Drug Development and Regulation

Flashcards about drug development and regulation.

Random Screening

Screening for biologic activity of large numbers of natural products, banks of previously discovered chemical entities, or large libraries of peptides, nucleic acids, and other organic molecules.

Chemical Modification

Chemical modification of a known active molecule, resulting in a “me-too” analog.

Rational Drug Designing

Rational design of a new molecule based on an understanding of biologic mechanisms and drug receptor structure.

Drug Screening

Define the pharmacologic profile of a drug through assays at the molecular, cellular, organ system, and whole animal levels.

Molecular Level (Drug Screening Assay)

The compound would be screened for activity on the target, e.g., receptor binding affinity to cell membranes containing the homologous animal receptors.

Cellular Level (Drug Screening Assay)

Determine whether the drug is an agonist, partial agonist, inverse agonist, or antagonist at relevant receptors.

Whole Animal Level (Drug Screening Assay)

Determine the effect of the drug on organ system models and disease models.

Lead Compound

The leading candidate for a successful new drug, found after drug screening.

Preclinical Safety and Toxicity Testing

Evaluation for potential risks before and during clinical testing, including identifying potential human toxicities and designing tests to define the toxic mechanisms.

Natural History of Disease

The progression of the disease process in an individual over time in the absence of treatment.

Single-Blind Design

Reduces subject bias; participants do not know what group they belong to or what treatment they are receiving.

Double-Blind Design

Reduces observer bias; the observer (or doctor) and the subject do not know the grouping, only the drug sponsor or drug manufacturer.

The Philippine FDA

The agency is responsible for licensing, monitoring, and regulation of cosmetics, drugs, foods, household hazardous products, medical devices and electromagnetic radiation emitting devices, pesticides, tobacco and related products, and vaccines for safety efficacy and quality in the Republic of the Philippines.

Investigational New Drug (IND)

A Notice of IND must be filed with the FDA once new drug is judged ready to be studied in humans.

Phase I (Clinical Trials)

Effects of the drug as a function of dosage (safe clinical dosage range).

Phase II (Clinical Trials)

Modest number of patients with the target disease to determine its efficacy (“proof of concept”).

Phase III (Clinical Trials)

Large number of patients to further establish and confirm safety and efficacy.

New Drug Application (NDA)

Phase 3 results meet expectations, application is made for permission to market the new agent.

Phase IV (Postmarketing Surveillance)

Low incidence drug effects are not generally detected before phase 4 no matter how carefully phase 1, 2, and 3 studies are executed.

Orphan Drugs

Drugs for rare diseases.