• [[ Whitepaper ]] Connecting With Rare Disease Patient Communities: Patient Advocates

    laureng Whitepapers April 19, 2018
  • [[Whitepaper ]] Disruptive Innovation in Rare Disease Markets

    laureng Whitepapers April 19, 2018
  • Choosing the Right Channel Strategy for Specialty Product Success

    Karina Kusova Clinical Development, Market Access, Orphan Drug Congress, Supply Chain & Manufacturing, Uncategorized, Whitepapers May 3, 2017
  • [Whitepaper] Challenging Conventional Patient Recruitment and Care Strategies for Rare Disease Studies

    Karina Kusova Clinical Development, Orphan Drug Congress, Uncategorized, Whitepapers April 10, 2017
  • [[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

    Karina Kusova Clinical Development, Featured on App, Orphan Drug Congress, Uncategorized, Whitepapers April 6, 2017
  • What’s New in Rare Cancer Research? (Spoiler Alert: A Lot!)

    Karina Kusova Advocacy, Clinical Development, Market Access, Orphan Drug Congress, Orphan Drugs Live October 10, 2016

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Regulation & Government

Regulation & Government

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UniQure Launches the West’s First Gene Therapy at $1.38 Million

In Clinical Development, Regulation & Government by CameronDecember 2, 2014Leave a Comment

Dutch firm UniQure finally brings to market their ground breaking lipoprotein lipase deficiency gene therapy in Germany with partner Chiesi After receiving approval for their …

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Extra 6 Months Market Exclusivity for Drugs Re-purposed for Orphan Indications

In Clinical Development, Regulation & Government by CameronNovember 25, 2014Leave a Comment

A bill submitted to the US House of Representatives would grant an extra six months of market exclusivity to products that are re-purposed to treat …

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EMA Orphan Drug Designations – November 2014

In Regulation & Government by CameronNovember 25, 2014Leave a Comment

See the FDA Orphan Drug designation for October here. EMA Drug: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome Orphan Designation: Adeno-associated viral vector serotype 10 carrying …

Regulation & Government / November 19, 2014

Rare Disease Lobbying Works, but Is It Worth it?

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Should We Be Worried About Pricing, Pipeline, or Policy?

In Market Access, Orphan Drug Congress, Orphan Drugs Live, Regulation & Government by CameronNovember 14, 2014Leave a Comment

If pricing isn’t going to be an issue in the next decade, then should we not be concerned with our development pipeline? Pricing, and the sustainability of high cost …

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FDA Orphan Drug Designations – October 2014

In Regulation & Government by CameronNovember 4, 2014Leave a Comment

Find the EMA’s orphan designation and approvals for October here. FDA – 10/01/2014 Generic Name:  PEGylated recombinant human hyaluronidase PH20 Trade Name:  N/A Orphan Designation:  Treatment of pancreatic …

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EMA Orphan Drug Designations – October 2014

In Regulation & Government by CameronOctober 20, 20144 Comments

Find the EMA’s orphan designation and approvals for September here. EMA – 09/10/2014 Drug: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid Orphan Designation: Systemic Sclerosis Sponsor: Inventiva EMA – 09/10/2014 Drug: …

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A Note To Techdirt and The Street: Read Up on the Orphan Drug Act

In Regulation & Government by Chris HackettOctober 14, 20142 Comments

Catalyst Pharma Accused of ‘Abusing’ the Orphan Drug System Abuse is the improper usage or treatment of an entity. Techdirt put out an article this week which highlights what …

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