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Moving Beyond the QALY in Patient-Centered Value Frameworks: But, in What Direction?

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Nancy et al's slides on QALY Patient-Centered Value Frameworks for ISPOR.

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Moving Beyond the QALY in Patient-Centered Value Frameworks: But, in What Direction?

  1. 1. Moving Beyond the QALY in Patient-Centered Value Frameworks: But, in what direction? Shelby D. Reed, PhD Duke Clinical Research Institute, Durham, NC, USA F. Reed Johnson, PhD Duke Clinical Research Institute, Durham, NC, USA Nancy Devlin, PhD Office of Health Economics, London, UK Sachin Kamal-Bahl, PhD Innovation Center, Pfizer Inc., Collegeville, PA, USA May 23, 2017 ISPOR, 22nd Annual International Meeting Boston, Mass
  2. 2. Introduction Traditional CEA QALYs C ICER    Disease-focused Broader frameworks
  3. 3. Traditional value frameworks Second Panel on Cost-Effectiveness Analysis • Addition of an Impact Inventory • Prioritized use of CEA for practical decision making over the role of theory • Recommends generic preference-based measures and community-derived preference weights Impact Inventory Formal healthcare sector Health outcomes Medical costs Informal healthcare sector Patient time costs Unpaid caregiver time costs Transportation Non-healthcare sectors Productivity Consumption Social services Legal/criminal justice Education Housing Environment Other impacts
  4. 4. ASCO Value Framework Points Clinical Benefit Death Overall survival Disease progression 0 – 100 Toxicity Grade 1/2 Grade 3/4 0 – 20 Bonus Points Tail of the curve 0 – 20 Palliation 0 – 10 Quality of life 0 – 10 Treatment-free interval 0 – 10 Net Health Benefits 0 – 170 Cost $ Scoring Rubric for Advanced Disease Source: Schnipper LE, et al. J Clin Oncol 2016;34:2925-2934. Points Clinical Benefit Death Overall survival Disease-free survival 0 – 100 Toxicity Grade 1/2 Grade 3/4 0 – 20 Bonus Points Tail of the curve 0 – 20 Net Health Benefits 0 – 140 Cost $ Scoring Rubric for Adjuvant Setting
  5. 5. Broader value frameworks Other Elements of Value Real option value Value of knowing Equity Value of hope Scientific spillovers Impact on others Insurance benefit Dosing regimen Clinical Outcomes Cost Value Adapted from Garrison LP, et al. Value Health 2017 and Neumann PJ, 2016.
  6. 6. Applications for value frameworks Society Health care system Providers People Patients • Overall budget allocation decisions • Resource allocation • Coverage decisions • Practice guidelines • Shared decision-making • Selecting insurance-benefit options • Advocacy • Shared decision-making
  7. 7. Key Questions • Are value frameworks patient-centered? • Is anything missing from value frameworks? • Whose preferences should be represented in value frameworks? • How should preferences be measured? • How should preferences be used in value frameworks?
  8. 8. Reed • Patient preferences should serve as the basis for value frameworks
  9. 9. Are value frameworks patient-centered? • Need to define “patient-centered” and “value” • What meaningful patient-centricity isn’t • Patient-reported outcomes • Satisfaction surveys • Therapeutic-area public meetings • What value is • How much of one desirable outcome one is willing to give up in return for another desirable outcome • Relative preference weights define the rates at which a decision maker would accept tradeoffs • Patient-centered values reflect patients’ trade- off preferences
  10. 10. Is anything missing from value frameworks? What should be included in value frameworks? • Medical – treatment benefits and harms • Emotional – life satisfaction, relationships • Social – effects on caregivers, family, friends • Societal – benefits and harms external to medical decision makers • Existence values • Altruistic benefits and harms • Social equity Factors that affect patient and nonpatient welfare, broadly defined
  11. 11. Whose preferences should be represented in value frameworks? • Patients and nonpatients who care for and care about patients • Why not general taxpaying public? • Individualistic ethic: patients are the best judge of their own welfare • Perceived benefits rely on faulty assessments of likelihood of eventually being a beneficiary • Weak altruistic valuations of well citizens of the welfare of ill citizens • Hence distortion of the relationship between societal benefits and societal costs
  12. 12. How should preferences be measured? • Trade-off values revealed by observed decisions • Where patients’ decisions are too constrained to be informative, controlled stated-preference experiments When asking the public to assist in determining health priorities, we should use techniques that allow people to reveal their true preferences. If not, why bother asking them at all? Amiram Gafni (Social Science and Medicine, 1995)
  13. 13. How should preferences be used in value frameworks? Post-marketingPhase IIIPhase IIbPhase IIa Clinical Trials Phase I Pre- clinical Phase Candidate Profiling Phase Discovery Phase Investigational new drug identification Development decision making Regulatory submission Reimbursement and Care Identify investment priorities Benefit-risk evaluations Net benefit of limited health-care budgets and capacity PATIENT VALUE WEIGHTS
  14. 14. Sachin • Industry perspective
  15. 15. Moving beyond the QALY: Toward a patient-centered assessment of value Sachin Kamal-Bahl, Ph.D. VP and Head, Innovation Center, Patient & Health Impact, Pfizer Inc. 23rd May, 2017.
