Macro-HTA vs. Micro-HTA: Necessary But Not Sufficient

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Macro-HTA vs. Micro-HTA:Necessary But Not Sufficient

Symposium on the Role of HTA in Decision Making

ISPOR Singapore

September 6, 2016

Lou Garrison, Ph.D.Professor, Pharmaceutical Outcomes Research and Policy Program,

Department of PharmacyAdjunct Professor Departments of Global Health and Health Services

University of Washington

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Acknowledgements

• OHE Report Co-Authors: Adrian Towse, Nancy Devlin, Emma Hawe– Sponsor: Pharmaceutical Research and Manufacturers

Association

• MSH Report Co-Authors: Joseph Babigumira, Rebecca Bartlein, Hiep Nguyen– Sponsor: USAID under the terms of cooperative agreement

number GHN-A-00-07-00002-00 with Management Sciences for Health.

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Agenda

• Introduction to Health Technology Assessment (HTA)

• Two Studies of HTA in Emerging, Low-Income, and Middle-Income Countries

• Principles of HTA: The Importance of Process

• Defining Value in HTA

• Dealing with Macro-HTA Issues.

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Definition of HTA

EUnetHTA (2008) report:

a health technology is ‘any [health] intervention that may be used to promote health, prevent, diagnose or treat disease, or for rehabilitation or long-term care. This includes pharmaceuticals, devices, procedures and organizational systems used in health care’

Our working definition:

Health technology assessment (HTA) is the structured analysis of health care technologies—medicines, vaccines, medical devices, diagnostics, healthcare procedures, and health systems—performed for the purpose of providing input into regulatory, coverage/formulary (including essential medicines lists), and/or reimbursement policy decisions.

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Why are we doing HTA?

• Most countries aspire to provide “Universal Health Coverage” (UHC) for their citizens.

• This implies a need to define what services and products will be provided: in other words, what goes in the “benefit package” or “minimum benefit package.”

• Can differentiate between:– “Macro-HTA”—health systems design, number of

hospitals, size of medical workforce– “Micro-HTA”-

• Appraisal of specific technologies, e.g., drugs and devices

• Preparation of treatment guidelines.

Key Messages aboutHealth Technology Assessment (HTA)

• Evolution—HTA is not new, but it has nearly 30 years of history—an evolutionary one.

• Globalization—HTA is being applied in more and more countries, and the number of competent practitioners is growing--globally.

• Variety—How HTA is used varies markedly, but depends on incentives to use the information.

• Challenge—HTA operates in the political sphere and its role and performance in any given country will depend on how it is institutionalized and organized.

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Health Technology Assessment: Principle vs. Practice

• In principle, not just about pharmaceuticals

– In practice, drugs have been the focus

• In principle, not just about costs

– In practice, it has been about costs and cost-effectiveness (and budget impact)

• In principle, it’s a scientific approach to resource allocation

– In practice, it’s often about politics.

HTA as an Economic Production Process and Economic Good

• The process of HTA can usefully be thought of as a “technology” or production process.– As such, one can ask, whether it is “technically” efficient, obtaining

maximum output given the resources used.

• The output of the process is “information”—a “public” good, in economic jargon. Indeed—a global public good– One can also ask whether the the production is “economically”

efficient, i.e., is it technically efficient, being produced a minimum cost, and in the right quantity?

• Public goods create incentive to be a “free-rider”

Economics says: “public goods” will be

undersupplied by private markets. Incentives like

patents and subsidies are needed.

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Agenda





• Dealing with Macro-HTA Issues.

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Two HTA Landscape Reports (2011, 2012)

Source: Towse et al., OHE, 2011 Source: Garrison et al., UW/MSH, 2012

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Source: Towse et al., OHE, 2011

OHE Study: Five Key Observations onHTA Challenges

1. HTA may have a role in assisting the health care system to reconcile rapidly expanding demand with more slowly expanding resources in a more explicit and transparent way, and in promoting public debate about priorities.

2. HTA of individual technologies is not a substitute for the reform of health care systems. Where health care systems create obviously bad incentives, this type of micro-HTA is unlikely to compensate for these failings.

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OHE Study: Five Key Observations onHTA Challenges

3. ‘One size fits all’ HTA processes and methods are unlikely to be appropriate for emerging markets. There needs to be clarity over the purpose of HTA – and the methods and processes which are adopted need to be fit for purpose.

3. HTA and pricing regulations work hand in hand: the approach to HTA should be appropriate to, and work sensibly in combination with, the particular approach to pricing technologies.

