Exam 2: GCP And Marketing Approvals - BLA, NDA, sNDA, aNDA, OTC, IDE Flashcards
The study protocol is a study plan on which all clinical trials are based. It is designed to protect the health, safety and psychological well-being of the trial participants, and to answer specific research questions about the behavior, safety and efficacy of the drugs. What is not typically included in a study protocol?
A) The study protocol includes a description of the statistical design and analysis that will be used to arrive at a sound conclusion about the efficacy of the investigational drug
B) The study protocol includes the floor plan of the manufacturing facility for the investigational drug.
C) The study protocol includes a description of the justification or purpose of the clinical study
D) The study protocol includes a description of the types of treatment and tests of efficacy and safety to be performed during the clinical study
E) The study protocol includes a description of the length of the clinical study and the schedule of administration of the investigational drug.
B) The study protocol includes the floor plan of the manufacturing facility for the investigational drug.
What is not true of the Institutional Review Board (IRB)?
A) The IRB takes responsibility for initiating, managing, and financing a clinical investigation
B) Any change in the study protocol must have approval from IRB, except in life threatening situations
C) The IRB is designated by the trial site (institution) to approve, monitor, and review biomedical and behavioral research protocols involving humans
D) The IRB is an independent group that ensures the protection of the rights, safety, and well-being of human subjects
E) The IRB conducts risk-benefit analysis to determine whether clinical research should proceed
A) The IRB takes responsibility for initiating, managing, and financing a clinical investigation
What is not a role or responsibility of the clinical trial investigator?
A) The clinical investigator obtains informed consent from the trial subjects
B) The clinical investigator reports adverse events within 10 days to the sponsor and IRB
C) The clinical investigator adheres to the protocols inclusion and exclusion criteria for choosing trial participants
D) The clinical investigator files the IND prior to initiating the clinical trial
E) The clinical investigator maintains study files for at least 2 years after drug marketing approval
D) The clinical investigator files the IND prior to initiating the clinical trial
_______________ is a document given by the investigator to the sponsor to provide the sponsor with information about the qualifications of the PI, and at the same time remind the PI of his or her regulatory responsibilities.
A) FDA Form 483
B) Informed Consent
C) Statement of Investigator
D) Investigators Brochure
E) FDA form 1570
C) Statement of Investigator
Jesse Gelsinger was a clinical trial participant in one of the first clinical trials on an investigational gene therapy approached approach that tested adenovirus as a vector to introduce a functional gene to replace a defective gene. He died as a result of a massive immune response to the AAV. What is not a GCP violation that the investigators involved were found to have committed?
A) Jesse had a health condition that should have excluded him from participating in the trial
B) One of the clinical research associates had a criminal record
C) Investigators ignored previous reports of serious adverse reaction in two other patients
D) There was Inadequate informed consent documentation regarding deaths in monkeys
E) There were conflict of interests, i.e., scientists and the hospital were getting research funding from the company sponsoring the trial
B) One of the clinical research associates had a criminal record
What is not a reason why the FDA might impose a clinical hold on a trial?
A) While the trial is being conducted, unreasonable adverse health effects on patients started to show up
B) The FDA has questions about the quality or sterility of the investigational drug based on information in the chemistry, manufacturing and controls portion of the IND submission
C) The FDA finds issues with pre-clinical animal safety data in the IND submission
D) The FDA finds that the study protocol or design proposed in the IND submission does not provide sufficient assurance that risks to the of health study participant would be minimized
E) The trial sponsor has not paid the required PDUFA fee
E) The trial sponsor has not paid the required PDUFA fee
Of about 5000 drugs that companies investigate as a drug candidate, only 1 will successfully make it to market. What is the top cause of drug failure?
