How New Drugs Are Evaluated (Sickle Cell)

Question 1

What is a drug?

Answer 1

A chemical substance of biological or biotechnological origin used for prevention, diagnosis, or treatment of disease in man or animal.

Question 2

Define a new drug.

Answer 2

A drug already approved by the licensing authority, proposed to be marketed with modified or new indications, dosage forms, and route of administration.

Question 3

Why are new drugs developed?

Answer 3

Due to aging population, increased prevalence of communicable diseases, emerging diseases, outbreaks, more efficacy, fewer side effects, and convenience.

Question 4

List methods and approaches in new drug discovery.

Answer 4

• Serendipity
• Folk remedies
• Replacement therapies
• Rational drug design
• High throughput screening

Question 5

What are the stages of new drug evaluation?

Answer 5

• Drug discovery
• Early development
• Full development

Question 6

What is the first step in new drug development?

Answer 6

Discovery and Development.

Question 7

What is the purpose of preclinical research?

Answer 7

To determine whether a drug has the potential to cause serious harm or toxicity.

Question 8

What are the two types of studies in preclinical research?

Answer 8

• In Vitro
• In Vivo

Question 9

What is the primary objective of Phase I trials?

Answer 9

To determine an acceptable range of dose(s) and schedule(s) for a new drug.

Question 10

What is the focus of Phase II trials?

Answer 10

To determine if the new drug has any beneficial activity and is worthy of further testing.

Question 11

What is the goal of Phase III trials?

Answer 11

To compare experimental or new therapies with standard therapy or competitive therapies.

Question 12

What is the significance of a control group in clinical trials?

Answer 12

It minimizes bias and allows credible inference from the trial.

Question 13

What does an NDA application entail?

Answer 13

It demonstrates that a drug is safe and effective for its intended use and includes all relevant data from preclinical to Phase 3 trials.

Question 14

What is the objective of Phase IV trials?

Answer 14

To confirm the efficacy and safety profile in large populations during practice and detect unknown/rare adverse drug reactions.

Question 15

True or False: Clinical trials provide complete information about a drug’s safety at the time of approval.

Answer 15

False

Question 16

List the phases of clinical trials and their corresponding objectives.

Answer 16

• Phase I: Safety, Doses, PD, PK - small number of healthy volunteers (20 to 100)
• Phase II: Efficacy and side effects - several hundred patients (with the disease)
• Phase III: Efficacy and monitoring of adverse reactions - large-scale testing (hundreds to thousands of patients)
• Phase IV: Post market survey - large-scale testing (hundreds to thousands of patients)

Question 17

What is Sickle Cell Disease (SCD)?

Answer 17

A devastating condition caused by an autosomal recessive inherited hemoglobinopathy resulting in vaso-occlusive phenomena and hemolysis.

Question 18

What genetic abnormality causes Sickle Cell Disease?

Answer 18

A substitution of the amino acid valine for glutamic acid at the sixth position on the beta globin chain.

Question 19

What are the clinical pathologies associated with Sickle Cell Disease?

Answer 19

• Anemia
• Vasoocclusion
• Chronic organ damage

Question 20

What are the two main categories of pharmacotherapy for Sickle Cell Disease?

Answer 20

• Disease Modifiers
• Supportive Care

Question 21

Fill in the blank: The _____ is the gold standard for establishing a control in clinical trials.

Answer 21

Randomized Control Trial (RCT)