Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
Diagnosing rare diseases can take 3 to 15 years, while about 95% lack approved treatments. Families are advocating for regulatory changes to speed access to therapies as new digital tools aim to improve clinical trials.
The global biotechnology market is undergoing a transformation from discovery-based science to precision engineering, driven by AI, synthetic biology, and gene editing technologies, with projected growth of 13-15% over the next five years.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06935019 |
TRIAGE-GS: Towards Reducing Inefficiencies Affecting Genetics Encounters Through Genome Sequencing |
ENROLLING_BY_INVITATION | NA |
| NCT05657405 |
Observational Study of Advanced Data Analytics in Genetic Conditions |
RECRUITING |
