1. Health Technology Assessments and their Impact on Benefit Plans, Considering the Patient Perspective
Frank DeFelice, MPA
Director Global HTA Public Policy,
Merck
Roundtable Discussion on UHC in Latin Amercia & the Caribbean: Health Benefit Plans
December 6, 2013

2. Outline
Key Challenges and Considerations for Universal Health Coverage (UHC)
Underlying Success Factors
The Role of HTA in achieving more optimal patient-centred health systems
Health Plan Experience with HTA
Limitations to adopting HTA from other jurisdictions and Essential Elements when considering HTA development
Conclusions 2

3. 1. Key Challenges to Achieve Universal Coverage
Achieving improved access to appropriate services in a fiscally constrained environment
Scale of the need for basic health care
Demographic changes (e.g. aging)
Epidemiological changes such as chronic disease burden
Emerging health technologies …
Siloed approach to health technologies, health system, societal impacts, and innovation

4. Underlying Success Factors for UHC: Local Tailoring, Partnership Approach and Evaluation
Shared Goal: Expanding access while preserving incentives to innovation and sustainability
Every Country is unique and tailored approaches are required
Progress on UHC will be enhanced by a partnership approach on priority policy areas
Standardized evaluation metrics to be developed for stakeholder monitoring of progress
Evidence from Research to underpin approach
Enhanced data and health information systems are a necessity

5. 2. The Role of HTA in Health Benefit Plans
HTA has often been seen as a tool to address cost concerns in the health system.
HTA when done effectively:
addresses a wide range of elements including clinical, patient, and societal impacts.
involves stakeholders in key parts of process design and implementation
takes a cross-health system approach (not solely focused on one technology)
There are widely accepted best practice principles for HTA.
Growing use of HTA globally with variation in approach (QALY-based v. relative assessment of value), application and how utilized (for guideline development, inform reimbursement)

6. HTA/CER System Development
Canada – existing HTA evaluation at national and provincial level
US – new CER system advancing
Mexico – existing HTA procedure for devices; under development for pharmaceuticals
Australia – long established HTA system
Brazil – New HTA body put in place to improve transparency
South Africa – HTA guidelines for pharmaceuticals put in place early 2011
Russia – increasing academic community interest in HTA
China – increasing academic community and government interest in HTA
Japan – increasing academic community and government interest in HTA
Korea – existing HTA evaluation as part of P&R process
Taiwan – existing non-mandatory HTA evaluation as part of P&R process
Thailand – existing HTA department, unclear impact on P&R
Turkey – existing HTA evaluation for pharmaceuticals; unclear impact on P&R
EU – Pan-European assessment under consideration; EUnetHTA advancing ; reform of long-established HTA systems (UK, France, Germany)
Established System
System in development
System under consideration 6 6 6

7. Key HTA Principles: How Widely Are They Adopted?

8. Structure of HTA program +
Key Principle
CMS (US)
NICE (UK)
IQWiG (Germany)
CADTH (Canada)
Anvisa (Brazil)
DHTA (Taiwan)
HIRA (Korea)
Structure of HTA program 1
The goal and scope of the HTA should be explicit and relevant to its use ++ + 2
HTA should be an unbiased and transparent exercise 3
HTA should include all relevant technologies 4
A clear system for setting priorities for HTA should exist
Methods of HTA 5
HTA should incorporate appropriate methods for assessing costs and benefits 6
HTAs should consider a wide range of evidence and outcomes 7
A full societal perspective should be considered when undertaking HTAs 8
HTAs should explicitly characterize uncertainty surrounding estimates 9
HTAs should consider and address issues of generalizability and transferability
Notes:
“+” signifies that the organization “supported” the principle in question in written guidelines or other form, regardless of whether they actually follow it.
“++” means that the organization “implemented” the principle in published reports, and decisions based on these reports demonstrate adoption of the specific principle.

9. Processes for conducting HTA
Key Principle
CMS (US)
NICE (UK)
IQWiG (Germany)
CADTH (Canada)
Anvisa (Brazil)
DHTA (Taiwan)
HIRA (Korea)
Processes for conducting HTA 10
Those conducting HTAs should actively engage all key stakeholder groups ++ 11
Those undertaking HTAs should actively seek all available data + 12
The implementation of HTA findings needs to be monitored
Use of HTA in decision making 13
HTA should be timely 14
HTA findings need to be communicated appropriately to different decision makers 15
Link between HTA findings and decision-making processes needs to be transparent and clearly defined
Notes:
“+” signifies that the organization “supported” the principle in question in written guidelines or other form, regardless of whether they actually follow it.
“++” means that the organization “implemented” the principle in published reports, and decisions based on these reports demonstrate adoption of the specific principle.

