Rare Diseases: A New Purpose

How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: nmurad@partners4access.com The US National Institutes of Health describes drug repurposing as “studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other…

P4A to present Cell & Gene Therapy insights at WODC 2018

Partners4Access, a leading market access consultancy specializing in orphan drugs, cell and gene therapy today announced its participation at the World Orphan Drug Congress, an industry conference that brings together rare disease stakeholders under a common platform. The three-day event is being held between November 6-8, 2018 in Barcelona, Spain. P4A’s Sophie Schmitz, Managing Partner…

LentiGlobin: a potential gamechanger in thalassaemia therapy

By Jack Rawson, Analyst Email: jrawson@partners4access.com October 2018 turned out to be a good month for β-thalassemia patients seeking new and effective treatments. The European Medicine Agency (EMA) accepted US-based biotech firm bluebird bio’s marketing authorization application (MAA). The new gene therapy – LentiGlobin – could be a gamechanger for patients with transfusion-dependent β-thalassemia (TDT).…