Portfolio access agreements – Lessons learnt from Vertex Pharma’s Orkambi

By Jack Rawson, Analyst Email: jrawson@partners4access.com Road to commercialization of Vertex’s CF portfolio The European Commission recently approved Vertex’s combination regimen Symkevi (tezacaftor / ivacaftor; marketed as Symdeko in the US). This is their third disease- modifying Cystic Fibrosis (CF) drug on the market.  The first was Kalydeco (ivacaftor) in 2012, which was the first…

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…

Partners4Access Briefing: November 2018 update on the European Commission regulation proposal on health technology assessment

By Christina Poschen, Consultant Email: cposchen@partners4access.com Background Since 2004, the European Union’s centralised drug approval is in place, allowing manufacturers to submit just one application to gain marketing authorisation in all European Union Member States. Although there were voluntary agreements and working groups to further harmonize health technology assessment, so far, there are 28 different systems…

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).…

CAR-T reimbursement: Lessons learnt from NICE assessments

By Max Rex, Analyst Email: mrex@partners4access.com It’s been difficult to keep up with the flurry of reimbursement news coming out of England relating to the recently approved CAR-T therapies Kymriah (Novartis) and Yescarta (Gilead). Despite only having gained EMA (European Medicines Agency) approval in late August this year, England’s medicines watchdog, NICE (National Institute of…

NICE balks at Spinraza price, rejects reimbursement in its draft guidance

By Christina Poschen, Consultant Email: cposchen@partners4access.com Biogen’s Spinraza, a treatment for the rare genetic disease spinal muscular atrophy (SMA) is currently making headlines for its high price and promising outcomes.  Various European countries including Spain, Italy as well as The Netherlands and Belgium just made the product available to their patients or confirmed they will…