Crafting business models to enable sustainable development of N-of-1 therapies — 2024 NEXT Report
May 24, 2024
When traditional pharma decides that therapies for N-of-1 and ultra-rare diseases is too risky, not-for-profits and public benefit corporations are able to step in to fill the gap for sustainable development of those therapies. Innovative
Genomics Institute (IGI), an academic research partnership, provided alternative pricing models for this sustainable development that could also maximize access, prioritize affordability, recover costs for development, and sustain the organization, while also supporting future drug development.
This is one of the several topics covered in 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
- The need for regulators to exercise their flexibility to address ultra-rare therapies
- How drug developers or working to rethink payment models for one-time, curative therapies
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