How Cross-Sector Collaboration Accelerates Rare Disease Innovation

Published: 10 Jun 2025

Reading Time: 3 minutes

“Everything we’ve done has been through collaboration — it’s the only way we’re going to make progress fast enough for patients.”
This quote from Dr. David Fajgenbaum, physician-scientist and co-founder of the Castleman Disease Collaborative Network, underscores the most urgent truth in rare disease research: no single institution can solve this alone.¹

Over 300 million people globally are affected by more than 10,000 known rare diseases, yet fewer than 5% have an FDA-approved treatment.² Despite scientific breakthroughs, progress remains stalled—often not because of lack of knowledge, but because of disconnected systems.

Why Innovation Stalls

Rare disease research faces three systemic barriers:

Fragmented Data: Patient records, genomic data, and clinical trial results are dispersed across systems and institutions, limiting their collective value.
Misaligned Incentives: Academic researchers often prioritize publication over application; biopharma focuses on profitability; and hospitals prioritize operational efficiency—leaving gaps in patient-centered development.
Trust Deficits: Marginalized communities, frequently underrepresented in clinical trials, lack trust in systems that exclude their lived experience.

Why Collaboration Is the Answer

The field of rare disease doesn’t just benefit from collaboration—it demands it. Smaller patient populations and higher uncertainty require broader partnerships to ensure timely, inclusive, and effective outcomes.

In her Harvard Business Review article on alliances, Rosabeth Moss Kanter makes the case clearly:

“The collaborative enterprise is the true path to innovation and value creation. No one can go it alone anymore.”⁴

At its best, collaboration brings:

Speed: Shared resources and pooled knowledge reduce duplication.
Credibility: Diverse stakeholder input improves trial design and equity.
Access: Patient advocacy groups increase relevance and trust.
Sustainability: Broader engagement attracts government, nonprofit, and industry support.

RECOMMENDED READ: Designing Ethical Health Tech for Rare Diseases

Case in Point: Castleman Disease

Dr. David Fajgenbaum’s own story demonstrates what’s possible. Diagnosed with a deadly subtype of Castleman Disease while in medical school, he quickly discovered that few researchers or institutions were coordinating efforts.

Rather than wait, he built a model for collaboration. The Castleman Disease Collaborative Network (CDCN) unified clinicians, patients, and researchers around shared goals—enabling them to identify and test a potential treatment using a repurposed FDA-approved drug.

“I realized that no one was coming to save me. I had to take action… We needed to stop working in parallel and start working together.”¹

That mindset shift accelerated research and improved survival—proof that strategic collaboration can close life-threatening gaps.

What Effective Collaboration Requires

At Elevate Impact, we’ve worked with hospitals, academic institutions, and health equity agencies to build collaborative infrastructures that function across silos. Five essential pillars have emerged:

Co-Designed Goals: All stakeholders, including patients, should shape research priorities and trial outcomes.
Shared Language: Standardizing definitions of “equity,” “success,” and “impact” ensures strategic alignment.
Leadership Development: We must train healthcare leaders in systems thinking, communication, and cultural humility—not just clinical expertise.
Technology-Enabled Exchange: Platforms that support asynchronous, peer-verified knowledge sharing help innovation scale faster.
Outcomes-Based Metrics: Progress must be measured not just in approvals or publications, but in lives impacted, disparities reduced, and knowledge transferred.

View Elevate Impact solutions.

A National Imperative

The U.S. Food and Drug Administration now mandates that sponsors submit Diversity Action Plans to ensure clinical trial populations reflect real-world demographics.⁵ And the NIH’s Accelerating Medicines Partnership is using public-private partnerships to pool data and co-fund trials in rare neurological disorders.⁶

Meanwhile, the EveryLife Foundation reports that the total economic burden of rare disease in the U.S. exceeds $1 trillion annually, with most of it borne directly by families through lost income, medical costs, and delayed care.²

These figures aren’t just sobering—they’re solvable. But only if we connect the dots.

Conclusion

The future of healthcare innovation won’t be defined by what any one entity achieves in isolation—but by how well we work together.

Cross-sector collaboration is no longer optional. For patients with rare diseases, it is the fastest path to hope, discovery, and survival. We need to reimagine research not as a pipeline but as a network—one where information flows freely, insights are co-created, and lives are transformed in real time.

Because the cure won’t come from silos. It will come from systems—built together.

References:

Fajgenbaum, D. (2019). Chasing My Cure: A Doctor’s Race to Turn Hope Into Action. Ballantine Books.
EveryLife Foundation. (2021). The National Economic Burden of Rare Disease Study. https://everylifefoundation.org
National Institutes of Health. (2021). Collins, F. “On COVID-19 and the Power of Collaboration.” NIH Director’s Blog.
Kanter, R. M. (1994). Collaborative Advantage: The Art of Alliances. Harvard Business Review. https://hbr.org/1994/07/collaborative-advantage
U.S. Food & Drug Administration. (2022). Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials.
National Institutes of Health (2023). Accelerating Medicines Partnership: Rare Neurological Diseases Program. https://www.ninds.nih.gov