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

Contact Us – and Thanks!!

www.pcori.orginfo@pcori.org

Q&A

Kim McClearyManaging Director,

FasterCuresMODERATOR

Joe SelbyExecutive

Director, Patient-Centered Outcomes Research Institute

Kyle BrownCEO and Founder, PatientCrossroads

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