4. The relevance and positioning of any role for HTA in a health care system depends on the development stage and structure of that health care system.

UW-MSH Study: Five Key Findings on HTA Challenges

1. The use of HTA in the surveyed low- and middle-income countries is extremely limited for the most part.

2. There is a strong need for consensus building among the stakeholders and raising awareness of the importance and use of HTA, prior to starting a process to strengthen HTA

3. Building capacity and an increaseing quantity and quality of human resources for HTA is critical.

4. It will be important to develop standards and policies to enforce the use of HTA.

5. Other than for regulatory review, micro-HTA is probably not that helpful or necessary in the absence of functioning public health insurance delivery and insurance system.

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Agenda





• Dealing with Macro-HTA Issues

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15 Key Principles for Conducting HTA (1-8)

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

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

SOURCE: Drummond MF, Schwartz JS, Jonsson B, Luce BR, Neumann, PJ, Siebert U, Sullivan SD. Key principles for the conduct of HTA for resource allocation decisions. International Journal of Technology Assessment in Health Care, 24:3 (2008), 244–258

* Red highlighting suggests process and governance principle.

15 Key Principles for Conducting HTA (9-15)

9. HTAs Should Consider and Address Issues of Generalizability and Transferability

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

13. HTA Should Be Timely

14. HTA Findings Need to Be Communicated Appropriately to Different Decision Makers

15. The Link Between HTA Findings and Decision-Making Processes Needs to Be Transparent and Clearly Defined

SOURCE: Drummond MF, Schwartz JS, Jonsson B, Luce BR, Neumann, PJ, Siebert U, Sullivan SD. Key principles for the conduct of HTA for resource allocation decisions. International Journal of Technology Assessment in Health Care, 24:3 (2008), 244–258

* Red highlighting suggests process and governance principle

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Essential Elements of a Technologyand Outcomes Assessment Initiative

(Emanuel, Fuchs, Garber, JAMA, 2007)

1. administrative independence

2. dedicated funding

3. production of objective and timely research

4. use of reliable methods

5. widespread dissemination

6. a governance and organizational structure that lend it legitimacy.

Guidance for Priority Setting in Health Care Checklist: Potential Criteria in Addition to Cost-Effectiveness

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*GPS-Health Checklist

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Agenda






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Insurance Perspective (Garber & Phelps, 1997)

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• “Implicit in our discussion is the assumption that CE analysis is used

to improve decision making at an individual level.

• Ordinarily an apparatus like CEA analysis is unnecessary for

individual consumption decisions, in the absence of externalities

or public considerations.

• In health care, however, the familiar informational failures are

sufficient reason for CE analysis to be performed as an aid to

individual decisions.

• A more common application, however, is for decisions about the

scope of health insurance: the technique can be used to help

determine which forms of health care should be reimbursed by a

private or governmental insurer, or provided by a health maintenance

organization.

• The optimal CE criterion is equivalent to determining optimal

coverage for an actuarily fair insurance policy, under perfect

information.”

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Defining Economic Value for HTA: Standard Definition

What is “economic value”?

• “Value”= what fully informed patients would be willing to pay (WTP) for a new medicine based on:

1) any cost savings,

2) life years gained (LYs),

3) improvements in quality of life or morbidity

(2+3)Quality-adjusted life years--QALYs

Source: Garrison, L., Mestre-Ferrandiz, J. and Zamora,

B., OHE and EPEMED, Forthcoming, June, 2016

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Agenda






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The Theory—and Problem—of the Second Best(Lipsey and Lancaster, 1956)

“The general theorem for the second best optimum states that if there is introduced into a general equilibrium system a constraint which prevents the attainment of one of the Paretian conditions, the other Paretian conditions, although still attainable, are, in general, no longer desirable.”

• In other words, if there are distortions due to problems in macro-

structure and incentives in the health care system, it may not be

appropriate to follow the MB=MC rule.

• We need to be careful in applying CEA in systems with macro-

structural problems.

• Prime example: paying doctors by letting them sell medicines.

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Challenges and Next Steps

• Expanding beyond CUA

– Identifying all relevant elements

• How and how much to measure/monetize—or not?

– Utilize multi-criteria decision analysis (MCDA) or mixed model (quantitative and qualitative)?

• Decision-making

– Rules (e.g., threshold) vs. type of deliberative process?

• How to consider or deal with macro-structural distortions and incentives

Thank you!

[email protected]

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Documents

Macro-HTA vs. Micro-HTA: Necessary But Not Sufficient