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Two decades of orphan product development.
Marlene E Haffner1, Janet Whitley, Marie Moses
1Office of Orphan Products Development, US Food and Drug Administration, Room 15A08, 5600 Fishers Lane, Rockville, Maryland 20857, USA. mhaffner@oc.fda.gov
Nature Reviews. Drug Discovery
|October 3, 2002
Summary
The Orphan Drug Act (ODA) has significantly improved public health over 20 years by funding innovative biotechnology products. Advances in pharmacogenomics are expected to identify more rare diseases, further increasing the impact of ODA incentives.
Area of Science:
- Biotechnology
- Genomics
- Pharmacogenomics
Background:
- The Orphan Drug Act (ODA) is a major source of funding for clinical research at the US Food and Drug Administration.
- The ODA has been instrumental in developing numerous innovative biotechnology products over the last two decades.
- Evolving understanding of the human genome is leading to advancements in identifying rare diseases.
Purpose of the Study:
- To evaluate the impact of the Orphan Drug Act (ODA) incentives on public health.
- To highlight the role of ODA in fostering biotechnology innovation.
- To discuss the future implications of pharmacogenomics in identifying orphan diseases.
Main Methods:
- Analysis of ODA's contribution to biotechnology product development.
- Review of public health outcomes linked to ODA-funded research.
- Assessment of the potential for pharmacogenomics to expand the definition of orphan diseases.
Main Results:
- The ODA has demonstrably enhanced public health through its funding mechanisms.
- Significant advancements in biotechnology have resulted from ODA incentives.
- Pharmacogenomics is poised to identify a greater number of orphan diseases.
Conclusions:
- The Orphan Drug Act has been highly successful in promoting the development of orphan drugs and improving public health.
- Continued investment and research, particularly in pharmacogenomics, will likely uncover more rare conditions.
- The ODA remains a critical policy for addressing unmet medical needs in rare diseases.