FDA Drug Approval Process

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Questions and Answers

What is the main difference between drug substance and drug product?

Drug substance is for preclinical studies, while drug product is for post-marketing surveillance.

Drug substance contains active drug ingredient, while drug product includes inactive ingredients. (correct)

Drug substance is intended for treatment, while drug product is the finished dosage form.

Drug substance only affects the body structure, whereas drug product treats diseases.

What is the primary role of an IRB in the drug approval process?

To compare standard review and priority review of NDAs

To review and approve clinical trial protocols (correct)

To manufacture generic drugs

To conduct post-marketing surveillance

Which term best describes drugs that have the same active ingredient but may differ in inactive components and appearance?

Pharmaceutical equivalents (correct)

Therapeutic equivalents

Bioequivalents

Pharmaceutical alternatives

What do priority reviews of NDAs aim to do compared to standard reviews?

Expedite the approval process for drugs addressing unmet medical needs Signup and view all the answers

Which stage of drug development involves studies on human subjects to assess safety and efficacy?

Clinical trials Signup and view all the answers

What defines a substance as a drug according to the Food, Drug, and Cosmetic Act (FDCA, 1938)?

Intended for use in the diagnosis, cure, mitigation, or prevention of disease in man or animals Signup and view all the answers

What costs and timescales are associated with drug development and approval processes?

High costs with long timescales Signup and view all the answers

What does a finished dosage form contain?

Both active drug ingredient and inactive ingredients Signup and view all the answers

What is the purpose of the Drug Discovery phase in the drug development process?

To find a promising molecule that could become a drug Signup and view all the answers

What is an important criterion when selecting a target molecule for drug development?

Target molecule's potential to interact with drugs Signup and view all the answers

What is the purpose of preclinical testing in the drug development process?

To ensure that the drug candidate is safe for human testing Signup and view all the answers

What does the Investigational New Drug (IND) application involve?

Requesting FDA authorization to administer an investigational drug to humans Signup and view all the answers

In drug development, what does ADMET stand for?

Absorption, Distribution, Metabolism, Excretion, and Toxicology Signup and view all the answers

What percentage of compounds typically survive the preclinical phase and reach clinical testing?

~0.01% Signup and view all the answers

What happens during the Drug Development phase after identifying a lead compound?

Scaling up production for clinical testing Signup and view all the answers

What is the primary goal of Target Identification in drug development?

To understand the underlying cause of a disease Signup and view all the answers

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

To allow clinical testing on humans before approval Signup and view all the answers

Who can submit a Sponsor-Investigator IND?

Physicians Signup and view all the answers

What kind of situation warrants the use of an Emergency Use IND?

Urgent life-threatening medical situations Signup and view all the answers

What is the purpose of an Institutional Review Board (IRB) in clinical trials?

To protect the rights and welfare of participants Signup and view all the answers

In what phase is an Exploratory IND Study (Phase 0) conducted?

Phase I Signup and view all the answers

When does an Investigational New Drug (IND) become effective?

30 days after submission Signup and view all the answers

What is the purpose of a Treatment IND?

To provide promising experimental drugs for grave conditions Signup and view all the answers

Who approves Investigational New Drug (IND) applications at clinical trial sites?

Institutional Review Boards Signup and view all the answers

Under what circumstance might an Emergency Use IND be used?

For urgent life-threatening medical situations. Signup and view all the answers

Study Notes

Drug Differences

A drug substance is the active ingredient that provides pharmacological activity, while a drug product is the final therapeutic formulation containing the drug substance along with inactive ingredients (excipients).
Drugs with the same active ingredient but varying in inactive components and appearance are termed "branded drugs" or "generic drugs."

Role of an IRB

The primary role of an Institutional Review Board (IRB) is to ensure the protection of human subjects involved in clinical trials, assessing the ethical and safety aspects of proposed studies.

NDA Review Types

Priority reviews of New Drug Applications (NDAs) aim for expedited assessment, facilitating faster access to important new therapies compared to standard reviews which follow a traditional timeline.

Human Subject Studies

Clinical trials involve studies on human subjects to evaluate the safety and efficacy of new drugs, typically occurring after successful preclinical testing.

Drug Definition under FDCA

A substance is defined as a drug under the Food, Drug, and Cosmetic Act (FDCA) if it is intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease, or if it affects the structure or function of the body.

Costs and Timelines

Drug development can cost upwards of billions of dollars and typically spans more than a decade from discovery to market approval.

Finished Dosage Form

A finished dosage form contains the active pharmaceutical ingredient, along with excipients necessary for formulation and delivery (e.g., tablets, capsules).

Drug Discovery Purpose

The Drug Discovery phase aims to identify and develop new drug candidates through biological research and preliminary evaluation.

Target Molecule Selection

An important criterion for selecting a target molecule is its relevance to a specific disease or condition, ensuring potential therapeutic impact.

Preclinical Testing Purpose

Preclinical testing assesses the safety and biological activity of drug candidates using laboratory and animal studies before initiating human trials.

IND Application Components

The Investigational New Drug (IND) application involves detailed documentation of drug composition, manufacturing, animal studies results, and planned human trial protocols.

ADMET Acronym

In drug development, ADMET stands for Absorption, Distribution, Metabolism, Excretion, and Toxicity, key factors in evaluating a compound’s pharmacokinetics.

Preclinical Phase Survival Rates

Typically, only about 10% of compounds survive the preclinical phase and advance to clinical testing.

Drug Development After Lead Compound Identification

Following the identification of a lead compound, extensive testing and optimization of the drug must occur, focusing on formulation and preclinical evaluations.

Target Identification Goal

The primary goal of Target Identification in drug development is to discover and validate biological targets associated with disease pathways for potential therapeutic intervention.

Purpose of an IND

An Investigational New Drug (IND) allows for the shipment of investigational drugs across state lines and initiates clinical investigations in humans.

A Sponsor-Investigator IND can be submitted by qualified investigators conducting trials that sponsor their own clinical research.

Emergency Use IND Situation

An Emergency Use IND is warranted when there is an immediate need for a therapeutically promising agent for a serious or life-threatening condition when no alternative is available.

IRB Role in Clinical Trials

The IRB’s purpose in clinical trials includes evaluating research proposals to protect the rights and welfare of participants.

Exploratory IND Study Phase

Exploratory IND Studies (Phase 0) are conducted prior to Phase I trials to assess pharmacodynamics and pharmacokinetics in a small number of subjects.

IND Effectiveness

An Investigational New Drug (IND) becomes effective 30 days after submission unless the FDA places a clinical hold on the application.

Treatment IND Purpose

A Treatment IND enables the use of investigational drugs for patients with serious or life-threatening diseases who lack other treatment options.

IND Approval Authority

Investigational New Drug (IND) applications at clinical trial sites are approved by the FDA.

Emergency Use IND Circumstances

An Emergency Use IND may be utilized when immediate treatment is necessary, and there's no satisfactory alternative, granting access to investigational drugs outside of standard protocols.

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Description

Explore the drug development and approval process, from preclinical studies to post-marketing surveillance. Learn about drug substances, drug products, IRBs, NDA reviews, and generic drug approval. Understand terms like bioequivalents, pharmaceutical equivalents, and therapeutic.