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CURE OM Patient Registry Update - 2019 CURE OM Symposium

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Dr. Richard Carvajal gives an update on the development of the CURE OM Patient Reported Ocular Melanoma Registry.

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CURE OM Patient Registry Update - 2019 CURE OM Symposium

  1. 1. CURE OM Patient-Powered Registry: Capturing Patient Reported Real World Data to Improve Ocular Melanoma Outcomes
  2. 2. Messages from our Patients and Caregivers… Support Hope Advocacy Research Partnership Collaboration Community Education
  3. 3. CURE OM Patient Powered Registry Patient Registry • Organized system that uses observational study methods to collect uniform data to evaluate specified outcomes for a defined population that serves a predetermined purpose Overall Objective: To build a patient developed and led registry of prospectively collected real world data that can be utilized by all stakeholders to generate the real world evidence necessary to improve outcomes for those affected by ocular (uveal and conjunctival) melanoma Real World Evidence • Evidence derived from real world data through the application of research methodology Stakeholders • Patients and family members • Clinicians • Researchers • Pharmaceutical companies • Regulatory agencies • Payers
  4. 4. Swift et al. Clin Transl Sci (2018) 11, 450-460. • Data relating to patient health status and/or the delivery of heath care • Sources: EMR, admin and claims data, home monitoring devices, wearable technologies, patient registries, clinical trials Real World Data
  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 since as early as 1995. These registries are distinguished from researcher-generated registries in that the registry and the research it yields is managed by patients and family members themselves, often through a disease advocacy organization 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 5
  6. 6. Emerging Emphasis Upon RWE Dec 2018 March 2019
  7. 7. Benefits of a Patient-Reported Registry  Share data with all parties – patients, clinicians, researchers, providers, partner organizations and industry  Data obtained directly from the patient or his/her legally authorized representative  Data entered from anywhere in the world  Brings together the patient population – platform for uniting patients around the world  Enhances patient education and understanding of disease  Acts as a vehicle for the patient community’s voice – patients, family members and caregivers become an integral voice in research  Patients can relate, compare and learn from other patients’ journeys  Helps patients, their families and caregivers further understand their condition so they can take a more active role in their care and advocacy Patient Benefits
  8. 8. Benefits of a Patient-Reported Registry  Share data with all parties – patients, clinicians, researchers, providers, partner organizations and industry  Real world data  Act as a single source of information that records the patient’s full journey with OM  Supports fundamental epidemiological research  Documents geographic practice variations  Document patient preferences  Supports health and social services planning  Post-marketing surveillance  Demonstrates value Benefits to OM Research
  9. 9. Real World Evidence & Drug Reimbursement: The 4th Hurdle Quality Safety Efficacy Value For regulatory approval (FDA, EMA) For reimbursement (NICE, HTA)
  10. 10. • The availability of real world contemporary outcomes data would aid in the development of more robust biostatistical trial designs • Optimal trial methodologies for demonstration of efficacy and safety in rare tumors may differ from those used in more common conditions. • Real world contemporary outcomes data may be used to support the use of novel study designs that: – Are acceptable to the regulatory authorities; – Minimize the number of individuals treated on control arms with limited efficacy; and, – Permit those randomized to a control arm earlier access to the investigational therapy. Real World Evidence & Clinical Trial Development
