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Porphyria Educational Services

PORPHYRIA EDUCATIONAL BULLETIN Vol.1 No. 7 February 1999
FOCUS: Clinical Trials & Medical Research

Investigational Drugs:

Before a pharmaceutical company can initiate testing in humans,
the phamaceutical manufacturer must conduct extensive preclinical or
laboratory research.

This research typically involves years of experiments in animal and human
cells.

The compounds are also extensively tested in animals.

If this stage of testing is successful, a pharmaceutical company then
presents this statistical data to the Food and Drug Administration (FDA).
At this point they are requesting approval to begin testing the drug in
humans. This is called an Investigational New Drug application (IND).
Each step in the process is quite lengthy, but assures us of many
checks and balances along the way. However even after a drug is approved
it is very often contraindicated in porphyrics.

Costs of Research and Clinical Trials:

Many porphyrics are interested in clinical research but wonder how much
is the cost.

Who pays for clinical research?

Funding for clinical research comes from both the federal government
and from private industry. Much of private industry includes the chemical
companies which produce everything from petroleum, paints, drugs
and other bio-tech.

The U.S. Government entity for medical research is known as the National Institutes
of Health. The sponsors of a specified research hire physicians, who may work
in a wide variety of health-care settings, to conduct the clinical trial.
The physicians are typically paid on a per-patient basis.
The medical care is often provided free to the patient.
Patients may also be paid a small fee to participate in a clinical trial.


ACTUAL HUMAN DRUG TESTING

Do you wonder how experimental drugs are tested in humans?
Clinical testing of experimental drugs is usually completed in three
phases.

Each individual successive phase involves a larger number of
Once the FDA has granted a New Drug Approval (NDA), the
pharmaceutical companies also conduct a post marketing or
what they call a late phase three/phase four study..

So what are all these phases?
The SAFETY Phase:

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).

These volunteers are usually paid for participating in the study.

The study is designed to determine what happens to the drug in the human
body. This is to ascertain 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 percent of experimental drugs pass this initial phase of testing.

The EFFICACY Phase:
Once a drug has been shown to be safe, it must be tested for efficacy.
This second phase of testing may last from several months to two years, and
involve 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. By doing it in this fashion,
the study can provide the pharmaceutical company and the FDA comparative
information about the relative safety of the new drug, and its effectiveness.
Only about one-third of experimental drugs successfully complete both phase
I and phase II studies.

The Adverse Reaction Phase:
In the third phase study, 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 effectiveness, benefits,
and the range of possible adverse reactions.
Most phase III studies are randomized and blinded trials.

This third phase study typically last several years.
Seventy to 90 percent of drugs that enter phase III studies successfully
complete this phase of testing.
Once this phase study is successfully completed, a pharmaceutical
company can request FDA approval for marketing the drug.

Post-Marketing or Late Phase Three/Phase Four Studies:

Inlast phase studies,pharmaceutical companies have several objectives.
The objectives include that
(1) studies often compare a drug with other drugs already in the market;
(2) studies are often designed to monitor a drug's long-termeffectiveness
and impact on a patient's quality of life; and
(3) many studies are designed to determine the cost-effectiveness of a drug
therapy relative to other traditional and new therapies.

PARTICIPATION IN CLINICAL RESEARCH

Should you participate in clinical research?

People participate in clinical research for a variety of reasons.

People who volunteer for phase II and phase III trials can gain access
to promising drugs long before these compounds are approved for the
marketplace. For instance at the present time heme arginate which is afar
better Intervention Therapy for treatment of acute porphyria attacks is now
undergoing trials in the United States, although all ready approved in
Europe and Australia.

Volunteers typically will get excellent care from the physicians during the
course of the study. This care will most likely be free.

The patient's rights and safety are protected in two important ways.
First, any physician awarded a research grant by a pharmaceutical
company or the NIH must obtain approval to conduct the study from
an Institutional Review Board.

The review board, which is usually composed of physicians and lay
people. This review board is charged with examining the study's
protocol to ensure that the patient's rights are protected, and that
the study does not present an undue or unnecessary risk to the patient.
Second, anyone participating in a clinical trial in the United States is
required to sign an "informed consent" form.

This "informed consent" form details the nature of the study.
The form clearly states the risks involved, and what may happen to a
patient in the study.

The informed consent tells patients that they have a right to leave the
study at any time.

Patients considering participating in clinical research should talk about
it with their physicians and medical caregivers.
Also if you are considering participating you should also seek to
understand the credentials and experience of the individuals and the
facility involved in conducting the study.

Other questions to ask include:

How long will the trial last?
Where is the trial being conducted?
What treatments will be used and how?
What is the main purpose of the trial?
How will patient safety be monitored?
Are there any risks involved?
What are the possible benefits?
What are the alternative treatments besides the one being tested in the trial?
Who is sponsoring the trial?
Do I have to pay for any part of the trial?
What happens if I am harmed by the trial?
Can I opt to remain on this treatment, even after termination of the trial?

TERMINOLOGY IN CLINICAL RESEARCH
A glossary of clinical research terms.

Clinical Trial
A carefully designed investigation of the effects of drug, medical
treatment, or device on a group of patients.

Clinical Investigator
A medical researcher in charge of carrying out a clinical trial's protocol.
Researchers are usually doctors, nurses, pharmacists or other health care
professionals.

Coordinator
This individual manages the conduct of the clinical trial.

Food and Drug Administration
A government agency that enforces laws on the manufacture, testing, and use
of drugs and medical devices.
**All drugs and medical devices must be approved by the FDA before they can
be used by the general public.

Informed consent
A discussion of all procedures, benefits, risks, and expectations of a
clinical trial between clinical investigators and potential patients.
The FDA requires all patients to sign an informed consent form before
participating in a trial.

Institutional Review Board (IRB)
A board consisting of health care professionals from the institution where
the clinical trial takes place, as well as members of the local community.
The board scrutinizes all trial activities including recruitment,
advertising, and potential risks. The IRB also makes sure that FDA
regulations are being followed in a particular trial.

Investigational treatment
The drug or medical device that is tested during a clinical trial.

Phases
Drugs and medical devices must pass three segments or phases of testing
before they can be eligible for FDA approval.

Principle investigator
A medical professional who is overseeing the treatment of the patients in
the clinical trial.

Protocol
A plan that sets guidelines for a trial and usually involves several
different trial locations. A protocol is usually designed by the sponsor of
a clinical trial.

Sponsor
The pharmaceutical company, research institution, or other health
organization that funds a clinical trial and designs its protocol.

Standard treatment
An FDA-approved treatment currently in wide use. In trials involving new
treatments, there may be no pre-existing treatment at all. In these cases,
the lack of any treatment is considered the standard treatment. Generally,
the goal of a clinical trial is to introduce an investigational treatment
that is safer and more effective than the standard treatment.