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

The FDA banned D

as a contraceptive after co

Developed in the 1950s,

The FDA first approved

contraceptive.

The FDA's decision

poses places the United

including England and F

estimated 1.5 million wo

contraceptive by the Wor

ly, the drug is prescribec

(IHS), creating the uniq

(FDA) forbids the use ofl

use for contraception.

Many drugs develo[

United States after unwal

tists developed clozapin~

research beginning in

hypertension, found its

researching decongestar

had taken a dose ofcloni,

low blood pressure.

Rifampicin, develol:

proven success against

veloped in an original

derived by modifying a

based on studies condut

Social and political

drugs, such as pentami

treatment of AIDS pa

obtained FDA approval i

THA, an innovative dru

disease patients at 17 mc

1989.

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prescription drugs, phy

for the treatment and cu

the doctor should not h

special drug products

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medical profession. A !

available.