Background: Age-related macular degeneration (AMD) is the leading cause of blindness among Americans over 40 years of age and irreversible vision loss in those over 65 years of age in the United States. While new agents have seen success in clinical trials, real-life evidence suggests that there is a wide disparity between real-life outcomes and the benefits seen during the clinical trials, especially over the long term. One proposed cause of this is that current clinical trial design for AMD focuses on endpoints that are easily quantifiable to show therapy success, such as improvements in visual acuity, contrast sensitivity, and color vision. While these endpoints may show improvement, they do not always correlate to outcomes that are personally important to the patients, such as reading speed, recognition of family and friends, mental well-being, and the independence of being able to continue driving and working.
Solution: The Angiogenesis Foundation proposes to convene a national summit of patients who have experienced vision loss from AMD, AMD patient caregivers, vision advocacy leaders, vision researchers and clinicians, industry leaders, and patient-centered outcomes research experts to develop an action plan for integrating patient perspectives and values into future research projects and clinical trial design, educating and empowering patients and their families to bring their voices to AMD advocacy, and improving usage of current patient-reported outcomes measurement (PROM) tools. The foundation will recruit motivated representatives from the patient, caregiver, advocacy, and research communities as well as experts on patient-centered outcomes research to share their perspectives and suggestions with stakeholders from the other groups.
The summit participants will:
- Develop strategies to integrate patient-centered outcomes into future AMD research.
- Identify important patient-reported outcomes measures to be considered for future research endpoints.
- Suggest questions to improve existing patient-reported outcomes measurement tools.
A long-term objective of this summit will be the formation of a community of patients, caregivers, researchers, and clinicians who are capable of discussing, disseminating, and engaging in PCOR in AMD care while ensuring patient needs and values are represented in healthcare decision making. This community will be champions for PCOR and be a voice for the consideration of patient needs and values in research and treatment decisions.
Output: The Angiogenesis Foundation will use the outcomes of the summit to develop an authoritative white paper report. A professional medical writer will record and transcribe the perspectives of multiple stakeholders to generate an initial draft of the white paper report, which will then be circulated among summit participants who will serve as co-authors to further develop the report.
Patient and Stakeholder Engagement Plan: Patients, family members, caregivers, vision researchers, patient advocates, and vision advocacy leaders will participate in every step of the summit development and execution. Their initial involvement will begin with a steering committee that will recommend participants and provide guidance on the draft agenda. The steering committee will include broad stakeholder representation, each contributing to the shaping of the program and the dissemination of the summit proceedings through a white paper report.