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# Rare Disease Therapeutic Modalities Analyzed
- URL: https://www.fdaweb.com/rare-disease-therapeutic-modalities-analyzed/
- Published: 2020-01-03T12:00:00.000Z
- Updated: 2026-09-14T15:57:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145757

Authors from FDA and several other organizations and academic centers analyze progress and challenges in developing new therapies for rare diseases. A *Nature* drug discovery review [article](https://www.nature.com/articles/s41573-019-0049-9.epdf?shared%5Faccess%5Ftoken=-JPEXuFHsXOtBaclpGzr39RgN0jAjWel9jnR3ZoTv0Mfbqb0p6X45A9rxDp2dNAs0TASxlyl3qZYas1K7sv5g7mq-10bMl0qTsLyzFvoP%5FoXXp%5FaCxclEua%5F9PnhIGIRxKjr7LrfapdNw-ckX3plxw%3D%3D&ref=fdaweb.com) says that addressing the translational gap is a multifaceted challenge for which a key aspect is the selection of the optimal therapeutic modality for translating advances in rare disease knowledge into potential medicines known as orphan drugs.

The article discusses the technological basis and rare disease applicability of the main therapeutic modalities including small molecules, monoclonal antibodies, protein replacement therapies, oligonucleotides, and gene and cell therapies, as well as drug repurposing.

For each modality, the authors consider its strengths and limitations as a platform for rare disease therapy development and describe the clinical progress so far in developing drugs based on it. They also discuss selected overarching topics in the development of therapies for rare diseases, such as approval statistics, engagement of patients in the process, regulatory pathways, and digital tools.