an introduction to resources - national health council · 2020-04-18 · research on patient...

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An Introduction to ResourcesELISABETH M. OEHRLEIN, PHD, MS

2:30-3:15 PM

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Copyright © 2019 National Health Council. All rights reserved.

NHC Value Initiative◦ Patient-Centered Value Model Rubric◦ Get-Ready Check List◦ NHC Value Workgroup ◦ Health Economics & Value Assessment Education◦ A Dialogue on Patient-Centered Value Assessment:

Overcoming Barriers to Amplify the Patient Voice◦ Research on patient involvement in HTA and patient

perspectives on value

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Value Model Rubric

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NHC Rubric Development• 2016 NHC convened panel of

member organizations, payers, pharma, professional and research organizations

• Identify characteristic of patient centeredness that would illustrate high quality engagement

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Characteristics of MeaningfulPatient Engagement in

Model Development

Characteristics of Patient Centeredness in

Model Development

Patient Partnership§ Direct relationships and

partnerships are bidirectional, reciprocal, and continuous.

§ Communications are open, honest, and clear. Engagement goals, participants, methods, desired impacts, and actual impacts are clearly outlined and transparent.

§ Created by engaging, informing, and actively listening to people with chronic conditions.

§ Respectful of and responsive to patient preferences, needs, and values in context of their own social worlds.

Transparency

Inclusiveness

Diversity

Outcomes

Data Sources

High LowHigh Low

Value Model Rubric

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Planning Drafting and Refinement

Dissemination and Implementation Evaluation Update and

Maintenance

Domains of a patient-centered value model

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Patient partnership

Transparency to patients

Inclusiveness of patients

Diversity of patients/populations

Patient-centered data sources

Patient-centered outcomes

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Patient Partnership

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Characteristic High Engagement Low EngagementDirect input is collected from a wide range of patients as suited to the disease, population, and context

Patient survey, interviews of disease-specific patient advocacy group staff, and/or use of a disease-specific registry

Only include use of data from randomized controlled trials reporting clinical outcomes.

Consideration High Patient-Centeredness

Low Patient-Centeredness

Rationale to substantiate the adequacy of the ratio of patient to non-patient participants is provided

Of a 10-member advisory committee, 2 members were patients and 1 was a family caregiver, and rationale was provided for stakeholder composition

No patients served on an advisory committee and no rationale was provided for stakeholder composition

Patients should be involved in every step of the development and dissemination processes


Transparency to Patients

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Characteristic High Engagement Low EngagementThe process for selection of patient representatives is transparent

The specific criteria used to identify, select, and invite patient representatives were disclosed, along with a rationale

No systematic process and/or criteria for selecting patient representatives was provided

Assumptions and inputs – and each step in the process – should be disclosed in an understandable way and in a timely fashion

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Inclusiveness of Patients

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Consideration High Patient-Centeredness Low Patient-Centeredness

The draft framework/model is vetted with a broad coalition of stakeholders, including patients

A broad coalition of patient organizations was given appropriate time to vet the framework/model

Notification of public comment period(s) was not widely distributed

Perspectives drawn from a broad range of stakeholders, including the patient community, should be reflected


Diversity of Patients/ Populations

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Characteristic High Engagement Low EngagementDiversity of the patient population is acknowledged and considered

Thoughtful consideration was given to differences in patient perceptions of value across relevant patient subpopulations, including populations at-risk and those with early-and late-stage disease

The model assumed the patient population is homogenous and takes a ‘one-size-fits-all’approach

Differences across patient subpopulations, trajectory of disease, and stage of a patient’s life should be accounted for

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Outcomes Patients Care About

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Consideration High Patient-Centeredness Low Patient-CenterednessEconomic inputs are considered in the context of a patient’s experience

The model incorporated costs from a variety of stakeholder perspectives, including patient out-of-pocket costs

Only cost issues from the payer perspective were included without rationale for exclusion of patient costs

The outcomes integrated should include those patients who have been identified as important and consistent with their goals, aspirations, and experiences


Patient-Centered Data Sources

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Characteristic High Engagement Low EngagementExisting sources of patient-generated health data (e.g., patient registries or patent-reported outcomes) are identified and considered

