people known to be taking the drug, but they also
could uncover potential new uses for the therapy
to treat other conditions.
The Clinical Trial Process
+ + Strict protocols direct each study.
Each trial follows an extensive and carefully
monitored protocol, which is a plan that spells out
the entire study process. This details how many
volunteers are needed, who is eligible, what tests
will be used to evaluate participants’ health at the
beginning and throughout the study, how long the
study will last, what information will be recorded
and other considerations. Similar to how a team
delivers your medical care, a staff of doctors,
researchers, nurses and study coordinators carry
out clinical trials. The principal investigator,
typically a doctor, leads the entire team and trial.
+ + Informed consent is obtained before you
enroll. Before you join a study, you’ll meet with
the trial team so they can discuss relevant aspects
of the protocol, answer your questions and go
over a consent form. This document, referred
to as informed consent, lists your rights as a
volunteer, the potential risks of participation
and what you can expect during the study. Once
you understand what the trial entails and you
feel comfortable and want to enroll, you sign the
informed consent. This isn’t a binding contract;
you can still withdraw at any time for any reason.
(You also can refuse study tests or treatments,
but doing so may exclude you from continuing in
the trial.)
conference. (You can inquire with your trial team
and search online for the published results.) At trial
conclusion, teams also determine next steps for
the therapy. They may recommend evaluation in
the next phase of clinical trial testing, or, equally
as important, they may suggest no further study
because safety and efficacy data are not compelling.
People who have enrolled in a placebo-controlled
trial often wonder whether they received placebo
or the study drug. If the protocol allows, this
information is made available when all participants
complete the trial. Some participants also want
to know if they can continue to get a study drug
once a trial ends. Often this is not possible, but the
trial protocol and informed consent documents
spell out in advance what will happen should this
question come up. And if the therapy continues
to the next phase of clinical testing, people who
have participated in earlier research may be given
an option to continue on with it. Because of many
factors, though, enrolling in the next study phase
may not guarantee that a person will continue
the study drug. (A new study may require re-
randomization, for example.)
+ + Independent committees review and approve
studies. An Institutional Review Board (IRB) —
an independent group of physicians, researchers
and others (usually at least one “non-scientific”
person who represents the patient voice) —
evaluates and approves each study’s protocol and
informed consent document. This committee also
monitors ongoing study activities. The IRB is in
place to protect the rights and welfare of people
participating in a study.
What Happens When the
Study Ends
Once a clinical trial is over, the trial team analyzes
the data, distills key findings, and publishes the
results or presents them to other researchers at a
Chapter 1 — Clinical Research Basics
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