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.
a drug from bench to bedside is a risky and expensive proposition. The
development of a new drug is estimated to cost many hundreds of millions
of dollars; as a result, decisions about funding a drug-development
program are based as much on economics as on science and medicine.Decisions
to invest and reinvest at all stages of development are driven by the
imperative to generate an attractive return on the capital invested,
whether by venture-capital and public investors or by pharmaceutical