PCORI Update: Lenore Arab, PhD, MS

Value of Information: Starting the Discussion

Doug Bell, MD, PhD12/04/2012

Director, Dr. Lenore Arab

Project Coordinator, Robin FariaStudy Coordinator, Erica Sasman

The Rapid Response Team:

Who We Are

Outline

PCORI Evolving Structure PCORI Status of Current Applications Currently Open RFAs: January & February

2013 LoIs Future PCORI Disease Priority Areas Value of Information Analyses- why you

should care, and what it might entail

PCORI Organization: Divisions

Status of Current Proposals

Topic Improving Methods for Conducting Patient-

Centered Outcomes Research Due Dates

LOI (required): 1/15/2013; 6/15/2013; 10/15/2013 Application: 3/13/2013; 8/15/2013; 12/17/2013

Funding $250,000 direct per year for three years (shorter

duration encouraged)

PCORI Funding Opportunity 5: Inaugural Cycle

Specific Questions of Interest1.Development of methods for patient centeredness2.Research in methods to conduct systematic reviews of

patient-centered comparative effectiveness research.3.Development of methods for generating, selecting, and

prioritizing topics for research and for including patients and stakeholders in the peer‐review process.

4.Development and refinement of general analytic methods.

5.Development and refinement of design‐specific analytic methods.

Improving Methods for Conducting Patient-Centered Outcomes

Research I

Specific Questions of Interest, Continued6. Research that determines the validity and

efficiency of data sources commonly used in PCOR.7. Research related to Patient-Centered Outcomes

(PCOs) and Patient-Reported Outcomes (PROs).8. Research in methods to enhance the

reproducibility, transparency, and replication of PCOR research.

9. Research that evaluates and compares strategies for training researchers, patients, and other stakeholders in the methods of patient‐centered outcomes research.

Improving Methods for Conducting Patient-Centered Outcomes

Research II

PCORI Funding Opportunity, Cycle III – 3rd Opportunity

Topics Assessment of Prevention, Diagnosis, and Treatment

Options Improving Healthcare Systems Communication and Dissemination Research Addressing Disparities

Due Dates LOI (required): 2/15/2013 Application: 4/15/2013

Funding $500,000 direct cost per year for xx years?

Purpose of Survey: to actively engage faculty interested in PCORI research and find ways to further help the research community in areas of interest

Incentive: two winners per week for $50 gift card Preliminary Results on Week 1 of Survey:

34 respondents First winners to be announced Thursday Cycle II and Cycle III Interest:

PCORI Interest Survey

Assessment of

Prevention

Communication &

Dissemination

Improving Healthcare Systems

Addressing Disparities

Accelerating Patient-Centered Outcomes

Yes 3 0 1 2 1 1No 17

Undecided 13 3 2 3 6 5Total 33 3 3 5 7 6

PCORI Staff Choice of Disease Priority Areas

Douglas Bell, MD, PhD

Value of Information: Starting the Discussion

Parameter Treatment A(95% CI)

Treatment B(95% CI)

Cure rate 94%(86.0 to 98.6%)

90%(83.5 to 95.0%)

Life expectancy if cured 20 years

Life expectancy if treatment fails

5 years

Costs of managing treatment failure

$50,000

Overall complication rate 20%(10.0 to 27.5%)

5%(2.1 to 10.0%)

Mortality rate after complication 10%(8.2 to 12.0%)

