New Drug

Question 1

Registration

Answer 1

• Company owners that do manufacturing, preparation, propagation, compounding or processing of drugs must register with HHS (owner name, place of business) for each establishment
• Compounders don’t have to register as manufacturers if they don’t “manufacture, prepare, propogate, compound or process drugs or devices for sale other than regular course of retail dispensing/selling drugs/devices” which means only if there is a prescription or reasonable expectation of a prescription
• If compounder deemed manufacturer, then FDCA requirements of registration, GMP, labeling/packaging, NDA, etc. Under DQSA, compounders outside regular course of business can register to avoid FDCA requirements.

Question 2

Legend Drug

Answer 2

RX only legend required on labeling

• FDA classifies RX only drugs if MD supervision required
• OTC required to have adequate directions for use that inform consumer about indication, use, etc
• Behind the counter - not official 3rd classification but they can only be sold by a pharmacist

Question 3

Investigational New Drug

Answer 3

First step to marketing/manufacturing a new drug is submit an investigational NDA to FDA (30 days to approve)
-animal studies, proposed clinical protocols for human tests

Question 4

Phase 1

Answer 4

• Tests SAFETY, toxocology, pharmacokinetics, and pharmacology
• Small group of healthy humans

Question 5

Phase 2

Answer 5

• Tests EFFECTIVENESS and also dosage, relative safety, AEs

- Larger group of humans (~100+) with dz or sx

Question 6

Phase 3

Answer 6

• Tests EFFECTIVENESS larger scale, last phase
• Larger group of humans (1000+), placebo group, multiple test centers, double blinded etc
• Success means filing NDA with FDA for market approval

Question 7

Phase 4

Answer 7

Postmarketing surveillance: healthcare professionals encouraged to report AEs/problems and manufacturers submit yearly reports on new info @ drug
-determines if drug should stay on the market

Question 8

Exception for Immediate, Life-Threatening Diseases

Answer 8

FDA Modernization Act and FDCA allow investigational new drugs be accessible outside clinical trials to treat patients with immediate fatal dz without satisfactory alternatives and its reasonable to expect effectiveness and not undue harm/risk

Question 9

Risk Evaluation and Mitigation Strategy (REMS)

Answer 9

• FDA Amendment Act 2007 gives FDA authority to require REMS on a pending NDA if it believes it will help ensure benefits outweigh the risk
• Can also require postmarket REMS if pt safety issue arises
• Require distribution of med guides, patient insert, communication plan for health care professionals, prescriber training, restricted settings for dispensing

Question 10

Expedited Approval (Fast Track)

Answer 10

-Can be granted if intended to treat a serious or life-threatening condition and address unmet medical need

Question 11

Review Drug Classifications

Answer 11

Type P: priority review d/t no other drugs to treat illness or significant advantage over current drugs
Type S: standard review (new one similar to current)
Type O: treats an orphan disease (

Question 12

Chemical Classifications

Answer 12

Type 1: new/unique molecular structure 
Type 2: new derivative of approved drug
Type 3: new formulation of approved
Type 4: new combo
Type 5: new manufacturer
Type 6: new indication for approved
Type 7: already marketed without approved NDA
Type 8: OTC switch
Type 10: new indication in distinct NDA, not consolidated

Question 13

Compassionate Use

Answer 13

• FDA can authorize investigational drug/device to use for dx, monitoring, or tx for serious/emergent situations
  1. Licensed MD determines nothing comparable/adequate and risk not greater than benefit
  2. FDA approves sufficient safety/effectiveness to support
  3. FDA determines that provision won’t interfere with trial
  4. Sponsor/investigator submits to HHS Secretary the clinical protocol for drug use in pt/group

Question 14

Naming New Drugs

Answer 14

US Adopted Names Council (USANC) w/HHS Secretary final approval
-short, distinctive, not easily confused, hints indication/chemical class

Question 15

National Drug Code (NDC)

Answer 15

Drug Listing Act 1972 requires registerees to provide list of drugs manufactured, prepared, propagated, compounded, processed for commercial distribution

• Three segments:
• First segment is assigned by FDA and identifies manufacturer/distributor
• Second segment manufacturer assigns for strength, dosage form, and formulation
• Third segment manufacturer assigns for package size

Question 16

SNDA

Answer 16

Supplemental new drug application when changes to be made on approved product
***SNDA is Submitted by the SAME company

Question 17

ANDA

Answer 17

Abbreviated new drug application is submitted when brand patent expires to market generic

• Requires proof that the new drug is similar to brand in pharmacokinetics, bioavailability, and clinical activity
• **ANDA is Submitted by ANOTHER company

Question 18

Drug Recalls

Answer 18

Selling recalled product is FDCA violation b/c its adulterated/misbranded
-Manufacturer REQUIRED to notify wholesalers/pharmacies written recall notices of Class I and II

Question 19

Class I Recall

Answer 19

Most serious, with risk of serious AE or death

-Involves pharmacies AND patients

Question 20

Class II Recall

Answer 20

Temporary or reversible AEs but serious AEs are rare

-Involves pharmacies only usually

Question 21

Class III Recall

Answer 21

Unlikely to cause AEs

Question 22

Current Good Manufacturing Practice

Answer 22

Regulations of minimum standards of pharm manufacturing

-Manufacturer must be registered with FDA and submit manufacturing/production in its NDA