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Orphan Drugs: An Update on Key Selected Pipeline Developments for Rare Diseases
IQVIA Pipeline Link
Jul 17, 2023
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  • Orphan Drugs: An Update on Key Selected Pipeline Developments for Rare Diseases
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A rare disease is defined as a condition that affects less than 200,000 people in the USA and fewer than 1 in 2,000 people in the EU. The development of orphan drugs was previously rare due to the small size of patient populations, which acted as a barrier for commercial development in the research and development of rare diseases. Regulatory bodies in major markets have introduced new legislation with the aim of providing incentives for the development of medicines for rare diseases, including the US FDA's Orphan Drug Act in 1983, followed by Japan in 1993 and the EU in 2000. These incentives, including extended market exclusivity (7 years in USA and 10 years in EU), and the advancement in new technologies have driven the growth in ground-breaking research against rare diseases and the development of orphan drugs.

The Orphan Drug Act has resulted in a greater number of orphan drugs approved in the USA since its enactment, driven by the approval of rare oncology products and secondary indications. As of March 2023, there have been 6,506 orphan drug designations granted by the US FDA of which 1,144 have led to orphan-designated approval from the agency. This review provides an insight into key selected significant therapeutic developments in rare diseases with data sourced from IQVIA Pipeline Link, a proprietary drug pipeline database. 

Key selected orphan drugs in development

Rare disease drug development has recently focused on hematology, neurology, infectious disease, metabolism/endocrinology, rheumatology/immunology, cardiovascular, gastroenterology, pulmonary and ophthalmology therapeutic categories. There are currently several key orphan drugs in development that may emerge as the next rare disease therapies.

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