Clinical Trials Demystified 
By Michael Plontz


It seems that every time you turn on the television or open a magazine these days there is a new drug being touted as the new miracle cure-all. This is especially true of allergy and heartburn medications. How do scientists and researchers know what works and what does not work? Potential new drugs are first thoroughly evaluated in the laboratory with computer models. After this phase of testing is completed, additional research is then performed on laboratory. 

Eventually, the new drugs must be tested on human beings. This is done in a process called a clinical trial. It may sound as though a research facility is being sued, but itís even more complicated than that.

It was in 1938 that the Federal Food, Drug and Cosmetic (FDC) Act was passed which made evidence of safety mandatory before marketing was possible. The Kefauver-Harris Drug Amendment passed in 1962, required that a drugís effectiveness, as well as its safety, had to be proven. The Food and Drug Administration (FDA) makes the final decision.

So what is the process? Clinical trials, also known as investigational drug studies, are a carefully designed research study to determine whether new treatments or drugs are safe and effective. These trials involve using people, but only after extensive laboratory and animal testing show positive results.

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

  • Who may participate

  • Test, procedure, 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. A procedure or drugís 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 amount of people tested to between 1000 and 3000. Safety, effectiveness and side effects are again monitored with special emphasis on safety. Phase IV occurs after the drug or treatment has been marketed. Information about their effect 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 single 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 patientsí rights are protected and that the trial is ethical. 

In addition to government watchdogs as a check and balance, informed consent must be given by any and all participants. This would include:

  • Why is the research being conducted

  • What is the desired end result

  • What will the trial entail and how long will it last

  • What are the risks

  • What are the benefits

  • Are there other treatments available

  • May I leave the trial at any time (the answer here is absolutely)

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 researcher 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. Once the study is set up and the participants are chosen, the clinical trial resembles any other experiment using a control group and either a blind or double-blind study. 

Clinical trials with human volunteers as subjects are always necessary before a drug can be released and approved for use. Great strides have been made in many different areas of medicine including multiple sclerosis, Parkinsonís disease, cancer and Alzheimerís and HIV and AIDS 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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