10 Jan 2024

Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”

The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradigm. The mission of the Rare Disease Moonshot is rooted in boosting public-private collaborations. Many challenges in modern medicine are too big for one organization to tackle alone. They require true collaboration between a diverse network of companies, policymakers, regulators, academics, civil society groups, and patients. Rare diseases present one such challenge.

Key Takeaways

  • There is no treatment for 95 percent of rare diseases, and in many cases, no science to translate into solutions for patients.
  • The Rare Disease Moonshot will enhance the translational research ecosystem, optimize clinical trials and regulatory pathways, and boost infrastructures.
  • New recommendations on “research needs” in clinical trials, diagnostics, and translational research have been published.
  • Public-private partners support calls for a European Action Plan on Rare Diseases.

Read the full article here