--General
Information--
Orphan Drug Designation
A drug becomes an "orphan" when it receives
orphan designation from the Office of Orphan
Products Development, FDA. An orphan
drug is a drug intended to treat diseases or
conditions affecting fewer than 200,000 people per year
in the United States. The costs of research and
development of an orphan drug may exceed sales of the
drug when it is marketed. Therefore, the Orphan
Drug Act was passed as an incentive to develop
and manufacture drugs that treat rare conditions. Orphan
designation qualifies the sponsor to receive
certain benefits, such as 7 years marketing exclusivity,
from the U.S. Government in exchange for developing the
drug. However, the drug must successfully complete the
new drug approval process and be approved by the U.S.-FDA
before these benefits can be realized. More than one
sponsor may receive orphan drug designation of the same
drug for the same rare disease or condition. When another
sponsor seeks orphan drug designation for a subsequent
drug for the same rare disease or condition, an
explanation of why the proposed variation may be
clinically superior to the first must be made.
Are orphan products approved
faster than other drugs?
Historically, the approval time for orphan products as a
group has been considerably shorter than the approval
time for all drugs. This is due to the fact that many
orphan products receive expedited review because they are
for serious or life-threatening disease.
How much do orphan drugs cost?
The cost of orphan products is determined by the sponsor
of the drug and is not controlled by the FDA. The costs
of orphan products vary greatly. Generally, health
insurance will pay the cost of orphan products that have
been approved for marketing.
The above information is reprinted from the FDA
Office of Orphan Products Development: http://www.fda.gov/orphan/index.htm
Clinical Trials
Testing Drugs in People
by Ken Flieger
"Most of us understand that drugs intended to treat
people have to be tested in people. These tests, called
clinical trials, determine if a drug is safe and
effective, at what doses it works best, and what side
effects it causes--information that guides health
professionals and, for nonprescription drugs, consumers
in the proper use of medicines.
Personal Participation in Clinical Trials
Clinical trials are carried out at major medical research
centers such as teaching hospitals, at specialized
clinics ....., and even in doctors' offices. Although
they often involve hospitalized patients, many clinical
trials are conducted on an outpatient basis, with
participants more or less going about their normal
activities.
Although investigational drug studies vary widely, some
things should be expected by participants in virtually
any clinical trial...... Tests to assess disease status
might be more frequent. Participants are often required
to keep detailed records of their symptoms and follow
strict schedules.
Trials in Humans
Phase 1
Number of Patients: 20-100
Length: Several months
Purpose: Mainly safety
Percent of Drugs Successfully Tested*: 70 percent
Phase 2
Number of Patients: Up to several hundred
Length: Several months to 2 years
Purpose: Some short-term safety but mainly effectiveness
Percent of Drugs Successfully Tested*: 33 percent
Phase 3
Number of Patients: Several hundred to several thousand
Length: 1-4 years
Purpose: Safety, dosage, effectiveness
Percent of Drugs Successfully Tested*: 25-30 percent
* For example, of 100 drugs for which investigational new
drug applications are submitted to FDA, about 70 will
successfully complete phase 1 trials and go on to phase
2; about 33 of the original 100 will complete phase 2 and
go to phase 3; and 25 to 30 of the original 100 will
clear phase 3 (and, on average, about 20 of the original
100 will ultimately be approved for marketing).
The above information and additional information can
be found at the FDA website: http://www.fda.gov/
Additional information on clinical trials for patients
with rare disease or conditions is provided by the Office
of Rare Diseases of the NIH and can be accessed
at the following sites:
http://rarediseases.info.nih.gov/ord/ct-about.html
"Testing Drugs in People" originally
appeared in the July-August 1994 FDA Consumer . The
companion article "Skeptic's Guide to Medical
Breakthroughs" was taken from the November 1987 FDA
Consumer Special Report on New Drug Development in the
United States (January 1995). Ken Flieger is a writer in
Washington D.C.
AMDA,
Acid Maltase Deficiency Association,
is not involved in the selection of patients for clinical
trials.
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