Clinical Trials - Part I

A few days ago the buzz was that an oral medication that was “in trial” for treatment of pulmonary hypertension, failed – not true, actually it hit a bump in the road. There is a lot that goes into a having a trial or a study; the so called research. There are rules and regulations, procedures to be followed and I will try to list a few.

Trials or research programs have certain guidelines that are followed – the participant or patient must be sick enough to be in the program but strong enough to be able to endure the process.

The FDA requires that people be told:

• That the study involves research of an unproven drug, biologic (such as a vaccine, blood product, or gene therapy) or device
• The purpose of the research
• How long the participant will be expected to participate in the study
• What will happen in the study and which parts of the study are experimental
• Possible risks or discomforts to the participant
• Possible benefits to the participant
• Other procedures or treatments that might be advantageous to the participant instead of the treatment being studied
• That the FDA may look at study records, but the records will be kept confidential
• Whether any compensation and medical treatments, if any, are available if the participant is injured, what those treatments are, where they can be found, and who will pay for the treatment
• The person to contact with questions about the study, participants' rights, or if the participant gets hurt
• That participation is voluntary and that he or she can quit the study at any time without penalty or loss of benefits to which the participant is otherwise entitled.
• And of course, the infamous “consent form”

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:

• In Phase I trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
• In Phase II trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
• In Phase III trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
• In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

Trials can be as short as 3 months or may last a few years.

More to follow within the next few days.

Merle - OHPA
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