patients registries - ispor · presentation registries support rmp ... design of edc tool...
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Patients RegistriesCarlos Peralta
Sources of “Real World Data”
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EBM & RCT placed at the top of the evidence hierarchy• Limited controlled experiment that not generalize to population,
settings or conditions not reflected in the trial.• Impracticality of basing all of medicine on RCTs• Expensive
ISPOR “Real World” Task Force Report• Supplements to traditional registriation RCTs• Large simple trials (practical clinical trials)• Registries• Administrative data• Health surveys• Electronic records and medical chart reviews
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Clarify some distinctions between registries andclinical trials
Clinical Trials
is an experiment
active intervention
randomization procedure
inclusion and exclusion criteria
homogeneous group
adhere to a strict schedule of visits
conduct protocol-specific tests and measurements
Registries
observational study design
does not specify treatments
observe
few inclusion and exclusion criteria
broad range of patients
tests and measurements a provider customarily uses
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Patient registry is anorganized system that uses observational studymethods to collect uniform data (clinical and other)
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Purpose
to describe the natural history of disease
to determine clinical effectiveness or cost effectiveness of health care products and services
to measure or monitor safety and harm
to measure quality of care.
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Planning
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Key steps in planning a patient registry• articulating the purpose of the registry• determining whether the registry is an appropriate means for
addressing the research question• identifying stakeholders• defining the scope and target population• regulatory and research practice considerations• assessing feasibility• assessing securing funding.
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Registry Design
Key points to consider in designing a registry include• formulating a research question (hypotheses)• choosing a study design• translating questions of clinical interest into measurable exposures
and outcomes• choosing patients for study,• Including deciding whether a comparison group is needed• determining where data can be found• Deciding how many patients need to be studied and for how long
Data Elements
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It is important to determine which elements are absolutely necessary and which are desirable but not essential.
choosing measurement scales (validated)
data map
data collection tools
Overall, choice of data elements should be guidedby parsimony, validity, and a focus on achieving theregistry’s purpose.
Data Sources
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Primary data are collected for direct purposes of the registry
Secondary data are comprised of information that has been collected for purposes other than the registry, and they may not be uniformly structured or validated with the same rigor as primary data
Ethics, Data Ownership, andPrivacy
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The Common Rule is the uniform set of regulations on the ethical conduct of human subjects research
The research purpose of a registry, the status of its developer, and the extent to which registry data are individually identifiable largely determine applicable regulatory requirements.
ethical and legalconcerns include transparency of activities,oversight, and data ownership.
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Patient and ProviderRecruitment and Management
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Factors that motivate participation include:• perceived relevance, importance, or scientific credibility of the registry• Incentives for participation
well-planned strategies for enrollment and retentionare critical.
Data Collection and QualityAssurance
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Requirements for quality assurance should be defined during the registry’s inception and creation.
developing a quality assurance plan is recommended.
Adverse Event Detection,Processing, and Reporting
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Regardless of whether or not AEs constitute outcomes for the registry, it is important to develop a plan for detecting, processing, and reporting AEs for any registry that has direct patient interaction.
The sponsor has mandated reporting requirements, and the process for detecting and reporting AEs should be established and registry personnel trained on how to identify AEs and to whom they should be reported.
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Analysis and Interpretation
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Core questions.
•Who was studied?•How were the data collected, edited, and verified, and how were missing data handled?
•How were the analyses performed?•The representativeness of the actual population to the target population is referred to as generalizability.?
Different perspectives
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From a Marketing perspective, it's the perfect way to engage your customers and discover real-time and actionable insight into how your drug is performing. It's a perfect tool for post marketing research and surveillance.
From a Clinical perspective, it's a unique and powerful method for the collection of observational or epidemiologic data for your Phase IV clinical trial and registry needs, and is becoming a widely adopted business tool in the pharmaceutical, medical device, biotechnology, academic and private healthcare settings.
From a Private Provider and Academic Medical Center perspective, it's an effective tool for gathering post-approval research to accelerate innovation, produce faster knowledge gains and alert companies to potential problems, patient safety issues and serious adverse eventconcerns, quicker than any other available tool.
Turning the marketing wheel
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Create momentum
Create Noise
Build a community
Position your product or service
Turning the marketing wheel
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KOL relationshipPhysician
relationship New indication
Off Labelmonitoring
Real worldUsage trends
Key physicianpresibers
Reimbursementsupport
Trade Publication
SymposiumPresentation
SalesRepresentatives
User meetingpresentation
RegistriesSupport RMP
Peer review articules
Supplementssymposium
Summary
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A patient registry is• an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure and that serves predetermined scientific, clinical, or policy purpose(s).
