High Cost Treatments / Limited Resources Seeking a Balance

1. High Cost Treatments / Limited Resources Seeking a Balance Rocky Mountain/ACP/AMA Internal Medicine Meeting Banff, Alberta – November 24, 2012 Stuart MacLeod, MD, PhD, FRCPC Professor, Pediatrics, University of British Columbia Child & Family Research Institute, Vancouver

2. 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

3. evolution or extinction

4. New technologies are receiving increasingly close scrutiny. Ultimately public-private views must align in a true risk sharing partnership to optimize benefit.

5. Applied health research & evaluation for decisionmakers: Perspective matters • 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 public health decision-makers physicians patients Reimburse? Recommend treatments? Healthcare direction Treat my patients? What to choose? « The best for my patients » « The best for me » « The best for my population » J-P Collet, CFRI, Vancouver, 2008

6. Key messages ▪ complexity ▪ measurement ▪ uncertainty and choice ▪ prioritization ▪ implementation

7. 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.

8. VALUING INNOVATIVE PRODUCTS: a deliberative process that considers context • 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. The real world is a special case. (Andrew Herxheimer, Oxford University)

9. Key issues 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.

10. The academic milieu is inadequately supportive of innovation • 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.

11. 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 cent since 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.

13. LIFE IN THE BALANCE How do researchers and policy-makers decide on the value of health? DanielCresseylooks at Britain’s National Institute for Health and Clinical Excellence Nature 2009;461(17);336-9

14. 2012 Health Canada CADTH initiative ▪ HTA collaborative steering committee ▪ inclusive of provinces, regions and stakeholders ▪ charged with preparation of Health Technology Strategy 2.0 (renewal of 2004 strategy) ▪ seeksagreement on transparent decision making shift from HT assessment… … to HT management

15. Health technology management 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

16. Pan Canadian health technology strategy 2.0 ▪ 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

17. Closing the know-do gap Both valleys can easily be made deeper and wider by overzealous investigators, regulators, government decision-makers.

18. Valuing health outcomes: Factors warranting consideration alongside cost benefit • 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

19. 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.

20. HIGHER SOCIAL VALUE C A LOWER INCREMENTAL COST PER QALY HIGHER INCREMENTAL COST PER QALY B LOWER SOCIAL VALUE The relationship between social value and incremental cost per quality-adjusted life-year (QALY) M F Drummond, 2007

21. The challenge in achieving appropriate valuation of outcomes by decision-makers 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.

22. The Ontario experience: An example of best practice

23. OHTAC decision determinants • overall clinical benefit • consistency with expected societal ethical values  value for money •  feasibility (ease) of adoption

26. HEALTH SYSTEM SUSTAINABILITY Leadership and Innovation The data deluge The Economist 25 Feb 2010

27. Knowing what works in health care: A roadmap for the nation January 24, 2008 In 2009 the Obama administration committed $1.1B to comparative effectiveness research.

28. There ain’tnothin’ 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.