Prospects for rapid advances in the development of new medicines for special medical needs.

Broadly speaking, the goals of the US Food and Drug Administration (FDA) special-designation programs--orphan, priority review, accelerated approval, and fast track--have been to expedite and sustain development and facilitate authorization of new medicines for unmet medical needs through so-called "push-pull" incentives. Although generally successful over time, their success has been confined to certain therapeutic areas and, within those areas, certain diseases. Times have changed. The research and development (R&D) burdens and public health urgency that acted as an impetus for the FDA to intervene more actively for certain disease areas are now broadly experienced across many disease areas. This betokens the need for the FDA to make designation and implementation decisions with a view that reaches beyond the immediate horizons of political expediency and patient advocacy to encompass the broader expanse of factors that now influence R&D decisions--global competitiveness, the needs of investors, emerging sponsors, and patient-focused drug development.