  16. 16. 16 Sachin Kamal-Bahl is an employee of Pfizer Inc. The opinions expressed in this presentation are the presenter's own and do not necessarily reflect the views of the company. Sachin Kamal-Bahl was not paid for this presentation.
  17. 17. 17 Importance of multiple perspectives in value assessment • Different stakeholders in the health system perceive and experience value very differently • Maximizing value in healthcare means maximizing value for all stakeholders – so incorporating all perspectives into the broader process of value assessment is key • These multiple perspectives likewise reflect the different levels of healthcare decision-making • Regardless of perspective, however, value frameworks should be patient-centered Individual Patients and providers making joint decisions about treatment Payer Population- level coverage decisions Societal Public policy, government programs, etc
  18. 18. 18 Why should value frameworks be patient-centered? Economics teaches that the consumer of a good is the one who determines its value – though the healthcare marketplace is complex, the ultimate consumer of healthcare is the patient Economic argument Value should include all relevant factors, and a given health good or service has many attributes outside of clinical outcomes. To understand the impact of individual level-preferences, the patient perspective is key Methodological argument The goal of healthcare and the health system is to maximize patients’ health and wellbeing. Value frameworks should do the same, and this requires placing patients at the center Ethical argument
  19. 19. 19 What does it mean for value assessment to be patient-centered? • Guidance statements on value assessment emphasize patient perspectives • Examples: NPC, PhRMA, NHC, Avalere-Faster Cures • Commonly emphasize the need for patient centricity, with some explicitly focused on patient perspective (e.g. Avalere-Faster Cures’ Patient Perspective Value Framework) • Patient-centered approach could include many elements, such as: • Partnering with patients to identify key elements of value in a given disease therapy context • Inclusion of value dimensions outside of clinical outcomes that are important to patients • Flexible enough to estimate relative value of treatments at sub-population or ideally the individual patient level • Designed to identify the treatment likely to be most valuable to a patient
  20. 20. 20 Despite guidance to the contrary, current value frameworks fall short on patient-centricity Patient-centric parameters Value Framework ICER ASCO MSK Drug Abacus NCCN Inclusion of patient perspective Limited Limited Public health benefit X Mode of administration X Accounts for patient preferences X X Ability and willingness to pay Caregiver Burden Other ancillary benefits important to patients Meets standards of NHC Value Model
  21. 21. 21 Why is patient centricity missing from value assessment? • Reliance on efficacy data from RCTs, not RWE or PROs • RCTs provide the most controlled setting for testing clinical effectiveness but results may not be translatable to patient-level value due narrow inclusion criteria and treatment contexts that do not fully reflect real world treatment considerations • RCT endpoints are designed to achieve goals such as FDA approval, and endpoints that are important for clinicians and clinical trials are not always meaningful for patients • Many HTA approaches take a population perspective appropriate for payers, but exclude elements of value that patients care about (e.g., caregiver burden, productivity, value of hope, insurance value, etc.)
