Not long after experiencing a rare disease in my immediate family for the first time, I found myself wondering—what more could I do?
At the time (2009), I didn’t know the term “rare disease” or that millions of people around the world were facing the same heartbreak and uncertainty that I felt when my father was diagnosed with Glioblastoma (GBM). I was 27 and in the early years of my career working in drug discovery and development. I had yet to realize that an entire industry sector was dedicated to helping patients like my dad. My grandfather passed away from GBM in 2019. My best friend in 2023 by the same disease.
In 2013, I took my first role in orphan drug regulations—and everything changed. In 2016, I co-founded RareMoon Consulting. As chance would have it, my co-founder had also lost two friends to GBM and shared the same focus: to make a difference. We have since dedicated our careers and professional focus to using our experience and understanding of orphan drug regulations to navigate the unique challenges of rare disease drug development.
Every day, we get to work alongside a team dedicated their entire careers to fighting for patients like my dad—scientists, doctors, and regulatory specialists who have been with us since day one. They have spent their entire careers in the rare disease space, guiding programs from concept to clinic and ultimately into the hands of patients. I am proud to collaborate with these individuals who write and operate with excellence, who navigate complex endpoint and manufacturing challenges, help justify a path forward when non-clinical models are unavailable, and ultimately work to overcome these common challenges many of our clients face.
I am humbled to be part of such a passionate group of people, a team of partners in the fight against rare diseases.
Rare diseases aren’t just part of what we do—they are why we exist.
The Challenges of Rare Disease Drug Development
The stakes are high, the timelines are critical, and the hurdles are unique.
- Small Patient Populations – Traditional clinical trial models don’t apply. Data is limited, and recruitment can be challenging.
- Limited Regulatory Precedent – With so few approved therapies for rare diseases, companies must navigate a path with little historical data or information.
- Complex Science – Many rare disease treatments involve gene therapies, cell therapies, or precision medicine approaches requiring subject-matter expertise.
- Uncertain Regulatory Expectations – The FDA, EMA, and other health agencies recognize the urgency of rare disease therapies, but the evolving nature of regulations requires strategic, early engagement.
For biotech teams developing orphan drugs, these challenges can feel overwhelming. As a team dedicated to supporting orphan drugs and advanced therapies, we regularly engage with regulators. Our extensive experience and deep expertise enable us to anticipate challenges, navigate complexities, and operate within—and occasionally around—the regulatory framework, bringing life-changing therapies closer to patients.
Our Commitment
Our mission is clear: To guide orphan drug and advanced therapy programs through the regulatory framework, driving them toward pivotal milestones that support biotech innovation and ultimately benefit patients.
Our commitment is unwavering: To push forward—alongside regulators, biotech innovators, and patient advocates—because we believe in the mission of bringing novel therapies to those who need them most. RareMoon’s team isn’t just part of the rare disease ecosystem; we are deeply embedded in the journey, working relentlessly to overcome obstacles and deliver solutions that make a difference.
On Rare Disease Day and every day, we remain committed to making a lasting impact in this space—because when we work together, we don’t just advance science, we change lives.
Sincerely,
Sabrina
