An unexpected medical problem that happens during treatment with a drug
or other therapy. Adverse events may be mild, moderate, or severe, and
may be caused by something other than the drug or therapy being given.
Also called adverse effect [adapted from Cancer.gov dictionary]
When describing a drug, “approved” usually means that your country’s
regulatory agency allows it to be prescribed to patients by doctors.
In medicine, the ability of an intervention (for example, a drug or
surgery) to produce the desired beneficial effect.
In research studies, an event or outcome that can be measured
objectively to see if the intervention being studied is beneficial. Some
examples of endpoints could be better disease control (like A1c control
in diabetes), relief from symptoms, or living longer.
A list of characteristics that a person cannot have in order to be
eligible for a research study. Examples of exclusion criteria are being
outside the allowed age range, taking certain medications, or having a
certain disease or condition other than the one being studied.
A list of characteristics that a person must have in order to be
eligible for a research study. Examples of inclusion criteria are being
within the allowed age range, having certain disease characteristics,
and being able to willingly consent to joining the study.
A process that is part of every medical research study, in which
patients are given important information to help them decide if they
want to participate in the trial. It includes discussing possible risks
and benefits. Patients are also given any new information that might
affect their decision to continue. Also called consent process.
Institutional review board (IRB)
A group of scientists, doctors, clergy, and patient advocates that
reviews and approves the detailed plan for a research study in people.
IRBs are meant to protect the people who take part. They check to see
that the trial is well designed, legal, ethical, does not involve
unneeded risks, and includes a safety plan for patients. There is an IRB
at every health care facility that does medical research, and every
research study is reviewed by an IRB before it can begin.
A research study that is done to learn about the effect of an
intervention, or action taken to improve health. The intervention could
be a drug, medical device, procedure, vaccine, or even diet changes,
exercise, or education. The study could include a group of participants
that don't receive the intervention.
Describes any drug or treatment being tested in a research study. An
investigational medication has not been approved by Regulatory
authorities for the use in which it is being studied. It means that it
has been tested in the laboratory and can be tested in people according
to the rules of your country’s regulatory agency. A drug that is already
being prescribed for one disease or condition can be considered
investigational in other diseases or conditions. The word “experimental”
may also be used.
A research study that collects information from participants as they go
about their usual medical treatment or activities. There is no attempt
to affect the outcome.
A person who enrolls in a research study. The word “participant” is used
to reflect the fact that the person has voluntarily decided to
participate in the trial. You may see the word “subject” or “patient”
used in place of participant.
Medical research studies are usually conducted in phases (1, 2, 3, or 4)
that build on one another. Each phase is designed to answer certain
questions. In earlier phases, the focus is on safety and how a drug or
treatment works. In later phases, the focus is on how well a drug or
treatment works and its safety.
An inactive substance or other intervention that looks the same as, and
is given the same way as, the active drug or treatment being tested. The
effects of the active drug or other intervention are compared to the
effects of the placebo. Often when a placebo is used, it is given with
the standard of care, so that no one in the trial goes without
treatment.
A written document that describes why a research study is being done and
how it is to be conducted. It outlines how study participants and the
integrity of the data collected will be protected. The doctors and staff
on a study team use this document as a guide to what they need to do
during the research study.
An independent government body that regulates a particular sector of the
economy or society. Each country has its own regulatory agencies.
Agencies such as the Food and Drug Administration (FDA) in the United
States, European Medicines Agency (EMA) in the European Union, and
Pharmaceuticals and Medical Devices Agency (PMDA) in Japan are involved
in regulating medical research.
A test of how well new medical approaches work in people. Research
studies test new methods of screening, prevention, diagnosis, or
treatment of a disease. Also called a clinical trial or clinical study.
In a research study, it is the period of time during which tests are
performed to confirm that you are eligible for (able to participate in)
the study and that the study is right for you.
A person, company, institution, group, or organization that oversees or
pays for a research study and collects and analyzes the data, but does
not actually conduct the investigation.
Treatment that is accepted by medical experts as a proper treatment for
a disease and that is widely used by healthcare professionals. Also
called best practice, standard medical care, and standard therapy.
Any medicine that is being studied in a research study. See
investigational for more information.
The hospital, clinic, or doctor’s office where a research study takes
place. A study site must go through an approval process to be part of a
research study.. Study sites are often “centers of excellence” and have
the expertise and resources to be able to carry out the study properly.
Who can join a research study?
Everyone! There are research studies for healthy people, people with a
medical condition, and people of all ages and backgrounds.
Why is there an effort to promote more diversity in medical research?
Because humans are diverse! All different types of people need
medicines. That means that for a new medicine to truly help the people
it is intended for, a diverse group of people need to be part of
research studies for that medicine.
How do I know my privacy is protected if I decide to join a research
study?
The researcher protects your identity and medical information just like
your doctor does. When research information is shared with the sponsor,
your name and other direct identifiers are removed so the sponsor does
not know who you are.
Who is watching out for me in a research study?
Many different people! These include the sponsor the study (for example,
a pharmaceutical company), your country’s regulatory agency, an
Institutional Review Board at the medical center where the study is
taking place, the team of study doctors, nurses, and others who carry
out the study, and even your own doctor, who can help make sure the
study is right for you.
Your safety is everyone's number one priority!
How can I find a research study that is appropriate for me?
You can Search for available trials on your own. One place to start is
ClinicalTrials.gov, which lists research studies that are being done all
over the world.
Talk to your doctor or trusted medical professional. If you are
interested in a specific research study, your doctor can reach out to
the doctor involved in the study. Your doctor can also search for
studies that might be right for you.
You can also call the hotline or visit the website listed on a research
study poster or brochure in your doctor’s office or hospital.
What happens after the study?
When a research study ends, your participation typically ends too. In
some cases, there may be an option to keep taking the study treatment as
part of what is called a “long-term extension study”. In other cases, a
study may end early because the treatment becomes “approved”, which
means doctors can prescribe it for their everyday patients.
If I don’t qualify for a study, can I try again for the same or
different study?
Every research study is different. If you don’t qualify at first, ask
the study doctor if you can be screened again in the future. You can
also see if you are eligible for a different study. You typically cannot
be in 2 studies at the same time.
What if I join a study and don’t want to stay in it?
Being in a research study is 100% your choice! You can leave at any
time, for any reason. There are no penalties and it does not affect your
regular medical care. You should talk to the study staff about why you
are leaving–there may be solutions for any problems that arise. The
study doctor may ask you to come back for follow-up visits for your
health and safety.