Data on patient-reported outcomes were used, and the sources well described

No effort was made to identify sources of patient-reported data on physical function, though this was identified by patients as the outcome of highest priority

Variety of credible data sources are used allowing for incorporation of new information and account for the diversity of patient populations and patient-centered outcomes, especially those from real-world settings and reported by patients directly

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Rubric Notes§ Groups support the intent behind value frameworks § Do not wish to waste resources on unnecessary treatments§ But, patients want information to make informed decisions on

the basis of what is or is not valuable to them§ Rubric is intended to assist all stakeholders and support

developers in conceptualizing plans for meaningfully engaging patients

§ Rubric should be considered a guide for patient-centeredness good practices

§ Living document, to be refined over time on the basis of feedback from patient, patient group, framework developer, and others

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Step 1. Advance Preparation

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Search: Who are the framework developers working in your area of interest? Assess: Understand the processes of each framework-development organization working in your area of interest.Learn: Familiarize yourself with the developer’s website, materials, and past value-related work.Investigate: What is the FDA review timeline for products in your disease area, and how will it impact the timeline for value framework development?Align: Does the developer’s work align with the National Health Council Value Model Rubric?

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Step 2. Decide Whether to Engage Articulate: Everyone involved should understand the issues.Evaluate: Internally evaluate your organization’s appetite for engagement.

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Step 3. Consider Coalitions and Partnerships Mobilize: Bring together relevant organizations and stakeholders

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Step 4. Refine Your Strategy Assemble: Your strategy is best substantiated with data and facts.

Gather: Information from your community is a key asset.

Search: Fill gaps in data where you are able.

Articulate: State your intentions clearly.

Re-evaluate

Message: Have your message ready to deliver.

Prepare: Be prepared for some negativity toward patient groups.

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Step 5. Engage with Developers

Approaching a developer: Have a game plan mapped out.

Objective(s): Define your objectives for the conversation. Communication with Developers

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Step 6. Follow Through Follow-up: Demonstrate your continued commitment.

Visibility: Use every opportunity to be viewed as an important stakeholder

Communication: Keep your constituents informed.

Reliability: Commit for the long haul.

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NHC Value Work Group• Representatives from NHC patient-group members and non-member patient organizations by invitation

• Meets monthly by teleconference to share and learn from each other’s’ experiences, and to stay up-to-date on topics related to value assessment

• Upcoming topics:• Communications on Value and Value Assessment

• Multi-criteria decision analysis

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A Dialogue on Patient-Centered Value Assessment◦ Articulate a shared vision

for what marks success in enhanced patient centricity in VA and

◦ Outline tangible, feasible actions toward achieving that success. The actions may be on the part of patient groups, VA developers, or both in collaboration

22https://www.nationalhealthcouncil.org/dialogue-patient-centered-value-assessment-overcoming-barriers-amplify-patient-voice

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What is a shared vision for what marks success in enhanced patient centricity in value assessment?• Ultimate goal of patient-centered VA is for patients to have access to

treatments they need at prices they can afford.• Exists when patients are engaged, respected, and understood

throughout the entire process and their input is incorporated and guides decision making.

What are tangible, feasible actions that can be taken to achieve that success?• Patient groups: engage VA bodies when developing or updating

registries and/or surveys • VA bodies: data needs can be communicated to relevant patient groups

in advance of a VA (the earlier, the better). • Together: Following an appraisal, the VA body and patient groups can

debrief about how data submitted by patient groups were/were not useful, and how data collection or presentation can be improved for future use

https://www.nationalhealthcouncil.org/dialogue-patient-centered-value-assessment-overcoming-barriers-amplify-patient-voice


Health Economics & Value Assessment Education

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• Increase patient-community capacity to engage on value, especially regarding value frameworks and assessments, as well as other value-related research, programs, and initiatives.