10. What elements of HTA are being captured? EUnetHTA Core Model --

11. Achieving more Optimal HTA: Patient-Centred & Innovation integrated
a. A focus on the patient is required in health system design and
health policy development and this extends
to HTA programs and processes.
b. Health care policy and program decision-making needs
to factor in innovation impacts.
If carried out in line with best practice
principles, HTA can be a useful tool to achieve
the value to patients and society
and to encourage innovation.

12. Towards More Patient Centred Health Care: Putting the Patient First
Accountability to:
Patients,
Citizens,
Society
Informed multi-stakeholder
involvement including patients,
providers, industry is essential

13. What Patient Centred should be
The experience (to the extent the informed, individual patient desires it) of transparency, individualization, recognition, respect, dignity, and choice in all matters, without exception, related to one’s person, circumstances, and relationships in health care*.
*Berwick, Don.”What patient centred should mean: Confessions of an extremist, Health affairs
March 25,2011

14. What is Patient-Based HTA?*
HTA Questions HTA Processes
Focus on
Patient’s
Problems
Allowing patient participation
Take
Patient’s
Perspective
Building on patient-physician partnerships
Accommodate
Patient’s
Preferences
Empowering patient to improve their health
*Bridges, John F.P, and Jones C,. Patient-based HTA: A vision of the Future,
IJHTAHC, 2007

15. Patient Based HTA*
*Bridges, John F.P, and Jones C,. Patient-based HTA: A vision of the Future,
IJHTAHC, 2007

16. Patients and carers can contribute to HTA:
How can patients’ perspectives in HTA be a route to robust evidence and fair deliberation?*
Patients and carers can contribute to HTA:
By providing evidence about their experiences and preferences
Through participation in the HTA process
*Facey et al. International Journal of Technology Assessment in Health Care, 2010,

17. Patient and Other Stakeholder Involvement Recognized
High Level Pharmaceutical Forum (Europe)
Highlights the key role of stakeholders including industry
Stakeholder involvement built in to the HTA process in Ireland (HIQA), Scotland (SMC), & England/Wales (NICE).
Importance of Stakeholder involvement recognized in Canada, Australia, and the US.
Patient Centred Focus of the Patient-Centred Outcomes Research Institute (PCORI) in the US

18. The full range of perspectives is needed
Aim is to have the full range of perspectives considered in HTA processes, with objective assessment that considers all stakeholders views and inputs. This requires a transparent system and education for all stakeholders, including patient advocates and interested health professionals.
Most systems invite some stakeholder participation but often exclude contribution at key stages of the evaluation process*
*A Comparative Analysis of the Role & Impact of HTA, CRA. Report for EFPIA,
PhRMA, EuropaBio, and Medicines Australia, 2011

19. Quantifying Patients’ preferences and outcomes in HTA
The number of instruments & approaches is growing…
EuroQol EQ5D, SF-36, SF-6D
Discrete Choice Experiments*
Analytic Hierarchy Process (AHP) –ranking of patient and provider preferences on different endpoints and relative weighting applied**
OxPAQ participation & activity measure
Increasing call for patient relevant endpoints (Patient Reported Outcomes (PRO’s))***;
Secondary Endpoints e.g. Health Related Quality of Life (HRQL), satisfaction, preferences less impactful***
Greater understanding of the data required***.
Qualitative aspects may need to be taken further into account
*See Professor Mandy Ryan, Chair in Health Economics, Aberdeen University
**Danner, M et al. Integrating patients’ views into HTA: AHP as a method to elicit patient
preferences (IQWiG based), IJHTAHC, October, 2011
***ISPOR Panel on PRO’s Presentation PROs when & who, November, 2011

20. Patient Access varies considerably across HTA-utilizing nations
International – Positive Reimbursement Percentages
(Based on all CDR-approved drugs also available in other countries)
Drug Reimbursement (Drug Type)
Canada’s Reimbursement %
International Avg
International Positive Reimbursement Percentage
51%
64%
First in class (positive reimbursement percentage)
40%
63%
JODR Drugs (subset of “non-CDR” drugs)
55%
65%
Canada CA Ireland IE Scotland SCT
Australia AU Italy IT Slovak Rep. SK
Austria AT Israel IL Slovenia SI
Belgium BE Japan JP Spain ES
Czech Rep. CZ Republic of Korea KR Sweden SE
Denmark DK Luxembourg LU Switzerland CH
Estonia EE Netherlands NL Turkey TR
Finland FI New Zealand NZ United Kingdom GB
France FR Norway NO USA US
Germany DE Poland PL Wales WLS
Greece GR Portugal PT
* Source: International Report on Access to Medicines 2009/10, Wyatt Health Management for Rx&D 20

