Despite ramped-up hiring, FDA continues to grapple with hundreds of vacancies
By Sydney Lupkin and Sarah Jane TribbleThe Food and Drug Administration has more than 700 job vacancies in its division that approves new drugs, and top officials say the agency is struggling to hire...
View ArticleOrphan Products Grants Program
Objective: To support the clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases or conditions where no current therapy exists or where the proposed product...
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Securing an orphan drug designation
by George UnderwoodGetting an orphan designation isn't as simple as just targeting a rare disease. Tim Coté, former director of the FDA's Office of Orphan Products, tackles the most common mistakes...
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The President Signs 21st Century Cures into Law; Highlights of Drug and...
By Michelle L. Butler & James E. ValentineThe 21st Century Cures Act (Act) was passed by the House on November 30, 2016 and the Senate on December 7, 2016. The President signed it into law on...
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Thanks to 21st Century Cures, the Sun is Coming out Tomorrow for Orphan Drugs...
12/16/2016 by Alan Minsk, Kalie Richardson | Arnall Golden Gregory LLPThe 21st Century Cures Act, a bill that includes major Food and Drug Administration reform, was passed by the Senate last week...
View ArticlePart 2 FDA Expedited Programs Series: Breakthrough Therapy
ObjectiveTo expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement...
View ArticleDrugs For Rare Diseases Have Become Uncommonly Rich Monopolies
As heard on NPR's Morning Edition, a look at the Orphan Drug Act and its lucrative incentives to drug companies, with mention of Dr. Timothy Cote.Sarah Jane Tribble & Sydney LupkinFull audio and...
View ArticleRare Disease Day: Its Significance & 7 Most Rare Diseases in the World
Source: NDTV Food Not many of you may be aware of the fact that the last day of February each year is marked as Rare Disease Day,. It is celebrated worldwide to draw attention to various kinds of...
View Article'Burdensome' FDA Drug Approval Process Fuels Innovation, Saves Lives
David Kroll, ContributorSource: Forbes.comhttps://www.forbes.com/sites/davidkroll/2017/02/28/burdensome-fda-drug-approval-process-fuels-drives-innovation-saves-lives/#1a49e34176e0 I watched two...
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Orphan drug sales will outpace all other meds, thanks to high prices
By: Ed SilvermanSource: Pharmalot https://www.statnews.com/pharmalot/2017/02/28/orphan-drugs-prices/As payers continue to foot the bill for orphan drugs, sales of these rare disease treatments are...
View ArticlePart 3 FDA Expedite Programs Series: Fast Track Designation
ObjectiveTo facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. CriteriaAn unmet medical need is defined as providing a therapy...
View ArticleHow to Prepare a Pre-Request for Designation (Pre-RFD): Draft Guidance for...
This guidance is intended to assist sponsors in obtaining a preliminary assessment from the U.S. Food and Drug Administration (FDA or Agency) through the Pre-Request for Designation (Pre-RFD) process....
View ArticleGlobal orphan drugs market to reach $ 209 billion by 2022
BS B2B Bureau | London, UK Source: Business Standard - http://www.business-standard.com/content/b2b-pharma/global-orphan-drugs-market-to-reach-209-billion-by-2022-117030700621_1.htmlWorldwide orphan...
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GOP Senators Ask GAO To Investigate High Prices For Orphan Drugs
By: Sarah Jane Tribble & Sydney LumpkinSource: NPR - http://www.npr.org/sections/health-shots/2017/03/07/518980280/gop-senators-ask-gao-to-investigate-high-prices-for-orphan-drugsBuilding on weeks...
View ArticleRare Diseases at a Glance from the Viewpoint of the European Medicines Agency...
Source: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jspGobal dimensionBecause rare diseases are a global issue, the Agency works closely with its...
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The FDA’s Rare Diseases Program: Driving the development of orphan drugs
Written By: Dr. Nicola DaviesSource: thepharmaletterRare diseases are characterized by the US Food and Drug Administration (FDA) as those conditions that affect less than 200,000 people in the American...
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Rare Disease Parent Meets with President Trump
By: James RadkeSource: Rare Disease ReportOn Monday March 13th, rare disease parent Stan Summers of Box Elder County, Utah talked with President Trump about the struggles that many rare disease...
View ArticleCoté Orphan Expands Services Through Expertise - Dr. Michael Klein, former...
FOR IMMEDIATE RELEASE: May 1, 2017 Coté Orphan LLC(202) 759-9599info@coteorphan.com Coté Orphan Expands Services Through ExpertiseDr. Michael Klein, former Director...
View ArticleCoté Orphan Expands FDA Expedited Program Service Offering
FOR IMMEDIATE RELEASE: May 1, 2017 Coté Orphan LLC(202) 759-9599info@coteorphan.comCoté Orphan Expands FDA Expedited Program Service...
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