FDA Office of Orphan Products Development Developing Orphan Drug Products Marlene E. Haffner, M.D.,...
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FDA Office of Orphan Products Development FDA Office of Orphan Products Development Developing Orphan Developing Orphan Drug Products Drug Products Marlene E. Haffner, M.D., M.P.H. Marlene E. Haffner, M.D., M.P.H. Director, FDA Office of Orphan Products Director, FDA Office of Orphan Products Development Development 2 nd International Conference on Rare Diseases & Orphan Drugs Carlos III Health Institute Madrid, Spain October 25, 2006
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Transcript of FDA Office of Orphan Products Development Developing Orphan Drug Products Marlene E. Haffner, M.D.,...
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
Developing Orphan Drug Developing Orphan Drug ProductsProducts
Marlene E. Haffner, M.D., M.P.H.Marlene E. Haffner, M.D., M.P.H.Director, FDA Office of Orphan Products DevelopmentDirector, FDA Office of Orphan Products Development
2nd International Conference on Rare Diseases & Orphan DrugsCarlos III Health InstituteMadrid, SpainOctober 25, 2006
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
Drug Development ProcessDrug Development Process
Discovery
Preclinical Development (Animal Testing)
Clinical Development (Human Testing) Phase 1, Phase 2, Phase 3
New Drug Application/Approval
Post-marketing trials
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
Steps to Drug ApprovalSteps to Drug Approval
Submission of INDTitle 21, Code of Federal Regulations, Part 312Evaluation of toxicology dataEvaluation of chemistry, manufacturing & controls (CMC)Clinical trials, phase 1, 2, and 3NDA
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
PHASE 1PHASE 1
20-100 Patients
Testing mainly for safety
PHASE 2PHASE 2Up to several hundred patients
Testing for some short-term safety but mainly for effectiveness.
PHASE 3PHASE 3Several hundred to several thousand patients
Safety, dosage, effectiveness
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
A New Drug Application A New Drug Application (NDA) contains the following: (NDA) contains the following:
• Non-clinical studies
• Clinical studies
• CMC information
• Proposed labeling
• Additional information
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
Scientific Advice
Review ofApplication
OOPD/Review Division InteractionOOPD/Review Division InteractionOrphan Designation NDA
OOPD FDA Review divisions
AssessmentsFollow-up
Category Experts
Rare Disease Experts
Interaction with Interested Parties
Result: Result:Orphan Designation Marketing ApprovalResult:
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
OOPD Web Site
Overview of the FDA Office of Orphan Products Development
Guidelines for designation applicationList of designated and approved orphan
productsGrant application informationList of ongoing orphan grant studies
http://www.fda.gov/orphan Your Link to:
Telephone contact: 301-827-3666 or 1-800-300-7469
FDA Office of Orphan Products DevelopmentFDA Office of Orphan Products Development
Meet the OOPD StaffMeet the OOPD Staff
