This document discusses the use of real-world evidence (RWE) to support payer decisions for Alzheimer's treatments. It proposes developing a multi-modal data access platform that includes 1) a natural history model of Alzheimer's across disease stages, 2) appropriate health economic and budget impact models, and 3) new standards for collating and evaluating RWE. Big data insights from RWE could help reduce uncertainty for health technology assessment bodies and payers regarding clinical and economic outcomes over the short and long term for Alzheimer's treatments. However, meaningful use of outcomes-based agreements requires infrastructure for data collection and ability to collect meaningful data to address current lack of trust in RWE.

1. THE ROAD TO 2025: Delivering Next Generation Alzheimer’s
Treatments
27-28 October 2016, Lausanne, Switzerland
Beyond Clinical Trial Data: How Can the Evidence Be Developed to Support the Payer Community?
Gaining Real World Evidence to Support Payer Decisions:
A European Example
Public-Private Partnership to Drive Real World Evidence
Frederic de Reydet de Vulpillieres - Global Patient Access, Novartis
Kristin Kahle Wrobleski - Global Patient Outcomes & Real World Evidence, Lilly
Martin Pan – Global Medical, Alzheimer’s Disease, Biogen 1

2. 2
Real world Outcomes across the AD spectrum for better care:
Multi-modal data Access Platform
1. Develop a natural history model across
disease stages
2. Develop appropriate health economic and
budget impact models
3. Set new standards for the collation and
evaluation of RWE to inform decisions

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Large Data Set from
several sources
Natural History of
Disease and Economic
Modelling
Harmonize Collation
Methods and
Analytical Tools

5. THE ROAD TO 2025: Delivering Next Generation Alzheimer’s
Treatments
27-28 October 2016, Lausanne, Switzerland
Beyond Clinical Trial Data: How Can the Evidence Be
Developed to Support the Payer Community?
What can HTA bodies and payers gain from Big Data insights?
Dr Jacoline Bouvy, National Institute for Health and Care Excellence
(NICE), United Kingdom 5

6. HTA bodies and payers
• Deciding on coverage/reimbursement of new
technologies
– Clinical effectiveness (HTA)
– Cost-effectiveness (HTA)
– Affordability (payers)
– Budget impact (payers)
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7. Using RWE
Strengths
• Real-life conditions reflective of use of
the drug in populations once launched
• Reflects the effectiveness of the drug in
an actual, day-to-day clinical setting
• Allows for assessment of outcomes not
captured adequately within clinical
trials
• Better captures adherence/compliance
to treatment
• Often captures outcomes in a longer
time scale than RCTs
Weaknesses
• Lack of defined methodological
standards
• Great heterogeneity in the studies
conducted to date
• Data access/privacy issues
• Low internal validity: e.g. lack of
randomisation leads to selection bias –
confounding factors mean large sample
sizes required and statistical adjustment
• …

8. RWD & AD: possible roles
• Reduction of uncertainty
• Clinical outcomes: short-term versus long-term
• Economic impact of disease
• Use of (successful) outcomes-based managed
entry agreements limited
– Infrastructure for data collection
– Ability to collect meaningful data
– Lack of trust
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