  22. 22. 22 Measuring value from the patient perspective is complex Depending on disease area and the patient, a treatment may have a wide range of outcomes that matter to patients (effects on mobility, energy level, adverse events, symptom improvement). Relative importance of these depends on patient preferences Health outcomes Traits of therapies themselves are determinants of value – mode of administration, length of course of therapy, etc Treatment attributes Growing literature supports the importance of including additional sources of value that are important to patients, for example: Non-conventional sources of value Hope provided by chance at low- probability but high-value outcome Spillovers to family and caregivers
  23. 23. 23 How should patient preferences be incorporated into value assessments? • To maximize scientific rigor, patient preferences should be elicited using stated preference techniques • Ideally, value frameworks should be flexible enough to accommodate the preferences and risk attitudes of individual patients • More granular outcomes data are also necessary – CER studies of specific subpopulations, better use of PROs to track non-clinical outcomes, etc
  24. 24. 24 Advancing methods for measuring value at the patient level is an important scientific priority • The QALY continues to be used as the central metric of health utility, but it neglects important dimensions of value • Methods must be developed to incorporate the broad array of attributes of importance to patients in a way that is scientifically rigorous • Methods and tools are also needed to allow for individualized assessment of value, based on a patient’s preferences, to aid in decision making
  25. 25. Thank you for your attention! Sachin.Kamal-Bahl@pfizer.com
  26. 26. Nancy • There is a role for both patients’ preferences and societal preferences
  27. 27. Are value frameworks patient-centred? What is a ‘value framework’? • Sets out the criteria relevant to a decision • Sets out the relative importance of those criteria in decision making • Provides a structured framework for assessing options about which choices are being made US value frameworks are ‘new’ in one sense – but • are just a kind of structured decision making (eg MCDA) • In making decisions affecting resource allocation, they have precedents in prioritisation frameworks used to support • eg PBMA use by budget holders in England, Canada, New Zealand • eg. NICE methods guides and social value judgement documents Are value frameworks patient-centred? Not necessarily Should they be? It depends what decision is being made, and who is making it
  28. 28. Whose preferences should be represented in value frameworks? • The person(s) affected by the decision • Where a patient is choosing (between treatments or providers), that individual patients’ preferences are clearly the relevant ones • For benefit risks assessments, the preferences of the group of patients for whom that treatment is aimed are relevant. • Note that this implies averaging in some way • ‘Patients’ preferences’ unlikely to be homogeneous • Where reimbursement is concerned, and budgets are limited: the preferences of both the patients who might benefit from a new technology, and those who would have benefited from the next best technology foregone, are relevant. • Foregone opportunities include spending on preventive measures that reduce the risk of healthy people becoming ill.
  29. 29. Who are the ‘patients’ whose preferences are relevant? Choice of treatment or provider, by a patient Benefit risk assessment Reimbursement decisions What is being decided, by who?Whose preferences are relevant? Individual preferences The average preferences of the relevant group of patients The average preferences of the relevant group of patients, and the average preferences of all other patients/potential patients ie society
  30. 30. How should preferences be measured? • Revealed preferences - where feasible • Stated preferences: • Though subject to various framing effects. • ‘Theory of constructed preferences’ (Fischoff 1991; Slovic 1995; Robinson and Bryan 2013) • Intelligent, new generation of methods for preference elicitation needed – focussed on getting people to think harder. - Deliberative/reflective valuation approaches (eg. ‘Personal Utility Functions’ – Devlin et al 2016).
  31. 31. Is anything missing from US value frameworks? Arguably, two things: (a) An appropriate basis for the weights applied to criteria. These should reflect the preferences of those affected by the decision. (b) A failure to recognise that there is no ‘one size fits all’ framework that will be relevant to all decisions and decision makers in health care • The things that matter to individual patients ≠ the things that matter to society • E.g externalities; distributional considerations; ethical considerations
  32. 32. How should preferences be used in value frameworks? • Preference data are fundamental to value frameworks (the clue is in the name!) • ‘Value frameworks’ meaningless unless they are based on preferences about (a) what criteria matter and (b) what trade offs decision makers are prepared to make between them. • Whose preferences are relevant depends on what decision is being made • Individual choices = individual preferences • Collective choices = aggregations of individual preferences • Use of value frameworks to support decisions affecting resource allocations between patients involve benefits gained and foregone - there is arguably a role for both societal preferences and patients’ preferences “Health economic guidelines could require analysis of benefit in terms of QALYs based on both patient and general public preferences” (Versteegh and Brouwer 2017) • Same applies to other elements of benefit beyond the QALY
  33. 33. Thank you! • Enquiries about this presentation are welcome: • ndevlin@ohe.org
  34. 34. Audience participation (necessary component for ‘successful’ Issues Panel) • FYI, I asked Jessica at ISPOR about the option of using the new polling app for the Issues Panel. It’s a pilot case, and they are not extending the option to Issues Panels. • The last ‘presenter’ could be the audience. We could pose the same questions to them.

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