Patient registries, whether disease-based or product-focused, can provide your organization, as well as the health community, with invaluable data about the natural history of a disease under standard care practices and/or the safety and effectiveness of a product. These data can then be presented as evidence to:
- Meet safety requirements and manage benefits and risks
- Provide evidence to meet evolving coverage or reimbursement requirements
- Generate scientific evidence and publications
- Develop Treatment Guidelines
- Demonstrate good product stewardship
Links to some existing registries for conditions identified through newborn screening or may be targets for newborn screening:
- Coordination of Rare Diseases at Sanford (CoRDS)
- Cystic Fibrosis
- Fabry registry
- Gaucher registry
- NBS Connect
- MPS I registry
- Myotonic Dystrophy (DM) or Facioscapulohumeral Muscular Dystrophy (FSHD)
- PKU registry
- Pompe registry
- Primary Immune Deficiencies
- Urea Cycles Disorders