The European Medicines Agency (EMA) hosted a workshop on 29 April 2026 to discuss regulatory expectations for clinical trial endpoints in Geographic Atrophy (GA), a debilitating eye condition with high unmet medical need. The workshop brought together regulators, patients, healthcare professionals, academics, and industry stakeholders to identify clinically relevant endpoints that demonstrate patient benefit, moving beyond reliance on imaging-based surrogate endpoints.
The workshop, organized by the European Medicines Regulatory Network (EMRN), focused on potential primary functional endpoints such as microperimetry, reading speed, visual acuity, and contrast sensitivity. Sessions also addressed imaging endpoints and their validation as surrogates, patient-reported outcomes (PROs) and quality of life measures, and emerging endpoints including AI-based image analysis. The agenda reflects EMA's proactive role in facilitating drug development for GA by aligning stakeholder perspectives on acceptable endpoints for marketing authorization.
Policy orientations and trade-offs
The workshop signals a regulatory push toward functional endpoints that directly measure patient benefit, which may increase the complexity and cost of clinical trials compared to relying on imaging surrogates. However, this approach could lead to more meaningful evidence of efficacy, potentially accelerating patient access to treatments that truly improve vision-related quality of life. The emphasis on PROs and AI-based analysis introduces both opportunities and challenges: PROs capture patient perspectives but can be subjective, while AI offers objective quantification but requires validation and regulatory acceptance.
Impact on stakeholders
- Patients: Positive impact if functional endpoints lead to approval of treatments that improve visual function and quality of life. However, delays in endpoint validation may slow access to new therapies.
- Pharmaceutical industry: Moderate negative impact due to increased trial costs and longer development timelines to meet new endpoint requirements. However, clearer regulatory guidance reduces uncertainty.
- Regulators (EMA and national authorities): Positive impact as the workshop helps harmonize expectations across the network, improving consistency in evaluation.
- Healthcare professionals: Positive impact as validated functional endpoints provide better tools to assess treatment benefit in clinical practice.
Expected institutional follow-up
The workshop is expected to inform future EMA guidelines on GA clinical trial design. Further research on endpoint validation, particularly for AI-based and functional measures, will likely be encouraged through regulatory science initiatives and collaboration with academic consortia.