A) The investigational drug exhibited unacceptable toxicity to test animals
B) The investigational drug just was not effective in humans
C) Unfavorable pharmacokinetics
D) The new drug will not be able to compete successfully with the earlier marketed drug
E) The test drug has unacceptable adverse effects in human trials
C) Unfavorable pharmacokinetics
The Common Technical Document contains:
A) Provided to clinical trial investigators in US, EU or Japan, to provide data and information about the investigational drug to be tested
B) Data and information submitted as part of a requirement prior to field testing of crops engineered to produce novel proteins
C) Data and information provided to clinical investigators in US, EU or Japan to guide the conduct of a clinical trial
D) Data and information submitted as part of marketing applications for a pharmaceutical for human use for US, EU or Japan.
E) Provided by investigators to trial sponsors in US, EU or Japan, to provide data and information about the qualifications of the investigators
D) Data and information submitted as part of marketing applications for a pharmaceutical for human use for US, EU or Japan.
In which type of marketing application would it be typical for the FDA to require efficacy data for a marketed drug being submitted for use in a new indication, and not require additional safety data?
A) Over-the-counter drug application
B) Abbreviated new drug application
C) Orphan drug application
D) Supplemental New Drug Application
E) Biologics license application
D) Supplemental New Drug Application
What type of information is not contained in a New Drug Application (NDA) which is submitted to the FDA?
A) The NDA should provide evidence that the drug is safe and effective in its proposed use(s)
B) The NDA should provide evidence that methods used in manufacturing assured the drug’s quality
C) The NDA should provide evidence that there are no alternatives to the use of primates in safety tests
D) The NDA should provide results of drug safety from pre-clinical and clinical studies
E) The NDA should prove that the drug’s proposed labeling (package insert) is accurate and informative
C) The NDA should provide evidence that there are no alternatives to the use of primates in safety tests
New Drug Applications (NDA) have chemistry, manufacturing and controls (CMC) section that tells the whole story about the candidate drug. Which type of information is included in the CMC?
A) The ingredients or components of the drug product
B) Evidence that there is a substantial pool of patients that is in need of the new drug
C) The changes in gross morphology of test animals
D) Evidence that the clinical trial protocol was designed to minimize risks to trial participants
E) Evidence that there are no alternatives to the use of primates in safety tests
A) The ingredients or components of the drug product
Before the target PDUFA date, the FDA center in charge of a drug approval will assemble an _______________ to help with the decision making on the marketing application
A) Independent Review Board
B) Justice League
C) Poison Squad
D) Advisory Committee
E) Muckrakers
D) Advisory Committee
Many investigational drugs are now in clinical trials for preventing or treating COVID19 and the safe and effective ones will be submitted for marketing approval. Approval of small molecule drugs like remdesivir, is evaluated by CDER, while the approval of biologic products have been split between CDER and CBER. Which type of biologic product is assigned to CDER?
A) A coronavirus vaccine which uses a virus to deliver nucleic acids that are then transcribed and translated inside patients’ cells.
B) Monoclonal antibodies produced by Regeneron which was used to treat former President Trump
C) Live vaccines prepared using adenovirus engineered to display the COV2 spike protein
D) Allergenic extracts used for the diagnosis of food allergies
E) Blood from people who have recovered from COVID-19 to treat patients with COVID-19
B) Monoclonal antibodies produced by Regeneron which was used to treat former President Trump
When is a complete response letter (CRL) issued by the FDA in response to a drug marketing application?
A) A CRL is issued if the drug is approved for marketing
B) A CRL is issued if there were no safety issues observed during the clinical trials
C) A CRL is issued if the drug failed to demonstrate effectiveness
D) A CRL is issued if the required prescription drug user fee has not been paid
E) A CRL is issued if the clinical trial plan in a proposed IND is unacceptable
C) A CRL is issued if the drug failed to demonstrate effectiveness
What is not correct about patents and marketing exclusivity?
A) Orphan drug designation adds 7 years of market exclusivity from generic competition
B) Marketing exclusivity can be concurrent with a patent
C) Patents are granted by the USPTO and expire 20 years from date of filing
D) Marketing exclusivity can extend protection from generic competition beyond the patent life.
E) Marketing exclusivity is granted by the USPTO upon drug approval to protect the drug from generic competition
E) Marketing exclusivity is granted by the USPTO upon drug approval to protect the drug from generic competition
