According to FDA (Food and Drug Administration) “Clinical trials, also known as clinical studies, test potential treatments in human volunteers to see whether they should be approved for wider use in the general population. A treatment could be a drug, medical device, or biologic, such as a vaccine, blood product, or gene therapy. “

Drugs development

What is a drug?

A drug is a substance (or mix of substances) which when administrated to a live organism will change some physiologic processes, or simply being put a drug taken will make some change into the body.

How are the drugs design and develop?

The major steps followed are:

– the discovery of the drug

– preclinical trial

– clinical trial

Discovery of the drug Begins in the lab when a drug is discovered.

From several thousands of drugs created only one will reach the market as an approved drug.

The Preclinical Trial

First, the drug is tested in the lab in vitro for optimizing the dosage for the preclinical trial.

The preclinical trial is done in vivo and in vitro to determine the basic characteristic of the drug.

(In this stage is tested the effect the drug have on the organism and the effect of the body on the drug, drug absorption, distribution, metabolism, excretion, and toxicity).

If the preclinical trial is successful the drug will go on the clinical trial.

The Clinical Trial

Clinical trials are testing the newly developed drug in human volunteers either healthy or with diseases.

The phases in this of the clinical trial are:

• The zero phase

– a very small numbers of volunteers (around 10)

– the volunteers are preferable to be healthy and the dosage is very low to prevent the possible toxicity

– the duration of this phase is short (few days)

– the main objective is to monitor the effects of the body on the drug, and if possible the effects of the drug on the body

• The first phase

– the numbers of volunteers are increasing to few tens (around 20)

– they can be healthy or with the specific disease

– the dosage is increased but is still subtherapeutic levels

– the duration of this phase is greater than the previous one (couple months)

– the goal is the evaluation of the safe dosage, toxicity, and parameters for clinical monitoring

• The second phase

– the number of volunteers is increasing to a few hundred

– the volunteers have the specific disease

– at this stage, the volunteers are divided into 2 even 3 groups receiving placebo or standard treatment or/and the full dose of the new drug depending on the protocol

– this phase can take between few months to a couple of years

– the main objective is to compare the new drug efficiency vs standard treatment or placebo

– now is expected to be seen the most common side effects

• The third phase

– the number of volunteers is increasing to a few thousand

– the volunteers have the specific disease

– same as the previous stage the volunteers are divided into 2 even 3 groups receiving placebo or/and the full dose of the drug depending on the protocol

– this phase can take up to a few years

– the main objective is to confirm the new drug efficiency and safety vs standard treatment or placebo

– now is expected to be seen some rare side effects

– the successful completion of the 3rd phase will allow the drug to go on the market for general population use.

• The fourth phase or post-market research

– the number of volunteers is increasing to several or even tens of thousands

– the volunteers have the specific disease

– same as the previous stage the volunteers are divided into 2 even 3 groups receiving placebo or/and the full dose of the drug depending on the protocol

– now is expected to be seen the rarest side effects

Who is involved in Clinical Trials?

As the process of Clinical Trials became more complex so the parties involved.

The main parties are:

The sponsors – the entity who paid for the studies {the sponsor can be a governmental agency like:

National Heart, Lung, and Blood Institute (NHLBI) and other National Institutes of Health (NIH) Institutes and Centers U.S. Departments of Defense and Veterans Affairs; private companies – pharmaceutical corporations; universities; and nonprofit organizations.

The participants or the volunteers – the people who meet criteria for the study and consent to participate.

The overseer or FDA – the main role to keep everybody safe, is monitoring and follow the study in order to keep all the parties out of harm way.

What is the role of government in Clinical Trials?

The main role of government related to Clinical Trials is to ensure the safety of the public.

FDA is a federal agency and the main way government can monitor, and regulated the process of clinical trials.

FDA is the one which needs to approve the new product (drug) on the US market.

As FDA stated, “The drugs that do must undergo the agency’s rigorous evaluation process, which scrutinizes everything about the drug – from the design of clinical trials to the severity of side effects to the conditions under which the drug is manufactured.”