Health Technology Assessment:

Towards a More Patient-focused Approach

Murat Aşık, MD, MBA

May 21st 2012 Moscow

Outline

• Definition of HTA

• Industry Contributing to the Dialogue

• Existing Principles

• HTA in Practice

What is HTA?

• Health Technology Assessment (HTA) encompasses the formal, explicit assessment of the (clinical) effectiveness

and cost-effectiveness of health technologies

• It is one of the tools of evidence-based medicine• Ideally, it should be applied to all interventions – not

just to new technology • It should be about providing safe and effective care to

patients• HTA should be free from undue political interference,

but also accountable. This means that the evaluating body should be independent of the payer.

Criteria for Assessment and Decision-Making

Criteria AT AU BE CA CH D FI F NL NO NZ SE UK

Therapeutic benefit x x x x x x x x x x x x x

Patient benefit x x x x x x x x x x x x x

Cost - effectiveness x x x x x x x x x x

Budget impact x x x x x x x x x x

Pharmacological/innovative characteristicsx x x x x X

Availability of therapeutic alternatives

x x x x x x x x

Equity considerations x x x x x

Community need x x

Public health impact x x

R&D x x

Government priorities x

Source: DIMDI, HTA-Bericht, Zentner, Busse, 2005

HTA - What it is not?

• Not just cost-effectiveness analysis (CEA)

• Not an endpoint

• Not a substitute for competition

• Not primarily about cost-containment

• Not just limited to pricing and reimbursement

• Not just when technology is to be launched

HTA is not new….

• HTA for medical technologies established in the US in the 70’s

• First European agency – Sweden 1987 (SBU)

• Today – agencies in most countries but legal mandate of many is unclear

• “Fourth hurdle” for drugs▫ Australia 1992

▫ UK,NICE 1999

▫ Sweden, LFN 2002

▫ Germany, IQWG

But governments’ demand for HTA is

new and increasing..

• Cost-containment

• Concern over the budget impact

• Pricing and reimbursement of products

• Pressure from patients and doctors

• Desire for ”objective” criteria

• Networks for exchange continue to evolve but data is not common

Does HTA really support decision

making?

• More in centralized, less - in decentralized systems

• Professional expertise of the decision making body is of vital importance, rather than the formal requirements and guidelines

• Research capacity uneven/skill shortages common (especially in health economics)

• Process is evolving (France, Germany, UK)• Studies by EUROMET (2000 and 2004) indicate

still limited influence

HTA – Potential Benefits -If

• could replace price controls

• used instead of reference pricing for patented products

• not completely inflexible and tied to RCT

• modeling allowed

• allowed faster initial adoption

• not limited to measuring QALYs or equivalent

• provided correct incentives for R&D

HTA – Potential Drawbacks

• Used in addition rather than instead of other forms of control

• Unsuitable for targeted products• Leads to reimbursement based on defined

indications• The high cost of HTA is an impediment

(especially for small companies or orphan drugs)• Could reduce incentives for innovation

HTA Policy Position

• Industry supports HTA using sound scientific methods and appropriately applied for improving patient health outcomes and overall health care system efficiencies.

• Industry believes HTA can play a valuable role in the dissemination of new medicinal and scientific information and help improve quality of care on an ongoing basis.

• Industry believes in the observance of several principles for successful HTA.

Contributing to the Policy Dialogue

• We will continue to provide constructive input to help shape HTA reforms.

• We believe it is important to focus on --

▫ incorporating the basic principles of sound HTA:

transparent, science-based, stakeholder inclusive, with consideration of broad societal value and patient quality of life; and

▫ the features and processes that achieve key results of:

improved and timely access for patients to innovative medicines, feasible data requirements, and innovation integration.

HTA in Action

101 for HTA Public Policy

• HTA using sound scientific methods and appropriately applied for improving patient health outcomes and overall health care system efficiencies.

