Embed Size (px)
DESCRIPTION
Best practices and considerations for conduct of Late Phase Studies.
Citation preview
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
o Key objectives and critical
differences of Late Phase
research
o Early planning: maximizing
product potential
o Selecting a strategic partner:
benefits and considerations
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
Phase II/III vs. Late Phase
o Objectiveso Protocol designo Study Populationo Sites outreacho Data collectiono Regulatory documents and submissionso Site agreementso Monitoring
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
Early Planning for Late Phase
FOCUSED. TRUSTED. GLOBAL.
Clinical to Commercial
o Know the Stakeholderso Research unmet needso Understand diverse objectiveso Consider industry advances
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
Starting Up
o Feasibilityo Start-up timelineso Product approval statuso Submission requirementso Site communication and training
FOCUSED. TRUSTED. GLOBAL.
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!
FOCUSED. TRUSTED. GLOBAL.
FOCUSED. TRUSTED. GLOBAL.
Data Management
o Electronic Data Captureo Electronic Health Recordso Direct Data Entry by patientso Multiple data sourceso Large data volumes
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
Selecting a Service Provider
FOCUSED. TRUSTED. GLOBAL.
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
FOCUSED. TRUSTED. GLOBAL.
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!
FOCUSED. TRUSTED. GLOBAL.
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
