Medical biotechnology including diverse discipline dedicated to improving human health

Pharmacogenomicsinfluence of genetic variation on drug response in patients by correlating gene expression or SNP with a drug's efficacy or toxicity Gene therapy use of DNA as a pharmace

Pharmacogenomics(influence of genetic variation

on drug response in patients by correlating gene expression

or SNP with a drug's efficacy or toxicity)

Gene therapy (use of DNA as a pharmaceutical agent

to treat disease)

Diagnostics (determine or identify a possible disease or disorder)

Drug production (use for treatment of infectious, cardiovascular diseases…)

Medical biotechnology

including diverse discipline dedicated to improving human health

Diagnostics and gene therapy

It mostly focuses on:

Disease prevention

Disease diagnosis,

Disease treatment.

A disease is a condition that impairs normal bodily functioning Examples of diseases include:

heart disease, cancer, diabetes, HIV/AIDS, inherited diseases,…

Pharmaceutical industry

The Food and Drug Administration (FDA) is a US regulatory agency

that oversees food, drugs and devices It also monitors clinical trials and

approves new drugs that have succeeded

Pharmaceutical companies are those licensed to discover, develop,

market, and distribute drugs

Drugs (according to the FDA) are defined as substances intended for use in

diagnosis, cure, treatment, or prevention of disease

Drug Production

1. Screening: is a laborious and costly process—only 1 in every 5,000 to 10,000 compounds screened eventually becomes an approved drug

To screen an antibiotic, it is first placed in a bacterial culture If the antibiotic is effective, it is next tested on infected laboratory animals to study the safety and efficacy of the new drug

2 Selecting: A new drug is selected for testing on humans only if it either promises to have therapeutic advantages over drugs already in use or is safer.

3 Clinical investigations, or "trials," of the drug on human patients First: a small group of healthy volunteers to determine and adjust dosage levels, and monitor for side effects.

Second: a larger group of volunteers or carefully selected patients.

Third round: a very large panel, sometimes upwards of 10,000 individuals.

4. Approval of The U.S Food and Drug Administration's (FDA), and Center for Drug Evaluation and Research (CDER) for commercial use.

The entire process, from the first discovery of a new compound to FDA approval, can take over a decade and cost hundreds of millions of dollars

Preclinical Testing

The first step is to identify a potential drug candidate, called a lead compound.

Once a lead compound has been identified in the lab, it begins years of testing to evaluate its safety and efficacy

In vitro studies are carried out to identify the way it interacts with its

target

After that, tests on model organisms (animals that have the targeted disease) take place This usually lasts around 6-7 years.

Preclinical Testing

Many preclinical tests include pharmacokinetics - the study of

how drugs move through living organisms

Four processes are examined in pharmacokinetic studies:

absorption, distribution, metabolism & excretion.

Clinical Trials

• Begin after the use of model organisms

• Most are classified into 3 phases: I, II, & III

• All participants go through informed consent process (given facts

about risks & benefits)

Clinical trials are a process of testing products prior to approval of a drug

or treatment plan for widespread use in humans

Phase I

• Initial studies of drug in a small number (20 -80) of healthy volunteers to determine its safety

• Intended to determine safe dose range

• Exceptions are made for highly toxic treatments (ex cancer or HIV) - in this case appropriate patients selected

•Pharmacokinetic studies determine dosing & limitations of use

Take 6 months - 1 year to complete

Phase II

• Larger groups of people with the disease (100 - 300) are used to study

efficacy

Usually about 2 years to complete

Phase III

• Medicine is tested in large, randomized, controlled trials with larger numbers

(1000 - 5000) of patient volunteers using blind & double blind studies

events)

Can take 1-4 years.

New Drug Application (NDA)

• If findings from clinical trials prove safe and effective, then company files

new drug application (NDA) with FDA

• Data must support that drug is more effective than placebo (a substance

with no pharma-cological effect) or drug offers alternative to existing

medication

• About 80% of drugs that make it to Phase III studies get approved.

Phase IV/

Ongoing studies

• Even after a product is on the market, studies & safety monitoring continue

• FDA requires periodic reports including cases of adverse events

• Phase IV studies (post-marketing) evaluate long-term safety, how it affects particular

groups (elderly/children), & comparisons with established treatments

Continues for years

Occasionally, new indication (intended use for drug) may be found.

Drug development process

Pharming (farming and pharmaceutical ) refers to the useful pharmaceuticals that are product of exogenous

genes transfered into host animals or plants that would not express those genes.

Recombinant pharmaceuticals :

Large quantity, Purified,

Eating (plants) or drinking the milk

Number (and percentage values) of recom-binant proteins approved as

biopharma-ceuticals in different production systems

Accumulated number of recombinant biopharmaceuticals obtained in different production systems.

Recombinant pharmaceuticals

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