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The need for regulators to exercise their flexibility to address ultra-rare therapies — 2024 NEXT Report

June 25, 2024

The path to rare disease drug development comes with built-in obstacles as it is, but when seeking therapies for ultra-rare and hyper-rare therapies, the path becomes even more difficult. Science and advances in technology are paving the way for drug developers, patient advocacy organizations, and patient families to realize their goals of treatments, but often, regulators and payors are the only obstacles remaining.

This is one of the several topics covered in the 2024 NEXT Report.

Download the 2024 NEXT Report

The NEXT report provides an overview of developments across the rare disease landscape and highlights trends in research, diagnosis, development, and treatment, as well as the changing regulatory and financial environment. Other topics covered include:

  • The need to think differently about the challenges for rare, ultra-rare, and hyper-rare conditions
  • The promise and challenges of emerging gene-editing and other genomic medicines
  • How changing sequencing technologies are improving diagnostic success
  • How non-profit and for-profit entities are crafting business models to enable sustainable development of N-of-1 therapies
  • How drug developers or working to rethink payment models for one-time, curative therapies

Download the 2024 NEXT Report

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