• HTA can play a valuable role in the dissemination of new medicinal and scientific information and help improve quality of care on an ongoing basis.

• Principles for successful HTA should be determined beforehand and should be agreed upon by all relevant stakeholders.

Principles & Results-Based Approach

• HTA approach needs to be based on widely accepted principles (i.e. evidence-based, transparent, inclusive, based on societal impact…) and achieve tangible results:

▫ Timely and broad access for patients to new medicine and a supportive innovation environment

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Principles• HTA should be scientifically sound, rigorous, predictable,

replicable, transparent, inclusive and fair.

• HTA should be applied to a broad range of interventions to best impact the efficiency and quality of the health care system.

• HTA should focus on diseases & conditions with a high clinical and economic burden and those technologies that provide the greatest opportunity for health & health system improvement.

• HTA should be consistently applied and adhered to; where the outcome of an evaluation is positive, local or national payers should commit the necessary funding

• When HTA is undertaken, it should be done independently from the regulatory bodies and from those making reimbursement or coverage decisions.

Principles

• Value should be defined broadly since clinical, economic, and social value are all important.

• HTA should reflect and balance input from key stakeholders

• New technologies should be evaluated in the context of the benefits and harms of existing treatment options and HTA should assess the full range of effects/benefits.

• HTA should not delay the introduction of new technologies to the market.

• HTA should be compatible with policies that couple early access to approved technologies with consideration of additional data at a later time if/when such data become available.

Patient access key to any HTA reform

• Efforts for central relative efficacy role in the short term. Potentially relative effectiveness assessment in future?

• Net overall benefit and an improvement to patient access need to be demonstrated for any centralized relative efficacy assessment.

• CER experience and independent PCORI established in the United States suggest value in keeping the regulatory process distinct from HTA processes.

No new hurdles to access

• Any new central level review of relative efficacy or relative effectiveness should not be an additional hurdle to national level review.

• Assurance would be required that national HTA agencies and public payers would support any proposed central review and follow central recommendations.

Current HTA Landscape

• Countries across Europe are at different levels of adopting benefit/risk assessments via mechanisms such as Health Technology Assessment (HTA)

• HTA is a dynamic, rapidly evolving process, embracing different types of assessments that inform real-world decisions about the value (i.e., benefits, risks, and costs)of new and existing technologies**

• There is great interest among many agencies in Europe to broaden their roles in HTA application

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**Drummond et al, Key Principles for the improved conduct of HTA for resource allocation decisions, International Journal of Technology Assessment in Health Care, 2008

Current HTA Landscape

• Major Emerging HTA Issues in Europe:▫ Harmonization and Centralization?▫ Pan-National v. National?▫ Who does what? ▫ Relative Efficacy (RE)

• EMA Roadmap* seeks to:▫ facilitate access to medicines by improving its

assessment of benefits and risks▫ take greater account of patient experience in decision-

making progress

*The European Medicines Agency Road Map to 2015: The Agency’s Contribution to Science, Medicines, Health, Draft for Public Consultation, January 26, 2010

Assessing European Experience in

International Context

• HTA record mixed in application of good HTA practices* and impact on patient access

• JAMA article reports Canada rejection rate of over 50% and Australia 46% (v. UK at 13 %)

• Patient access worse for first drugs for a disease (86% rejection) and first-in-class drugs(68%) in Canada**

• New Zealand 2nd to last, Canada 6th to last, Scotland 8th last and Australia 9th last in access to new medicines.***

• New Zealand and Canada 4th and 5th to last for biologics***

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*Key Principles for improved HTA supported & used by HTA organizations?, Neumann, PJ, Drummond, MF et al. IJTAHC, 26:1 (2010)** Axia Research Report on CEDAC Recommendations, April, 2009***Rx&D International Report on Access to Medicines, Wyatt Heath Management,2008/2009

Key Considerations• Need to ensure patient impact is paramount:

▫ Timely & Broad Access to Needed New Medication▫ Patient voice in the process▫ Patient Impact Assessed▫ Significant value placed on patient health

improvement• Transparency and fairness• Multi-stakeholder involvement (patients, providers,

industry…) in policy design and program implementation

• Strong science & evidence-based decision-making• Cross-health system impacts considered (beyond drug

budgets)• Innovation Impact & Value Considered

Issues• Human Resource

▫ How many Health economists are there in the industry, SSI and academia?

