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Melanoma Therapeutics Foundation
Clinical Trials
Clinical
Development – Testing of
new investigational compounds in humans is done in stages
in order to maximize benefit and minimize harm to patients.
All
stages of the process are conducted in close communication
with the FDA or other appropriate regulatory agencies
that have oversight of the final drug approval.
- Phase I Clinical Trials – First
exposure of humans to the Development Compound.
These safety studies
provide evidence of patients’ tolerance for escalating
doses of administered drug and are meant to carefully
monitor the
appearance of adverse side effects. Trial protocols are
often designed to gain insights into the drug’s
PK and PD profiles in patients. Any evidence of activity
and
efficacy
are also noted to help guide the selection and treatment
of patients in later stage clinical trials.
- Phase II Clinical Trials – Designed to demonstrate
the drug’s effectiveness in the selected patient
population, to fine tune final drug dose and administration
regimen and
to determine appropriate remedies for common adverse
side effects that may be encountered. At times, studies
are designed
as Phase IIA to specifically determine
dosing requirements or Phase IIB where
efficacy is measured.
- Phase III Clinical Trials – Large, well controlled
studies to demonstrate clear efficacy when drug is administered
at optimal dose and regimen. Often conducted in a randomized and blinded fashion, these studies require treatment of a
large number of patients in order to demonstrate a statistically
significant benefit attributed to the new drug as compared
to either standard of care or placebo control. Upon approval
of a new drug, Phase III trials are also employed to expand
the use of the drug for new indications.
- Phase IV Clinical Trials – At times requested by
the FDA or initiated by the sponsoring company for competitive
or marketing reasons, these studies are conducted post launch
for surveillance of safety and to support ongoing use of
a new drug. Because large numbers of patients in the general
population are exposed to the drug for extended periods of
time, adverse side effects that are rare or are associated
with long term use of the drug may become apparent as a result
of these Pharmacovigilance studies.
New Drug Application – Upon completion of the appropriate
testing in human clinical trials, a New Drug Application (NDA)
is filed with the FDA. The goal of the NDA is to ensure that
enough information is provided for the FDA to determine whether
the new drug is safe and effective and the benefits outweigh
risks associated with its use. In addition, the FDA will determine
if the product label and packaging insert are sufficient and
appropriate and that the manufacturing process is adequately
robust to maintain the new drug’s identity, strength,
quality and purity.
NEED – ACCELERATED
DEVELOPMENT
LINKS
FDA, Food and Drug Administration http://www.fda.gov/
CEDR, Center for Drug Evaluation and Research http://www.fda.gov/cder/
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