Patient Centered Outcomes Research (PCOR) A Framework for Meaningful - - PowerPoint PPT Presentation

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Patient Centered Outcomes Research (PCOR) A Framework for Meaningful Assessment of Value C. Daniel Mullins, PhD Professor, University of Maryland School of Pharmacy Disclaimer: The views expressed in this presentation are solely those of the

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Patient‐Centered Outcomes Research (PCOR)

A Framework for Meaningful Assessment of Value

  • C. Daniel Mullins, PhD

Professor, University of Maryland School of Pharmacy

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Disclaimer: The views expressed in this presentation are solely those of the speaker and do not necessarily represent the views of the Patient‐Centered Outcomes Research Institute (PCORI), its Board of Governors or Methodology Committee. Acknowledgment: Parts of this presentation are based on work completed under contract PCORI‐SOL‐PCWG‐002, funded by the Patient‐ Centered Outcomes Research Institute (PCORI).

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Take‐Away Points

  • Patient‐centered outcomes research (PCOR) allows the

patient’s voice to be heard

  • Because PCOR provides more meaningful data, patients

and their health care providers

– Can make better treatment decisions – May be more adherent to evidence‐based recommendations – May contribute to reductions in wasteful spending

  • Value and return on investment (ROI) depends on

– Perspective – What is included in “costs” of disease

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Objectives:

  • Describe methods for comparative‐effectiveness research (CER)
  • Describe methods for patient‐centered outcomes research (PCOR)
  • Examine how the value proposition and cost‐effective analysis (CEA)

varies based on the types of costs included in CEA

  • Define the joint role for CEA and PCOR in formulary decision making

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Objectives:

  • Describe methods for comparative‐effectiveness research (CER)
  • Describe methods for patient‐centered outcomes research (PCOR)
  • Examine how the value proposition and cost‐effective analysis (CEA)

varies based on the types of costs included in CEA

  • Define the joint role for CEA and PCOR in formulary decision making

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Comparative Effectiveness Research (CER)

Definitions of Comparative Effectiveness Research

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Comparative Effectiveness Research (CER)

  • CER is the process of evaluating the impact of

different options for treating a given medical condition for a particular set of patients

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Comparative Effectiveness Research (CER)

CER is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical condition, or to improve the delivery of care.

‐Institute of Medicine (IOM)

Sox HC, Greenfield S. Comparative effectiveness research: a report from the Institute of Medicine. Ann Intern Med. 2009 Aug 4;151(3):203‐5.

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Comparative Effectiveness Research (CER)

  • Active comparators (i.e. not a placebo comparator)
  • Broad patient population
  • Outcomes that are meaningful to patients
  • Innovative Methods
  • Clinical trials

Electronic health records

  • Registries

Insurance claims datasets

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Comparative Effectiveness Research (CER)

“What differentiates CE from conventional medical research is that it is demand‐driven rather than investigator‐driven. Demand is defined by the needs of decision makers, including public and private payers, clinicians, patients, and professionals.”

Chalkidou K, Anderson G. Comparative Effectiveness Research: International Experiences and Implications for the United States July 2009 Available at www.academyhealth.org

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The Need for Additional Evidence

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Best Case Situation:

Threshold for Action vs. Definitive Answer

Scientific Evidence Threshold for Action Evidence Needed for Definitive Answer on Patient Outcome

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Scientific Evidence Threshold for Action Evidence Needed for Definitive Answer on Patient Outcome

Worst Case Situation:

Threshold for Action vs. Definitive Answer

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CER Closes the Evidence Gap in order to get to the Threshold for Action

Scientific Evidence Threshold for Action Evidence Needed for Definitive Answer on Patient Outcome Other data sources Clinical trials

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Filling the evidence gap

  • It’s important to fill the right gap/answer the

right question

  • Simultaneously “too little”

and “too much” info

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Patient‐Centered Outcomes Research (PCOR)

Definitions of Patient‐Centered Outcomes Research

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Patient Centered Outcomes Research (PCOR)

CER Patients PCOR

“PCOR involves the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions, as well as the delivery of health care in ‘real world’ settings.”

