed - - PowerPoint PPT Presentation

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Research Triangle Institute The Use of Conjoint Analysis to Elicit Patient Preferences in Selecting Treatment Endpoints F. Reed Johnson, PhD Distinguished Fellow and Principal Economist Research Triangle Institute Integrating Stakeholder

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Research Triangle Institute

RTI International is a trade name of Research Triangle Institute.

www.rti.org

The Use of Conjoint Analysis to Elicit Patient Preferences in Selecting Treatment Endpoints

  • F. Reed Johnson, PhD

Distinguished Fellow and Principal Economist Research Triangle Institute Integrating Stakeholder Preferences in Comparative Effectiveness Research

August 27, 2012

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RTI International Research Triangle Institute

Comparative Effectiveness Research

  • Compares the benefits and harms of alternative

interventions

  • Assists patients, physicians, and regulators to make

informed decisions

Institute of Medicine, 2009

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RTI International Research Triangle Institute

Comparisons for whom?

  • Comparing benefits and harms and making informed

decisions requires identifying relevant endpoints

  • Increased concern about patient involvement in protocol

development

  • “When asking the public to assist in determining health

priorities, we should use techniques that allow people to reveal their true preferences. If not, why bother asking them at all?” Gafni, Social Science and Medicine, 1995

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RTI International Research Triangle Institute

Health-State Utilities

Standard Gamble/Time Tradeoff EQ-5D Tariffs QALYs

CEA, reimbursement

Stated Preferences

Discrete Choice Tailored Preference Weights, HTE, MAR, MAB, WTP

CEA, CBA, licensing, adherence, clinical guidelines

Elicitation Formats Example Instruments Uses

Patient- Reported Outcomes

Likert Scale SF-36 HRQoL Scores

CEA, licensing

Types of Self-Reported Data

Metrics

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RTI International Research Triangle Institute

Health-State Utility versus Preference Utility: Determinants

PREFERENCE UTILITY

  • Clinical Outcomes
  • Duration
  • Treatment factors

–

Side Effects/Tolerability

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Dosage Method/Frequency

–

Cost

  • Process factors

– Health-Care Setting

–

Physician interactions

  • Personal factors

– Age, gender, education, etc.

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Health history

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Financial circumstances

HEALTH-STATE UTILITY

  • Clinical outcomes
  • Duration

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RTI International Research Triangle Institute

Labels

  • Conjoint (consider jointly) analysis
  • Discrete-choice experiments
  • Stated-choice surveys

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Choice-Experiment Methods

  • Treatment alternatives consist of combinations of

features.

  • Preferences among treatment alternatives depend on

the relative importance of features.

  • Respondents state preferences for series of

constructed, hypothetical treatment alternatives.

  • Statistical model estimates preference weights

consistent with observed choices.

  • Preference weights quantify relative importance as the

willingness to accept tradeoffs.

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Example Benefit-Risk Tradeoff Question

Osteoarthritis

Feature Treatment A Treatment B

PAIN STIFFNESS STOMACH PROBLEMS Occasional mild symptoms. Treat with over

  • the -

counter medicines Frequent moderate symptoms. Treat with a prescription medicine RISK OF BLEEDING ULCER 1 patient out of 100 (1 %) will have a bleeding ulcer 5 patients

  • ut of

100 (5 %) will have a bleeding ulcer RISK OF HEART ATTACK or STROKE 5 patients out of 100 ( 5 %) will have a stroke 15 patients out of 100 ( 15 %) will have a heart attack

No Stiffness Extreme Stiffness No Stiffness Extreme Stiffness No Pain Extreme Pain No Pain Extreme Pain

Efficacy Mild- Moderate Side Effects Serious Side-Effect Risks

Which treatment would you choose if these were the only options available?

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1.00 0.72 0.0 0.2 0.4 0.6 0.8 1.0 "Best" "Satisfactory" 1/day 2/day

Why are T2DM patients inadherent?

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Glucose control

 = +0.28

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Why are T2DM patients inadherent?

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1.00 0.72 0.89 0.28

0.0 0.2 0.4 0.6 0.8 1.0 "Best" "Satisfactory" 1/day 2/day

Glucose control Number of Injections

 = +0.28  = -0.61

Hauber AB, Mohamed AF, Johnson FR, Falvey H. Treatment preferences and medication adherence of people with type 2 diabetes using oral glucose-lowering agents. Diabet Med. 2009;26:416-24.

