What Are Clinical Trials?
A clinical trial is a drug study
sponsored by a pharmaceutical or biotechnology company.
There are cases, however, where health-related
government agencies provide funding and other resources
for a clinical trial. The purpose of these studies is to
find out whether a medication is safe to use and
effective against various diseases or medical
conditions. In order to study the medication, several
questions need to be answered first. For example, what
patient population or disease is the drug meant to
treat? What criteria should be used for accepting
participants into the study? What general and
disease-specific information are the study doctors going
to obtain? Essentially, once these and other important
study questions are answered, the study doctors
(investigators) are chosen, the regulatory documents are
approved, and the study is ready to begin.
Why are clinical trials important?
There are a several reasons why clinical trials are
important. First, the participant may have a positive
response to the study medication, and their disease or
condition may improve. In addition, participants receive
free laboratory and medical testing. More often than
not, the study medication is in development because it
works differently than other drugs on the market, and
therefore, it may present an alternative course of
treatment for the patient. Second, participation in a
clinical trial helps manufacturers make informed
decisions about whether to pursue getting a particular
drug approved by the Food & Drug Administration (FDA).
Finally, data from one patient (who completes the entire
study) can be an important part of a drug development
program. In the best case scenarios, these data can help
get an exciting new drug approved by the FDA, and
ultimately, continue the industry's efforts in
developing medications that are safer, more effective,
and work faster than any before them.
How are a participant's rights and safety protected?
The FDA is the governing agency that develops the
policies and guidelines for all medical research,
regardless of manufacturer, study phase, or drug type.
There are also independent Institutional Review Boards (IRB)
that review and approve all study-related documents,
such as protocols, Informed Consent forms, physician
credentials and eligibility, and patient recruitment
materials, such as print advertisements and public
service announcements.
What are the phases of the clinical trial process?
There are three primary phases an investigational drug
has to go through before it can be approved by the FDA,
and a fourth phase that an approved drug may enter.
Phase I
In this phase of a clinical trial, the manufacturer
wants to find out how the drug works in healthy study
participants. Mode of action (how the drug exerts its
effects), safety, and side effects -- these are some of
the main issues that are of the utmost concern to drug
makers at this stage of the clinical trial process. It
is important to note that in Phase I studies, the
overall safety of the medication in patients has not
been established.
Phase II
A drug reaches Phase II only when the FDA has reviewed
the Phase I data and concludes that the drug is safe for
patients, and that its clinical activity may be
beneficial against a particular disease or condition. At
this point, a larger group of patients are enrolled and
condition- or disease-specific rating scales are used to
record data.
Phase III
This is an important turning point for drug
manufacturers. The medication has already past the
rigorous testing and review process required by the FDA,
and now it's ready to be studied in an even larger
patient population, with even more advanced rating
scales and clinical measures. In recent years, there has
been a growing industry trend to not only measure
clinical effectiveness at this phase, but also measure
"real world" results. For example, if a patient shows
clinical improvement after entering the study, how are
their activities of daily living improving (e.g., the
patient can work in the garden more often or attend
their child's games on a regular basis)? Most
medications that reach Phase III will at least be
considered for approval by an FDA advisory board.
Phase IV
At this phase in development, the drug has already been
granted FDA approval. Phase IV studies are often
performed to either identify an additional use for an
already approved drug, or to gather additional safety
information from a larger group of patients. In some
cases, Phase IV studies are implemented to establish
effectiveness in a subgroup of patients, for example,
patients over age 65. |