21. Summary of Key Considerations
A patient focus requires:
Patient voice in the process
Patient Impact Assessed
Appropriate value & weighting placed on patient perspective and outcomes
Timely & Appropriate Access to Needed New Medication
Transparency
Multi-stakeholder involvement (patients, providers, industry…) in policy design and program implementation
Health Literacy (e.g. HTA Patient Training at LSE)
Cross-health system impacts considered (beyond drug budgets)
Productivity & Societal Impacts captured and given sufficient weighting
Innovation Impact & Value Considered

22. 3. Limitations when Considering HTA Development Based on other countries
Transferability. Inappropriate transfer of HTA evaluations can result in incorrect decisions, patient access delays to medical advances, and to less efficient use of health care resources
HTA Capacity(technical expertise & financial resources)
Unique country context and priorities
Data capacity
Differences in Comparator, practice patterns of patient population, inadequate reporting…
Health Information Systems lacking

23. When is Formal HTA Appropriate? Essential Elements*
The health care system should be effective in providing quality services to all citizens
The use of HTA should have a clear objective and be predicated on the current capacity in the existing health system
There should be adequate financial resources to establish and sustain the HTA system
There should be an adequate HTA workforce
There should be well functioning health information systems
There should be clarity in the purpose and process of a proposed HTA system
HTA should be tailored to ensure appropriate customization to the individual characteristics of a country
*HTA in Emerging Markets. PhRMA International Position Paper,May 2013

24. 4. Conclusions: HTA Development is moving forward but how it is used varies; let best practice principles guide the way
Increased health care spending, health system reforms to broaden coverage, and growing need to access modern technology gives a strong impetus to HTA*
HTA systems are constantly evolving even amongst early adopters
Although NICE remains a prominent model, it is also a model in transition
Some forms of HTA are converging but significant methodological issues remain*
Despite some convergence of approaches, the motto appears to be “Globalize the evidence, localize the decision”*
Adopting widely accepted best practice principles is preferable approach when developing HTA.
*CRA,Latin America Industry Workshop Presentation, May 2013

25. Impact of HTA Considering the Patient Perspective
Questions or Comments

26. Back Up Slides

27. Use of HTA by Private Plans
HTA plays an increasing role in translating emerging technologies into clinical practice and policy.
Private payers are important users of HTA whose decisions impact adoption and use of new technologies.
Payers relied more extensively on HTAs for reviews of personalized medicine (64%) than for other technologies.
Most payers (82%) valued expertise of reviewers and rigor of evaluation as HTA strengths
Key reported shortcomings for personalized medicine were limited availability of reviews (73%) and limited inclusion of nonclinical factors (91%), such as cost-effectiveness or adoption of technology in clinical practice.
(HTA & private payers coverage of personalized medicine.Am J Manag Care. 2011;17(5 Spec No.):SP53-SP60)

28. Use of HTA among private plans in the US
Insurers coverage and reimbursement policies have traditionally lacked sufficient transparency
The Academy of Managed Care Pharmacy (AMCP) produced its first guidance document on evidence requirements to support formulary listing in 2000.
WellPoint released a set of evidence requirements to improve transparency on the type of data required to make value-based, health-care decisions on drug therapy for its members.
HTA in Health-Care Decisions in the United States, Value in Health, S. Sullivan et al, Vol 12, 2009

29. Use of HTA among private plans in the US*
HTA will continue to evolve to accommodate the needs of health insurers.
It will always be shaped in part by the political landscape that will in turn be influenced by public perceptions of HTA as either a system that improves patient care or simply restricts access to promising medical technologies.
Such perceptions will influence the level of public financing of HTA, but they will not change the real need that payers have for controlling health spending at the same time ensuring access to effective medical technologies. The medical technology industries will need to grapple with the seeming paradox of meeting regulatory and licensing requirements while building an evidence-base necessary to satisfy health-care payers.
The evidence summary reports by organizations such as the Cochrane Collaboration, UK-NICE, DERP, and the CADTH now are more similar than they are distinct. As a result, decision-makers in the United States have begun to rely on these sources in their HTA processes, and are thus less prone to develop ad hoc or nontransparent approaches to evidence appraisal.
*HTA in Health-Care Decisions in the United States, Value in Health, S. Sullivan et al, Vol 12, 2009