The Food and Drug Administration (FDA), in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (MCERSI), hosted the Advancing the Development of Pediatric Therapeutics (ADEPT) 10: Addressing Challenges in Neonatal Product Development – Leveraging Rare Disease Frameworks in Washington DC last week. This public workshop brought together regulators, researchers, clinicians, industry leaders, and patient advocates to examine how lessons learned in rare disease drug development can be applied to neonatal therapeutics—one of the most complex and underserved areas in medicine.

I was honored to be invited to present on a panel session as the President of the NICU Parent Network and author of several books and research papers in the neonatal advocacy space.

A central theme of the ADEPT 10 discussion was the shared challenge between neonatal and rare disease product development: small patient populations, ethical constraints, limited clinical trial endpoints, and high vulnerability of patients. The workshop explored how rare disease frameworks—such as flexible trial designs, adaptive endpoints, and meaningful patient-reported outcomes—can help accelerate safe and effective therapies for neonates while maintaining rigorous regulatory standards.

As a NICU parent author, speaker and advocate, I was invited to address a critical question:

How do we protect and empower parents in the design of studies for therapeutics targeting rare diseases or neonatal intensive care populations?

My response emphasized that families must not be treated as passive participants, but as essential partners in research design. Studies should prioritize outcomes that parents can observe, understand, and connect to their child’s daily life—such as improved breathing with less support, better feeding tolerance and growth, reduced length of hospital stay, and progress toward developmental milestones appropriate to corrected age.

Equally important is a focus on reducing harm and treatment burden. For families navigating the NICU, success is not defined solely by survival, but by fewer invasive procedures, reduced exposure to pain and sedation, lower rates of adverse events, and shorter dependence on mechanical ventilation or IV therapies. These outcomes matter deeply to parents and should be recognized as legitimate, measurable indicators of therapeutic benefit.

Parents must also have a direct voice in the data itself. Validated parent-reported outcomes—such as symptom improvement, parental stress and anxiety levels, decision regret, and clarity of communication—offer critical insight into whether a study truly serves families. When parent perspectives are systematically collected and valued as data, studies become more ethical, transparent, and aligned with real-world impact.

Finally, neonatal studies must look beyond discharge or short-term survival. Families live with the long-term consequences of early medical decisions, making outcomes such as neurodevelopmental progress, ongoing technology or therapy needs, functional independence, and overall quality of life essential endpoints. ADEPT 10 reinforced that when rare disease frameworks are thoughtfully applied—and when parents are meaningfully included—we can advance neonatal therapeutics in ways that are not only scientifically sound, but also compassionate, ethical, and truly patient- and family-centered.

It was a brief trip in and out of Washington, DC in under 24 hours, but a meaningful one. I could not stay for the second day due to work commitments, but I was told it was as powerful as Day 1.

What continues to ground and guide me—as a mother, an educator, and an advocate—is the responsibility I feel to share my lived experiences with those shaping policies that affect children and families. These moments matter, because decisions made in these rooms ripple far beyond them. I carry many roles, and together they make me whole. I am a mother, an educator, and an advocate, and I remain deeply grateful for every opportunity to serve in these ways for as long as I am able.

You can learn more about the ADEPT event by clicking here.

You can learn more about my work as President of the NICU Parent Network by clicking here.

Thank you for reading and please connect with me if I can help your advocacy efforts.