  • AHRQ PA-12-114

http://grants.nih.gov/grants/guide/pa-files/pa-12-114.html

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Patient Centered Outcomes Research (PCOR)

CER Patients PCOR

“Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care

  • ptions. This research answers patient-centered questions such as:
  • 1. ‘Given my personal characteristics, conditions and preferences, what

should I expect will happen to me?’

  • 2. ‘What are my options and what are the potential benefits and harms of

those options?’ …”

PCORI (http://www.pcori.org/assets/PCOR-Definition-Revised-Draft-and-Responses-to-Input.pdf) 18

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PCOR is a Win for Everyone

PATIENT RESEARCHER

Informed decision making Meaningful research Not a “one- size-fits-all” result

Interaction Continuous

Topics and research questions Translate/disseminate results Comparators and

  • utcomes

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Objectives:

  • Describe methods for comparative‐effectiveness research (CER)
  • Describe methods for patient‐centered outcomes research (PCOR)
  • Examine how the value proposition and cost‐effective analysis (CEA)

varies based on the types of costs included in CEA

  • Define the joint role for CEA and PCOR in formulary decision making

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Take‐Away Points

  • Patient engagement at every step transforms

CER‐> PCOR

  • Patient engagement is a continuous process
  • Patient engagement requires trust & respect
  • Pre‐engagement is a critical component
  • Patient engagement should involve diverse

patients including hard‐to‐reach patients

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10‐Step Process for Conducting CER *

  • 1. Topic Solicitation
  • 2. Prioritization
  • 3. Framing the Question

Public Announce‐ ments

Patient Forums Delphi Process

* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587‐8.

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* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587‐8.

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  • 4. Selection of Comparators

and Outcomes

  • 5. Creation of Conceptual

Framework

  • 6. Analysis Plan
  • 7. Data Collection

In‐person Meetings Focus Group Interviews Telecon‐ ferences Electronic Social Media Telephone Calls

10‐Step Process for Conducting CER *

* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587‐8.

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* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587‐8.

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  • 8. Reviewing & Interpreting

Results

  • 9. Translation
  • 10. Dissemination

Teach‐Back Method Critique Documents (e.g. Patient Guides) Media

10‐Step Process for Conducting CER *

* Based on Mullins CD, Adbulhalim AM, Lavallee DC. Continuous Patient Engagement in Comparative Effectiveness Research. JAMA 2012; 307(15): 1587‐8.

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Patient Engagement: Why?

  • Makes PCOR meaningful

– Framing the question so it is understood – Framing the answer so it is understood – Framing for cultural appropriateness – Does meaningful imply significant or impactful?

  • Helps with patient activation

Having the essential knowledge, skills and confidence for managing one’s own health and healthcare

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Patient Engagement: Why?

  • Public health could be improved

– Patients have unique insights – Priority setting is important – Pragmatic considerations

  • It shows an honest commitment to PCOR

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Patient Engagement: How?

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The journey begins by meeting patients where they are

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Patient Engagement: How?

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There are others along the journey who can help:

  • Community Members
  • Leaders
  • Organizational Partners
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Patient Engagement: How?

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Identifying Partnering Explaining Doing Updating

Building/Maintaining Trust and Respect Building/Maintaining Trust and Respect

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Objectives:

  • Describe methods for comparative‐effectiveness research (CER)
  • Describe methods for patient‐centered outcomes research (PCOR)
  • Examine how the value proposition and cost‐effective analysis (CEA)

varies based on the types of costs included in CEA

  • Define the joint role for CEA and PCOR in formulary decision making

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The (Patient‐Centered) Value Equation

Historically, productivity is typically not included (or used for calculating cost sharing) in formulary decision making

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The (Patient‐Centered) Value Equation

  • Indirect costs often half the total cost of illness
  • If indirect costs are considered, the “value” of

reducing amenable morbidity is twice the amount when indirect costs are added to direct medical costs savings

  • From a cost‐effectiveness viewpoint, the

incremental cost‐effectiveness ratio is cut in half

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The (Patient‐Centered) Value Equation

