Orphan drugs—or orphan diseases?

Abstract

In the beginning, the question was that of how to provide drugs for rare diseases; and the beginning in this country was perhaps around 1972 when the 'Pharmaceutical Working Party' of the National Economic Development Office discussed the matter and agreed to keep it under review [1]. I was permitted to raise the issue more publicly at a symposium under the chairmanship of the late Sir Derrick Dunlop in 1973 [2], while also in the USA the Intra-agency Committee on Drugs of Limited Commercial Value was established in 1973, under the direction of the Food and Drug Administration (FDA), to ascertain such needs and where the obstacles lay to their being met. Nothing came of this and the committee was disbanded 2 years later.