PGx and Drug Develop/ Study Design Stev

Pharmacogenetics is study of variability in drug response due to genetics While Pharmacogenomics is a more broad term which includes all genes in the genome which may determine drug response

• True

• False

FDA responsible for protecting the public health by:

• Ensuring the safety, efficacy and security of human and veterinary drugs, biological products and medical devices

• Ensuring the safety of our nation's food supply, cosmetics and products that emit radiation

• For advancing the public health by helping to speed innovations that make medical products more effective, safe and affordable

• By helping the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health

• All of the above

If a drug developer plans to develop and promote a medication, the drug developer has to submit an application to the FDA:

called Investigational New Drug (IND) which includes different components: animal study data, manufacturing information, clinical protocols (study plans), data from any prior human research, information about the investigator

• True

• False

The Components of Drug Development and Drug Approval Process includes:

•Preclinical studies

and

•Clinical studies:

–Phase I, II, III, IV

• True

• False

Phase 1:

–Purpose: safety and dosage

–Study participants: 20 to 100 healthy volunteers or people with the disease/condition

–Last about several months

• True

• False

Phase 2:

–Purpose: efficacy and side effects

–Study participants: up to several hundred people with the disease/condition

–Lasts between several months to 2 years

• True

• False

•Phase 3:

–Purpose: efficacy and monitoring of adverse reactions

–Study participants: 300 to 3,000 volunteers who have the disease or condition

–Last about 1 to 4 years

• True

• False

Phase 4:

–Purpose: safety and efficacy

–Study participants: Several thousand volunteers who have the disease/condition

–No length of time determined (Post-Market Safety Monitoring)

• True

• False

Drug Development and Precision Medicine consist of:

•Rapid identification and interpretation of genetic variants which underline single patients’ disease can:

– Help with providing window into patient specific mechanisms which cause and/or contribute to a disease development

– Allow “precise” targeting of these specific mechanisms

• True

• False

During Pharmacogenomics and Drug Development, there are different benefits, challenges and improvements in drug discovery and development process with pharmacogenomics:

• Drug approval process changed due to impact of genetic-based procedures based on proper identification of patients for specific treatments

  •Need to identify if there are specific variants for which treatments work

• Identification of molecular targets can help with identification and optimization of unique therapeutic strategies

• Pharmaceutical companies can identify specific molecular targets

• All of the above