Review questions and answers

Review questions

2.1 Which of the following is true for the drug development and regulatory process?

A.      A drug’s sponsor must submit an IND application before an FIH trial of a drug

B.      An IND application must precede an NDA submission to the FDA

C.      An NDA approval must precede a corresponding ANDA submission

D.      All of the above

E.       None of the above

2.2 Indicate which of the following statements is TRUE and which is FALSE.

A.      The FDA can approve new formulations without phase III clinical trials.

B.      In phase III clinical trials, only a small number of patients are enrolled.

C.      New drug substances are extracted from plants or animals or syn-thesized in laboratories.

D.      The CDER is responsible for the approval of vaccines.

E.       The ANDA requires full clinical and nonclinical testing.

F.       The ANDA can be filed for biological products.

G.      The BLA is approved by the CBER, whereas the NDA is approved by the CDER.

2.3 A. Define the following terminologies: FDA, IND, NDA, CDER, FIH, CBER, and BLA.

B. List the different steps involved in the drug development and approval process.

C. In which phase of drug development are healthy subjects evaluated?

2.4 A. What are the specific responsibilities of the CDER and the CBER?

B. What information does the FDA require in an IND application?

C. What are the goals of phase I, II, and III trials?

D. Why is the postmarketing surveillance necessary?

2.5 What are the three key components of pharmaceutical development?

A.      Bioavailability: To ensure that the drug has reproducible and clin-ically desired bioavailability from a dosage form.

B.      Stability: To ensure that the drug product is stable at the labeled storage conditions for the duration of its assigned shelf life.

C.      Manufacturability: To ensure that the drug product can be manu-factured reproducibly and robustly at a commercial scale.

D.      Tolerability: To ensure that the drug is tolerated by the subjects and there are no significant adverse effects.

E.       All of the above

2.6 Match the stage of drug development in the left column with the key deliverables of that stage in the right column. Write the letter of row in the left column in front of the corresponding row in the right column.

2.7 Interspecies dose scaling for small-molecule compound is generally carried out using which metric?

A.      Body weight

B.      Body surface area

C.      Body fluid volume

D.      Muscle weight

E.       Fat tissue weight

2.8 Clinical studies carried out in which phase are also called first-in-human studies?

A.      Phase I

B.      Phase II

C.      Phase III

D.      Phase IV

2.9 Which of the following may not be a typical postcommercialization activity?

A.      Investigation of a new drug–drug combination product

B.      Investigation of a new drug–device combination product

C.      First phase III clinical trial to support commercialization of an

D.      NCE

E.       Investigation of an approved drug’s ability to treat a new disease indication

Answer:

2.1 D.

2.2 A. False

B. False

C. True

D. False

E. False

F. False

G. True

2.3 A. FDA: Food and Drug Administration; IND: investigational new drug application; NDA: new drug application; CDER: Center for Drug Evaluation and Research; Biologics: viruses, therapeutic serum, toxin, antitoxin, vaccines, blood, blood components or derivatives, allergic products, or analogous products, applicable to the preven-tion, treatment, or cure of a disease or condition of a human being.

B. Refer to the chapter.

C. Healthy subjects are evaluated in phase I clinical trials of drug product development.

D. A lead compound is the one that shows high bioactivity and low toxicity.

2.4 A. The CDER evaluates prescription, generic, and OTC drug prod-ucts for safety and efficacy before they can be marketed. It also monitors all human drugs and biopharmaceuticals once they are in the market, and removes products from the market that may not be manufactured properly or may cause harm to patients. The CBER regulates biologics not reviewed by the CDER, such as vac-cines, blood and blood products, gene therapy products, and cel-lular and tissue transplants.

B. Refer to the chapter.

C. Refer to the chapter.

D. Postmarketing surveillance is necessary as it may contribute to the understanding of the drug’s mechanism or scope of action, indi-cate possible new therapeutic uses, and/or demonstrate the need for additional dosage strengths, dosage forms, or routes of administra-tion. Post marketing surveillance studies may also reveal additional side effects, and rare, serious and unexpected adverse effects.