ORPHAN DRUG APPROVAL LAWS

Course overview

RARE DISEASES AFFECT OVER 25 MILLION INDIVIDUALS IN THE UNITED STATES AND HAVE HISTORICALLY LACKED PHARMACEUTICAL DEVELOPMENT ATTENTION DUE TO DECREASED POTENTIAL FOR PROFIT. THE FOOD AND DRUG ADMINISTRATION (FDA) QUALIFIES A RARE DISEASE AS ONE WHICH AFFECTS FEWER THAN 200,000 PEOPLE, OR AS A CONDITION THAT AFFECTS GREATER THAN 200,00 PERSONS IN THE UNITED STATES BUT FOR WHICH THERE IS NO REASONABLE EXPECTATION THAT PHARMACEUTICAL COMPANIES WILL RECOVER THE COSTS INCURRED DURING DRUG DEVELOPMENT FOLLOWING PHARMACEUTICAL SALES. THE ORPHAN DRUG APPROVAL LAW OF 1983 INCENTIVIZED PHARMACEUTICAL COMPANIES TO DEVELOP DRUGS FOR RARE DISEASES BY OFFERING MARKET EXCLUSIVITY AND TAX CREDITS. THIS ACTIVITY REVIEWS THE HISTORY OF THE ORPHAN DRUG APPROVAL LAW AND DISCUSSES ASSOCIATED ETHICAL CONCERNS AND CLINICAL SIGNIFICANCE.