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CURE OM Patient Registry Update


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Presentation by Chad Kimbler and Carla Tressell. Presented at the 2018 Eyes on a Cure: Patient & Caregiver Symposium, hosted by the Melanoma Research Foundation's CURE OM initiative. 

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CURE OM Patient Registry Update

  1. 1. CURE OM Patient-powered Registry Presented by Chad Kimbler & Carla Tressell
  2. 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. 1
  3. 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. 2
  4. 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 3
  5. 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 4
  6. 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” 5
  7. 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. 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 7
  9. 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 8
  10. 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 9
  11. 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 10
  12. 12. Example – Registering with Patient Registry 11
  13. 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 12
  14. 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 13
  15. 15. Example – Patient Registry 14
  16. 16. Example – Patient Registry Data Results See Study Data Results Example Registry Data – Demographics: 15
  17. 17. Example – Patient Registry Data Results Example Registry Data – Diagnosis: 16
  18. 18. Example – Patient Registry Data Results Example Registry Data – Risks: 17
  19. 19. Example – Patient Registry Data Results Example Registry Data – Treatments: 18
  20. 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.” 1 9