• Well-designed and well-performed patient registries can provide a real-world view of clinical practice, patient outcomes, safety, and comparative effectiveness and serve a number of evidence development and decisionmaking purposes
NOVARTISRegistry
Introduction
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CRO team
Project Manager
Database Manager
Data Manager Lead
Statistician
Agenda
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CRO responsibilities
CPO responsibilities
Communication strategy
Training to physicians
EDC user access
Management of missing query responsesand data entries
SAE reporting and reconciliation
Support of sites
Summary
CRO responsibilities
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Design of EDC tool (Electronic Data Capture)
Queries Management: Electronic only
SAE reconciliation
Reporting: Electronic only
Help Desk: questions regarding EDC tool
Development and distribution of newsletters • 2 times per year, published via email and on a website
Develop training materials & provide training to CPO representatives
Statistical Analysis
CPO responsibilities: EDC support
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Provide training to CPO and site staff • CRO is not involved in training of CPO and site staff
Provide user access information about CPO and site staff
Query management – Follow-up with sites• Frequently consult CRO Web Reports for site status• Motivate compliance with data collection• Support with delayed and missing queries
SAE management: Resolve discrepancies and follow-up with sites
Help Desk for EDC questions• Out of Help Desk operating hours: support sites in case of emergencies • Contact help desk in case of issues with EDC tool
Communication strategy
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NOVARTIS Global
CROProject Manager
Study progressStrategy & timelinesIssues, budgets
Study sitesPhysicians and nurses
• Set up activities• Site Selection• Patient enrolment• Site training• Study progress• Site motivation• Query resolution
support• Follow-up of missing
SAE reports
NOVARTISCPOs
Query management
CRO Data Management
User Access List
Newsletters Web Reports
Site training material
Training to Physicians
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All physicians have to be properly trained before CPOs request access to EDC databaseAll physicians have to be properly trained before CPOs request access to EDC database
NOVARTIS Global
NOVARTIS CPO representatives
Study sitesphysiciansand nurses
TrainCRO Train
CRO provides training material to Novartis• Test database and user names / passwords• Presentation slides• Q&A log
CRO provides training material to Novartis• Test database and user names / passwords• Presentation slides• Q&A log
EDC user access
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CROTest database
PhysiciansSite staff Novartis CPO staff / Global
User
CROEDC database
• Only for training, only dummy data entry possible
• Each user has separate user name / password
• Only for training, only dummy data entry possible
• Each user has separate user name / password
• Only sites with complete approval, properly trained and ready for data entry
• Different access levels for Novartis Global, CPOs and sites
• Only sites with complete approval, properly trained and ready for data entry
• Different access levels for Novartis Global, CPOs and sites
EDC user access –training / test database
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CROData Management
NOVARTIS Global
NOVARTIS CPOs
Provide user test accounts
Provide user test accounts
User
Distribute to relevant users
EDC user access –for data entry
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CROData Management
Provide EDC user access information
and updates
NOVARTIS Global
NOVARTIS CPOs
Approve user access
Arrange user access
• Country• First name / last name• Function (principal physician, study nurse)• Email address
• Within 1 day / 24 hours• User receives user name and passwordin 2 separate emails
• Within 1 day / 24 hours• User receives user name and passwordin 2 separate emails
Missing query responses / data entries
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CROData Management
NOVARTIS Global
Study sitesphysiciansand nurses
NOVARTIS CPOs
Follow-up with sites
Send monthly reminders to check Web Reports
Inform about problem sites
Send reminders to resolve query / enter data
Follow-up with CPOs
Check Web Reports
If no action after 2 weeks
If no action after 4 weeks
If no action after 8 weeks
Reminders for data collection / queries
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Data collectionCRO/ EDC
reminderto site
2 2 weeksweeks beforebeforeexpectedexpected datadata entryentry
ExpectedExpecteddatadata entryentry
2 2 weeksweeks afterafterexpectedexpected datadata entryentry
4 4 weeksweeks afterafterexpectedexpected datadata entryentry
CROEDCreminder
to site
CRO/ EDCreminder
to site
CPO follow-up with site
2 2 weeksweeks afterafterqueryquery sentsent
4 4 weeksweeks afterafterqueryquery sentsent
CRO/ EDC: Query email
to site
CRO/ EDC reminder
to site
CPO follow-up with site
WithinWithin 2 2 weeksweeks afterafterdatadata entryentry
Query management
SAE reporting
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General procedure
Physicians reports SAE to Novartis Safety Desk
Physicians enters SAE into EDC tool and indicates whether reported to Novartis Safety Desk
Potential problems - Discrepancies
SAE not reported to Novartis Safety Desk: immediate action required by CRO (CPO to support FU)
SAE not entered into EDC tool
Different information in Novartis Safety Database and EDC database
SAE reporting and reconciliation
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CROData Management
Reports SAE
NOVARTIS CPOLocal Safety Desk
Enters SAE in eCRF
Study sites
NOVARTIS Global Safety
Reconciles SAEs
Provide monthly listings
SAE discrepancies
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CROData Management
Report SAE
NOVARTIS CPO
Enter SAE in eCRF
Study sites
NOVARTIS Global Safety
Check Web Reports for pending query response /
data entries
Send query to sites
If query confirms discrepancy, request safety database amendment
Follow-up with sites
If SAE not reported, request confirmation
Support of sites
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CRO‘s Help Desk
Supporting questions regarding use of the EDC tool - Not for medical issues
Operating time: Monday to Friday, 9 to 5 CET
Mailbox out of operating time:• Sites can leave message, will be followed-up within 24 hours• Mailbox message will advise to contact local Novartis CPO for urgent
cases
CPO‘s Support
CPOs to assist sites in resolving issues, if possible
Summary – CPO responsibilities (1)
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Train relevant CPO staff Before staff train other staff or physicians
Inform Novartis CTL about dates of local training meetings
At least one week before local training meeting to ensure test database is operational on that day
Q & A log Forward any questions about EDC tool to Novartis Global for the Q&A Log
As appropriate
Train physicians Before physicians enter data
Training
If required, re-train new NVS staff and physicians
Before new NVS staff trains other NVS staff or physicians Before physicians enter data
Complete EDC access list received from Novartis Global and return to Novartis Global
At least 1-2 days before planned enrollment
Whenever there are changes to the EDC access list, provide updated list
As appropriate
EDC user access information
Summary– CPO responsibilities (2)
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Check Web Reports and identify sites with late / missing queryresponses / data entries > 4 weeks
Monthly
Follow-up with the sites in case of late / missing query responses / data entries > 4 weeks
On regular basis and as required
SAE that are not reported to Safety Desk
Follow-up with sites about non-reported SAEs As required
EDC site issues Support sites in resolving EDC issues during out of operating hours of EDC help desk
On regular basis
Late / missing query responses / data entries
Questions and Close
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