  • Personalized medicine

– Tailored therapy

  • Biomarkers often do not exist
  • Other patient‐level factors

– What is important to patients – Adherence and downstream costs

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Objectives:

  • Describe methods for comparative‐effectiveness research (CER)
  • Describe methods for patient‐centered outcomes research (PCOR)
  • Examine how the value proposition and cost‐effective analysis (CEA)

varies based on the types of costs included in CEA

  • Define the joint role for CEA and PCOR in formulary decision making

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Goals of an Evidence‐Based Approach

  • Better Heath Care

– Health care coverage – Access to care

  • Better Quality

– Pay‐for‐performance – Integrated care models – Improve health outcomes – Appropriate drug therapy (evidence‐based therapy)

  • Improved Efficiency

– Identify wasteful spending in the system – Decrease unnecessary ED and hospitalizations – Eliminate unnecessary or inappropriate medications

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How PCOR Informs Evidence‐Based Medicine

  • PCOR answers relevant questions

– Head‐to‐head comparisons not comparisons to placebo – “Hard outcomes” rather than intermediate endpoints

  • PCOR provides evidence on

– Effectiveness – Routine clinical practice – Broader patient populations

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Health Technology Assessment (HTA)

– Definitions

  • Health technology assessment (HTA) is a

multidisciplinary process that summarizes information about the medical, social, economic, and ethical issues related to the use of health technology in a systematic, transparent, unbiased, robust manner

  • Its aim is to inform the formulation of safe, effective,

health policies that are patient focused and seek to achieve best value

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Health Technology Assessment (HTA)

“A form of policy research that examines short‐ and long‐term consequences of the application

  • f a health‐care technology.”

(O’Donnell et al., 2009)

O’Donnell J, Pham S, Pashos C et al. Health Technology Assessment: Lesson Learned from Around the World – An Overview. Value in Health. 12 (2): S1‐S5

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Health Technology Assessment (HTA)

Evidence of safety Efficacy Patient‐ reported

  • utcomes

Real‐world effectiveness Cost and cost‐ effectiveness

Properties assessed include:

Health Care Cost, Quality, and Outcomes: ISPOR Book of Terms. Lawrenceville, NJ: International Society for Pharmacoeconomics & Outcomes Research, 2003

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Health Technology Assessment (HTA)

HTA also assesses the impact of the following:

Health Care Cost, Quality, and Outcomes: ISPOR Book of Terms. Lawrenceville, NJ: International Society for Pharmacoeconomics & Outcomes Research, 2003

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Health Technology Assessment (HTA)

The scientific methods and data must be:

  • Transparent
  • Credible
  • Consistently

applied

O’Donnell J, Pham S, Pashos C et al. Health Technology Assessment: Lesson Learned from Around the World – An Overview. Value in Health. 12 (2): S1‐S5

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Health Technology Assessment (HTA)

Quality

  • The aggregate of quality ratings for individual studies,

predicated on the extent to which bias was minimized.

Quantity

  • Magnitude of effect, numbers of studies, and sample

size or power.

Consistency

  • For any given topic, the extent to which similar findings

are reported using similar and different study designs.

http://www.ahrq.gov/clinic/epcsums/strengthsum.htm#Rating

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CER/PCOR

  • Active comparators (i.e. not a placebo comparator)
  • Broad patient population
  • Outcomes that are meaningful to patients
  • Innovative Methods
  • Clinical trials

Electronic health records

  • Registries

Insurance claims datasets

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CER/PCOR + Pharmacoeconomics

  • Active comparators (i.e. not a placebo comparator)
  • Broad patient population
  • Outcomes that are meaningful to patients
  • Innovative Methods
  • Clinical trials

Electronic health records

  • Registries

Insurance claims datasets

44 Potential for reduction in wasteful spending

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  • Market incentives historically are misaligned

– Potential for underuse or overuse of drugs – Potential for high economic barriers to appropriate use, particularly when focus is only on direct medical costs

  • PCOR helps to assure that formulary and coverage decisions

consider what is important to patients

– What do patients value? – What are my options and what are the potential benefits and harms of those options?

  • Value‐based purchasing depends on

– Value to whom – Cost to whom – Which costs are considered

Summary

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