FOREWORD
by Nicholas Bachynsky, M.D., Ph.D
Orphan drugs are drugs available outside the United States but n0t
permitted within this country and they are drugs needed to treat rare
diseases or disorders affecting a small number of patients. The term
"orphan drugs" troubles me because it suggests that these drugs are
different from other substances available to doctors. The implication is
that these drugs have not been fully tested or adequately documented.
Let's put these myths to rest. First, pharmaceutical research out-
side the United States is competent, scientific, and thorough. Many
foreign drug firms have been in business longer than American phar-
maceutical companies. Second, we can adequately research the effects
of drugs used for diseases by testing small patient populations. Third, it
is not uncommon for doctors to use a drug effectively for diseases other
than the one for which it was developed. Once we learn the chemical
properties of a new drug, we can readily adapt it to new therapeutic
uses.
This book, Orphan Drugs, lists 192 generic (and 1,535 brand name)
drugs available outside the United States that have passed the scientific
tests. The Sources Directory lists 241 companies licensed or chartered
in their countries to produce pharmaceuticals. These drugs should be
readily available to doctors in the United States, but their use has not
earned FDA approval.
There was a time in history when a doctor regularly prescribed
combinations of drugs to be compounded by a pharmacist. We can still
do this, but most of us are reluctant to create our own compounds, even
though they may be the best remedies. Arbitrary government regula-
tions create an atmosphere of fear. The doctor and the pharmacist are
wary of legal repercussions from the use of an unlabelled compound
prepared without FDA approval although the chemistry works and
studies have proven that drugs in combinations are generally safer and
less costly than drugs administered separately.
The Food and Drug Administration is the governmental agency
responsible for placing restrictions on the use of pharmaceuticals in the
United States. The FDA has a history of beneficial policies, but it has an
abysmal career in its response to crises. Rather than react to scientific
evidence, too often it responds to worst-case scenarios. It tends to
impose unwarranted restrictions where a safe and effective drug ha
been used irresponsibly or implausibly and contrary to a doctor's
advice. In the classic case of tl~alidomide, a drug used worldwide as an
effective tranquilizer, the FDA banned the drug completely when it
should only have sought to prevent its administration to pregnant
women. On the other hand, there is no ban on cigarettes in spite of the
undisputed medical evide
single contributor to lunl
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treatment of AIDS pa
obtained FDA approval i
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available.
發表於2024-11-28
Orphans Drugs 2024 pdf epub mobi 電子書 下載
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Orphans Drugs 2024 pdf epub mobi 電子書 下載