From the Lab to Market

Unit 3.04

Understanding Biotechnology research & Development

How a New Drug Gets to Market

Americans have access to probably the safest and most advanced pharmaceutical system in the world

The FDA is the main consumer watchdog for this system.

The FDA evaluates new drugs before they can be sold to make sure they are safe and effective for their intended use

How a New Drug Gets to Market

Development of a new drug is a lengthy process

Drug Discovery & Testing

Drug development begins in the laboratory, where scientists look for compounds that treat or prevent disease

Biopharmaceuticals are naturally occurring human proteins or modified human proteins produced from generally engineered cells grown in culture

Drug Discovery & Testing

Once a company finds a drug, it thinks is promising, it begins pre-clinical studies that test the drug on animals or in other non-human test systems

Since animals have a much shorter lifespan than humans, valuable information can be gained about a drug’s possible toxic effects over an animal’s life cycle and on its offspring

Clinical Trials

Human studies designed to distinguish a drug’s effect from other influences

If the drug appears to be safe and effective after clinical trials with several thousand subjects the company files a New Drug Application (NDA) with the FDA before the drug can go to market

Clinical Trials

Phase 1 trials: The drug is tested for its interaction with the human system,

including the way it is absorbed, distributed in the body, metabolized and excreted.

These trials usually involve normal, healthy volunteers and take about a year to complete

Clinical Trials

Phase 2 Trials: Pilot studies that begin to define the effectiveness and

safety of the drug in selected populations of patients with the disease or condition to be treated, diagnosed or prevented.

Clinical Trials

Phase 3 Trials: Expanded clinical trials intended to gather additional

evidence of effectiveness for specific indications and to better understand safety and drug-related adverse effects

Clinical Trials

Phase 4 Trials: Studies that occur after a drug has received FDA approval to

be marked.

Performed to determine the incidence of adverse reactions, to determine the long-term effect of the drug

Study a patient population not previously studied

Marketing comparisons against other products and other users

NDA Review: How Long Does This Phase Take?

NDA is a comprehensive statement with information about the drug (thousands of pages long), companies must submit three copies of the report

The FDA is 60 days to conduct a preliminary review and decide if it has enough information to proceed with the NDA review

The NDA decision making process can take anywhere from 2 months – 7years, (average is 2 years)

Post-Marketing Surveillance

Monitor the ongoing safety of marketed drugs

Accomplished by reassessing drug risks based on new data collected after the drug is marketed and by recommending new ways of trying to most appropriately manage that risk

Pharmaceutical &

Biopharmaceutical Industries

From the beginning of civilization, people have been concerned about the quality and safety of foods and medicines

Regulations of food in the United States dates from early colonial times, and one of the earliest food and drug law was enacted by the state of Massachusetts in 1785

Many tragic events and concerns about food and drug safety have led to regulations about developing and manufacturing new products/medications

What are Regulations?

General and Permanent rules issued by the various departments and agencies of the executive branch of the federal government are published in the Code of Federal Regulations

Regulations state what may or may not be done or how something must be done and are enforceable by law

Regulations are vital to ensure quality and safety of the drugs marketed in the United States