IND 123 MIDTERM ACTUAL PAPER 2026
QUESTIONS WITH SOLUTIONS GRADED A+
◉ What are key attributes of a 'hit' compound? Answer: A hit
compound shows activity against a target in assays, but its
mechanism may be unclear.
◉ What distinguishes a 'lead' compound from a 'hit'? Answer: A lead
compound demonstrates pharmacological activity with therapeutic
potential and may require structural modifications.
◉ What role does the FDA play in preclinical studies? Answer: The
FDA reviews preclinical studies to ensure safety and efficacy before
clinical trials.
◉ What is the Declaration of Helsinki? Answer: A set of ethical
principles for medical research involving human subjects.
◉ What types of preclinical studies exist? Answer: Types include in
vitro studies, animal studies, and pharmacokinetic studies.
◉ What is an Investigational New Drug Application (IND)? Answer:
A request to the FDA for permission to start clinical trials on a new
drug.
, ◉ What are the components of an IND? Answer: Components
include drug composition, manufacturing information, and clinical
study protocols.
◉ What is the purpose of target selection in drug discovery?
Answer: To identify disease pathways linked to patient needs and
assess potential drug targets.
◉ What is 'druggability'? Answer: The ability of a target to bind a
drug molecule with high affinity and have favorable
pharmacokinetics.
◉ What is proof of concept in drug development? Answer:
Demonstrating that manipulating a target can change disease
biology.
◉ What is high-throughput screening (HTS)? Answer: A method to
test hundreds of thousands of compounds in parallel for activity
against a target.
◉ What are CFTR modulators? Answer: Drugs developed to correct
the function of the CFTR protein in cystic fibrosis patients.
QUESTIONS WITH SOLUTIONS GRADED A+
◉ What are key attributes of a 'hit' compound? Answer: A hit
compound shows activity against a target in assays, but its
mechanism may be unclear.
◉ What distinguishes a 'lead' compound from a 'hit'? Answer: A lead
compound demonstrates pharmacological activity with therapeutic
potential and may require structural modifications.
◉ What role does the FDA play in preclinical studies? Answer: The
FDA reviews preclinical studies to ensure safety and efficacy before
clinical trials.
◉ What is the Declaration of Helsinki? Answer: A set of ethical
principles for medical research involving human subjects.
◉ What types of preclinical studies exist? Answer: Types include in
vitro studies, animal studies, and pharmacokinetic studies.
◉ What is an Investigational New Drug Application (IND)? Answer:
A request to the FDA for permission to start clinical trials on a new
drug.
, ◉ What are the components of an IND? Answer: Components
include drug composition, manufacturing information, and clinical
study protocols.
◉ What is the purpose of target selection in drug discovery?
Answer: To identify disease pathways linked to patient needs and
assess potential drug targets.
◉ What is 'druggability'? Answer: The ability of a target to bind a
drug molecule with high affinity and have favorable
pharmacokinetics.
◉ What is proof of concept in drug development? Answer:
Demonstrating that manipulating a target can change disease
biology.
◉ What is high-throughput screening (HTS)? Answer: A method to
test hundreds of thousands of compounds in parallel for activity
against a target.
◉ What are CFTR modulators? Answer: Drugs developed to correct
the function of the CFTR protein in cystic fibrosis patients.
