FDA ’s Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases

AbstractThere are nearly 30 million Americans that suffer from at least one of the more than 7000 rare diseases identified to date. Therapies for treating, preventing, or diagnosing rare diseases have been limited due to various reasons. Incentives are provided to sponsors in an effort to promote the development of therapies for rare diseases and to encourage the availability of therapeutically superior drugs or biologics. This paper will discuss the mission of the Office of Orphan Products Development within the Food and Drug Administration (FDA), the specific programs within the office and the relation to incentives provided, achievements of the programs, and continued challenges in rare disease product development.
Source: Journal of Pharmacokinetics and Pharmacodynamics - Category: Drugs & Pharmacology Source Type: research

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Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
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Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
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Source: Journal of Medical Mycology - Category: Biology Source Type: research
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Source: Klinische Monatsblatter fur Augenheilkunde - Category: Opthalmology Tags: Klin Monbl Augenheilkd Source Type: research
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Source: Anti-Cancer Drugs - Category: Cancer & Oncology Tags: Case Reports Source Type: research
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Source: Revista Peruana de Medicina de Experimental y Salud Publica - Category: International Medicine & Public Health Tags: Rev Peru Med Exp Salud Publica Source Type: research
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Source: MDDI - Category: Medical Devices Authors: Tags: Regulatory and Compliance Source Type: news
Join a major event on rare diseases in 2020: The RE(ACT) Congress and IRDiRC Conference, 11-14 March 2020, Berlin - Germany. Young and professional scientists, patients &patient representatives, and other rare diseases stakeholders are welcome to discuss latest cutting-edge research, innovation, and policy with the rare disease leaders. The scientific program -inspired by IRDiRC principles, achievements, and activities foreseen in 2020- encompasses topics like diagnosis &artificial intelligence, molecular etiology, innovative clinical trials, patient engagement and drugs for all. Blackswan Foundation and IRDiRC...
Source: EUROPA - Research and Innovation: Events - Category: Research Tags: Health - D72D1707-FE0F-3C39-4E55830C194C2E7C Information and Communication Technologies (ICT) - 20C389EF-F27F-C500-874F52AB1E2A3208 Life sciences - 7DB37216-D9A7-5F19-1F4B02B58D353C4B Horizon 2020 - 719491AD-9C69-558D-B05B8AC79345ED08 Environment Source Type: events
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Source: NeuroRehabilitation - Category: Rehabilitation Tags: NeuroRehabilitation Source Type: research
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