Every year, thousands of newborns experience brain injury due to conditions such as hypoxic-ischemic encephalopathy (HIE), white matter injury, or intraventricular hemorrhage (IVH).
While therapeutic hypothermia (cooling) has improved outcomes for some with neonatal HIE, it remains the only approved treatment. For decades, progress in developing new therapies has been hindered by fragmented data, limited follow-up, and a lack of validated biomarkers that regulators can use to evaluate new drugs.
To change that reality, Hope for HIE has been a driving force collaborating with the International Neonatal Consortium (INC) under the Critical Path Institute (C-Path) to launch the Neonatal Brain Injury Collaborative (NBIC)—a global public-private partnership dedicated to accelerating drug development for babies with brain injury.
Read the full announcement from C-Path ›
The Neonatal Brain Injury Collaborative (NBIC) unites stakeholders from regulatory agencies, academia, industry, and advocacy to create the first structured pathway for qualifying biomarkers in neonatal brain injury, and working to develop standard for neonatal clinical trials for neonatal brain injury.
Currently, biomarkers such as MRI injury patterns, EEG background activity, and fluid markers (from blood or cerebrospinal fluid) are known to correlate with outcomes — but they are not yet validated for regulatory use. That distinction is critical.
Validated biomarkers must undergo rigorous, reproducible testing across datasets and populations to prove their reliability for prognosis, trial enrichment, and potential use as surrogate endpoints.
By bringing together previously siloed data and harmonizing standards across clinical and real-world settings, NBIC is paving the way for regulatory-grade evidence that can support FDA qualification programs and make HIE and other neonatal brain injury trials more feasible and faster to conduct.
“Together with Hope for HIE, INC is driving the collaborative science needed to bring new therapies to babies with brain injury.”
— Critical Path Institute
As the leading global organization for families affected by HIE, Hope for HIE plays a pivotal role in ensuring that research reflects what matters most to families: long-term outcomes, quality of life, and meaningful progress.
Betsy Pilon, Executive Director of Hope for HIE, represents the HIE community within INC’s Neonatal Brain Injury Collaborative, advocating for family-centered approaches in trial design, biomarker validation, and communication strategies.
Her leadership has been instrumental in ensuring that the lived experience of families is embedded in every stage of this scientific effort — from defining research priorities to interpreting outcomes.
In addition, HIE parent and volunteer, Michelle Stroe, has been serving as a patient advocacy representative on the Neonatal Adverse Events Severity Scale (NAESS) workgroup within INC.
In September 2025, Betsy Pilon brought the voice of HIE families to some of the most influential platforms shaping the future of pediatric and neonatal drug development.
At the Critical Path Institute’s 20th Anniversary Conference, Betsy joined leaders from the FDA, NIH, academia, and industry to discuss how authentic patient collaboration strengthens regulatory science.
She highlighted how the NBIC model—built on shared data, transparency, and trust—serves as a blueprint for other rare and complex pediatric conditions.
At the INC Day, many of Hope for HIE's medical advisory board members and industry partners also participated, with many collaborators meeting for the first time in-person.
📺 Watch the conference highlights: C-Path 2025 Conference Recording
Betsy was asked to represent patient advocacy in the FDA’s Interested Parties Meeting on the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) on September 15, 2025, presenting with fellow patient advocate, Deb Discenza of the PreemieWorld Foundation, INC Executive Director Kanwaljit Singh, MD, and NICHD Neonatal Research Network representative Augusto Schmidt, MD, PhD.
Her remarks underscored the urgent need for regulatory frameworks that support early-life data integration, longitudinal follow-up, and equitable access to innovation for newborns and infants.
By highlighting the challenges of conducting trials in fragile populations, Betsy emphasized how partnerships like INC and Hope for HIE can reduce barriers, improve communication, and ensure ethical and inclusive study design.
Rounding out September's focus on clinical trials, Betsy joined the IQ Consortium for a multidisciplinary discussion on how advocacy-regulatory collaboration can close the gap between unmet medical needs and drug development for children.
She spoke about the realities families face when navigating uncertain prognoses and limited treatment options — and how transparent, empathetic communication from researchers and clinicians builds trust that drives engagement.
Neonatal brain injuries remain a leading cause of death and lifelong disability. Yet for most affected families, there are still no approved drugs beyond cooling therapy. The NBIC’s collaborative science aims to change that.
By establishing a unified framework for data sharing and regulatory validation, NBIC will:
Future phases of the NBIC will expand to include biochemical and physiological biomarkers, integrate international data sources, and strengthen collaboration among academic, regulatory, and advocacy partners.
Hope for HIE remains deeply committed to ensuring that families are not just participants but partners in this global effort — shaping science that reflects their realities, values, and hopes for the future.
As this work continues, the message is clear:
When science and families work together, progress becomes possible.
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