Study Set Content:
No satisfactory alternative is available.
Treatment IND
is a rare disease or condition affecting fewer than 200,000 people in the United States and of which there is no reasonable expectation that R&D costs will be recovered by product sales.
Orphan disease
Examples of Orphan Disease
– Chronic lymphocytic leukemia – Cystic fibrosis – Gaucher disease
The sponsor can withdraw at
any time
Inactive status
Withdrawal or Termination of an IND
for reasons of safety, efficacy, or regulatory compliance
FDA termination
Approval to market the new product
General Content of the NDA Submission
General Content of the NDA Submission
Complete presentation of:
Preclinical results – Clinical results
Drug product labeling, Must meet specific labeling requirements in the (blank) and approved for each product by the (blank)
CFR, FDA
Labeling includes:
• Labels on immediate container • Package insert • Company literature • Advertising • Promotional materials
• Continued data collection for drugs after they are markete
Phase 4 Studies and Postmarketing Surveillance
Phase 4 Studies and Postmarketing Surveillance
May reveal additional:
Side effects – Serious and unexpected adverse effects – Drug interactions
• Continued clinical investigations
Phase 4 Studies and Postmarketing Surveillance
Better understand the drug’s MOA – Indicate possible new therapeutic uses – Demonstrate need for new formulations – Additional side effects, adverse effects and/or drug interactions
Continued clinical investigations
Adverse drug experiences must be reported to the FDA within(blank) working days of receipt of information.
15
The FDA may require to revise
product labeling or other actions
• SNDA. or
Supplemental New drug application
Changes in an application for a number of reasons.
Supplemental New Drug Application
Nonclinical laboratory studies and clinical investigations may be omitted, except for studies pertaining to drug’s bioavailability.
Abbreviated New Drug Application
Minor changes (editorial and others) can be made without prior approval.
Supplemental New Drug Application
