The Bioethical Implications of the Orphan Drug Act on Healthcare in the United States



Journal Title

Journal ISSN

Volume Title



Orphan drugs and diseases are a neglected academic and scientific research area. The Orphan Drug Act (ODA) of 1983 established profit incentives for pharmaceutical research and drug development. The ODA resulted in the development of approximately 177 medications for orphan diseases at a high cost to patients and physicians. The average profit margin for orphan drug distributors is 50% with a reported industry revenue of approximately $35.8 billion. The average orphan disease patient has a co-pay of 48% and deductible of 39% of the total annual cost of their medication. The annual cost per patient per year is over $6,000 in 91.8% of cases. Insurance companies, both private and public, will frequently challenge physician’s prescriptions in order to reduce overall cost of patient care. The artificial price inflation incentive created by the ODA has resulted in unaffordable medications, ineffective treatment, and a damaged patient-physician relationship. A healthcare system primarily incentivized by profit is ultimately incapable of prioritizing the well-being of patients’ lives. The issue is not the availability of resources, but rather the ethical system utilized to determine the allocation of resources. The first step in a long-term solution would be increasing patient-physician accessibility to diseases by lowering cost – as proposed by rejected HR 3678 “Preserving Access to Orphan Drugs”. A long-term shift from a profit-centered healthcare industry to a patient-centered one is necessary to the overall sustainability of patient care. The most resource-efficient and ethical way to achieve this is through a form of socialized healthcare.