Lesson10: Research&Development

Question 1

IND application must contain?

Answer 1

▪ Animal pharmacology and toxicology studies
▪ Manufacturing information
▪ Clinical protocols and investigator information

Question 2

Who could be the sponsor of IND?

Answer 2

The sponsor may be an individual (a sponsor–investigator), a pharmaceutical company, governmental agency, academic institution, or some other private or public organization.

Question 3

What are the objectives of FDA’s reviewing an IND application?

Answer 3

▪ To protect the safety and rights of the human subjects
▪ To help ensure that the study allows the evaluation of the drug’s safety and effectiveness

Question 4

What must be included in investigator’s brochure(IB) in order to gain FDA’s approval for IND?

Answer 4

To gaining the FDA’s approval for the IND, the investigator’s brochure (IB) must be reviewed and approved by the institutional review board (IRB) of each clinical site (e.g. hospital and medical center) where the proposed
clinical trials will be conducted.

Question 5

why needed pre-clinical stage ?

Answer 5

To ensure safety, efficacy of drug before use on human.

Question 6

What’s clinical trial ?

Answer 6

Clinical trial is a systematic investigation in human subjects for evaluating the safety, efficacy, and adverse effect of any new drug.

Question 7

What’s informed consent ?

Answer 7

Informed consent is mandatory document which is a process by which a participant voluntarily confirms to participate in clinical investigation with documented by means of a written, signed and dated informed consent form.

Question 8

What’s ethic ?

Answer 8

Ethical issues are required in the clinical investigation for respecting enrolled participations.

Question 9

What’s conflict of interest ?

Answer 9

Conflict of interest can bias the outcome of research so that
it should be considered a form for scientific misconduct.

Question 10

What are the three phases in clinical studies ?

Answer 10

▪ Phase I: small group of healthy people (20-50)
▪ Phase II: group of patients (100-300)
▪ Phase III: large group of patients (1,000-3,000)

Question 11

Synonym of Phase I ?

Answer 11

Fist-in-Human (FIH)

Question 12

Synonym of Phase IV ?

Answer 12

Post-Marketing Surveillance / Pharmacovigilance

Question 13

What’re the objectives of Phase I ?

Answer 13

➢ To identify a drug’s safety profiles including the safe dosage regimens
➢ To establish the tolerance of the drug in healthy human subjects at different doses
➢ To determine pharmacokinetic and pharmacodynamic profiles of drug

Question 14

Synonym of Phase II ?

Answer 14

Therapeutic exploratory trial

Question 15

What’re the objectives of Phase II ?

Answer 15

➢ To assess the effectiveness of the drug for a particular indication(s) and reconfirm toxicological profile
➢ To determine the common short-term side effects and risks associated with the drug
➢ Two key aspects of late-stage clinical development are randomization and blinding

Question 16

Synonym of Phase III ?

Answer 16

Therapeutic confirmatory trial

Question 17

What’re the objectives of Phase III ?

Answer 17

➢ To determine efficacy of the drug and identify adverse effects of the drugs
➢ To gather precise information on the drug’s effectiveness for specific indications
➢ To determine whether the drug produces a broad range of adverse effects
➢ To identify the best way of administrating and using the drug for the purpose

Question 18

What’re the information need to be contained in NDA ?

Answer 18

The NDA must contain all of the scientific information during the animal studies and human clinical trials.

Question 19

What require to be told in documentation of NDA ?

Answer 19

➢ The documentation required in an NDA is supposed to tell the drug’s whole story, including
▪ What happened during the clinical tests
▪ What the ingredients of the drug are
▪ The results of the animal studies
▪ How the drug behaves in the body
▪ how it is manufactured, processed and packaged.

Question 20

What’re the goals of NDA ?

Answer 20

The goals of the NDA are to provide enough information to permit the FDA reviewers to reach the following key conclusions:
▪ Whether the drug is safe and effectiveness in its proposed use(s)
▪ whether the benefits of the drug outweigh the risks
▪ Whether the drug’s proposed labeling is appropriate, and what it should contain.
▪ Whether the methods used in manufacturing the drug and the controls used to assure the drug’s quality are adequate to preserve the drug’s identity, strength, quality, and purity

Question 21

Key commercialization of R&D ?

Answer 21

➢ Key commercialization functions that intercept with research and development (R&D) are following:
▪ Marketing: identify the regions and countries where the drug product should be marketed, and also competitors
▪ Finance: provide cost-of-goods analyses
▪ Manufacturing: responsible for day-to-day operations in reproducibly producing

Question 22

What’re the objectives of Phase IV ?

Answer 22

➢ To indicate possible new therapeutic uses, and/ or demonstrate the need for additional dosage strengths, dosage forms, or routes of administration.
➢ To monitor of drug’s long-term safety in large population during commercial use
➢ To detect additional side effects, especially the rare events that may not be detected even in large-scale clinical trials
➢ To define the dosage and risk–benefit profile in patient populations

Question 23

What’s ANDA contained ?

Answer 23

An abbreviated new drug application (ANDA) contains data which is submitted to FDA for the review and potential approval of a generic drug product.

Question 24

What’s Generic drugs ?

Answer 24

Generic drugs are defined as products containing the same active ingredient as the branded drug, but likely having different inactive ingredients.