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Professor Adrian TowseDirector of the Office of Health Economics, London
Visiting Professor, London School of Economics
Presentation at ISPOR 19th Annual European CongressNovember 2, 2016
IS BALANCING VALUE DEMONSTRATION FOR PAYERS AND PATIENT INTERESTS A FEASIBLE NOTION?
A Health System Perspective
Balancing Value Demonstration
What is the role for patents? (i) • Principal agent theory
• Clinicians are imperfect agents for patients• Payers have an agency relationship with patients and with
the people who pay premiums / taxes to finance the system• Ex ante versus ex post willingness to pay
• Advocacy versus expertise• Is it a false dilemma? We must recognise that patients are
advocates. So are manufacturers. The role of the evidence assessment process is to challenge. However the patient voice must be there
• What HTA bodies should want to understand is the impact on the patient, and the preferences of the patient. They should be taken into account. They do not determine the decision
Balancing Value Demonstration
What is the role for patents? (ii)
• Incentives• Regulator and HTA body approaches will drive
investment further upstream• But companies need to collect evidence
• Qualitative versus quantitative approaches – the importance of evidence• Too often HTA bodies only look at costs and effects
because those are the only things they have evidence on. Quantification of “effect size” is important for “weight” to be given it in a deliberative process, whether structured or not.
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
4
What are “attributes” of value?
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
Toxicity/safety X X X X X
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
Toxicity/safety X X X X X
Treatment novelty X
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
Toxicity/safety X X X X X
Treatment novelty X
Condition rarity and condition burden X
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
Toxicity/safety X X X X X
Treatment novelty X
Condition rarity and condition burden X
Budget impact X
ACA/AHA ASCO ICER Sloan Kettering NCCN
Clinical benefit X X X X X
Toxicity/safety X X X X X
Treatment novelty X
Condition rarity and condition burden X
Budget impact X
Cost-effectiveness X X
Reproduced with permission from Peter Neumann, Tufts University
Balancing Value Demonstration
Garrison et al. (2016) EPEMED Report
HTAi Tokyo May 2016
Extract
HTAi Tokyo May 2016
Step : Results of weighting
Per cent‘Experts’ workshop
‘Patients’ workshop
Extent to which treatment is available in the absence of the new medicine 19.5 11
Disease’s mortality impact with current SoC 14 11.5
Morbidity and disability with the disease with current SoC 12 15
Impact of the disease on patients’ and carers’ daily lives with current SoC 8 15
Sub-total weight for impact of disease / extent of unmet need 53.5 52.5
Evidence of treatment clinical efficacy and patient clinical outcome 27.5 17.5
Drug safety 8 7.5
Social Impact of the treatment on patients’ and carers’ daily lives 11 17.5
Treatment innovation 0 5
Sub-total weight for impact of new medicine 46.5 47.5
Total 100 100
2
Balancing Value Demonstration
Examples of initiatives
• SMC uses a Patient and Clinician Engagement (PACE) process
• Myeloma UK establishes patient preference partnership with NICE
• The CADTH Scientific Advice Program includes patient input as part of the process for developing Scientific Advice
Evaluation Results
Patient Input Summaries
CADTH Review Protocols
119 things that matter to patients
89 / 119included
75%
Clinical Trials
61 / 119included
50%
67 / 119included
56 %
CDECRecommendation
& Reasons
Source: Brian O’Rourke. Reproduced with permission
Balancing Value Demonstration
References• Blinman, P., King, M., Norman, R., Viney, R. and Stockler, M.R., 2012.
Preferences for cancer treatments: an overview of methods and applications in oncology. Annals of Oncology, 23: 1104-1110.
• Garrison L, Zamora B, and Mestre-Ferrandez J. 2016. The Value of Knowing and Knowing the Value: Improving the Health Technology Assessment of Complementary Diagnostics. Available at: http://www.ohe.org/sites/default/files/WP_EpemedOHE_final.pdf
• Messina, J., Grainger, D.L., 2012. A pilot study to identify areas for further improvements in patient and public involvement in health technology assessments for medicines. Patient, 5: 199-211.
• Shah, K.K., Mestre-Ferrdandiz, J., Towse, A., and Smyth, E.N., 2013. A review of health technology appraisals: case studies in oncology. International Journal of Technology Assessment in Health Care, 29:101-109.
• Towse, A. and Barnsley, P. 2013. “Approaches to identifying, measuring, and aggregating elements of value.” International Journal of Technology Assessment in Health Care. 29(4), 360-364.