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Physician Versus Patient Preferences

Hepatitis B

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Lescrauwaet B, Mohamed AF, Johnson FR, Hauber AB. Do patients and physicians have similar preferences for health care decisions involving uncertain outcomes for chronic hepatitis B in Germany and Turkey? Poster presented at the International Society for Pharmacoeconomics and Outcomes Research 16th Annual International Meeting; May 2011. Baltimore, MD.

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RTI International Research Triangle Institute

Physician Versus Patient Preferences

Hepatitis B

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Lescrauwaet B, Mohamed AF, Johnson FR, Hauber AB. Do patients and physicians have similar preferences for health care decisions involving uncertain

  • utcomes for chronic hepatitis B in Germany and Turkey? Poster presented at the International Society for Pharmacoeconomics and Outcomes Research

16th Annual International Meeting; May 2011. Baltimore, MD.

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Physician Versus Patient Preferences

Hepatitis B

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Lescrauwaet B, Mohamed AF, Johnson FR, Hauber AB. Do patients and physicians have similar preferences for health care decisions involving uncertain

  • utcomes for chronic hepatitis B in Germany and Turkey? Poster presented at the International Society for Pharmacoeconomics and Outcomes Research

16th Annual International Meeting; May 2011. Baltimore, MD.

Most important Least important

German patients Renal toxicity Weight of evidence German physicians Efficacy Weight of evidence Turkish patients Weight of evidence Fracture risk Turkish physicians Renal toxicity Fracture risk

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Maximum Acceptable Risk Calculation

Renal Cell Carcinoma

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Wong MK, Mohamed AF, Hauber AB, Yang J-C, Liu Z, Rogerio J, et al. Patients rank toxicity against progression-free survival in second-line treatment of advanced renal cell carcinoma. J Med Econ. 2012 Jul 3. doi: 10.3111/13696998.2012.708689. [Epub ahead of print].

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Maximum Acceptable Risk Calculation

Renal Cell Carcinoma

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Wong MK, Mohamed AF, Hauber AB, Yang J-C, Liu Z, Rogerio J, et al. Patients rank toxicity against progression-free survival in second-line treatment of advanced renal cell carcinoma. J Med Econ. 2012 Jul 3. doi: 10.3111/13696998.2012.708689. [Epub ahead of print].

 = +0.84

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 = 0.84

Maximum Acceptable Risk Calculation

Renal Cell Carcinoma

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Wong MK, Mohamed AF, Hauber AB, Yang J-C, Liu Z, Rogerio J, et al. Patients rank toxicity against progression-free survival in second-line treatment of advanced renal cell carcinoma. J Med Econ. 2012 Jul 3. doi: 10.3111/13696998.2012.708689. [Epub ahead of print].

 = +0.84

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Maximum Acceptable Breast-Cancer Risk

Vasomotor Symptoms

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Severe to no symptoms Severe to mild symptoms Severe to moderate symptoms

0.025

Maximum Acceptable Risk

0.020 0.015 0.010 0.005 0.000 Absolute Risk Relative Risk

Johnson FR, Ozdemir S, Hauber AB, Kauf T. Women's willingness to accept risk for perceived vasomotor symptom

  • relief. J Womens Health. 2007;16(7):1028-40.
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Maximum Acceptable Breast-Cancer Risk

Vasomotor Symptoms

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Severe to no symptoms Severe to mild symptoms Severe to moderate symptoms

WHI Risk

0.025

Maximum Acceptable Risk

0.020 0.015 0.010 0.005 0.000 Absolute Risk Relative Risk

Johnson FR, Ozdemir S, Hauber AB, Kauf T. Women's willingness to accept risk for perceived vasomotor symptom

  • relief. J Womens Health. 2007;16(7):1028-40.
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Some Methodological Challenges

  • Hypothetical bias

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Inexperience with condition

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Socially acceptable responses

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Stated preference/revealed preference experiments

  • Cognitive challenges

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Effective description of clinical endpoints

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Surrogate markers

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Risk concepts

  • Consensus among researchers

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Experimental design

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Statistical analysis

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Discussion

  • Effective incorporation of patient perspectives in protocol

development requires quantification.

  • Idea of treating patient-preference measures as

evidence is novel for most clinicians.

  • DCE methods offer methods for quantifying relative

values of health endpoints.

  • Good validity and reliability for relatively simple trade-off
  • problems. Applications to more difficult problems is an

active area of research.

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