PCORI Update: Lenore Arab, PhD, MS Value of Information: Starting the Discussion Doug Bell, MD, PhD 12/04/2012

Director, Dr. Lenore Arab Project Coordinator, Robin Faria Study 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 Interest 1.Development of methods for patient centeredness 2.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, 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. Improving Methods for Conducting Patient-Centered Outcomes Research II

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?

 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 No17 Undecided Total

PCORI Staff Choice of Disease Priority Areas

Douglas Bell, MD, PhD Value of Information: Starting the Discussion

ParameterTreatment A (95% CI) Treatment B (95% CI) Cure rate94% (86.0 to 98.6%) 90% (83.5 to 95.0%) Life expectancy if cured20 years Life expectancy if treatment fails5 years Costs of managing treatment failure$50,000 Overall complication rate20% (10.0 to 27.5%) 5% (2.1 to 10.0%) Mortality rate after complication10% (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

OutcomeTreatment ATreatment B Mean life expectancy18.72 years18.40 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 $750 Simulation Number Net Benefits Treatment A Net Benefits Treatment B Maximum Net Benefits Preferred Strategy Opportunity Cost 1$4,180$4,306 B$0 2$2,273$2,415 B$0 3$7,095$4,507$7,095A$2,588 4$3,186$4,017 B$0 5$3,504$3,433$3,504A$72 6$5,698$6,740 B$0 7$4,762$3,718$4,762A$1,044 8$3,960$1,919$3,960A$2,041 9$5,071$5,964 B$0 10$1,904$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 = E  max j NB(j,  ) - max j E  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,  Minimal Modeling Approaches to Value of Information Analysis for Health Research, Metlzer, et al  Systematizing the Use of Value of Information Analysis in Prioritizing Systematic Reviews. Hoomans, et al  Value of Information and Research Prioritization. Rein. NORC at the U of Chicago,  Value of Information Literature Analysis: A Review of Applications in Health Risk Management. Yokota and Thompson, MDM,  Value of Information on Preference Heterogeneity and Individualized Care, Basu and Meltzer, MDM,  Value-of-Information Analysis for Patient-Centered Outcomes Research Prioritization, Myers et al., References

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