New England Journal of Medicine
April 17, 2014 Vol. 370 No. 16
http://www.nejm.org/toc/nejm/medical-journal
Perspective
The Calculus of Cures
R. Kocher and B. Roberts
Excerpt
In 2013, the Food and Drug Administration (FDA) approved 27 new drugs for marketing. Eight of these drugs are for orphan diseases, including six rare cancers. In fact, more than half of the 139 drugs approved by the FDA since 2009 are for orphan diseases and cancers.1 This disproportion is not solely the result of scientific breakthroughs; the economics of drug development and the business of health care delivery also play large roles. Although these drugs may end up being critically important to patients with the targeted diseases, we believe we must avoid systematically underinvesting in drugs in other important areas of medicine…