Integrating the Patient Perspective Into Value Frameworks Avalere Health An Inovalon Company August, 2017
Speakers and Agenda Josh Seidman, PhD Senior Vice President jjseidman@avalere.com @jjseidman 1. The Need for the Patient Perspective in Existing Value Frameworks 2. A Multi-Faceted Approach to Developing the PPVF 3. The Current State and Evolution of PPVF Pilots and Use Cases 4. Questions? 2
The Need for the Patient Perspective in Existing Value Frameworks
The Current Value Framework Landscape Value assessment critical as US healthcare transitions from volume to value Proliferation of value frameworks in 2015 Today s value frameworks range from focusing on oncology (e.g., ASCO, MSK, NCCN) to specialty drugs and devices more broadly (e.g., ICER) Developers refining frameworks in response to patients, payers, providers, and manufacturers o Industry (as represented by AdvaMed, BIO, NPC, and PhRMA) published guiding principles in Spring 2016 o Patient groups have also joined the conversation (e.g., NHC s Value Rubric and Avalere/FasterCures Patient-Perspective Value Framework, PPVF) ASCO: American Society of Clinical Oncology MSK: Memorial Sloan Kettering NCCN: National Comprehensive Cancer Network ICER: Institute for Clinical and Economic Review BIO: Biotechnology Innovation Organization NPC: National Pharmaceutical Council NHC: National Health Council 4
Frameworks Differ in Aim, Conditions, and Audience Intended Users Therapeutic Focus Stated Aim ASCO Providers Patients Oncology Develop physician-guided tool to assess benefits and toxicities of drugs in relation to their costs to assist in shared decision-making with patients ICER Payers Policymakers Multiple Provide a framework to guide payers in assessment of the value of drugs, medical devices, and procedures MSK Providers Patients Oncology Determine appropriate prices for cancer drugs based on multiple value components NCCN Providers Patients Oncology Provide visual representation of 5 key aspects of evidence 5
Under ASCO s Framework, Value Assessment Is Based on a Drug s Net Health Benefit Score Users are encouraged to evaluate the score and direct drug cost side by side Hazard ratio (HR) for death is the preferred statistic used to assess the efficacy of a treatment. All adverse events - scored by frequency and grade - are considered to assess toxicity. Clinical benefit score Net health benefit score Toxicity score Input from Healthcare Industries Bonus points* Provided for: Improvements in palliation of symptoms Treatment free intervals Improvements in QoL QoL: Quality of Life Schnipper L. E., et al. 2016. Updating the American Society of Clinical Oncology Value Framework: Revisions and Reflections in Response to Comments Received. Journal of Clinical Oncology. Link. 6
Drug Abacus Price Reflects Eight Domains, Modifiable within a Given Range Efficacy Toxicity Novelty R&D Improvement in overall OS or surrogate Frequency/ severity of side effects Mechanism of action / delivery / next-in-class Cost of development Rarity Pop Health Burden Unmet Need Prognosis Incidence of disease Years of life lost in US pop Social need for a drug Severity of the disease OS: Overall Survival DrugPricing Lab, Memorial Sloan Kettering. Methodology. Link. 7
NCCN s Value Framework Evaluates New Cancer Drugs Based on Five Categories of Evidence NCCN Evidence Blocks Categories and Definitions* 5 E Efficacy of Regimen/Agent 4 3 2 1 E S Q C A S Q C A Safety of Regimen/Agent Quality of Evidence Consistency of Evidence Affordability of Regimen/Agent *For example purposes only National Comprehensive Cancer Network To date, NCCN has published Evidence Blocks for 42 of their 47 clinical guidelines, which are a key resource that providers use when making treatment decisions. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) with NCCN Evidence Blocks. Link. 8
ICER s Value Assessment Framework Considers Both Long-Term Value and Short-Term Affordability Goal: Sustainable Access to High-Value Care for All Patients Long-Term Value for Money Short-Term Affordability Comparative Clinical Effectiveness Estimated Incremental Cost- Effectiveness Potential Budget Impact Other Benefits or Disadvantages Contextual Considerations ICER. Value Assessment Framework for 2017-2019. Link. 9
Payer Familiarity with ICER is High, but is Not the Driving Factor in Coverage Determinations ICER Familiarity Shown: All Payers 100% 1 6 15 80% Degree to which ICER factored into coverage plans Shown: Payers familiar, somewhat familiar, or not that familiar with ICER framework 9% 2% 0% 1% 60% 29% 40% 61 19% 20% 18 0% 40% Very Familiar Not That Familiar Never Heard of It Somewhat Familiar Not Familiar at All The The Single Biggest Determining Factor, 0% 0% A Significant Factor, But Not the Biggest Barely a Factor Among the Biggest Factors A Minor Determining Factor Not a Factor at All Do Not Know Totals do not add to 100% due to rounding Q: How familiar would you say you are with The Institute for Clinical and Economic Review s Value Assessment Framework, also referred to as ICER s Value Assessment Framework? Q: Overall considering all of the decisions surrounding therapies that your organization has made in the past year, how much have ICER s value frameworks factored in to your plans decision to cover a therapy? See Appendix for full survey methodology. 10
