Drug Development, Regulation, Safety Flashcards
GCP
Clinical trail research must meet good clinical practice (GCP) standards
-set of rules that dictate key aspects of clinical trials from study design, facilities, and personnel to proper documentation of data
What do clinical trails require review and approval of?
The IND and the IRB
Which phase of studies has an emphasis on safety with small patient groups, determine adverse effects, drug metabolism and excretion?
phase 1
Which phase of studies has an emphasis on effectiveness and safety?
phase 2
What phase of studies involves post-marketing studies that are imposed upon a pharmaceutical firm as a condition for drug approval.
Phase 4
Formal submission to the FDA to consider approving a new drug for marketing in the US.
NDA- new drug application- this includes all animal and human data and analyses of the data, as well as information about how the drug behaves in the body and how it is manufactured
Which schedule has a lower potential for abuse than schedule IV and consist of preparations containing limited quantities of certain narcotics.
schedule 5- antidiarrheal, antitussive, and analgesic purposes
IRB
Institutional Review Board- protection of human research subjects
- informed patient consent
- fully informed of their risks and have given informed consent
- informed that they have alternatives
Which schedule has moderate to low potential for physical and physiological dependence?
schedule 3
GLP
GLP- pre-clinical research must meet good laboratory practice standards (GLP)
-the set of rules that dictate study design, facilities, and personnel to proper documentation of data
Lead compound discovery
This is the first step in pre-clinical development. Assays can be developed to screen for molecules that activate or inhibit a molecular target
- drug targets are either receptors or enzymes
- lead compounds can be discovered in a variety of screening assays
Orphan drug status
An orphan drug is developed specifically to treat a rare medical condition (an orphan disease)- orphan drugs status can provide longer patent exclusivity, tax incentives, and financial incentives
-must affect less than 200,000 American citizens
PICO
P- patient, population
I- intervention/indicator
C- comparator/control
O- outcome
Which phase of studies involves larger trials power to assess safety and effectiveness?
phase 3
REMS
Risk evaluation and mitigation strategies- another tool the FDA uses to increase drug safety
IACUC
Institutional animal care and use committee- protection of animals in research
What are pre-clinical testing?
- In vitro studies
- IACUC
- Animal studies address safety and efficacy
In vitro testing
The pharmacological profile of the compound can be determined using in vitro assays. The goal of these experiments is to determine the potency and selectivity of the compound. Cell culture assays (activity, selectivity, toxicity) or tissue slices grown in culture (activity, selectivity, toxicity)
Which schedule has a low potential for abuse and low risk of dependence?
schedule 4- Xanax and Ambien
Medicinal chemistry
This is the second step in pre-clinical development. Derivatives of a lead compound are synthesized to search for a compound with the required properties of drug.
Drug properties- affinity, stability, and toxicity
Animal studies address safety and efficacy
experiments in animal models are used to address efficacy of a drug candidate and other issues, pharmacokinetic properties, adverse effects and toxicity
Which schedule has high potential for abuse, severe psychological or physical dependence?
schedule 2- cocaine, methamphetamine, methadone, oxycodone, methylphenidate
IND- when is one filed?
Investigational New Drug Application- if pre-clinical testing, both in vitro and in animals justifies human testing an IND application is filed
Which schedule has no accepted medical use and a high potential for abuse?
schedule 1- Heroin, marijuana
