By: MSF Staff and reviewed by the MSF Medical Advisory Board
It is often said that good things take time. Clinical trials may be a prime example. In the U.S., for example, it usually takes just over fourteen years and $500 million dollars for an experimental drug to make the arduous journey from laboratory to patient.
Sponsored by a pharmaceutical company, a health-related government agency, or sometimes a university, the purpose of a clinical trial is to determine the safety of a particular medication as well as its efficacy against various diseases and medical conditions.
Before a new drug can be studied in terms of its potential effect on humans, it is first subject to pre-clinical test-tube analysis and/or animal studies to establish toxicity. This information is then assessed for risk and benefits to determine its acceptability for humans.
Next, the sponsor of the proposed drug will submit a plan to the FDA for testing on humans. This must include all pertinent information regarding animal testing, drug composition and manufacturing, qualifications of study investigators as well as a guarantee of protection for trial participants. This information is called the Investigational New Drug Application (IND). The treatment trials, which fall into three distinct phases, can now begin.
Phase I provides initial exposure of humans to a particular drug. This phase is concerned primarily with how the drug works in healthy participants. Is it safe? What are the side effects? What will the appropriate dosage be? The FDA will then evaluate all data before the drug progresses to the next phase.
Phase II incorporates a larger group of participants in order to better assess the efficacy of the drug. These participants are patients exhibiting a particular condition or disease. Rating scales are used to record data specifically geared toward the effects of the drug on the patient's disease or condition, as well as side effects.
Phase III compares proposed treatments with existing drugs. The FDA verifies all information obtained from the clinical trials thus far and also inspects the manufacturing facility to ensure that the highest possible standards of quality are being met. Much of the information provided on medication labels or inserts will be obtained during this phase.
Despite the risks involved, there are countless reasons why participation in these trials can prove beneficial. Participants receive free medical testing from specialists who have the most current information on their disease. There is the potential for a positive response to the medication, leading to improvement of a participant's condition. Also, manufacturers become more knowledgeable, their decisions more educated, creating safer, more effective medications.
(Last reviewed 7/2009)