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Long before a new medication or modality is brought to market, a series of research
studies or clinical trials are conducted to make sure the
therapy is safe for patients and effective for the disease
state it is designed to treat. Clinical trials are organized
tests of medicines and new treatment options and rely on patient
volunteers who try the new or changed treatment. Clinical
trials can study medicines that have not been approved by
the US Food and Drug Administration (FDA) for sale in the
US, or drugs that are on the market but are being tested for
different dosages or even a new disease state. Generally,
pharmaceutical or biotechnology companies sponsor clinical
trials, although some health-related government agencies can
also provide funding and other resources.
Why Participate in a Clinical Trial?
The process of collecting information in the study will allow
your physician to find out more about your disease and the
effects it has on you. You may benefit from better treatment
after the trial. You may receive free laboratory and medical
testing. Even if the trial may not benefit you directly, the
information gathered may be of help to other patients with
the same condition. Many patients want to be part of the effort
to potentially reduce the suffering of others.
Are Clinical Trials Safe?
Clinical trials are conducted in a healthcare setting (a hospital
or clinic) and are typically monitored by a trained healthcare
professional. The FDA develops strict policies and guidelines
for all medical research, regardless of manufacturer, study
phase, or drug type, and also reviews clinical trials. Independent
Institutional Review Boards (IRB), a committee made up of
physicians, ethicists, and members of the general public and
administrators, also review and approve all study-related
documents, such as protocols, informed consent forms, physician
credentials and eligibility, and patient recruitment materials,
including advertisements and public service announcements.
The IRB is held responsible for determining that a trial does
not represent an unreasonable risk to patients who are participating.
Are You Eligible?
Your personal physician is responsible for your healthcare
and is the best person to help you decide whether or not you
are eligible to participate in a trial.
The Clinical Trial Process
A drug under investigation must pass through three phases
of study before it can be approved by the FDA and possibly
a fourth phase after being approved.
Phase I: The manufacturer wants to find out how the drug works
in healthy study participants. Phase I studies are primarily
concerned with assessing the drug’s safety. This initial Phase
of testing in humans is done in a small number of healthy
volunteers (20 to 100), who are usually paid for participating
in the study. The study is designed to determine what happens
to the drug in the human body (how it is absorbed, metabolized,
and excreted). A Phase I study will investigate side effects
that occur as dosage levels are increased. This initial Phase
of testing typically takes several months; about 70%
of experimental drugs pass this initial Phase of testing.
Phase II: Once a drug has been shown to be safe, it must be
tested for effectiveness. This second phase of testing may
last from several months to two years and involves up to several
hundred patients. Most Phase II studies are randomized trials.
One group of patients will receive the experimental drug while
a second “control” group will receive a standard treatment
or placebo. Often these studies are “blinded”—neither the
patients nor the researchers know who is getting the experimental
drug. In this manner, the study can provide the pharmaceutical
company and the FDA with comparative information about the
safety and effectiveness of the new drug. Only about one-third
of experimental drugs successfully complete both Phase I and
Phase II studies. Safety continues to be evaluated. Now the
study group is even larger and the trial uses advanced rating
scales and clinical measures.
Phase III: A drug is tested in several hundred
to several thousand patients. This large-scale testing provides
the pharmaceutical company and the FDA with a more thorough
understanding of the drug’s safety, effectiveness, benefits,
and the range of possible adverse reactions. In recent years,
there has been a growing industry trend to not only measure
clinical effectiveness at this phase, but also measure quality-of-life
results. Most Phase III studies are randomized and blinded
trials and typically last several years. Between 70% and 90% of drugs in Phase III studies successfully complete
this phase of testing and move to the FDA for marketing approval.
Most drugs that reach Phase III will at least be considered
for approval by an FDA advisory board.
Phase IV: These studies identify an additional use for an
already approved drug, or 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.
After the Study
When the clinical trials are complete, the FDA rules whether
to continue or halt the drug’s development. If it remains
in development, the manufacturer may build in a “compassionate
use” (also called “expanded access”) extension for the study.
This allows patients to receive drugs that have not yet been
approved by the FDA.
Usually an investigational drug can be considered for compassionate
use when the patient will benefit from the use of the drug;
there is no drug commercially available to successfully treat
the patient’s disease state; the patient currently does not
meet eligibility requirements to be treated on an investigational
protocol using this drug; no investigational protocol exists;
or the patient does not have access to a physician participating
in such a protocol. Often, patients requesting compassionate
access may include those who are too sick to participate in
the controlled studies.
