The offices of Allergy & Asthma Consultants of NJ-PA conducts various
Research Studies in Allergic Disorders and Asthma.
If you are interested in participating in any of our Research Studies,
please call our Research Co-coordinator Tracy Devito at
The Importance of Clinical Research
Every day, thousands of people volunteer to
take part in clinical research trials. It is because of their willingness to
participate in these studies that modern medicine is able to meet the
challenge of continuing to find new, improved treatments and cures. But what
exactly are clinical research trials? Basically, these are scientific
studies of medication use in people, designed to answer specific questions.
These studies are the basis by which medical researchers and pharmaceutical
companies develop new and improved therapies for human diseases.
People choose to participate in clinical
research for a variety of reasons. Some enjoy the opportunity to further
science; others desire early access to new, still experimental medications;
some use clinical research to gain access to highly sought after physicians
or specialists; or to obtain medical care not covered by their insurance
plans. Others simply enjoy the camaraderie, education and close medical
attention afforded by frequent visits to the site where the research is
being conducted. Patients may be paid for their participation in studies.
Should you volunteer?
Clinical research is not for everyone. Some people may be hesitant to
participate in research due to concerns about the safety of the study
medication and how well it may work, their own time constraints and various
other concerns. To help you make a more informed decision regarding your own
or your child’s participation in clinical research studies, it’s important
to understand the process of drug development; U.S. Food and Drug
Administration (FDA) requirements for the development and approval of new
medications; and federal regulations that protect the rights of clinical
New drug testing
To ensure that drugs are safe for humans, drug testing is done as follows:
- Drugs are initially developed and
evaluated in the laboratory to ensure that each drug possesses the desired
characteristics and actions.
- If a drug appears to have the desired
properties, it is then tested in animals to determine what, if any, side
effects the drug may cause. A wide range of doses are administered to test
for side effects, both at the estimated human dose level as well as in
large overdoses to determine possible toxic effects.
- The candidate drug is also given to
pregnant animals to determine whether it has any adverse effects on the
developing fetus. This is to help determine whether the drug would be safe
for pregnant women.
- If the candidate drug appears to be safe
in test animals, it is then ready to be tested in humans.
Clinical studies on humans
Before an experimental medication is tested in humans, an Investigational
New Drug (IND) application must be filed with the FDA. After reviewing all
of the laboratory and animal data, the FDA will either approve the IND if
they think the drug appears safe enough to be tested in humans, or deny the
application if the risks appear unacceptably high. If the IND is approved,
the first phase, called a Phase I trial, of advanced drug development
begins. This phase usually involves testing the drug in normal adults,
followed by adults with the disease targeted by the drug, to determine the
safety of the drug at various doses.
Phase I trials are followed by larger Phase
II trials in which the effectiveness and safety of the drug is tested over a
range of doses. These are generally multi-centered, placebo-controlled,
multi-dose, blinded trials. This means that the exact same study is
conducted in multiple medical centers across the country. Individual
subjects participating in the trial may be on one of several doses of the
real medicine or on an inactive placebo (fake). Neither the investigator
(research doctor) nor the subject know what the subject is actually getting.
The purpose of these studies is to determine scientifically whether the drug
is effective compared with a placebo, and if so, what the optimal dose might
be. This approach is usually the only way to know for sure if a drug is safe
Once an optimal dose is determined, even
larger multi-center Phase III trials are conducted to establish the safety
and effectiveness of long-term treatment (usually up to 12 months). After
successful completion of Phase III trials, the pharmaceutical company
submits to the FDA a new drug application containing all of the data
collected from all of the trials. After thorough evaluation of each piece of
data, the FDA acts on the application, approving the new drug only if
safety, efficacy and drug stability are proven. The whole process, from the
pre-clinical laboratory stage to final approval, takes about 10 years.
Frequently, after a drug is FDA-approved and
is marketed, research will be done to determine the best way to use the new
medication. These are called post-marketing, or Phase IV, trials. Typical
examples of a Phase IV trial would include comparisons between two drugs on
the market, or comparisons between two different time/dose schedules for
taking a single drug.
Who watches out for the patients?
All research involving human subjects must be reviewed and approved by an
Institutional Review Board (IRB). The IRB reviews the research protocol, the
informed consent form (the document explaining the study, including risks
and benefits, to the potential study subjects), known information about the
drug (including reports of unexpected adverse events), and any potential
advertising planned for recruitment of subjects.
The job of the IRB is to ensure that the
protocol and recruitment techniques are ethical, the potential subjects are
fully informed about the procedures, risks, benefits and alternative
treatments available before they agree to participate in the study, and that
the subjects are not placed at unacceptable risk. People interested in
becoming a part of such a research study should first read the informed
consent document carefully, and then ask the study personnel any additional
questions before agreeing to participate.
Most research protocols have built in safety
monitoring for subjects. These may include blood or urine tests, x-rays,
electrocardiograms (which monitor heart activity), and other tests. Many
protocols also have automatic "kick out" criteria, instructing investigators
to withdraw a subject from the study if the disease being studied worsens
significantly. These safeguards help to prevent research participants from
experiencing increases in their disease symptoms as a result of receiving a
placebo or ineffective dose of the study drug.
Research participants should not agree to
take part in a study unless they intend to complete it. However, subjects do
have the right to drop out of a study at any time. Typical reasons for
dropping out include unacceptable side effects from the drug, worsening of a
subject’s medical condition, or changes in work schedules that make it
difficult to attend the clinic according to the study schedule.
The study doctors (investigators) may also
withdraw someone from a study if they feel that it is not in the study
subject’s best medical interest to remain in the study. This usually happens
if the subject’s medical condition is getting worse, or if new information
about serious adverse effects becomes known. The IRB also has the power to
terminate a study if they determine that the study procedures or medications
pose too much of a risk for subjects. The pharmaceutical company may
terminate a study if they discover that the investigational drug is not
appropriate for further development. This usually occurs as a result of the
drug being too toxic or ineffective.
How do I decide which study to take part
Most research centers do several studies at a time, so you may be eligible
for more than one study. Try to find a good match between your own needs and
the needs of the study. Ask not only whether you fit the study
inclusion/exclusion criteria, but also whether the study meets your medical
and scheduling needs.
For instance, if you are interested in
participating in an asthma study, but you know that your nasal allergies
will flare up during the study, you’d probably want to choose a study that
allows you to take your nasal medications. If you are particularly concerned
about safety issues, a Phase III or post-marketing Phase IV trial may be
more desirable for you than an earlier Phase I or II trial, since more is
known about a drug’s side effects by that time. Or, if you have a very tight
schedule, a study with short visits would work better than one with several
long visits. Most research subjects seem to enjoy participating in studies,
and many choose to participate in additional studies after finishing a
If you are interested in participating in
research studies, see your physician or nearest research center for
information on clinical trials taking place in your area.
can provide you with more information on clinical research trials.
The content of this brochure
is for informational purposes only. It is not intended to replace evaluation
by a physician. If you have questions or medical concerns, please contact