Findings
The FDA’s increasing commitment to patient-focused drug development (PFDD) and patient engagement in translational research presents a significant opportunity to improve the clinical trials enterprise and enhance participation by patient groups . Patient groups can play important roles in improving the entire therapeutic development enterprise, from study endpoint selection that reflects outcomes meaningful to patients, to recruitment and retention in clinical trials, and more effective postmarketing safety . However, there is a lack of clarity about how, when, and by whom patients or patient groups should be engaged during the therapy development process, and which patients or patient groups should be engaged . Metrics by which the value of such engagement, in terms of regulatory and market success, might be measured are also lacking .

Recommendations
PFDD and patient engagement in research should be considered an effort to extend the benefits of incorporating patient insight and experiences, as well as desires and preferences, from bench to bedside and back . The therapeutic development process should meaningfully engage patients throughout, though specific guidance on implementation methods is needed .

Regulatory Considerations
The paper does not provide specific regulatory considerations or recommendations. The focus remains on identifying the opportunity and gaps in current patient engagement approaches rather than detailing regulatory pathways or compliance requirements.