High Cost Treatments / Limited ResourcesSeeking a Balance

High Cost Treatments / Limited ResourcesSeeking a Balance

Stuart MacLeod, MD, PhD, FRCPCProfessor, Pediatrics, University of British Columbia

Child & Family Research Institute, Vancouver

Rocky Mountain/ACP/AMA Internal Medicine MeetingBanff, Alberta – November 24, 2012

Disclosure

• Until August 2012 SMM was vice president research coordination and academic liaison for BC Provincial Health Services Authority.• SMM has served as a consultant to Health Canada, European Community FP-7, Canadian Agency for Drugs and Technology in Health and Ontario MOH.• Speaking fee was received in 2011 from Eli Lilly (Canada) Inc.• consultant to Purdue Pharma (USA) in 2012 re trial of management of chronic pediatric pain.• member of Data Monitoring Board, ApoPharma Ltd 1995-2012

evolution or extinction

New technologies are receiving increasingly close scrutiny.

Ultimately public-private views must align in a true risk sharing partnership to optimize benefit.

• Does it work in real life?• For whom?• Better than current treatments?• Is it safe?• Compared to what?• At what cost?

questions about health interventions

« The best for my patients »

« The best for my population »

« The best for me »

physicians

Treat my patients?

patients

What to choose?

public health decision-makers

Reimburse?Recommend treatments?Healthcare direction

J-P Collet, CFRI, Vancouver, 2008

Applied health research & evaluationfor decision makers: Perspective matters

Applied health research & evaluationfor decision makers: Perspective matters

▪ complexity▪ measurement▪ uncertainty and choice▪ prioritization▪ implementation

Key messagesKey messages

Determination of Benefit and Risk is at the core of present decision-making

about therapeutic and diagnostic innovation.

It is essential to preserve a public policy environment that fosters incremental innovation.

• evidence-informed vs evidence-based • increased focus on comparative effectiveness• observational studies that go beyond RCTs• effective use of colloquial evidence (Lomas et al)• EPOC approach (Cochrane Effective Practice

and Organization of Care Group)

Expectations of evidence based medicine must be tempered by reality.

VALUING INNOVATIVE PRODUCTS: a deliberative process that considers context

VALUING INNOVATIVE PRODUCTS: a deliberative process that considers context

The real world is a special case. (Andrew Herxheimer, Oxford University)

1. The new economy requires innovation to drive competitiveness and improved productivity.

2. Different levels of government seek different returns on investment.

3. Investment in evaluation and implementation science lags behind the cost driver of new technologies.

4. Academic attitudes to technologic advance in health (commercialization) is erratic and sometimes hostile.

Key issuesKey issues

• Human resource plans impede recruitment of researchers in applied, evaluative, or implementation science.

• University research is often not well aligned with public/patient (eg, CIHR-SPOR)

• It is easier to accept the dogma of evidence based medicine than to pursue an approach that recognizes culture and values.

• Many academics openly oppose the innovation/ commercialization agenda.

The academic milieu is inadequately supportive of innovation

The academic milieu is inadequately supportive of innovation

Globe and Mail 16 September 2011

by KEVIN LYNCH and MUNIR SHEIKH

Wanted: culture of innovation

“Productivity isn’t everything,” Paul Krugman once wrote in his New York Times column, “but in the long run it is almost everything.” Strange then, with Canada’s poor productivity and innovation performance compared with that of the U.S., that we remain complacent. Where’s our sense of urgency?

Productivity growth is the dividend produced by innovation. So our difficulties are placed in sharp relief when we see that our productivity growth has dropped substantially, from average growth of close to 3 per cent annually from 1961 to 1980, to under 1 per centsince 2000. And that gap between Canada and the U.S. has widened in the past decade, despite the relative improvement of macroeconomic fundamentals in our country.

LIFE IN THE BALANCEHow do researchers and policy-makers decide on the value of health?

Daniel Cressey looks at

Britain’s National Institute for Health and Clinical Excellence

Nature 2009;461(17);336-9

▪ HTA collaborative steering committee

▪ inclusive of provinces, regions and stakeholders

▪ charged with preparation of Health Technology Strategy 2.0 (renewal of 2004 strategy)

▪ seeks agreement on transparent decision making

shift from HT assessment… … to HT management

2012 Health Canada CADTH initiative2012 Health Canada CADTH initiative

macro: governments and regional health authorities ▪ regulate introduction and use, including coverage

meso: hospitals ▪ address issues re acquisition and impact analysis ▪ prioritization of resource use

micro: providers ▪ participate in field evaluation ▪ establish clinical practice guidelines

Health technology managementHealth technology management

▪ assess new technology as a cost driver

▪ address sustainability of the healthcare system

▪ consult with potential end-users and decision-makers

▪ emphasize contextualization of evidence

▪ training and assurance of human resources for evaluation and implementation science

Pan Canadian health technology strategy 2.0Pan Canadian health technology strategy 2.0

Both valleys can easily be made deeper and wider by overzealous investigators, regulators, government decision-makers.

Closing the know-do gapClosing the know-do gap

• lack of, or inadequacy of, alternative treatments• seriousness of the condition (rights of rescue)• affordability from the patient perspective• financial implications for government• equity objectives• social values• potential overall impact of innovation

Valuing health outcomes: Factors warranting consideration alongside cost benefit

Valuing health outcomes: Factors warranting consideration alongside cost benefit

Daniels and Sabin 1998

There are four elements of legitimacy and fairness in public decision-making: stakeholder involvement publicity (transparency, dissemination) revision or appeal leadership, including accountability for reasonableness

Health Affairs 1998;17:50-64.

M F Drummond, 2007

LOWER INCREMENTAL COST PER QALY

HIGHER INCREMENTAL COST PER QALY

HIGHER SOCIAL VALUE

LOWER SOCIAL VALUE

A

B

C

The relationship between social value and incremental cost per quality-adjusted life-year (QALY)

The relationship between social value and incremental cost per quality-adjusted life-year (QALY)

climbing a Wall of Fear

• inertia may be safer• high cost of innovation• interplay of values and evidence• divergence in social values• ambiguity re ‘effectiveness’• lack of consensus on methods• constraints on access to data

Resolution requires an innovative approach to risk sharing agreements.

The challenge in achieving appropriate valuation of outcomes by decision-makersThe challenge in achieving appropriate

valuation of outcomes by decision-makers

The Ontario experience:     An example of best practice

· overall clinical benefit

· consistency with expected societal ethical values

value for money

feasibility (ease) of adoption

OHTAC decision determinantsOHTAC decision determinants

Leadership and Innovation

The data deluge  The Economist 25 Feb 2010

HEALTH SYSTEM SUSTAINABILITYHEALTH SYSTEM SUSTAINABILITY

Knowing what works in health care: A roadmap for the nationJanuary 24, 2008

In 2009 the Obama administration committed $1.1B to comparative effectiveness research.

There ain’t nothin’ in the middle of the road ‘cept yellow lines and dead armadillos.

Jim Hightower, Texan senator

The Canadian health system in its      present form is not sustainable.