Looking Forward, Payers Generally Anticipate Using the ICER Framework in Some Capacity Health plans can leverage value assessments at many points during the decision-making process, from supplementing P&T committee evaluations to strengthening price negotiations Likelihood of Using ICER Framework in the Next Year Shown: All Payers 15% 50% 33% 2% Very Likely Somewhat Likely Not Very Likely Not Likely At All Q: Separate from how you described the plan s decisions in the past year, were there any instances where an ICER value framework was especially relevant to deciding on a therapy? Q: How likely are you to use ICER s value frameworks in a year from now See Appendix for full survey methodology. 11
How Frameworks Could Be More Appealing to Payers Frameworks for the payer audience will need to have methodological transparency, consider total treatment cost, integrate real-world data, and facilitate physician-patient dialogue Considerations that would make payers more likely to use an external value framework Shown: All Payers Having a better understanding of the value framework s methodology 68% If the value framework considered total treatment cost If the value framework considers real world data as well as randomized controlled trial data 57% 56% If the value framework can be used at the point of care between physician and patient 40% If the value framework considers the patient perspective 31% Frameworks that extend across many therapeutic areas 19% None of the above 4% Q: Which of the following would make you more likely to use a value framework in the future? (Select all that apply.) See Appendix for full survey methodology. 12
A Multifaceted Approach to Developing the Patient-Perspective Value Framework
The Patient-Perspective Value Framework Initiative WHERE WE VE BEEN AND WHERE WE RE GOING In Phase I, the PPVF Steering Committee built PPVF to fill a gap in the value framework space PPVF to be implemented and used by various decisionmakers seeking to define and measure value from the patient s perspective Avalere analysis determined that existing value frameworks do not adequately incorporate and measure the patient s perspective on value In Phase II, Avalere and FasterCures aim to refine, test and validate the PPVF and apply it to real-world settings 2015 Phase I Phase II 2016-2017 2017-2018 Phase III 2018 - Forward Identify Gap in Existing Frameworks Build PPVF Version 1.0 Refine, Test, & Validate Implement PPVF: Patient-Perspective Value Framework Additional information available here: http://avalere.com/expertise/life-sciences/insights/releasing-the-draft-avalerefastercures-patient-perspective-value-framework 14
Phase I Approach: An Iterative, Collaborative Process PHASE I FOCUSED ON BUILDING THE FRAMEWORK P4C Conference Steering Committee formed Draft PPVF Released 100 public comments received Patient outreach Collaboration with other framework developers PPVF Version 1.0 release Nov 2015 June 2016 Nov 2016 Dec 2016 Jan 2017 Feb 2017 May 2017 Value frameworks emerged as a pressing topic to various stakeholders Coalesced around a common goal: defining value from the patient s perspective Received over 60% of comments from patients and patient advocates Conducted conditionspecific focus groups and surveys Interactive meeting with framework developers including ASCO and NCCN P4C: Partnering for Cures 15
Multi-Stakeholder Steering Committee Guides PPVF Initiative The steering committee convenes regularly to provide feedback on the PPVF and guide next steps * * * In addition to the above organizations, leadership and staff from across the Centers for Medicare and Medicaid Services (CMS) will participate in meetings and provide input to the framework. * denotes a verbal commitment formal membership is being finalized 16
Overview / The Patient-Perspective Value Framework The PPVF has five broad domains, which contribute different types of information to the framework Patient Preferences Quality & Applicability of Evidence Patient-Centered Outcomes Patient & Family Financial Considerations Usability & Transparency 17
Multifaceted Approach to Gathering Input on the PPVF We conducted the below activities to pressure test the PPVF beyond our steering committee: Feedback Mechanism Participants % Patients/ Caregivers Public Call for Feedback 96 63% Roundtable with Patient Advocates 12 100% American Heart Association/American Stroke Association (AHA) Citizen Scientist Think Tank 4 100% Michael J. Fox Foundation Survey 31 97% Cancer Support Community Focus Group 8 100% American Heart Association/American Stroke Association (AHA) Survey 119 94% TOTAL 270 84% Moving forward, we will heavily rely on human-centered design to implement and test the PPVF in real-world settings 18
