late phase outsourcing - clinical studies

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Planning and Executing a Late Phase Trial: Setting Expectations and Maximizing Outcomes

Alexander Artyomenko MD PhD3rd Annual Outsourcing in Clinical Trials Europe

Zurich, 14 May 2013

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o Key objectives and critical

differences of Late Phase

research

o Early planning: maximizing

product potential

o Selecting a strategic partner:

benefits and considerations

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Example definitions Late Phase Studies

o Any study conducted on a marketed, fully approved

product

o Studies capturing clinical data and addressing real

world issues of safety, effectiveness, health outcomes,

reimbursement and risk management

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Late Phase Research Objectives

o Long-term real-world safety data and risk management FDA/EMA mandated Adverse claims New endpoints, health outcomes, effectiveness

o Maximize market penetration New comparators and dosage forms Label extension

o Provide access to yet unregistered drugs EAP/Named Patient/Compassionate use studies

o Health economics and Patient Reported Outcomes Reimbursement

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Interventional/Non-interventionalLate Phase Study Types

o Randomized Clinical Trials

o Observational epidemiologic studies

o Expanded Access Programs

o Post-Authorization Safety/Efficacy studies

o Health Economics and Patient-Reported

Outcomes

o Product and disease registries

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Regulatory Frameworko GCP and GPPo ISPE Guidelines for GPPo CIOMS International Ethical Guidelines for Epidemiological

Studieso Eudralex Volume 9A: Guidelines on Pharmacovigilance for

Medicinal Products for Human Useo GRACE Principleso Country-specific HEOR guidelines

o No harmonized legal framework across countries due to a lower risk to subjects

o High emphasis on scientific credibility and data protection

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Phase II/III vs. Late Phase

o Objectiveso Protocol designo Study Populationo Sites outreacho Data collectiono Regulatory documents and submissionso Site agreementso Monitoring

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Late Phase Studies/Non-interventional

o Capture real-world data/standard of careo Analyse data from a large number of patientso Less per patient costs than in a clinical trialo Increase disease and/or drug awarenesso Hypotheses generation and academic

research supporto Create and support stakeholder relationships

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ChallengesLate Phase Studies/Non-interventional

o Attributing causality to outcomeso Retrospective and prospective data

collectiono Diversity of regulation, standards of care

and cultures

Maximizing Product Potential

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Early Planning for Late Phase

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Clinical to Commercial

o Know the Stakeholderso Research unmet needso Understand diverse objectiveso Consider industry advances

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Engaging Broader Audience

o Phase I/II Safety vs. Long-term Safety Outcomes

o Efficacy vs. Effectiveness Real-World Data

o Cost and Value Compliance Health Economics

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Maximize Study Potential

o Focus on key objectives Impact on design, timelines, risks and costs

o Consider designs Follow-up extensions Health Economics and Patient-reported outcomes Genetic/biomarker testing

o Target geographies Market access KOL support Product launch

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Starting Up

o Feasibilityo Start-up timelineso Product approval statuso Submission requirementso Site communication and training

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Monitoring

o Risk assessment during protocol development

o Focus on patient safety and data quality

o Targeted on-site activitieso Timely data entryo Remote site managemento Patient recruitment…and

retention!

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Data Management

o Electronic Data Captureo Electronic Health Recordso Direct Data Entry by patientso Multiple data sourceso Large data volumes

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Safety Reporting

o Increased demand for real-world safety datao Focus on the events of special interest, SAEs

and SUSARs o Data mining beyond trials

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Selecting a Service Provider

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Selecting a Strategic Research Partner

o Therapeutic expertiseo Local knowledge and global reacho Broad scope of serviceso Prior positive experienceo Operational expertise and efficiencieso Market access expertiseo Greater flexibility

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Continuous Collaboration

o Facilitates early planningo Leverages previous learning o Ensures process clarityo Utilizes innovative technologieso Streamlines project executiono Maintains organizational focus

Saves time and money!

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Late Phase Research

o Therapeutic, regulatory and operational

expertise is essential

o Diverse objectives require early planning

o Strategic partnership brings significant

efficiencies to all stakeholders

Thank you!

Dr Alexander Artyomenko MD PhD

a.artyomenko@medpace.comwww.medpace.com