  11. 11. IMCgp100 Program Registration Strategy Accelerated approval strategy: Single arm trial approval strategy is based on either ORR or OS with RWE as comparator IMCgp100-102: Second or third line in the metastatic setting IMCgp100 at RP2D from Ph I N ~ approx 150 Interim Analysis Final Analysis Real World Evidence (ORR, OS via one of three platforms: Meta- analysis, global patient registry or Flatiron derived comparator) Opportunities for approval IMCgp100-202: First line metastatic setting R Investigator Choice (dacarbazine, pembrolizumab or ipilimumab) N = approx 109 Sample size Interim Analysis Final Analysis re-estimation Primary endpoint: Median OS Traditional approval strategy: Randomized study of IMCgp100 v. Investigator choice with OS as endpoint IMCgp100 at RP2D from Ph I N = approx 218 Opportunities for approval
  12. 12. Timeline of Registry Activities March 2016 CureOM Unite Campaign Launch Hired Registry Expert Consultant Formed Interdisciplinary Steering Committee March 2017 First Steering Committee Working Group Meeting Engaged and Surveyed Stakeholders; Identified Funding Sources Formalized Registry Objectives and Protocol Vetted Multiple Technology Platforms and Innovation Models Nov 2018 Formally Presented Proposal to MRF SAC Progress towards Seed Funding March 2019 Second Steering Committee Working Group Meeting
  13. 13. Interdisciplinary Steering Committee Patient/Family Advocates Linda O’Brien Chad Kimbler, MA Carla Tressel Shana Ratner, MD Ocular Oncologists Bertil Damato, MD, PhD Dan Gombos, MD Ivana Kim, MD Miguel Materin, MD Medical Oncologists Richard Carvajal, MD Marlana Orloff, MD Nursing Renee Zalinsky, RN Barbara Perez, BSN, RN Social Work Alison Petok, MSW, MPH Melanoma Research Foundation CureOM Sara Selig, MD, MPH Jacqueline Kraska Biostatistics Codruta Chiuzan, PhD Patient Reported Outcomes Ethan Basch, MD, MSc Epidemiology Ashley Schoenfisch, PhD
  14. 14. Overall Objectives 1. To develop and utilize a standardized global patient-reported online registry to provide data regarding the incidence, prevalence, demographics, risk factors, genetics, natural history, treatments and patient experience (patient preferences and quality of life) from diagnosis through the lifetime of OM. 2. To provide patient centered data to enhance the care of individuals with OM. 3. To facilitate collaborative efforts between patients, clinicians and researchers to improve the outcomes for patients affected by this disease. 4. To develop a virtual OM biospecimen repository (future goal).
  15. 15. Eligibility and Enrollment Targets Recruitment: Start in the United States Over time, achieve global coverage, starting with international collaborators in UK, Canada, Europe and Australia Enrollment target of 400 recruits annually (1,200 over the first 3 years) Inclusion Criteria: Diagnosis of OM Ability to provide informed consent Exclusion Criteria: No conclusive diagnosis of OM
  16. 16. CURE OM Patient Registry Experience Inform and Engage Register Consent Complete Profile Knowledge
  17. 17. Recent Accomplishments Added three new disciplines to interdisciplinary registry steering committee: Biostatistics, Epidemiology, and Patient-Reported Outcomes experts. Secured transformational funding partnership to support support launch and multiyear maintenance. Opened new partnership opportunities including funding partners as well as international participants. Presented and engaged stakeholders and partners at three international scientific meetings. – Including obtaining feedback from a wide range of stakeholders as well as an oral presentation at the 2019 International Society of Ocular Oncology meeting.
  18. 18. Registry of registries… Academic Natural History Registry
  19. 19. • Demographics • Family history • Social history • Con meds • Diagnostic procedures/results • Therapeutic procedures/results • Pathology results • Molecular results • Laboratory results • Imaging results • Surveillance strategies • Treatment/Toxicity • Biospecimen repository • Symptoms • Quality of life • Diet/supplements • Lifestyle/Fitness/Stress • Environmental exposures • Occupational exposures • Impact upon daily life • Perception of disease • Perception of treatments • Treatment effectiveness • PRO-CTCAE • Economic impact • Ratings of care Academic Registry Patient Registry Relationship Between Registry Efforts
  20. 20. Next Steps 1. Confirm technology partner 2. Finalize data dictionary 3. Build platform 4. Obtain IRB approval 5. Continue outreach, education and fundraising efforts 6. Launch!!!
  21. 21. Thank you

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