The presentation by Adrian Towse at the Genesis Conference discusses the economics of risk-sharing in personalized medicine, highlighting the elements of value such as reduced drug adverse effects and increased treatment effectiveness. It emphasizes the need for institutional arrangements, alignment of incentives, and flexible pricing mechanisms to ensure the effective assessment and use of personalized medicine technologies. The document identifies challenges in diagnostic uptake and discusses examples of performance-based risk-sharing arrangements utilized in various healthcare systems.

1. Adrian Towse
Director
Office of Health Economics
London, UK
Presentation to Genesis Conference 2014, 9 December, Westminster, London The economics of risk-sharing and personalised medicine

2. Presentation to Genesis Conference December 9, 2014
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Agenda
•What are the elements of value in personalised medicine?
•What institutional arrangements are needed for the assessment of value?
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Flexible pricing, coverage with evidence development and PBRSAs
•Challenge of diagnostic uptake

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Presentation to Genesis Conference December 9, 2014
Value
1. Reducing drug adverse effects
2. Reducing time delays in selecting optimal Tx
3.Increasing adherence or willingness to start Tx
4. Enabling Tx effective in a small fraction to be made available
5.Informing, reducing uncertainty about value
Framework to assess value of personalised medicine in the context of treatment
Source: Garau et al. (2013)

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Examples of Pathways for Value in Personalised Medicine
Source: Garau et al. (2013)

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Agenda
•What are the elements of value in personalised medicine?
•What institutional arrangements are needed for the assessment of value?
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Flexible pricing, coverage with evidence development and PBRSAs
•Challenge of diagnostic uptake

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Agenda
•What are the elements of value in personalised medicine?
•What institutional arrangements are needed for the assessment of value?
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Flexible pricing, coverage with evidence development and PBRSAs
•Challenge of diagnostic uptake

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Aligning incentives
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Aligning incentives – what is needed?
•A greater willingness on the part of payers to accept prices that reflect value.
•A greater willingness on the part of drug and test developers to generate evidence to demonstrate that their price provides value
•Realistic expectations on all parties around the standards for evidence. This involves the use of CED and real world evidence collection for both drugs and diagnostics.
•Public investment to complement the efforts of payers and manufacturers, recognising the limitations for both to invest in evidence collection on all of the questions that matter.
•A need for price flexibility for drugs as evidence of their value for different groups of patients emerges over time
•Consideration of some form of IP protection (for example, data exclusivity) for diagnostics to cover evidence of clinical utility
Source: Towse and Garrison (2013)

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Agenda
•What are the elements of value in personalised medicine?
•What institutional arrangements are needed for the assessment of value?
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Flexible pricing, coverage with evidence development and PBRSAs
•Challenge of diagnostic uptake

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Need for flexible pricing and more outcomes-based PAS

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Presentation to Genesis Conference December 9, 2014
UK AMS Pricing and Reimbursement Recommendations
Recommendation 14: Flexible, value-based pricing and reimbursement system
•Adjust prices over time—for increases and decreases in value
•Need to be able to handle single biomarker and large platform multiple biomarker tests
•Should consider cost per QALY and cost-offsets
•But also consider:
oValue of greater certainty of response
oValue of improved adherence
oValue of great uptake

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Performance guarantees are not new …..
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Performance based risk sharing arrangements
To manage utilization in the real world
To provide evidence regarding decision uncertainty
- Outcomes guarantees - Money back guarantees - Conditional treatment continuation
Cost sharing arrangement
- Budget capping
- Utilization capping
- Discounts
- Price/volume
Payer-producer/provider
arrangement
Coverage with evidence development
Performance linked reimbursement
Intermediate endpoint
Clinical endpoint
- Only with research
- Only in research
- Process of care
Pre-specified agreement
No pre-specified agreement
Source: ISPOR Task Force Report

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Performance based risk sharing arrangements
To manage utilization in the real world
To provide evidence regarding decision uncertainty
- Outcomes guarantees
- Money back guarantees
- Conditional treatment continuation
- Budget capping - Utilization capping - Discounts - Price/volume
Coverage with evidence development
Performance linked reimbursement
Intermediate endpoint
Clinical endpoint
- Only with research
- Only in research
- Process of care
Pre-specified agreement
No pre-specified agreement
Examples of PBRSA Schemes
UK: MS RSS
Aus: Bosentan
UK: Votrient
Fr:glitazones risperidone
US Medicare: LVRS, PET, PTAS
Italy: oncology schemes
UK: Velcade, Lucentis
Source: Adapted from ISPOR Task Force Report
Cost sharing arrangement

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UK Multiple Sclerosis Risk Sharing Scheme: Test Case or interesting anomaly?

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Agenda
•What are the elements of value in personalised medicine?
•What institutional arrangements are needed for the assessment of value?
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Flexible pricing, coverage with evidence development and PBRSAs
•Challenge of diagnostic uptake

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Presentation to Genesis Conference December 9, 2014
France (INCa) – An approach to clinical implementation
•28 regional platforms
•Partnerships between several laboratories located in University hospitals and cancer centres
•Cooperation between pathologists and biologists
•Compensation of local pathologists for sample shipment
•Free of charge to patients and hospitals
•Public/private partnerships for molecular testing
•Early phase network of 16 early phase clinical trial centers (CLIP2)
Source: Buzyn 2013.

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France – estimates of economic impact of molecular testing
PFS = progression free survival
•Focus on cost-offset arising from not treating non- responder subgroups patients identified through testing.
•May explain willingness to fund the INCa initiative Source: Calvo 2011

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Summary
•What are the elements of value in a diagnostic test?
•A summary of nine case studies
•What institutional arrangements are needed for the assessment of value?
•Options for appraising stratified medicines and diagnostics
•Generating the evidence, linking it to value, getting the technologies used
•The need to align incentives
•Recognise diverse forms of evidence
•Flexible pricing and coverage with evidence development
•Diagnostic uptake – not yet resolved in the NHS