patients registries - ispor · presentation registries support rmp ... design of edc tool...

Patients RegistriesCarlos Peralta

Sources of “Real World Data”

2 | Presentation Title | Presenter Name | Date | Subject | Business Use Only

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


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

Patient registry is anorganized system that uses observational studymethods to collect uniform data (clinical and other)


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.

Planning


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.


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


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


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


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.

Patient and ProviderRecruitment and Management


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


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


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.

Analysis and Interpretation


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


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.

http://www.strategicpatientregistry.com/phase_IV.html




http://www.strategicpatientregistry.com/patient_safety_sae.html

http://www.strategicpatientregistry.com/patient_safety_sae.html

Turning the marketing wheel


Create momentum

Create Noise

Build a community

Position your product or service

Turning the marketing wheel


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


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


CRO team

Project Manager

Database Manager

Data Manager Lead

Statistician

Agenda


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


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


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


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


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


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


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


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


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



Reminders for data collection / queries


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


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


CROData Management

Reports SAE

NOVARTIS CPOLocal Safety Desk

Enters SAE in eCRF

Study sites

NOVARTIS Global Safety

Reconciles SAEs

Provide monthly listings

SAE discrepancies


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


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)


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)


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


patients registries - ispor · presentation registries support rmp ... design of edc tool...

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