Changing the Business Model for Pediatric Rare Diseases

By: Mark Veich

‍

Every parent’s greatest hope is to see their child grow up healthy, happy, and thriving. But for families of children with rare diseases and cancers, that hope can feel like it’s slipping away. Each year, nearly 400,000 children worldwide are diagnosed with cancer, yet only a fraction of the treatments available are designed specifically for them. The reality is sobering: 95% of rare disease slack FDA-approved therapies and pediatric cancers often take a backseat in the world of drug development.

Why? Because for-profit drug development operates on a simple formula: pursue treatments that have the greatest market potential. For diseases that affect small populations—like rare pediatric cancers—pharmaceutical companies often conclude that the financial returns aren’t worth the significant risk and investment.This leaves families facing unimaginable decisions, relying on outdated treatments or options designed for adults that can be devastating to a child’s body.

This isn’t just a gap in the system—it’s a moral failure. And it’s one that CobiCure is uniquely positioned to address.

‍

Why CobiCure Exists

CobiCure is not your typical nonprofit. Founded with a mission to tackle one of the most overlooked crises in healthcare, CobiCure is dedicated to advancing treatments for rare conditions and diseases that affect children. But what truly sets us apart is how we do it.

CobiCure began by focusing on pediatric medical devices—a critical area of unmet need. Children require devices specifically designed for their growing bodies and unique medical needs, but too often they are not available. Innovation in this space is often deprioritized due to the same market forces that limit investment in pediatric therapeutics. Through partnerships and creative problem-solving, we’ve helped address these gaps by supporting the development of medical devices tailored for children. From this early success, we know our model can work.

Building on that foundation, CobiCure has expanded its mission to include early-stage therapeutics for pediatric cancers and other rare diseases. This natural progression allows us to address the continuum of needs in pediatric healthcare, leveraging the expertise and infrastructure we’ve already developed.

Unlike traditional nonprofits that fund research or rely solely on philanthropy, CobiCure is focused on collaboration. We bring together the cutting-edge infrastructure, networks, and expertise needed to develop profitable therapies and devices, but without the pressure to deliver big returns to investors. This model allows us to work faster, smarter, and with greater efficiency, while still creating a viable business.

The Profound Importance of Early Clinical Development

The road from ascientific discovery to an approved treatment is long, costly, and filled withuncertainty. Early clinical development is a challenging part of the journey,where critical questions about safety and efficacy are answered. It’s alsowhere most potential therapies are abandoned due to a lack of funding orinfrastructure to carry them forward.

But here’s thetruth: Once a therapy successfully clears early clinical trials, the likelihoodof success improves dramatically. This stage is where the foundation for hopeis laid—and it’s precisely where CobiCure focuses its efforts.

Through our unique model, we aim to de-risk these therapies and devices, making them attractive to larger partners who can carry them through later stages of development. While this approach is still in its early days, it combines the mission-driven purpose of a nonprofit with the operational rigor of a for-profit organization, offering a way forward for diseases and conditions that have long been neglected.

‍

The Potential and Unique Impact of CobiCure

CobiCure’s approach is built on the belief that innovation and compassion can work hand in hand to solve some of healthcare’s toughest challenges. By blending the best of nonprofit values with capabilities of a world-class healthcare investment firm, we are laying the groundwork for what could be a game-changing model for pediatric cancers and rare diseases.

‍

Why It Matters

This work isn’t just about advancing science—it’s about changing lives. It’s about children with rare pediatric cancers, life-threatening diseases, or conditions requiring innovative devices. These children often have no other options because the system has overlooked their unique needs.

Although we’re only at the beginning of our journey in therapeutics, our early success in medical devices gives us confidence in the potential of our model. Behind every statistic is a family, a community, a future. The work we’re doing at CobiCure isn’t just filling a gap—it’s providing a lifeline to those who need it most.

‍

How You Can Help

Raising awareness about the challenges of pediatric cancers, rare diseases, and the critical need for tailored devices is just the beginning. At CobiCure, we’re committed to not only starting this vital conversation but also delivering tangible solutions.But we can’t do this alone. It will require a community, and together, we can ensure that no child is denied the chance to grow up because the system failed them.

Help us spread the word. Share this story, follow our journey, and join us in our mission to transform the future of rare disease, pediatric cancer, and medical device innovation.

Together, we can make a profound and lasting impact.

Because every child deserves a chance at life.

‍