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FDA Orphan Designations At Record Levels; Resources Need To Keep Up

 
• By Kate Rawson

The development of drugs and biologics for rare diseases has reached a regulatory milestone that underscores industry’s interest in the orphan drug market. FDA’s Office of Orphan Product Development is now granting one new orphan drug designation, on average, every day of the year. That activity underscores the success of the Orphan Drug Act, but creates a new problem: Finding the funding to support it.

The regulatory pathway for nearly every product developed to treat a rare disease begins and ends with the Food & Drug Administration’s Office of Orphan Product Development.

That office reviews requests from sponsors for orphan drug designation at the front end of development, and – should the...

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Ocaliva: Still No Clarity On Why EU Court Opposed Revocation Of Approval

 
• By Neena Brizmohun

Advanz Pharma would have had to show that the European Commission’s decision to revoke Ocaliva’s conditional marketing approval risked causing serious and irreparable harm, according to lawyers from Van Bael & Bellis.

Final Chance To Have Your Say: Take Our Reader Survey This Week

 
• By Nielsen Hobbs

This is your final call to participate in the survey to better understand our subscribers’ content and delivery needs. The deadline is 20 September.

Shape Our Content: Take The Reader Survey

 
• By Nielsen Hobbs

We are conducting a survey to better understand our subscribers’ content and delivery needs. If there are any changes you’d like to see in coverage topics, article format, or the method in which you access the Pink Sheet – or if you love it how it is – now is the time to have your voice heard.

Brazil Pilots Digital Drug Pack Inserts

 
• By Francesca Bruce

A new pilot aims to take Brazil closer to ‘digital transformation.’

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COVID-19 mRNA Vaccine Labels Now Quantify Myocarditis Risk, But ACIP Wants Other Changes

 
• By Sue Sutter

New members of the CDC’s Advisory Committee on Immunization Practices suggested concerns beyond myocarditis, questioning whether test-negative observational studies are giving an incorrect picture of effectiveness.

Medicare’s Combo Products Negotiation Plan Conflicts With US FDA Standards, Firms Argue

 
• By Cathy Kelly

The plan could curtail a period of pricing flexibility for newer subcutaneous versions of important oncology drugs.

EU Pharma Overhaul May Reshape Aspects Of Drug Promotion And HCP Partnerships

 
• By Eliza Slawther

Lawyers explain how changes to rules around drug advertising and promotional activities under the EU pharma reform package could impact companies operating in the bloc, with one legal expert advising companies to “monitor developments closely.”