Cost of complication $10,000

Costs associated with fatal complication

$50,000

Input Variables and Values for Treatment A and B

Outcome Treatment A

Treatment B

Mean life expectancy 18.72 years 18.40 years

Mean costs $10,940 $10,725

Mortality from complications of treatment

2.0% 0.5%

Expected Values of Outcomes of Interest Given

Treatment A or B

Outcomes from 10 Simulations of Treatment A and B Assuming a WTP

Threshold of $750Simulation Number

Net Benefits Treatment A

Net Benefits Treatment B

Maximum Net Benefits

Preferred Strategy

Opportunity Cost

1 $4,180 $4,306 $4,306 B $0

2 $2,273 $2,415 $2,415 B $0

3 $7,095 $4,507 $7,095 A $2,588

4 $3,186 $4,017 $4,017 B $0

5 $3,504 $3,433 $3,504 A $72

6 $5,698 $6,740 $6,740 B $0

7 $4,762 $3,718 $4,762 A $1,044

8 $3,960 $1,919 $3,960 A $2,041

9 $5,071 $5,964 $5,964 B $0

10 $1,904 $5,123 $5,123 B $0

Expected value (mean of simulations 1-10)

$4,163 $4,214 $4,789 $575

An approach to research prioritization which uses Bayesian methods to estimate the potential benefits of gathering further information (through more research) before making a decision (Meyers, et al., 2012)

A tool of the decision sciences, estimates the potential economic losses associated [with] choosing suboptimal policies when that decision is made with uncertain information (Rhein, 2012)

A decision analytic technique that explicitly evaluates the benefit of collecting additional information to reduce or eliminate uncertainty. (Yokota and Thompson, 2004)

Value of Information

General Challenges to the Use of VOI for Research

Prioritization Resources needed to develop appropriate models

Personnel Time Computing Resources

Scope of VOI Prioritizing across disease areas Prioritizing within a condition Prioritizing specific comparative effectiveness

research Stakeholder Engagement

Lack of familiarity with the methodology Timing of VOI

Personnel Time Computing Resources

“minimal modeling appears to be most useful when an intervention affects quality of life alone” 3

Resources Needed to Conduct VOI

Difference between the expected value given perfect information and the value given current information

i.e. the upper bound of the opportunity cost of making a wrong decision; any effort to improve the quality of available data that costs less than the EVPI is worth pursuing

Expected Value of Perfect Information (EVPI)

Calculating EVPI

EVPI is calculated as the expected value of a decision made with perfect information minus the expected value of a decision made with the existing information:

EVPI = EmaxjNB(j,) - maxjE NB(j,)

Go/no go measurement to determine if additional research is appropriate

Comparison for cost effectiveness of research across different interventions

Using EVPI for Research Prioritization

The value of being able to know the outcome of another related uncertainty instead of the original value itself before making a decision. It is quantified as the highest price the decision-maker is willing to pay for being able to know the uncertainty before making a decision. Note that it is essentially the value of perfect information on the second variable.

Expected Value of Partial Perfect Information (EVPPI)

Using VOI by Research Sponsor, such as PCORI

Perform VOI prior to solicitation for research proposals and only request areas identified as having acceptable EVPI/EVPPI

Perform VOI after initial review of proposals as part of funding decisions with higher EVPI/EVPPI receiving higher priority for funding

Research sponsor could encourage or require investigators to include VOI analysis as part of rationale for proposals

Treatment Decision Tree

Justify additional research in advance of adapting specific interventions

Identifying which areas contribute the most to uncertainty

Common uses of VOI

Patient-Centered Outcomes Research Institute Board of Governors Meeting, www.pcori.org, 2012.

Minimal Modeling Approaches to Value of Information Analysis for Health Research, Metlzer, et al. www.ahrq.gov, 2011.

Systematizing the Use of Value of Information Analysis in Prioritizing Systematic Reviews. Hoomans, et al. www.ahrq.gov, 2012.

Value of Information and Research Prioritization. Rein. NORC at the U of Chicago, 2012.

Value of Information Literature Analysis: A Review of Applications in Health Risk Management. Yokota and Thompson, MDM, 2004.

Value of Information on Preference Heterogeneity and Individualized Care, Basu and Meltzer, MDM, 2007.

Value-of-Information Analysis for Patient-Centered Outcomes Research Prioritization, Myers et al., www.pcori.org, 2012.

References

Thank You!

Contact us at: RRT@mednet.ucla.edu or (310) 267-4258