1. CURE OM Patient-powered Registry
Presented by Chad Kimbler & Carla Tressell

2. The Importance of a Registry
National Institutes of Health – NIH Research Trials and You
Why are registries needed?
Registries can provide health care professionals and researchers with
first-hand information about people with certain conditions, both
individually and as a group, and over time, to increase our
understanding of that condition. Some registries collect information
that can be used to track trends about the number of people with
diseases, treatments, and more. Other registries invite people to sign
up to be contacted about participating in clinical research. These ask
very basic questions about health history that would help determine
whether someone is possibly eligible to join a research study.
Participation in a registry is likely to increase what we know about a
specific condition, help health care professionals improve treatment,
and allow researchers to design better studies on a particular
condition, including development and testing of new treatments.
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3. The Importance of a Registry
EURORDIS-NORD-CORD – Joint Declaration of Rare
Disease Patient Registries
EURORDIS, NORD and CORD, along with the patients they
represent in Europe and in North America, recognize that Rare
Disease Patient Registries constitute key instruments for increasing
knowledge on rare diseases, supporting fundamental clinical and
epidemiological research, and post-marketing surveillance of orphan
drugs and treatments used off-label. Furthermore, and of great
importance for patients and their families, they can be instrumental
in supporting health and social services planning. Rare Disease
Patient Registries are powerful, cost-effective instruments to
improve the overall quality of care, quality of life and survival of
patients.
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4. What is a Registry?
“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 a predetermined scientific, clinical, or policy
purpose(s).”
Registries for Evaluating Patient Outcomes: A User’s Guide – AHRQ
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5. What is a Patient-powered Registry?
To focus research more directly on patient and family
member needs, patient and family advocates and
organizations have created and operated “patient-
powered” patient registries and research networks since as
early as 1995. These registries and networks are
distinguished from researcher-generated registries in that
the registry (or network) and the research it yields is
managed by patients and family members themselves,
often through a disease advocacy organization or a
network of organizations that receives advice and input
from a scientific board of advisors.
AHRQ: Engaging Patients in Information Sharing and Data Collection: The Role of Patient-Powered
Registries and Research Networks
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6. CURE OM Registry Objectives
Top priority for patient community surveyed: Increase likelihood of
development of effective treatments
Capture and record global cases of ocular melanoma in a standardized
patient-reported online registry to help understand the disease demographics
and the patient experience from its onset to its end; including genetics, other
risk factors, symptoms, diagnosis, treatments, outcomes, patient preferences
and quality of life.
To enhance the understanding of the variability, progression, and natural
history of uveal melanoma, with the ultimate goals of better guiding and
assessing therapeutic intervention, and providing the data upon which to base
patient-centered recommendations on patient care to the medical community
that will improve the outcomes for patients affected by this disease around the
world.
Increase collaborative efforts between patients, clinicians, and researchers
that could lead to discoveries that will improve the outcomes for patients
affected by this disease.
Feedback from FDA: “important to have ‘patient-focused information on the
population that it will enroll. And look more broadly at patient-related issues or patient
preference”
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7. CURE OM Registry
Online research study sponsored by MRF/CURE OM
Led by patient advocacy organization
Powered by patient input and patient-reported data
Has a Principal Investigator (PI) to oversee the study
Developed in collaboration with OM experts
Has a research protocol, structured data set, consent form and
communication material
Registry study documents are all approved by an Institutional Review
Board (IRB)
Collaborates with all research sectors for the best outcomes
Strategically planned for achieving objectives, sharing results,
advancing research & long-term success6

8. CURE OM Registry Plan - Progress
Steps completed so far:
1. Commitment to fund & sponsor a patient powered
registry
2. Raised initial funds for initiative
3. Building resources & collaborations, including
international parties
4. Formed multi-disciplinary Registry Working Group (4
ocular oncologists, 2 medical oncologists, 2 research
nurses, 3 patients, 1 social worker, and 3 family
members – 1 from Huntersville Cluster)
5. Assessed Registry Platform providers: compared non-
profit, private & academic providers
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9. CURE OM Registry Plan - Progress
Steps completed so far:
6. Conducted Patient Community questionnaire for
input to direct initiative
7. Collaboration underway to partner with academic
multi-center, international researcher-reported
registry study.
8. Working Group started development of registry
protocol & data set
9. Outlined potential collaborations with industry and
initiated conversations with corporate sponsors,
including assessing responses from a survey that
was sent to them
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10. CURE OM Registry Plan – Next Steps
Next steps:
1. Working Group complete IRB approved Registry protocol
2. Working Group complete IRB approved Registry data set
3. Select registry platform provider & agree specifics for patient
registry & collaboration with academic research-reported registry
4. Secure sustainable funding
5. Develop an online educational seminar for the patient community
6. Develop a patient engagement and retention plan
7. Develop IRB approved communication material
8. Data Management Plan & Data Sharing Policy
9. Launch Registry & initiate day to day management
10. Share results of data with patient community and collaborators
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11. Participating in a Registry Study
Registry Study Process for Participants:
Step 1: Learn about the CURE OM Registry
Step 2: Register & Create an registry account
Step 3: Consent to participate in the study
Step 4: Take the study surveys/questionnaires
Step 5: See data results
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12. Example – Registering with Patient Registry
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13. Consenting with a Patient Registry
Consent to Participate in Study (online consent -
required before taking surveys):
Consent from patient or legally authorized
representative
Consent form based on registry protocol
Institutional review board (IRB) approved
Summary of participation, data safety and security &
conditions of informed consent
Registered participant ticks box/s of consent
(understanding of participation) to take study surveys
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14. Example – Patient Registry Data Set
Registry Study Surveys – Example of Data Set (Surveys) NIH
RaDaR CDEs (common data elements)
Organized into categories that include required and optional elements:
•Current contact information
•Sociodemographic information
•Diagnosis
•Family history
•Birth and reproductive history
•Patient-reported outcomes
•Medications, devices and health services
•Clinical research participation and biospecimen donation
•Communication and preferences
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15. Example – Patient Registry
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16. Example – Patient Registry Data Results
See Study Data Results
Example Registry Data – Demographics:
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17. Example – Patient Registry Data Results
Example Registry Data – Diagnosis:
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18. Example – Patient Registry Data Results
Example Registry Data – Risks:
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19. Example – Patient Registry Data Results
Example Registry Data – Treatments:
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20. Agency for Healthcare Research and Quality – Community Forum White
Paper
“Patient-powered registries (PPRs) offer new
directions for patient-centered outcomes research,
and contribute to translational science in important
ways. Experts agree that these registries are
transforming patient/caregiver support and advocacy
groups into research organizations. They also provide
patients and family members another way to become
engaged in research beyond the role of advisor or
informant to researcher-generated studies.”
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