Orphan Drug Policy (FDA)

Fast Track Designation

Fast Track Designation is granted to drugs being developed for the treatment of serious or life-threatening diseases or conditions where there is an unmet medical need. The purpose of the Fast Track Designation provision is to help facilitate development and expedite the review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

Sponsors of drugs that receive Fast Track Designation have the opportunity for more frequent interactions with the FDA review team throughout the development program. These can include meetings to discuss study design, data required to support approval, or other aspects of the clinical program. Additionally, products that have been granted Fast Track Designation may be eligible for priority review of NDA and the FDA may consider reviewing portions of a NDA before the sponsor submits the complete application (Rolling Review).


Orphan drug development: the increasing role of clinical pharmacology

Authors: Mariam A. Ahmed, Malek Okour, Richard Brundage, Reena V. Kartha


Over the last few decades there has been a paradigm shift in orphan drug research and development. The development of the regulatory framework, establishment of rare disease global networks that support drug developments, and advances in technology, has resulted in tremendous growth in orphan drug development. Nevertheless, several challenges during orphan drug development such as economic constraints; insufficient clinical information; fewer patients and thus inadequate power; etc. still exist. While the standard regulatory requirements for drug approval stays the same, applications of scientific judgment and regulatory flexibility is significantly important to help meeting some of the immense unmet medical need in rare diseases. Clinical pharmacology presents a vital role in accelerating orphan drug development and overcoming some of these challenges. This review highlights the critical contributions of clinical pharmacology in orphan drug development; for example, dose finding, optimizing clinical trial design, indication expansion, and population extrapolation. Examples of such applications are reviewed in this article.

Evolution along the Government - Governance Continuum: FDA's orphan products and fast track programs as exemplars of "what works" for innovation and regulation

C.-P. Milne,Joyce Tait

In governing the life sciences, there is a need to foster innovation, but also to control risk. While the scientific complexity and public unease with new healthcare technologies typically proscribe industry self-regulation, overly burdensome regulatory regimes can thwart the speed of innovation and the diversity of innovators necessary for a high-tech field to sustain itself. The premise of this article is that FDA has two programs, the orphan product and fast track programs for biopharmaceuticals, which serve as useful models of a regulatory system evolving towards a so-called ‘governance’ approach, which is characterized by push-pull incentives and a problem-solving philosophy. However, in this case, it also demonstrates features of old-style command-and-control ‘government,’ but with more control, and less command. The authors’ analysis presents evidence that these programs are exemplars of “what works.”


Smarter Regulation of Drug Development: FDA Orphan Products and FastTrack Programmes

Tait, J,Milne, CP

Advances in biomedical sciences are predicted to have enormous impact on the prevention, diagnosis, treatment and cure of disease and disability, for example pharmacogenomics, proteomics, nanotechnologies, micro-arrays, biomarkers, bio-informatics, synthetic biology, and tissue engineering. On the other hand, the R&D process is increasingly costly, unpredictable and inefficient, so that these advances will not automatically translate into improvements in health care.