BACE- Regulatory Compliance and Quality Systems

NDAs stand for

new drug applications

BLA stands for

biologics license applications

5 steps of drug development process in the US

1) Early drug discovery

2) Preclinical studies

3) Clinical development

4) FDA review

5) Post-market monitoring

What does “Preclinical studies” do in drug development

Before testing a drug in people, researchers must find out whether it has the potential to cause serious harm, also called toxicity.

2 types of preclinical research

1) in vitro

2) in vivo

GLP stands for?

Good laboratory practices

In vivo

studies conducted within living organisms

In vitro

studies occur in controlled, artificial laboratory environments like test tubes or petri dishes ( a good way to remember this is that, “in vitro” means “in glass” in latin)

what does ‘good laboratory practices’ mean

a set of international, mandated, non-clinical safety study standards ensuring the quality, integrity, and reliability of data used for regulatory approval of products like pharmaceuticals and chemicals.

The GLP regulations are found in ?

21 CFR Part 58.1 (Good Laboratory Practice for Nonclinical Laboratory Studies).

What do researchers do after clinical testing?

researchers review their findings and decide whether the drug should be tested in people.

What does ‘clinical research’ mean

studies, or trials, that are done in people. (after pre-clinical studies)

IND stands for?

Investigational New Drug Process

what does ‘Investigational New Drug Process’ mean

a process where researchers must go through before clinical research begins.

Drug developers are free to…. at any point in the drug development process.

to ask for help from FDA

The FDA review team has …. days to review the original IND submission.

30

IND protects volunteers who participate in clinical trials from .. in clinical trials.

unreasonable and significant risks

What is the developer responsible for, during clinical trials?

informing the review team about new protocols, as well as serious side effects seen during the trial.

What do researchers need to do after a clinical trial ends?

researchers must submit study reports.

What do developers need to do before filing a marketing application?

they must have adequate data from two large, controlled clinical trials.

How many phases do clinical trials have?

four,

• Phase 1 - Checking safety and dosage

• Phase 2 - Testing effectiveness and side effects

• Phase 3 - Confirming efficacy and comparing to standard treatments

• Phase 4 - Tracking long-term effects after approval

Goal of phase 1 in clinical trials

learning how the treatment works for humans and what is the best dose of the treatment

Goal of phase 2 in clinical trials

learn about how safe the treatment is for people to use

Goal of phase 3 in clinical trials

learn if the treatment can prevent/diagnose/treat the disease, also more about safety and side effects

Goal of phase 4 in clinical trials

FDA approve treatment for use in public, and researchers continue to look at the treatment’s benefits, side effects, and the best way to use the medicine

How long does phase 1 of clinical trials take?

less than a year

How long does phase 2 of clinical trials take?

a few months to 2 years

How long does phase 3 of clinical trials take?

1-3 years

How long does phase 4 of clinical trials take?

many years

how many participates are typically in phase 1 of clinical trials

20-100 who may be healthy or have the disease

how many participates are typically in phase 2 of clinical trials

~300 who have the disease

how many participates are typically in phase 3 of clinical trials

300-3000 people who have the disease

how many participates are typically in phase 4 of clinical trials

thousands of participants who have the disease

NDA stands for?

New drug application

what is a NDA

A application from drug developers to the FDA for them to approve/reject the new drug for markets

What needs to be included in a NDA

EVERYTHING ABOUT THE DRUG, from pre-clinical trials to phase 3 data. Also proposed labeling, safety updates, drug abuse info, patent info, any data

How long does it typically take for the FDA review team to make a decision on whether to approve the drug

6-10 months

How do new drugs get FDA approved?

developers send a NDA to the FDA review team

What does ‘labeling’ mean in the drug approval process

After FDA approval, FDA and developer work to make/refine prescribing info

is it possible to have complete info about the safety of a drug at the time of approval

no

when does the “true picture of a product’s safety” actually show up

the product’s lifetime in the marketplace,

What does regulatory compliance mean

the process of following the laws, regulations, and standards that apply to an organization’s industry and operations

primary regulatory body for drugs in the US

FDA

primary regulatory body for drugs in the european union

EMA

primary regulatory body for drugs in the US japan and china

PMDA and NMPA respectfully

FDCA stands for

Federal Food, Drug, and Cosmetic Act

EMA stands for

European Medicines Agency

PMDA stands for

Pharmaceuticals and Medical Devices Agency

NMPA stands for

National Medical Products Administration

product categories in biotech

Biologics, pharmaceuticals, and medical devices

Biologics category in biotech products

Includes vaccines, blood products, gene therapies, and monoclonal antibodies. These are typically subject to stringent regulatory oversight.

Pharmaceuticals in biotech products

Includes drugs produced through biotechnological methods, such as recombinant proteins and biosimilars.

Medical devices in biotech products

Biotechnology often intersects with medical devices, particularly with in vitro diagnostics and implantable devices.

Regulatory pathways for a biotech product

preclinical research, clinical trials, and marketing authorization

GCP stands for

Good clinical practice

Compliance requirements for biotech products

GLP, GCP, CMP, GDP

What is GCP

compliance requirement that covers the design, conduct, and reporting of clinical trials.

What is GMP and what does it stand for

Good manufacturing practice: compliance requirement that ensures that products are consistently produced and controlled to quality standards.

What is GDP and what does it stand for

Good Distribution Practice: compliance requirement that relates to the proper handling, storage, and distribution of biotechnology products.

GCMP stands for? and what does it mean

Current good manufacturing practice: same thing as GMP

There must be … every time a product is made.

systems to provide documented proof that correct procedures are consistently followed at each step in the manufacturing process

CFR stands for?

Code of Federal Regulations

what is CFR

the codification of the general and permanent rules published in the Federal Register by the executive departments and agencies of the Federal Government.

CFR is enforced by what federal agencies

FDA, EPA, IRS, DHS

EPA stands for?

environmental protection agency

IRS stands for?

internal revenue service

DHS stands for?

department of homeland security

How is the CFR organized?

divided into 50 titles that represent broad areas subject to Federal regulations
→ Each title is divided into chapters that are assigned to various agencies issuing regulations pertaining to that broad subject area.
→ Each chapter is divided into parts covering specific regulatory areas.
→ Large parts may be subdivided into subparts
→ Each part or subpart is then divided into sections — the basic unit of the CFR
→ paragraphs or subsections.

example of a CFR citation: 21 CFR 211.67 (a), what does the number ‘21’ mean

the title

example of a CFR citation: 21 CFR 211.67 (a), what does the number ‘211’ mean

the part

example of a CFR citation: 21 CFR 211.67 (a), what does the number ‘.67’ mean

the section

example of a CFR citation: 21 CFR 211.67 (a), what does the ‘a’ mean

the first paragraph or subsection

What does 21 CFR 211 show

the 10 fundamentals of CGMP