The Changing Face of Clinical
Trials - Patient-Centered
Research in the Internet Age
Cal Collins
Co-founder & CEO
OpenClinica, LLC
June 21, 2013
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What is “Patient-Centered Research”?
Part of a “learning healthcare system”?
A trendy buzzword?
Where the money is?
Cheaper and less rigorous than RCTs?
A major cultural shift in approaches to research?
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What is “Patient-Centered Research”?
It is generally defined as research
leading to evidence-based, patientcentered health interventions.
Umm, ok.
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3
Why is Patient-Centered Research Important?
It helps people make more informed healthcare decisions by directing
research toward questions that are important to patients, measuring
outcomes that are noticeable and meaningful to them, and producing
results that help them weigh the value of healthcare.
Due to a focus on 'real world' factors, it tends to be prominent in certain
research designs:
 Observational research
 Registries
 Pragmatic trials
But it has applications to the design and methods of explanatory
randomized clinical trials as well.
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Why is Patient-Centered Research Important?
Less than one-third of people who come in for a screening
end up completing a clinical trial.[1]
Employing new ways to engage patients and physicians
while increasing their level of knowledge and trust can
improve the sponsor’s ability to meet recruitment goals.
[1] Getz, Ken, The Gift of Participation: A Guide to Making
Informed Decisions About Volunteering for a Clinical Trial,
2007, p40.
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Why do people participate in trials?
“Big picture” factors:
 Access to needed treatment
 Altruism (advancing science, helping those with
similar conditions)
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Why do people participate in trials?
“On the ground” factors that influence decisions:
 Prior participation
 Relationships with researchers
 Trusted leaders
 Trust in organization
 Incentives
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Why DON’T people participate in trials?
 Recruitment/Matchmaking insufficient
 Not convenient
 Privacy concerns
 Cultural norms
 Confusing consent forms
 Lack of knowledge about potential to participate
 Control over data sharing/secondary use
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Source: http://dx.doi.org/10.1016/j.atg.2012.07.001
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What roles can technology play?
 Increase convenience (ePRO, web-based access to trial
materials, email reminders).
 Strengthen connections between participants and
researchers. Create infrastructure for coordinated teambased care, home-based continuous monitoring, including
wireless data reporting from medical devices.
 Enhance communication structures - provide trial-specific
information at the points in clinician and participant
workflow when they can best make use of it.
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What roles can technology play?
 Improve recruitment – social networks, cohort
identification through patient record mining
 Provide rewards/incentives to participation – gamification,
access to health information, risk factors, etc on mobile
devices
 Better secondary data use/feedback loops
 The (still elusive) promise of using the Electronic Health
Record system in research to identify participants and
capture clean, accurate trial data with less duplication
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What is Open Source? Why does it matter?
 Open Source Software is software that allows
programmers to freely run, read, redistribute, and modify
the software’s source code.
 We need new, innovative approaches to improving
health outcomes. We need to be integrated and patientcentric, in care and research.
 Open Source Software (OSS) can deliver improved
integration, quality, and efficiency in patient-centered
care and research environments.
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Why Open Source?
 When people can rapidly download and try things for
free, share ideas with a community, and see each others’
source code, a few surprising things happen:
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It’s easier to evaluate… all the way down to the code
Technologies get ‘mashed up’ in highly innovative ways
Best practices emerge and are shared quickly
Interoperability is easier
Business can focus on where they contribute the most
value, rather than on the ‘plumbing’
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Prominent Patient Centered OSS Technologies
 OpenDataKit (ODK) and OpenXData
 Mobile data capture by field workers and patients.
 http://opendatakit.org/
 http://www.openxdata.org/
 SMART
 The “App Store” for health care
 http://smartplatforms.org/
 tranSMART
 Translational research platform (based on i2b2
 http://www.transmartproject.org/
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Key Goals for Patient Centered Capabilities in
OpenClinica
Patients require a simple interface that can be used
without any training. As such, Participant Portal and
mobile device solutions must meet several goals:
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1.
Make it easy for participants to securely access assessment
forms and enter data in a timely fashion.
2.
Send the participant a reminder when his assessment is due or
overdue
3.
Make sure the participant completes the correct assessment
(assuming there are different questions at different time
points).
4.
Submit data back to the clinical trials database in real time
Approach
 Develop stand alone modules that leverage core
OpenClinica
 Study design is done in one place (OpenClinica) and
deployed to all modules, including event/CRF design,
skip logic, and study calendar => no separate study
build process
 Provide a simple, intuitive data entry interface and
submit data back to the OpenClinica instance.
 Provide participants the ability to complete the right
forms at the right time plus provide the management
tools needed to manage the patient responses.
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Participant Portal Overview
 Web-based survey and outcomes reporting by clinical
study participants.
 A standalone application that interfaces with existing
OpenClinica capabilities.
 Leverages the existing investment in OpenClinica’s
study design, data management, and extract
capabilities.
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Participant Portal Overview
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Participant Portal Overview
Portal forms support:
 Skip patterns
 Real-time data checks
 Multiple input types
 Repeating Groups*
 Simple UI with
customizable layout
 Design in OpenClinica
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* - not implemented for proof of concept
Tablet-based Data Capture
Needs:
 Tablet-based
surveys/instruments for
completion by patient
 Provide immediate
feedback on health risks
and conditions for
clinicians to use in the
patient visit
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Tablet-based Data Capture
 Uses existing open source components (OpenClinica, ODK), same
technology foundation as Participant Portal
 Uses OpenClinica CRF definitions and rules, no additional
programming/design necessary.
 Touch screen tablet, with support for other modes (SMS, IVR) in
the future.
 Standards-based form definition (CDISC ODM) provided by EDC
System, automatically converted to XForm for mobile client device.
 Open source technologies ‘mashed up’ in 9 month development
period
 ODK
 Dimagi TouchForms
 OpenClinica EDC
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Vision
True transformation of healthcare must deliver on potential
of research via integration into practice (translational
research and precision medicine)
PCAST report on Health IT, Dec. 2010:
Nations are creating a “health data
infrastructure in which patient
data… can be accessed in deidentified form by researchers and
public health agencies, and in which
a market for applications that
enhance EHR usability and patient
involvement can flourish”
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“Sound medicine needs to be based
on empirical evidence…
Aggregated deidentified
information could enable a wide
range of studies on such topics as
prevention strategies, frequency
complications in particular settings,
and the individuals response to
specific drugs based on genotype”
Thank You!
Questions?
Cal Collins
OpenClinica, LLC
ccollins@openclinica.com
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