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Clinical Trials Demystified

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It seems that every time you turn on the television or open a magazine these days there is a new drug being marketed.

How do scientists and researchers know what works and what does not work?

Investigational drugs are first thoroughly evaluated in the laboratory with computer models.  Eventually, the Investigational drugs must be studied on human beings. This is done in a process called a clinical trial.

In 1938, Congress passed the Food, Drug and Cosmetic Act (FDCA) requiring demonstratable evidence of safety before an investigational drug could be marketed. The Kefauver-Harris Drug Amendment passed in 1962, including efficacy, as well as safety to be proven prior to approval for marketing. The Food and Drug Administration (FDA) is the regulatory agency in the U.S. which makes the final decision.

So, what is the process?

Clinical trials, also known as investigational drug studies, are carefully designed research studies to determine whether investigational drugs are safe and effective. These trials involve using people, but only after an investigational drug has undergone rigorous, extensive laboratory and animal testing.

Clinical trial protocol is a set of rules on which the trial is based. Basic protocol consists of the following information:

Who may participate?

Potential testing, medical procedures, medication and dosage schedules

Length of study

Each clinical trial proceeds through four phases. In Phase I, scientists test a small group of 20-80 people for the first time. During this phase, a procedure or drug’s safety is evaluated, a safe dosage range is determined, and side effects are determined. In Phase II, the number of participants is increased to 100-300, and safety and effectiveness are further evaluated. Phase III increases the number of participants tested to between 1000 and 3000. Safety, effectiveness and side effects are again monitored with special emphasis on safety. Phase IV occurs after the study drug or treatment has been approved and marketed. Information about their performance in various populations is evaluated as well as any side effects associated with long-term use.

As one can imagine, the government regulates every facet of these trials with strict guidelines. Every clinical trial in the U.S. is approved and monitored by an Institutional Review Board (IRB) to make sure that potential benefits far outweigh the risks involved. An IRB is made up of a committee of physicians, community advocates, statisticians, and others to ensure that patients’ rights are protected and that the trial is ethical.

In addition to government watchdogs as a check and balance, all participants will go through the Informed Consent Process where, among other topics, the following will be discussed:

Age, current medical condition, type of disease being treated, and medical history will factor into the equation also. Participants must qualify before they can join a study. Many times researchers will want perfectly healthy volunteers while, in other instances, they will want volunteers with a specific disease. Inclusion criteria are factors that allow one to participate in a study while exclusion factors prevent that from happening.

Clinical trials with human volunteers as subjects are always necessary before an experimental drug can be released and approved for use. Great strides have been made in many different areas of medicine including Multiple Sclerosis, Melanoma, Parkinson’s disease, cancer, Alzheimer’s and ALS research. Participation in these studies helps make it possible for many people to live or lead a better life and allows the safe release of new treatments and cures for many diseases to become available.

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