New Drug" definition
A drug that is not generally recognized by experts
as safe and effective for use under the conditions recommended in the
drug's labeling.
New Drug" examples
New molecular entity
Drug already approved by FDA w/ new: AI, therapeutic indication,
formulation/manufacturer, dosing schedule/route of admin, combination
of products.
1. Pre-clinical studies
FDA approval not required prior
Assesses therapeutic effects and safety on animal subjects
Investigators/sponsors req'd to follow GLP
2. Investigational New Drug (IND) Application
1st application form to be submitted prior to administering "new
drug" to humans
It is illegal to distribute a drug unless it has an approved
_______ on file.
IND contents
Drug name
Composition
Methods of manufacturing and QC
Pharmacologic and pharmacokinetic data
How long does FDA have to decide if IND is suitable for testing?
30 days
3. Clinical Trials
Has 3 phases
FDA can terminate at any point if deemed necessary
Pt/representative must provide informed consent in each phase
Phase 1 Clinical Trials
Subjects: < 100 healthy individuals
Phase 1 Clinical Trials
Purpose is to evaluate drug safety, toxicity, absorption, metabolism,
and elimination
Phase 2 Clinical Trials
Hundreds of subjects affected by disease or symptoms of condition
Phase 2 Clinical Trials
Purpose is to determine effectiveness of drug and doses at which
efficacy occurs
Phase 3 Clinical Trials
Hundreds to thousands of subjects
Phase 3 Clinical Trials
Purpose is to collect all necessary data to meet safety and efficacy
standards for FDA approval.
Treatment IND
Allows for administration of IND to pts not enrolled in clinical
trial. Must be imminent, life-threatening w/ no current cure or where
current drugs are not effective.
Must have safety and efficacy data AND be in phase II or III of
clinical trial.
Must submit treatment protocol to FDA
4. New Drug Application (NDA)
Must include:
Reports of investigations showing drug safety and efficacy
Drug components & composition
Methods of manufacturing, processing, & packaging
Samples of drug and components
Any proposed labeling
Minimum length of time FDA reviews NDA
6 months
If approved, the NDA is ___________ and the drug may be ___________
by the manufacturer.
on file"; marketed
5. Post-marketing Surveillance
Subjects are all patients receiving drug
5. Post-marketing surveillance
Purpose is to report adverse effects on widespread population.
5. Post-marketing Surveillance
Concerned w/ long term safety profile in larger pt population
Manufacturer must maintain and est. post-marketing records &
provide annual reports.
FDA determines if drug stays on market
FDA MedWatch Program
System which HCPs can report any problems with use of drugs. Part of
Post-marketing Surveillance.
Type P
Priority" therapeutic classification
Major therapeutic gain
No other effective drugs available/has significant advantages
compared to current drugs
Type S
Standard/similar" therapeutic classification
Similar to other drugs on the market
Type O
Orphan drug" therapeutic classification
Chemical Classification
Types 1 - 6
Fast Tract Products
Accelerated review request if drug:
Intended to treat serious/life-threatening condition
Will fulfill unmet medical need Likely to provide
clinical benefit
Act that granted Fast Track Products
FDA Modernization Act of 1997
Abbreviated New Drug Application (ANDA)
May be submitted when "innovator" drug patent expires.
First applicant to complete demonstrating equivalent generic is
awarded 6 months exclusivity on sale of generic
ANDA requires _______ data than IND/NDA, but must prove
_______________ _______________.
less, Pharmaceutical equivalence
The Approval Process
Pre-clinical studies IND application Clinical
trials NDA Post-marketing surveillance
Supplemental New Drug Application (SNDA)
New use for existing drugs
Used when manufacturer wants to change drug's:
Synthesis production procedure manufacturing
location packaging labeling dosage
form/strength therapeutic indication
Orphan Drugs
Used for treatment of "rare diseases" affecting small pt population
Rare disease
Affects <200,000 persons in US
Orphan Drug Act of 1983
Offered tax relief, exclusive marketing rights, and faster NDA
approval to encourage development of orphan drugs.