The Current State and Evolution of PPVF Pilots and Use Cases
Phase II and Beyond: Future Applications of the PPVF Shared Decision Making Shared decision-making tool to support conversations between patients and clinicians Apply to Existing Frameworks Apply to existing value frameworks to better incorporate the patient perspective into value assessment Support Public Healthcare Programs Support public healthcare programs through shared decision making applications and others Strategic Internal Analyses Guide internal strategic analyses and potential future conditionspecific analyses Other Applications Each PPVF application will be used to inform other applications and future versions of the PPVF 20
Shared Decision Making Pilot / Developing a Shared Decision-Making Tool Aim: Develop/test decision-support tool that addresses the full spectrum of patientoriented value and fully integrates into the clinician s workflow and patient s life flow Phase 1 Phase 2 Phase 3 Phase 4 Identifying Partners, Pilot Sites and Conditions Developing the Shared Decision Making Tool Piloting the PPVF Decision Aid Evaluating the Intervention Partners Needed Content & Technology Clinical Payer (data) As we develop specific shared decision-making applications, we will pay special attention to overcoming the limitations associated with patient and clinician burden 21
Apply to Existing Frameworks Use Case / Collaborating with Other Framework Developers Aim: Integrate PPVF into other value frameworks to address gaps in patient-centered value considerations Map PPVF to Value Equation of Other Frameworks in collaboration with framework developers Evaluate methodologies of other frameworks Identify specific gaps in patientcentered value considerations Recommend Adjustments in collaboration with PPVF Steering Committee Develop targeted methodological recommendations to address gaps Partner to Implement Adjustments Provide support to implement and publish PPVF-based adjustments to other frameworks The PPVF Steering Committee has actively engaged with existing framework developers throughout the process 22
Support Public Healthcare Programs Pilot / Developing & Implementing a PPVF Checklist Aim: Develop/test a paper-based checklist that can be deployed as a patientfacing educational and engagement resource Develop & Test the PPVF checklist with patients & families using humancentered design Implement the PPVF checklist in real-world settings e.g., engage hospitals/ practices across HIINs and TCPI Evaluate the implementation and publish results Scale e.g., submit as a MIPS improvement activity for inclusion in the Quality Payment Program Focus group results with cancer patients and survivors showed they would benefit from a checklist outlining range of patient-centered aspects of value to be considered when making decisions about treatment options HIIN: Hospital Improvement Innovation Network TCPI: Transforming Clinical Practice Initiative MIPS: Merit-Based Incentive Payment System 23
Support Public Healthcare Programs Use Case / Developing Patient-Centered Measures Aim: Identify gaps in measures that are most meaningful to patients/families, among measures currently being used by public payers Map existing PROMs, patient experience measures, and shared decision-making measures against the PPVF Develop a report outlining identified gaps in patient-centered measures and suggestions for how to improve them/develop de novo measures Support public/private payers in guiding their measure identification, selection and development process The PPVF is a valuable tool to guide mapping exercises and gap analyses, given that it was developed in collaboration with and vetted by a multi-stakeholder Steering Committee PROMs: Patient-Reported Outcome Measures 24
Strategic Internal Analyses Use Case / Driving Patient-Centered Drug Development Aim: Partner with researchers and life sciences companies to evaluate the patient-centeredness of current and future healthcare products Select Therapeutic Area of Focus Identify areas that will have broad impact, e.g., cross-section of cancers and chronic conditions Conduct Gap Analysis Identify gaps in patientcentered evidence base within the therapeutic areas of focus Outline Recommendations Suggest next steps for improving evidencegeneration strategies for outcomes that matter to patients By collaborating with industry partners, the PPVF could ultimately drive patient-centeredness in the development of new healthcare products 25
Questions?
We Look Forward to Partnering with You Josh Seidman, PhD Senior Vice President jjseidman@avalere.com @jjseidman