The FDA has no formal regulations covering such programs.
However, for each compassionate access program, companies
make a proposal to the FDA showing that the program fulfills
minimum requirements for scientific and ethical merit and
targets a population with the disease that was included in
previous trials. At their own expense, drug companies provide
drugs free to patients and analyze and present data on their
treatment to the FDA. The drugs involved are usually in Phase
III of the drug approval process.
Questions to Ask Before You Enter a Clinical Trial
- How long will the trial last?
- Will medications prescribed by my regular physician interfere
with the study?
- Are there any tests requiring painful shots or blood samples?
What do I do if I want a friend/relative to join a study?
- Will I be paid?
- Will I be reimbursed for transportation expenses?
- What kinds of medical problems would prevent participation
in the clinical trial?
- Do I have to talk to my health insurance company before
enrolling in a study?
- What if I miss a dose?
- Can I drop out at any time?
- Are there placebos involved?
Terms Used in Clinical Trials
Baseline: A patient’s initial vital signs and clinical
symptoms are documented. The patient’s response is compared
back to baseline over time.
Clinical Trial Site: The facility where the trial is
being conducted. It can be a physician’s private practice,
a center dedicated solely to clinical research, or a hospital
Contract Research Organization (CRO): A clinical services
company involved with various steps in the clinical trial
process from study design to trial execution and data management.
Double-blind Trial: Many clinical trials are double
blind in design. This means that neither patients nor investigators/doctors
know whether an individual participant is on an active drug
or a placebo.
FDA (US Food and Drug Administration): The FDA is the federal agency responsible
for ensuring that drugs, biological products, and medical
devices are safe and effective for the general public. The
FDA reviews and oversees the manufacturing specifications
of these products, as well as the execution of clinical trials
in which these products are evaluated. The agency also ensures
that these products, once made commercially available, are
represented to the public in an accurate and informative manner.
Inclusion/Exclusion Criteria: Inclusion/Exclusion
Criteria is an actual section of a clinical trial protocol
that lists specific medications, conditions, and relevant
medical information that may, or may not, be allowable during
a trial. Age, blood pressure, and previously taken medications
are a few examples of patient-specific information that would
be compared to the inclusion/exclusion criteria of a protocol
to determine a patient’s eligibility.
Informed Consent Form: A form that outlines a patient’s
rights during participation in a clinical trial. It also discussesthe
potential risks and benefits associated with participation.
An Informed Consent form must be signed by the patient or
authorized caregiver before entrance into a study.
Institutional Review Board (IRB): An IRB is an independent committee, comprised of individuals
with diverse medical and non-medical backgrounds, that reviews
and approves all study-related documents. An IRB approves
protocols, Informed Consent forms, physician credentials and
eligibility, and patient recruitment materials, such as print
advertisements and public service announcements. IRBs work
closely with the FDA to ensure patient safety in a clinical
Open-label Trial: Later-phase studies (Phases III
to IV) often have an open-label design, meaning that all patients
receive active study medication— no placebo is dispensed.
Placebo: A substance containing no active ingredients
and having no pharmacological effects. Clinical trials often
compare an investigational medication to a placebo as a way
to differentiate patient response.
Principal Investigator (PI): The lead doctor in charge
of the clinical management of a clinical trial. As part of
the site selection process, the PI’s academic credentials
and area(s) of expertise are evaluated.
Protocol: A set of guidelines that must be followed
during a clinical trial. This includes information such as
patient eligibility, data to be collected during patient visits,
and included and excluded medical conditions and medications.
Randomization: The assignment of individuals to treatment
groups in such a way that each individual in a clinical trial
has an equal chance to be assigned to each treatment group.
(Also see Double-Blind Trial)
Study Coordinator: A key member of the clinical team
who works directly for the principal investigator. Responsibilities
usually include maintaining accurate patient documentation,
dispensing medication, and corresponding with patients.
For information on clinical trials, visit www.clinicaltrials.gov
Clinical trials for Complex regional pain syndrome on NHS Choices
Information provided by WHO International Clinical Trials Registry
ResearchMatch is a nonprofit effort that brings together researchers and people who are willing to learn more about research studies via a secure and convenient online web portal.
ResearchMatch has a simple goal—to bring together two groups of people who are looking for one another: (1) people who are trying to find research studies, and (2) researchers who are looking for people to participate in their studies. It is a free and secure registry that has been developed by major academic institutions across the country who want to involve you in the mission of helping today's studies make a real difference for everyone's health in the future.
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October 26, 2010