Nicole Sweeny, Shire joined us at the World Orphan Drug Congress USA 2016 to deliver her presentation on “Understanding the Unique Challenges and Opportunities in Rare …
Tackling the Rare Diseases therapy deficit –the nonsense mutation read-through agent
Mark Rothera, PTC Therapeutics, long time friend of the World Orphan Drug Congress USA, joined us once again in 2016. His presentation “Tackling the Rare …
Adaptive Biomedical Innovation: Innovating How We Innovate
Rare Disease research is innovative. The Orphan Drug industry has always embraced the NEW. This is why at the World Orphan Drug Congress USA, we …
Maintaining a relationship based on trust: finding common goals with a KOL and its institution
In a multi-stakeholder industry such as that of Orphan Drugs, communication is vital to ensuring strong, effective working relationships. Charles Stark from Emmaus Lifesciences joined …
Delivering Hope – A First-In-Class Orphan Drug Comes to Market
Blogged on behalf of ClinicalMind >> Hope is perhaps the one thing that 30 million Americans living with a rare disease have to hold on …
‘Significant Benefit’ in the European Regulatory Framework for Orphan Medicinal Products
Orphan Drug regulation has changed over the past 30 years. New legislation like Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures are …
Creating Medicines with Real Value for Orphan Diseases
We were thrilled to attend the RareVoice Awards in November 2016, where we met up with our Everylife Foundation colleagues to celebrate the amazing work …
Re-engineering the process rather than new legislations: Europe’s approach to get more, better, faster, cheaper orphan drugs
Despite great progress, scientific advances are not translated into approved therapies fast enough to cater to the patietns in need. Today’s regulatory system, either for MA …