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PCORI Update: Lenore Arab

, PhD, MS

Value of Information:

Starting the Discussion

Doug Bell, MD, PhD

12/04/2012

The Rapid Response Team:

Who We Are

Director, Dr. Lenore Arab

Project Coordinator, Robin Faria

Study Coordinator, Erica Sasman

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

PCORI Funding Opportunity 5:

Inaugural Cycle

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)

Improving Methods for Conducting

Patient-Centered Outcomes Research I

Specific Questions of Interest

1.

Development of methods for patient centeredness

2.

Research in methods to conduct systematic reviews of patientcentered 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 II

Specific Questions of Interest, Continued

6. 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.

PCORI Funding Opportunity,

Cycle III – 3

rd

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?

PCORI Interest Survey

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:

Yes

No

Undecided

Total

3

17

13

33

Assessment of

Prevention

Communication &

Dissemination

0

3

3

1

2

3

Improving

Healthcare

Systems

2

3

5

Addressing

Disparities

1

6

7

Accelerating

Patient-

Centered

Outcomes

1

5

6

PCORI Staff Choice of Disease Priority Areas

Value of Information: Starting the

Discussion

Douglas Bell, MD, PhD

Input Variables and Values for

Treatment A and B

Parameter

Cure rate

Life expectancy if cured

Life expectancy if treatment fails

Costs of managing treatment failure

Overall complication rate

Mortality rate after complication

Cost of complication

Costs associated with fatal complication

Treatment A

(95% CI)

94%

(86.0 to 98.6%)

20 years

Treatment B

(95% CI)

90%

(83.5 to 95.0%)

5 years

20%

(10.0 to 27.5%)

$50,000

10%

(8.2 to 12.0%)

5%

(2.1 to 10.0%)

$10,000

$50,000

Expected Values of Outcomes of

Interest Given Treatment A or B

Outcome

Mean life expectancy

Mean costs

Mortality from complications of treatment

Treatment A Treatment B

18.72 years 18.40 years

$10,940

2.0%

$10,725

0.5%

1

Outcomes from 10 Simulations of Treatment A and B Assuming a WTP Threshold of $750

Simulation

Number

Net Benefits

Treatment A

Net Benefits

Treatment B

Maximum

Net Benefits

Preferred

Strategy

Opportunity

Cost

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

4

5

2

3

6

7

8

9

10

Expected value

(mean of simulations 1-10)

$2,273

$7,095

$3,186

$3,504

$5,698

$4,762

$3,960

$5,071

$1,904

$4,163

$2,415

$4,507

$4,017

$3,433

$6,740

$3,718

$1,919

$5,964

$5,123

$4,214

$2,415

$7,095

$4,017

$3,504

$6,740

$4,762

$3,960

$5,964

$5,123

$4,789

B

A

A

B

B

B

A

B

A

$0

$2,588

$0

$72

$0

$1,044

$2,041

$0

$0

$575

Value of Information

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)

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

Resources Needed to Conduct

VOI

Personnel

Time

Computing Resources

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

Expected Value of Perfect

Information (EVPI)

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

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 = E

 max j

NB(j,

) max j

E

NB(j,

)

Using EVPI for Research

Prioritization

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

Comparison for cost effectiveness of research across different interventions

Expected Value of Partial Perfect

Information (EVPPI)

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.

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

Common uses of VOI

Justify additional research in advance of adapting specific interventions

Identifying which areas contribute the most to uncertainty

References

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.

Thank You!

Contact us at:

RRT@mednet.ucla.edu

or

(310) 267-4258

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