Abstract
BackgroundRegulatory and economic frameworks stimulated the research and development of orphan drugs, but very little has been done for devices necessary for the in-vivo diagnosis, prevention and treatment of life-threatening conditions with a low prevalence/incidence.DiscussionA general public consultation in Europe has shown a positive attitude towards an “orphan device” directive. The United States of America have a Humanitarian Use Device exemption, but Europe is still waiting for such a stimulating framework. Post-marketing surveillance (“materio-vigilance”) will be necessary for follow-up, patient-reported outcome measures (quality of life versus survival) needed and off-label use data available for patient-safety reasons.SummaryThe marketing period for devices is shorter than for medicinal products. Incentives are necessary to stimulate research and development of such “orphan devices” especially when surgical intervention is the only option.
Highlights
Regulatory and economic frameworks stimulated the research and development of orphan drugs, but very little has been done for devices necessary for the in-vivo diagnosis, prevention and treatment of lifethreatening conditions with a low prevalence/incidence
Orphan devices are medical devices intended for the invivo diagnosis, prevention or treatment of a very rare life-threatening or chronically debilitating condition
Several institutions (for example, the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Australian Therapeutic Goods Administration (TGA)) have put in place regulatory and economic frameworks to facilitate the development of orphan drugs (118 authorized by EMA to date), but much needs to be done for similar medical devices, especially in Europe
Summary
A general public consultation in Europe has shown a positive attitude towards an “orphan device” directive. Several institutions (for example, the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Australian Therapeutic Goods Administration (TGA)) have put in place regulatory and economic frameworks to facilitate the development of orphan drugs (118 authorized by EMA to date), but much needs to be done for similar medical devices, especially in Europe. A negative pressure ventilator (“Iron Lung”) is a medical ventilator that enables a patient to breathe when spontaneous breathing control has been lost or exceeds the patient’s ability This device was invented by Philip Drinker (1894–1972) and Louis Agassiz Shaw (1886– 1940) at the Harvard School of Public Health after an. When the pressure goes above that within the lungs, the air is expelled [2] This device is used for Ondine’s curse and other rare conditions in which failure of the medullary respiratory centers in the brain results in patients having no autonomic control of breathing. Poliomyelitis vaccinations (Jonas Salk 1952) have virtually eradicated new cases of poliomyelitis, and the iron lung has virtually disappeared from modern medicine
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