| FOREWORDby Nicholas Bachynsky, M.D., Ph.D Orphan drugs are drugs available outside the United States but n0tpermitted within this country and they are drugs needed to treat rarediseases or disorders affecting a small number of patients. The term"orphan drugs" troubles me because it suggests that these drugs aredifferent from other substances available to doctors. The implication isthat 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. Manyforeign drug firms have been in business longer than American phar-maceutical companies. Second, we can adequately research the effectsof drugs used for diseases by testing small patient populations. Third, itis not uncommon for doctors to use a drug effectively for diseases otherthan the one for which it was developed. Once we learn the chemicalproperties of a new drug, we can readily adapt it to new therapeuticuses. 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 theundisputed medical evidesingle contributor to lunl The FDA banned Das a contraceptive after coDeveloped in the 1950s,The FDA first approvedcontraceptive. The FDA's decisionposes places the Unitedincluding England and Festimated 1.5 million wocontraceptive by the Worly, the drug is prescribec(IHS), creating the uniq(FDA) forbids the use ofluse for contraception. Many drugs develo[United States after unwaltists developed clozapin~research beginning inhypertension, found itsresearching decongestarhad taken a dose ofcloni,low blood pressure. Rifampicin, develol:proven success againstveloped in an originalderived by modifying abased on studies condut Social and politicaldrugs, such as pentamitreatment of AIDS paobtained FDA approval iTHA, an innovative drudisease patients at 17 mc1989. Although safeguarccounter sales of pharmprescription drugs, phyfor the treatment and cuthe doctor should not hspecial drug products "family" of curatives ar medical profession. A ! available. |
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