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

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. Introduction
Traditional CEA
QALYs
C
ICER



Disease-focused Broader frameworks

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. 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. 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. 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. 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. Reed
• Patient preferences should serve as the basis for value frameworks

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. 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. 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. 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. 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. Sachin
• Industry perspective

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
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
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
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
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
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
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
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
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
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. Thank you for your attention!
Sachin.Kamal-Bahl@pfizer.com

26. Nancy
• There is a role for both patients’ preferences and societal preferences

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. 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. 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. 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. 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. 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. Thank you!
• Enquiries about this presentation are welcome:
• ndevlin@ohe.org

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.