▫ How much time is needed to have this potential?

• Civil Society Involvement

• Abundance of variable, verifiable data

• Cost of HTA

▫ It is expensive to develop this new, more transparent, structured and rational health policy.

▫ AHRQ (Agency for Healthcare Research and Quality) budget: $300 million US in 2004,

▫ The Swedish SBU annual budget of €8 million, and it employs 35 professionals.

▫ The Centre for Reviews and Dissemination (CRD, 5York), which undertakes work for NICE, has 58 employees with yearly budget of $60 million

▫ IQWIG. 2006 budget appr. $20 million

What is limiting Patient Access to Innovation?

• Cost effectiveness interpretation & drug budget impact focus

• Increasing &/or more burdensome clinical data requirements

▫ Long term outcome data

▫ Non-acceptance of surrogate markers

▫ Lengthier real world data

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NERA’s Global Principlesfor Better Health Care• Fair Access/Equity

▫ Access to essential health care services and the contributions of individuals

towards the cost of these services should be in line with the views of society about

fairness

• Efficiency

▫ The health care system should deliver the maximum improvement in health

outcomes given the available resources

• Responsiveness

▫ Services delivered and the level of funding should reflect citizens’ views, based on

high quality information

• Innovation

▫ The health care system should encourage appropriate product, diagnostic,

therapeutic, administrative and contracting innovation and its optimal

application

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IDEAL world decision making

• Equity

• Efficiency

• Responsiveness

• Innovation

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Implementation

Reevaluation

Cost/QALY threshold

Local epi data

Local costs

Discounting

Gold standard comparator

Endpoint Studies

Cost effectiveness analysis

Use of QALYs as parameter

Sensitivity analysis

PRINCIPLES OF

QUALITY HEALT CARE

SUPPORTS INNOVATION

PRINCIPLES OF

CONDUCTING HECON ANALYSIS

REAL world decision making

• Equity

• Efficiency

• Responsiveness

• Innovation

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Implementation?

Reevaluation?

Cost/QALY threshold?

Local epi data?

Local costs?

Discounting?

Gold standard comparator?

Endpoint Studies?

Cost effectiveness analysis?

Use of QALYs as parameter?

Sensitivity analysis?

PRINCIPLES OF

QUALITY HEALT CARE

DOES IT SUPPORT INNOVATION?

PRINCIPLES OF

CONDUCTING HECON ANALYSIS

The Way Forward• Patient impact assessment part of the approval process

(e.g. consider emerging methodologies)

• Feasible and Adequate requirements in terms of data & information requirements

• Full health care system impacts considered (eg savings for hospital and physician expenditures)

• Incentives for innovation that expedite access v. creating more burdensome regulatory hurdles

• Consider certain drugs to be fast-tracked by bypassing customary cost-effectiveness review. While still in development, the Innovation Pass in the UK is worth monitoring.

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Key Questions• Are the full health care system impacts being

considered?

• Have actual clinical practice considerations been adequately taken into account? How this medication could fit into practice v. a “yes/no” decision.

• Are the requirements feasible as well as adequate?

• Are Patient Impact Assessments and Appeal Mechanisms part of the process?

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Concluding Thoughts

• The value of pharmaceutical innovation rests ultimately in providing better health care, better quality of life, and the ability to live longer for patients.*

• Adequate and feasible requirements that focus on patients and place value on innovation will be most beneficial.

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*Pharmaceutical Research and its value to Canadians, David Griller, SECOR, Rx&D & SECOR, 2006

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