Keeping it Real: Utilization of real world data sources to generate RWE for better evidence-based decision-making 2017 CADTH Symposium Ottawa, ON - April 24th, 2017

Presented by Tarry Ahuja, PhD. Sr. RWE Scientist – Medical, Europe & Canada, Eli Lilly Canada Inc.

Scientific Disclosures

• Employed by Eli Lilly Canada Inc. o Sr. RWE Scientist – Medical, Europe & Canada,

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Objectives

• types of real-world data sources (strengths/limitations)

• what RWE can and cannot deliver • applications of RWE • CPCSSN – a real-world example

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Real World Data (RWD)

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The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) defines real-world data as “data used for decision making that are not collected in conventional randomized controlled trials (RCTs)”.

Sources of Real World Data (RWD)

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Real-World Data

(RWD)

Databases & Registries

Patient & Population

Surveys

Patient Chart

Reviews

Observational Data

Pragmatic Clinical Trial

Consumer Data &

Social Media

Real World Evidence (RWE)

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Question/ Hypothesis

Real World Data

Design and Analytics

Real World Evidence/ Insights

Strengths of RWE

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• real world/complex patients • representative population • large numbers/long duration • group comparisons • costs/resource utilization • patient-reported outcomes • more timely, less costly

Strengths

Weaknesses of RWE

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• not randomized: potential bias • many confounders • weak for treatment comparisons • high drop-out rates • variability in methodology

Weaknesses

Why RWE Research Is Needed

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• RCTs are not enough, customers want effectiveness data

• better understanding of long-term data is necessary

• certain bodies (ie. EMA) require PASS, PAESS, Pt. registries

• increased demand for ‘value’ beyond efficacy/safety: o mandatory post-launch requirements (FR, IT, NEC,..)

o outcomes-based agreements

o need to manage costs (RCT vs. RWE)

What RWE Can And Cannot Do

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RWE can…

• generate evidence of value

• establish effectiveness and comparative effectiveness

• validate safety profile or identify safety concerns

• determine real-world practice patterns and treatment strategies

• identify unmet need

What RWE Can And Cannot Do

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RWE cannot…

• influence labeling regarding efficacy of a product

• substitute the evidence generated through RCTs (complementary evidence)

Application of RWE in HTA

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RWE pre-launch

Understand Standard of Care

Identify Unmet Need

Budget Impact

Application of RWE in HTA

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RWE post-launch

Understand Safety

Utilization

Effectiveness

Application of RWE in HTA

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RWE post-launch

Understand Safety

Utilization

Effectiveness

• Outcomes based agreements • Adaptive pathways

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Real World Example of RWE in Action

Canadian Primary Care Sentinel Surveillance Network (CPCSSN)

Canadian Primary Care Sentinel Surveillance Network (CPCSSN)

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• over 1 million Canadian patients

• 11 PBRNs in 8 provinces, 1 territory

• captures EMR data (some as far back as 2003)

• anonymized data are merged into a national data set

Data Capture by CPCSSN

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RWD capture includes:

• patient characteristics and provider profile

• disease specific health outcomes

• socio-economic information

• medications

• laboratory data

RWE Generated with CPCSSN Data

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RWE Information:

• drug utilization (pre/post) in “real” patients

• understanding of co-morbidities

• holistic estimate of resource utilization

• assessment of safety data (adverse events), possible signals

• pan-Canadian generalizability

• dynamic feedback promotes optimal use of medications

Appropriateness of RWD

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Local RWD

Source

National RWD Source

• info on subpopulations • local treatment patterns • local access & reimbursement

• pan-Canadian perspective (generalizability)

• increased sensitivity (large N)

Challenges

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• identifying, accessing, and generating RWD and RWE

o where do data sources exist?

o what does good look like?

• acceptance of RWE by HTA, Health Canada, and payers

o evidence outside of RCTs

o framework for incorporating in to current process

• agreement on definitions and standards for RWD/RWE

Benefits

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• improved understanding in real-world setting

o effectiveness, treatment patterns, utilization

• potential for improved health-care spending

• improved access of medicines for appropriate patients

• collaboration across health system stakeholders

o HTA, regulators, industry, academics, HCPs, patients

Conclusions

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• RWD sources are increasing in quantity/quality in Canada

• RWE can provide information including; effectiveness, utilization, treatment patterns, and safety

• RWE has the potential to be a powerful tool within the HTA process, and when making decisions around access

• RWE may allow for better informed decisions

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