© #oc15europe 1 electronically collecting data from participants in clinical trials june 1, 2015...
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Electronically Collecting Data from Participants in Clinical Trials
June 1, 2015Ben Baumann
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Trajectory Terminology Benefits & Promises Barriers & Reality Technology Regulatory Content Data Management
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eCOA(electronic clinical outcomes assessment)
Patient Driven eData
Definition: an electronic assessment that may be influenced by human choices, judgment, or motivation and may support either direct or indirect evidence of treatment benefit.
Assessment = instrument = questionnaire = form…
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eCOA ModalitiesModality Definition Use When Examples
ePRO (Patient-reported outcomes)
Data reported electronically directly verbatim by subjects. Self report, or interview.
• Trained health professional not needed + self-report feasible and appropriate.
• Unobservable concepts (feelings, sensations)
• Pain intensity• Mood/feelings• Eating habits
ClinRO (Clinician-reported outcome)
Report from a health-care professional observing subject’s condition. Involves clinical judgement/interpretation.
Trained health professional needed
• tumor size• Parkinson’s patient who
cannot comment• palpating a spleen
ObsRO (Observer-reported outcome)
Measurement based on an observation by someone other than the patient or a health professional. Does not include medical judgment/interpretation.
Trained health professional not needed and self-report NOT feasible/appropriate.
• parent/infant• caregiver/elderly person
PerfO (Performance outcome)
Performance of a task by subject based on instructions administered by a HC professional
The specific task is required by protocol
• 6 minute walk test• Memory recall
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Why do it?
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• Better dataQuality
• Faster, less laborProductivity
• Engage patients in new ways
Enablement
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May Clinic Study
How would you describe your overall quality of life today?
0 1 2 3 4 5 6 7 8 9 10 As bad as can be
As good as can be
Score of 5 or less, 15 months survival Sore of 5+, 52 months survival Cannot ignore decrease of 2
3,704 cancer patients
This is a prognostic factor for cancer patient survival
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Incorporated QOL assessment into daily clinical oncology practice
Initiated conversations with patients Insomnia Stupid thoughts
Reduced emergent care
Improved survival
Improved quality of life
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Paper vs. ElectronicPaper
Piles of paper can be confusing
Don’t need to worry about the internet
Chance of getting lost/destroyed
Slow access to actionable information
Parking lot syndrome
eCOA More controlled experience
System prompts user automatically
Increased compliance (regulatory, patient) Requires secure login/password
Date/Time Stamped in Audit Trail
E-Source recorded in Audit Trail
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Drivers of eCOA Quality & Efficiency
Increased data quality (edit checks, data types, show/hides, required fields)
More efficient data collection (reduce need for data entry)
Improved patient compliance1,2
Auto-reminders Electronically monitor
Know exactly when something happens
Report deviations to sites immediately
Get data earlier to prevent problems
Better patient care
Cost savings
1 ePRO often produces compliance rates over 90%. See Meltzer EO, Kelley N, Hovell MF (2008). "Randomized, Cross-Over Evaluation of Mobile Phone vs Paper Diary in Subjects with Mild to Moderate Persistent Asthma". 2 Friedberg F, Sohl SJ (2008). "Memory for Fatigue in Chronic Fatigue Syndrome: The Relation Between Weekly Recall and Momentary Ratings". International Journal of Behavioral Medicine 15 (1): 29–33
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Drivers of eCOA (cont.) Technology trends
Ubiquity of internet access Mobile devices Responsive design Sensory devices Apple ResearchKit
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Drivers of eCOA (cont.) Patient engagement
Integrate into subjects’ lives, especially if BYOD Fewer dropouts More events Feedback
Improvement over time Comparison vs. the field
Engaged Patients
Increased Compliance
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eCOA in an ALCOA World
Attributable
Legible
Contemporaneous
Original
Accurate
Login / access code
Can control when things happen
It’s digital, structured
ePRO captured directly from subject
Edit checks, rules logic
CompleteConsistentEnduringAvailable
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Barriers to Adopting eCOA
Expense (or, at least perceived expense)
Devices / Logistical complexity
Usability
Fit with study population
Integrating with existing EDC/CDM Systems
“Translating” existing PROs to electronic form
Others? What has stopped you?
