Biotechnology and Pharmaceutical Investor Research
Phase I Trials


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Phase I (or Phase 1) clinical trials are the first step in a series of trials on human beings required by the FDA (Food and Drug Administration).

Biotechnology investors should be aware that successful Phase I trials are nearly meaningless to the value of a company's stock. Of course success is better than failure, but most drugs and therapies that pass Phase I trials fail during Phase II or Phase III trials. A Phase I trial in motion or completed only indicates that a company has a drug in its pipeline which it believes is worth the expense of clinical trials.

Phase I is first of all about safety. In fact, Phase I trials are usually conducted in healthy human subjects. Volunteers are closely monitored for side effects and adverse reactions. Usually there is an escalation of doses to determine what a maximal dose should be. Effectiveness of drugs may depend heavily on dosage, but higher dosages usually mean more side effects.

Phase I is also about science. Before starting a Phase I study medical scientists have usually done computer modeling, test-tube studies, and animal trials. They have hypotheses about what a substance will do in the human body. By carefully monitoring the metabolisms of patients they can test those hypotheses. A goal from these metabolic studies is to get enough information on the drug's pharmacological effects to allow for a well-designed Phase II study.

If the Phase I human subjects do have the target disease or condition, the trial can be used as an initial indicator of efficacy (effectiveness). But since these trials usually enroll only 20 to 80 patients the statistical significance of any efficacy results is limited.

It is also important to note that safety concerns do not go away just because a Phase I trial has good results and a Phase II trial is planned. Diseased subjects may have side effects and adverse reactions that are not seen in healthy Phase I subjects. Even drugs that have been approved for marketing have been recalled or had their indications limited because of adverse effects discovered when the drug was given to large numbers of patients.

More information:

FDA on Phase 1

FDA guidance for applying to do a Phase 1 study