Post on 14-Apr-2017
transcript
Amanda Cole, Economistacole@ohe.org
Why RWE Matters to Payers: Incorporating RWE in Health Economic Analysis to Maximise Value
7th DecemberHealth Economics Global Congress 2015
Why RWE matters for payersDecember 2015 2
Content
1. RWD and RWE – what and why?• What is RWD/RWE• Why might it be important for payers?
2. Do payers live in the ‘real world’?• Managed entry agreements (MEAs) to address uncertainty
and maximize value for payers
3. Challenges• Implementation• Governance• Methods to deal with messiness
Why RWE matters for payersDecember 2015 3
Preview: Five global challenges1
The environment for global R&D is evolving
Challenge 1: Increasing importance of specialised and stratified medicines
Challenge 2: Rising drug development costs
Challenge 3: Closer benefit/risk monitoring by regulators over a medicine’s lifecycle
Challenge 4: Increase in demand for RWE and relative effectiveness by payers, and regulators
Challenge 5: Disconnect between regulators and payers/HTA bodies evidence needs
1 Mestre-Ferrandiz, Towse, Pistollato and Lewis. 2014. Securing a future for innovative medicines: a discussion paper. London: ABPI.
Why RWE matters for payersDecember 2015 6
• Real world data (RWD) • Collected outside of an experimental clinical trial
setting: reflects outcomes in the ‘real world’.• Electronic health records, clinical registries,
surveys, observational data from cohort studies, etc. (messy data)
• Real world evidence (RWE)
1. RWD and RWE – What and why?
Why RWE matters for payersDecember 2015 7
1. Understanding outcomes in the ‘real world’• Efficacy Effectiveness• Representative populations (co-morbidities,
spectrum of ages, treatment delivery less controlled etc.); additional insights e.g. adherence, costs.
1. RWD and RWE – What and why?
Why RWE matters for payersDecember 2015 8
2. Evolving R&D pipeline• Declining R&D investment in drugs - often
attributed to rising cost of developing new products
• More specialised and stratified medicines – Highly targeted, small populations, high
uncertainty
1. RWD and RWE – What and why?
2 Daniel et al. (2015) Health Affairs, 34 (2) pp. 319-3273 Scannell et al. (2012). Nature reviews Drug discovery, 11 (3) pp. 191-200
Source: Daniel et al (2015)2:
Annual new drug approvals by FDA and Industry spending on R&D, 1994-2013
Reduced R&D productivityNumber of new drugs approved per $billion halved every 9 years since 19503
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3. Timing of decision-points for regulators and payers are changing
• Regulators: Accelerating access to medicines that have the potential to address a high unmet need (uncertainty vs. urgency)– E.g.: EAMS (UK), ATU (France), BTD (U.S)
• Payers: Making decisions based on a limited evidence base.
1. RWD and RWE – What and why?
Adaptive pathways
Why RWE matters for payersDecember 2015 10
1. Understanding outcomes in the ‘real world’2. Evolving R&D pipeline3. Timing of decision-points for regulators and
payers are changing
USING RWE TO MANAGE UNCERTAINTY
1. RWD and RWE – What and why?
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• In England the National Institute for Health and Care Excellence (NICE) produces evidence-based guidance to the NHS (payer).
• Decisions based on robust clinical and cost-effectiveness evidence
• [not selected if it is expected that there is insufficient evidence]
• Yes• Optimised • No• Only in research (we’re too uncertain)
2. Do payers live in the ‘real world’?
Managed entry agreements
Why RWE matters for payersDecember 2015 13
• Managed entry agreements (MEAs)• Performance-based risk-sharing arrangements (PBRSA), outcomes-based
schemes, pay-for-performance, patient access schemes...
• Formal utilisation of RWD to manage the entry of a product. Can be in many forms, for example:
• commitment to collecting confirmatory evidence of effectiveness in real-life settings
• linking reimbursement explicitly to patient outcomes
• capping expenditure • Ensures the payer receives value for money
2. MEAs
Why RWE matters for payersDecember 2015 14
Source: University of Washington PBRSA database. May 2015
2. MEAs
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2. MEAs• MEAs to address uncertainty
and maximise value for payers4
Clinical uncertaintyValue for moneyBudget impact
4 Garrison, L.P., Towse, A., et al. (2013). (5) pp. 703-719
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• Reimbursement based on outcome - ‘Rebate’ for non-responders
• Manufacturer pays for the medicine beyond a certain number of doses
• Reimbursed for a specified period of time whilst further evidence / RWD are collected
Performance-linked reimbursement
Coverage with evidence development
Outcomes guarantee
Dose capping
RE
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A2. MEAs
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• Substantial administrative burden• Can be complicated to negotiate with providers• Capacity for data collection
• Electronic health records, registries, patient identifiers for data linkage5
• Coverage with evidence development:• sufficient time to address resolvable uncertainty • Not straightforward to ‘de-list’ a drug (an ‘acquired
right’?)
3. Challenges: Implementation
5 Chapman & Karlsberg-Schaffer (2015). OHE Consulting report: https://www.ohe.org/publications/assessing-use-multi-indication-medicines-review-current-data-capabilities-uk
Why RWE matters for payersDecember 2015 19
• Who manages (and pays for) data collection / analysis?
• Information governance• Data protection: patient consent• De novo versus routinely-collected data• Audit or research?
3. Challenges: Governance6
6 Cole, Garrison, Mestre-Ferrandiz & Towse (2015) OHE Consulting Report. London: https://www.ohe.org/publications/data-governance-arrangements-real-world-evidence
Why RWE matters for payersDecember 2015 20
3. Challenges: Methods• Methodological challenges in dealing with
messy RWD• Incomplete data• Confounders and biases• Quality of data
• Precedent set by medical technologies evaluation?
Why RWE matters for payersDecember 2015 21
• Understanding outcomes in the real world• Being responsive to an evolving R&D pipeline• There is an expanding experience with regulatory
mechanisms that allow earlier access to medicines in development Payers need to be on board
• HTA moving from Yes/No Agreements• MEAs can explicitly link payment with value• There are implementation, governance and
methodological challenges to the implementation of MEAs.
ConclusionWhy RWE Matters to Payers
Why RWE matters for payersDecember 2015 22
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acole@ohe.org