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Adoption of eCOA 2009 FDA PRO guidance1
More trials are collecting COA data
Use of BYOD increasing, but mostly in post-approval studies
Scientific evidence growing to broaden use
Technology farther ahead of the regulations and adoption
1 http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf
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Success = Planning + Implementation
So, let’s break it down:
Formula for Success
• Patient experience• Content• Regulations• Technology
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Patient Experience
How will you engage your patients? Motivation Incentive Feedback
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Usability Number of forms/instruments Items per page Scolling Edit checks Required fields Skip logic Accommodating disabilities Offline Support: patients, devices, logistics
“Simple can be harder than complex: You have to work hard to get your thinking clean to make it simple. But it’s worth it in the end because once you get there, you can move mountains.”
- Steve Jobs
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Language
Will instrument be translated? Avoid idiomatic expressions Allowing for text length Left Right, Right Left
pressclickmouse
select
choose
enter
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Item Stems & Recall Making questions self contained
During the last week, how has the pain interfered with: Going to work Sleeping at night
During the last week, how has the pain interfered with going to work?
During the last week, how has the pain interfered with sleeping at night?
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Usability Testing Field-test involving patients (ePRO) and/or clinicians
(ClinRO) -- use the real thing
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Electrifying Existing COAs
1 FDA PRO Guidance 2009: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf
Must show eCOA version yields data which are equivalent or superior to paper version.
eCOA version must measure the same thing as paper
Need to show evidence of evidence of equivalence1
Level of evidence depends on severity of change
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Evidence of Equivalence
Table from ISPOR ePRO Task Force Paper: http://www.ispor.org/workpaper/patient_reported_outcomes/Coons.pdf
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Content: Leveraging Others’ Work C-Path PRO and ePRO Consortia (www.c-path.org)
Pre-competitive, FDA + industry Collaborate to generate measurement equivalence data Best practices
PROMIS (www.nihpromis.org/) Freely distributes instruments Tested, multiple languages
ISPOR (http://www.ispor.org/)
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Regulations: Available Guidance
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2009 FDA PRO GuidanceHow FDA reviews and evaluates existing, modified, or newly created patient-reported outcome (PRO) instruments used to support claims in approved medical product labeling.
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2013 FDA E-Source Guidance Assure reliability, quality, integrity, and traceability of data from electronic source
to electronic regulatory submission.
Promotes capturing source data in electronic form
Retention of electronic source data in FDA-regulated clinical investigations.
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2013 Mobile Medical Applications
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Mobile Medical Applications
Mobile App: A software application that can be run on a mobile platform, or a web-based software application that is tailored to a mobile platform but runs on a server.
Mobile Medical App: A mobile app that meets the definition of a device and is intended: To be used as an accessory to a regulated medical device
To transform a mobile platform into a regulated medical device (e.g., via a sensor or electrode)
FDA intends to apply its regulatory oversight to only those mobile apps that are medical devices and whose functionality could pose a risk to a patient’s safety if the mobile app were to not function as intended.
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Technology
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Choices, choices…
Web-based
Choice Considerations
Smart Phone • Typically used for ePRO• Well-integrated messaging (SMS, phone, email)• Smaller screens may present issues for certain
instruments• Logistics if provisioning
Tablets • Typically used in ClinRO settings• More readily emulate paper• Logistics if provisioning• Less validation burden
Digital Pen • Special pen, special paper• Still have paper• Legibility• Technology support
IVR • Limited types of questions• Recall time• Patients get frustrated
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App vs. Web Limitations and Liberties
Pushing updates
Validation
Data stored on device
Responsive design
What actually is the app?Web?
App?
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BYOD?Provide device?
Device Provisioning vs. BYODDevice BYOD
User may forget Already in habit of using
Greater control Unknown device configurations
Connectivity issues Already connected
Simpler validation Device checksResponsive design
Connectivity issues Participate already connected
Lots of logistics and support
Little to none
Hybrid BYOD / Provisioning an option
…or both?
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Role of Data Management Edit checks/controls at entry Required fields: complete data vs. accurate data Frequency of data collection EDC/CDMS integration Reviewing data How to handle data received too early / too late Clear policy with sites around changing ePRO data (e.g. visit dates,
basic demographics) Under control of investigator at all times Paper as back-up; how to handle Site Support, FAQ
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Putting it All Together
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Thinking About ROIPaper
Costs you are incurring: Printing
Mailing
Data Entry
Data Cleaning
Electronic
Costs you are incurring: Electronic system
Costs you are saving: Less data entry
Less data cleaning
Printing and mailing
Fewer site payments
Quality benefits: Increased accuracy
Removing interpretation
More time for patients
Faster study closeout
Vs.
Some estimates as high as $20 per page
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We Can Do This!Technology has advanced and we have the power to use
that technology to improve patient care.
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