Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?
A workshop entitled "Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?" was held at the 36th Annual Meeting of the American College of Toxicology in Summerlin, Nevada. The workshop was sponsored by Shire and Ultragenyx and was designed to present the nonclinical considerations for the development of various products for rare diseases. A panel of experts from industry and government highlighted the nonclinical considerations in developing toxicology programs supporting rare disease therapeutics, challenges in preclinical safety assessment, reviewed the current guidance, and presented the progress that has been made to date. The main learning from the workshop was that nonclinical testing of therapeutics targeting rare disease warrants special considerations, and early collaboration between sponsors and health authorities may help optimize the scope and timing of the supportive studies. Specific examples for nonclinical development programs for enzyme replacement therapy (ERT) were presented. Although the symposium focused on ERTs, the concepts are broadly applicable.
Jazz Pharmaceuticals Inc, Palo Alto, CA, USA krishna.allamneni@jazzpharma.com.
Krishna P
KP
Allamneni
Shire, Lexington, MA, USA.
Suezanne
S
Parker
BioMarin Pharmaceutical Inc., Novato, CA, USA.
Charles A
CA
O'Neill
Dimension Therapeutics Inc, Cambridge, MA, USA.
Teresa L
TL
Wright
US Food and Drug Administration, Silver Spring, MD, USA.
Sruthi
S
King
AbbVie Inc Research and Development Center, Worcester, MA, USA.
Laura
L
Andrews
D000818:Animals; D035843:Biomedical Research; D004353:Drug Evaluation, Preclinical; D056947:Enzyme Replacement Therapy; D006801:Humans; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000188:drug therapy*
enzyme replacement therapy; orphan indication; rare disease; safety assessment