CLINICAL TRIALS: AN EXPLANATION

Background to Clinical Trials

In the complex field of health-care, clinical trials are undertaken with a key, specific goal in mind; namely, to allow for the collection and analysis of safety data and efficacy (defined as the capacity to produce an effect) data on new and experimental drugs, medicines and medications – and for a whole range of illnesses, diseases and conditions.

These trials may only commence once satisfactory information has been carefully gathered and scrutinized on the quality of the product and its non-clinical safety; and approval for the use of the product has been officially granted and authorized by the relevant, governing health body.

Largely depending upon the particular nature, description and intended use of the medication at issue, those tasked with coordinating and undertaking the initial research generally first enlist the help of healthy volunteers and/or a select number of patients who will take part in small-scale studies.

And, if all progresses well, and adverse side-effects or unforeseen serious problems do not occur, these particular studies will then be followed by others of a larger size and wider scope. They may possibly even be international in nature, with the medical and scientific communities of several nations playing a variety of roles in the clinical trials.

As a result of the fact that the financial costs involved in clinical trials can often be significantly high, it is generally the case that those same costs become the responsibility of the relevant sponsor of the trials – which may be a biotechnology company or a pharmaceutical firm.

When it comes to the first-step – namely that of planning and coordinating a clinical trial - the relevant sponsor has to first identify the particular type of medication that is to become the subject of the trial.

Along with a panel of accredited experts (who are typically respected, credentialed physicians who are widely regarded for their experience at a clinical level), the sponsor will be responsible for making the decision regarding how - and under what particular circumstances - the new medication may offer benefit to the patient, and which particular kinds of patients might benefit most of all.

Defining the Trials:

The U.S. National Institutes of Health (NIH) officially organizes the nature of clinical trials into a series of clearly delineated areas.

They are as follows:

Prevention Trials: Basically, these are formulated to uncover more effective ways to (a) try and prevent disease developing in those people who have never suffered from the disease in question, and (b) prevent a recurrence of the disease in those patients who have previously been diagnosed with the relevant condition.

Screening Trials: These are focused upon determining the most advantageous ways to uncover particular medical conditions and diseases.

Diagnostic Trials: These are undertaken with the goal of finding superior ways and means of accurately diagnosing specific diseases and illnesses.

Treatment Trials: The purpose of treatment trials is to (a) evaluate new, experimental, and sometimes radical treatments, (b) to study newly-formulated combinations of various drugs, and/or (c) to explore new avenues of treatment.

Quality of Life Trials: Essentially, these are considered ways and means designed to significantly offer improvement and quality of life in those patients affected by chronic, serious illness and disease.

Compassionate Use Trials: These particular trials are offered to those patients whose options with the majority of other tried-and tested remedies have not proven to be successful.

An absolutely vital aspect of any and all clinical trials is to ensure that those who take part in such trials are carefully protected via a signed document that falls under the category of “Informed Consent."

Essentially, and from a specifically legal perspective, Informed Consent is the process by which a person is advised of important, central facts and procedures that the trial may potentially involve – thus giving the person the opportunity to carefully decide if they wish to take part in the trial or not.

Informed Consent documents and forms should always fulfill certain particular criteria, including the dates of the trial, its length in terms of days, weeks, months etc, its specific purpose, and the potential hazards and risks to health that may possibly be in evidence during the course of the trial.

It is important to note that an Informed Consent form is not a legally binding and restrictive contract: the person at issue is free to leave the trial at any time and for any reason that he or she wishes.

Clinical Trials: The Phases

Phase 1 Trials: These are defined as the very first stage of actual testing in human-beings. Generally, a relatively small group of healthy volunteers (in numbers from 20 to 80, usually) are selected for Phase 1 trials. This phase will typically include trials to carefully evaluate the safety and tolerability of the drug at issue.

Phase 2 Trials: At this stage, trials are undertaken on somewhat larger groups of individuals, and perhaps as many as several hundred; again to try and ascertain how effective the particular drug may be, and to continue the work that was initiated in Phase 1.

Phase 3 Trials: Generally, up to 3,000 patients may take part in such trials; the goal of which is to ascertain how effective the particular drug may be, in comparison to the currently available, and most successful, other forms of treatment.

Phase 4 Trials: Known also as Post Marketing Surveillance Trials, Phase 4 Trials are focused upon the continued technical-support for a new drug after permission for its sale and use has been officially granted.