Clinical Drug Development and Rationale Quiz

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Questions and Answers

What does the NPRA in Malaysia regulate?

Randomisation in clinical trials

Approval of medicines, medical devices, food, and cosmetics (correct)

Investigational new drug applications

Clinical trial protocols

What is the purpose of an Investigational New Drug (IND) application?

To monitor on-site patient data collection

To be filed prior to beginning clinical trials (correct)

To obtain drug approval after pivotal trials

To analyze and write reports on clinical trials

What is the function of the Investigational New Drug (IND) application according to the FDA?

It has 60 days to decide if there is enough information to continue NDA review

It is filed prior to beginning clinical trials

It contains extensive data on the investigational drug and results of clinical trials (correct)

It makes a final decision within 180 days

What is the role of the internal review board (IRB) in clinical trials?

Determine whether the study is ethical Signup and view all the answers

What is the purpose of randomisation in clinical trials?

Prevents selection bias and minimizes confounding Signup and view all the answers

What is the purpose of a placebo in a clinical trial?

Acts as an inert substitute for a treatment or intervention Signup and view all the answers

What does blinding refer to in a clinical trial?

Masking the patient and investigator from information about the treatment Signup and view all the answers

What type of blinding involves masking only the patient?

Single blind Signup and view all the answers

What effect does a placebo have on study participants?

Psychosomatic effects brought about by relief of fears, anxiety, or stress because of study participation Signup and view all the answers

Double blind in a clinical trial refers to which level of masking?

The patient and investigator are blind to the treatment information Signup and view all the answers

What is the primary purpose of pre-clinical testing in drug development?

To develop adequate data to support a decision for human trials Signup and view all the answers

Which phase of drug discovery involves the initial synthesis and analysis of a promising pharmaceutical or the development and analysis of a biopharmaceutical produced in living cells?

Lead optimisation Signup and view all the answers

What is one advantage of conducting in vivo experiments in pre-clinical testing?

Monitor the drug side effects on many organs in the body in one experiment Signup and view all the answers

What is a disadvantage of conducting in vitro experiments in pre-clinical testing?

Environment is not exactly same as in the body Signup and view all the answers

What type of data is collected during pre-clinical testing to support a decision for human trials?

Genetic toxicity and pharmacological effects Signup and view all the answers

What issue makes dissecting mechanisms of action difficult in in vivo experiments?

The drug remains in the system for a long period after administration Signup and view all the answers

Which advantage is associated with conducting in vitro experiments in pre-clinical testing?

Ability to control or modify experimental conditions Signup and view all the answers

What type of information can be obtained from conducting in vivo experiments?

Monitoring drug side effects on many organs in the body and determining drug selectivity for different organ systems Signup and view all the answers

What is a challenge associated with in vitro experiments due to the environment not being exactly the same as the human body?

Metabolite could produce a different or not effect/enhanced effect Signup and view all the answers

During which phase of drug discovery is it possible to use several different tissues in isolated tissue bath experiments?

Pharmacological profiling Signup and view all the answers

Study Notes

NPRA Regulation

The National Pharmaceutical Regulatory Agency (NPRA) in Malaysia oversees the regulation of pharmaceuticals and medical devices to ensure safety, efficacy, and quality.

Investigational New Drug (IND) Application

The IND application allows a pharmaceutical company to start clinical trials on a new drug in humans.
Its primary purpose is to ensure that there is enough evidence of safety and efficacy to justify human testing.

FDA's Function of IND Application

The Food and Drug Administration (FDA) reviews IND applications to ensure that clinical trials will not pose undue risk to participants.
The IND serves as a means for companies to provide comprehensive data about the drug, including preclinical studies and trial protocols.

Internal Review Board (IRB)

The IRB reviews and approves research protocols to protect the rights and welfare of participants in clinical trials.
It ensures that trials adhere to ethical standards and regulatory requirements.

Randomisation in Clinical Trials

Randomisation is used to eliminate selection bias, ensuring that treatment groups are comparable.
It enhances the validity of the trial outcomes by distributing variables evenly across groups.

Placebo in Clinical Trials

A placebo serves as a control to differentiate the effects of the experimental treatment from psychological effects.
Placebos help to assess the true efficacy of the drug being tested.

Blinding in Clinical Trials

Blinding refers to concealing information about the treatment assignment from participants or researchers to reduce bias.
It helps ensure the objectivity of results by minimizing expectations.

Patient-Only Blinding

Single blinding involves masking only the participant from knowing whether they are receiving the treatment or a placebo.

Placebo Effect

The placebo can induce a physiological response in participants, demonstrating the power of expectation and belief in treatment effects.

Double Blind

Double blind indicates that both the study participants and the researchers do not know who receives the treatment versus the placebo, minimizing bias on both ends.

Pre-Clinical Testing Purpose

The primary aim of pre-clinical testing is to assess the safety and biological activity of drug candidates before human trials.

Initial Phase of Drug Discovery

The initial synthesis and analysis of promising pharmaceuticals, including biopharmaceuticals produced in living cells, occur during early drug discovery phases.

In Vivo Experiments Advantage

Conducting in vivo experiments allows observations of the drug’s effects in a living organism, accounting for complex biological interactions.

In Vitro Experiments Disadvantage

In vitro testing does not replicate the entire biological context of an organism, potentially leading to incomplete data regarding drug efficacy and safety.

Data Collected in Pre-Clinical Testing

Pre-clinical testing gathers pharmacological data, toxicological profiles, and mechanisms of action necessary to justify advancing to human trials.

Mechanisms of Action Challenge

In vivo experiments can complicate the interpretation of mechanisms of action due to multiple interacting biological factors within a living system.

In Vitro Experiments Advantage

In vitro testing allows for controlled studies, making it easier to isolate specific interactions and effects of compounds.

In Vivo Information

In vivo experiments provide holistic data regarding drug absorption, distribution, metabolism, and excretion, contributing to a comprehensive understanding of pharmacokinetics.

In Vitro Environment Challenge

The artificial environment in in vitro experiments may not accurately mimic human physiological conditions, impacting the relevance of the findings.

Isolated Tissue Bath Experiments Phase

During the early phases of drug discovery, isolated tissue bath experiments can be performed, allowing the examination of drug actions on various tissues.

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Description

Test your knowledge about the process of clinical drug development, including the research and development phases in the lab, pre-clinical testing in animal models, and the various phases of testing in humans such as clinical trials. Explore the R&D drug discovery phase, including initial synthesis, analysis of promising pharmaceuticals, and the development and analysis of bio pharmaceuticals produced in living cells.