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Building Research-Ready Communities: A Closer Look at Patient Registries A FasterCures Webinar June 9, 2015
Transcript of Building Research-Ready Communities: A Closer Look at ... · Advocacy Groups –credit & funding...
Building Research-Ready Communities: A Closer Look at
Patient Registries
A FasterCures Webinar
June 9, 2015
Speakers
Kim McClearyManaging Director,
FasterCuresMODERATOR
Joe SelbyExecutive Director, Patient-Centered
Outcomes Research Institute
Kyle BrownCEO and Founder, PatientCrossroads
Kim McCleary
Managing DirectorFasterCures
MODERATOR
FasterCures is an “action tank” driven by a singular goal –to save lives by speeding up and improving the medical research system.
A center of the Milken Institute, we are a nonprofit and nonpartisan organization that collaborates with all the sectors of the medical research and development ecosystem.
WebinarsPurpose• Real-time sharing of
ideas, best practices, trends, and lessons learned
• Amplifies meaningful solutions, productive tools, and encourages action needed to spur medical progress
Who’s logged on?Nearly 600
registrants from:
2015 topics• R&D policy• Science of
patient input• Venture
philanthropy
Nonprofit40%
Healthcare/Industry30%
Academia/Research16%
Government/Public Sector
5%
Other9%
Patient Registries & Research Platforms
http://fastercures.tumblr.com/post/115947743289/navigating-the-landscape-of-patient-registries
http://fastercures.tumblr.com/post/115947743289/navigating-the-landscape-of-patient-registries
http://fastercures.tumblr.com/post/115947743289/navigating-the-landscape-of-patient-registries
Kyle Brown
CEO and Founder, PatientCrossroads
Kyle BrownCEO
CONNECT Registry Programwww.patientcrossroads.com
NIH National Down Syndrome Registry
NIH National Pregnancy Registry (in process)
Patient-Centered Outcomes Research Institute (PCORI) grant participant
RD-CONNECT European Union FP7 research grant partner
ClinGen – GenomeConnect
70+ Advocacy partnered registries spanning 300+ diseases
Pharmaceutical companies & biotechs
PatientCrossroads Registry Programs
www.patientcrossroads.comPatientCrossroadsTM
The world has more than 7,000 diseases.
How can researchers decide where to focus — without knowing how many people are affected, where they live, and the symptoms they experience?
www.patientcrossroads.comPatientCrossroadsTM
Current 32
In Process
12
Planned 10
Duchenne Muscular Dystrophy Registries
Patient registries show promise … but are fragmented
• Different types of organizations • Different consent
www.patientcrossroads.comPatientCrossroadsTM
• Different collection instruments • Different access policies• Different languages
Why so much fragmentation?
Researchers – publications & funding
Advocacy Groups – credit & funding
Pharma & Biotech – regulatory & competition
www.patientcrossroads.comPatientCrossroadsTM
# Re
cords
Data quality highlow
low
high
Patient opt‐in
Physician entered
Product registry
Patient opt‐in, verified
$ Co
sthigh
low
www.patientcrossroads.comPatientCrossroadsTM
Types of Patient Registries
www.patientcrossroads.comPatientCrossroadsTM
CONNECT Registry Program
Outreach Partners
Standardized Data Collection Medical Questionnaires
Confirmatory Testing
EHR Integration
Mobile Apps
Data Quality& Curation
Reporting &Analysis
Clinical TrialRecruitment
Long TermFollowup
Study Data Collection
Joe Selby
Executive Director, Patient-Centered Outcomes Research
Institute
A Closer Look at Patient Registries:the PCORI Perspective
Joe Selby, MD, MPHExecutive DirectorPatient-Centered Outcomes Research InstituteFasterCures Webinar – June 9, 2015
• PCORI‐funded research seeks to help patients and those who care for them maker better‐informed health decisions
• PCORI involves patients in formulating and prioritizing research questions it will fund
• PCORnet, the National Patient‐Centered Clinical Research Network, established to provide a more efficient platform for patient‐focused outcomes research
• Registries play a substantial role in PCORI’s overall portfolio and in PCORnet
Setting the Stage: PCORI & PCORnet
• PCOR Methods Research Program (40%)– Using registries for modeling or simulations– Registries include veterans living with HIV, pediatric IBD, back pain, stroke, cancer
– Example: how to improve use of patient registries to conduct CER• Assessing Diagnosis, Prevention & Treatment Options (40%)
– Use current disease/procedure‐specific registries (cancer, stroke, IBD) or hospital system registries and linking registries with EHR or Medicare/Medicaid data
– Some projects developing own registries by merging existing data sets– Some hospital registries used in recruitment or to follow study participants– One registry being used for risk prediction in chronic disease
• Addressing Disparities, Improving Health Systems (20%)
PCORI’s Portfolio: 28+ Studies Using Registries
• 29 networks involving 155 different organizations, 3,000 collaborators– 18 Patient‐Powered Research Networks (PPRNs) led by patient communities
– 11 Clinical Data Research Networks (CDRNs) based in healthcare systems