CHAPTER 1• Role of health economics• Introduction to basic terms CHAPTER 2

• The Mysterious QALY• Budget-Impact Analysis

CHAPTER 3• Diving into Value Frameworks• What patients and patient

groups need to know about Value Frameworks

• The NHC Get Ready Checklist

CHAPTER 4 (under development)• Heterogeneity of Treatment Effect• Sensitivity Analysis in Value

Assessment

https://www.nationalhealthcouncil.org/dialogue-patient-centered-value-assessment-overcoming-barriers-amplify-patient-voice

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Available modules• The Current Health Care

Environment and the Role of Health Economics

• Getting to Know the Lingo• Unlocking the Mysteries of the

Quality-Adjusted Life Year (QALY)• Budget Impact Analysis• Value Frameworks• The Patient Voice in Value: The

NHC Patient-Centered Value Model Rubric

• Steps to Activation: The NHC Get-Ready Checklist

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Clinical Outcome Assessment Series Live and Archived Online

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November2018

Patient-Reported Outcomes and Patient-Centered Outcomes: Is There a Difference? (Eleanor Perfetto, PhD, MS)

December2018

FDA’s Roadmap to COA Selection/Development for Clinical Trials (Laurie Burke, RPH, MPH)

January2019

Untangling the Terms: Endpoints, Items, Outcomes, PROs, PROMs, PRO-PMs… (Ashley Slagle, PhD)

February2019

Successful COAs: It all starts with the “concept of interest” and “context of use” (Rosie Love, MPH)

March2019

What do we mean by validation of a measure? (Eleanor Perfetto, PhD, MS)

April2019

Measuring Patient Experiences: Distinguishing Between Patient-Reported Outcomes (PROs) and Patient Preferences (Bennett Levitan and Pauline McNulty)

May2019

Lessons Learned From Developing A PRO Measure: The Experience Of One Patient Advocacy Group (Dory Kranz)

June2019

What is the Difference Between Health-Related Quality of Life (HRQoL) and Patient-Reported Outcomes (Laurie Burke, RPH, MPH)

July2019

Core Outcome Sets: What Are They? (Donna Messner, PhD)

September2019

Build New or Adapt Old? (Stacie Hudgens, MA, Chief Executive Officer (CEO) Strategic Lead, Regulatory & Access)

October2019

Patient Input to Guide Clinician-Reported Outcomes (ClinROs) (John Powers III, MD)

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Upcoming NHC Tools and Resources

• Glossary of Value Assessment Terms

• Finalized Fall 2019

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Upcoming NHC Tools and Resources

• The NHC Considerations Guide for Patient Organizations Developing Comments on Value Assessment

• Finalized Fall 2019

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ICER Patient Participation Guide

•In this guide:• What is ICER?• What does ICER do?• How you can participate in

ICER’s process

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https://icer-review.org/patient-participation-guide/

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IVI Partnering with Patients Principles and Commitments

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https://www.thevalueinitiative.org/wp-content/uploads/2018/04/2018-04-12.IVI-Partnering-with-Patients_Web-Version.pdf

Principle 1: IVI will involve multiple patient stakeholders in value assessment.

Principle 2: IVI will employ robust methods to identify and involve patient stakeholders.

Principle 3: IVI will partner with patient stakeholders at all stages of research.

Principle 4: IVI will continuously evaluate and refine patient partnership action.

Principle 5: IVI will commit resources for continuous patient engagement.


NCCN Resources• Submission Request to the NCCN Guidelines Panels

• NCCN Evidence Blocks™ User Guide

• NCCN Guidelines for Patients

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EUPATI HTA resources

33https://www.eupati.eu/webinar/webinar-experience-patient-involvement-hta-processes/

Experiences of patient advocates in HTA processes along with some questions and challenges with such involvement and work.

Guidance for patient involvement in HTA

Patient experiences abroad


HTAi Ethical Considerations for Patient Groups Collecting Information

34https://htai.org/wp-content/uploads/2018/02/PCISG-Resource-EthicsShortGuide_May16.pdf

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NPC’s Current Landscape: Value Assessment Frameworks

•Compares and contrasts key Value Framework characteristics • Intended purposes • Development processes • Methods• Elements of value (benefits

and costs)

35https://www.npcnow.org/issues/value/measuring-value/value-assessment-frameworks

Additional Resources?

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Questions?

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an introduction to resources - national health council · 2020-04-18 · research on patient...

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