Ceutics quiz 1.2

Common sources of new drug molecules

Chemical synthesis; natural products

Why chemical synthesis is important

Allows creation of new molecules not found in nature

Why natural products are important

Plants, microbes, marine organisms produce bioactive compounds

Activities included in pre-clinical development

Toxicology studies, PK/PD evaluation, formulation development

Purpose of toxicology studies

Evaluate safety risks before human exposure

Purpose of formulation studies

Develop the dosage form

Purpose of pre-clinical PK studies

Determine how the body absorbs, distributes, metabolizes, and excretes the drug

Phase I

Assess safety in small groups of healthy volunteers

Phase II

Evaluate efficacy + refine dosing in patients

Phase III

Confirm safety and efficacy in large, controlled trials

Phase IV

Long-term risk–benefit analysis after approval

Investigator’s Brochure (IND component)

Summaries for investigators on drug safety and rationale

Clinical Protocols (IND component)

Plans for human testing

Pre-clinical data (IND component)

Laboratory and animal study results

CMC information (IND component)

Chemistry, manufacturing, and controls

IND

Permission to begin human clinical trials

NDA

Approval for brand-name small molecule drugs

BLA

Approval for brand-name biologics

ANDA

Approval for generic drugs

sNDA

Approval for new use or new formulation of an already approved drug