• Registries are the backbone of the PPRNs and span a range of ~100 different conditions
• Half of 18 PPRNs focus on rare conditions
• Millions of patients cared for in participating systems
• PCORI is investing $275M to build this national resource
PCORnet: Added Opportunities to Use Registries
9 PPRNs Focus on Rare ConditionsPPRN Lead Organization Condition Population
Size
Duke University Juvenile Rheumatic Disease 9,000
ALD Connect, Inc. Adrenoleukodystrophy 3,000
Phelan‐McDermid Syndrome Foundation
Phelan‐McDermid Syndrome 737
Immune Deficiency Foundation
Primary Immunodeficiency Diseases 1,250
University of Pennsylvania
Vasculitis 500
Parent Project Muscular Dystrophy
Duchenne and Becker muscular dystrophy 4,000
Arbor Research Collaborative for Health
Primary Nephrotic Syndrome (Focal Segmental Glomerulosclerosis, Minimal Change Disease, Membranous Nephropathy, Multiple Sclerosis)
1,250
Epilepsy Foundation Aicardi Syndrome, Lennox‐Gastaut Syndrome, Phelan‐McDermid Syndrome, Hypothalamic Hamartoma, Dravet Syndrome, Tuberous Sclerosis
1,500
Genetic Alliance, Inc. 29 different genetic disorders, ranging from pseudoxanthoma elasticum to hereditary breast cancer
50‐50,000
Organization Condition PotentialParticipants
University of California, San Francisco Cardiovascular Health 100,000Cincinnati Children's Hospital Medical Center
Pediatric Crohn's Disease and Ulcerative Colitis 15,000
Crohn’s & Colitis Foundation of America
Inflammatory Bowel Disease (Crohn’s disease and ulcerative colitis)
30,000
Global Healthy Living Foundation Arthritis (rheumatoid arthritis, spondyloarthritis), musculoskeletal disorders (osteoporosis), inflammatory conditions (psoriasis)
50,000
American Sleep Apnea Association Sleep Apnea 50,000COPD Foundation COPD 50,000Accelerated Cure Project for Multiple Sclerosis
Multiple Sclerosis 20,000
University of South Florida Hereditary Breast and Ovarian Cancer 17,000Massachusetts General Hospital Major Depressive Disorder, Bipolar Disorder 50,000
9 PPRNs Focus on Common Conditions
• Building/enhancing a registry to enable research and deepen engagement with patient constituencies– Patients can propose research ideas– Researchers can interact with patients via the registry
• Test acceptability of and approaches to linkage between registry and EHR data
• Help patients connect with one another• Help patients understand the context and the bigger picture contribution they can make to research
• Examples from two PPRNs illustrate this well…
Role of Registries in PCORnet
Multiple Sclerosis Patient PortalNoteworthy elements:
Informed Consent = Prominent Options for other ways to be a part
Vasculitis Patient Portal
Noteworthy elements:Clearly stated expectations of participants
Offer of additional engagement opportunities
• Well‐designed technology• Recruitment, encouragement, and gratitude for participation• Collaborative relationships with researchers• Partnerships with a broad range of stakeholders
Patient-Powered Registries Working Well*
* REFERENCE: Workman TA. Engaging Patients in Information Sharing and Data Collection: The Role of Patient‐Powered Registries and Research Networks. AHRQ Community Forum White Paper. AHRQ Publication No. 13‐EHC124‐EF. Rockville, MD: Agency for Healthcare Research and Quality; September 2013.
PPRNs are collaborating with each another and CDRNS on research ideas, technology innovations, and enrollment approaches for registries
Creating a collaborative community is an essential PCORnet hallmark
PPRNs are collaborating with each another and CDRNS on research ideas, technology innovations, and enrollment approaches for registries
Creating a collaborative community is an essential PCORnet hallmark
• Well‐designed technology• Recruitment, encouragement, and gratitude for participation• Collaborative relationships with researchers• Partnerships with a broad range of stakeholders
PCORnet
Overlapping registries,e.g., obesity and asthma
A very “deep” ordetailed registry
Finding additionalRegistry members
Comparison studies
EMR and claims data on large, general populations
Potential Linkage of Patient Registries with Large Population Databases
• What are the intended uses of the registry:
–Natural history studies, time trend studies, practice patterns –then a representative registry is needed
– Intervention studies or patient‐reported outcomes – then a volunteer registry may be preferable if it’s not so important that patients are highly representative.
• Will you consent registry participants? What consents will you obtain? Biospecimen use? Repeated uses of data? Linkage of data to other sources such as insurance claims?
• How will you obtain EMR data? Claims data? Patient‐reported data? Biospecimens?
• How will you engage researchers, clinicians, patients?
Key Questions in Launching a Registry
Q&A
Kim McClearyManaging Director,
FasterCuresMODERATOR
Joe SelbyExecutive
Director, Patient-Centered Outcomes Research Institute
Kyle BrownCEO and Founder, PatientCrossroads
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