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…

NICE balks at Spinraza price, rejects reimbursement in its draft guidance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

Partners4Access expands top leadership with new appointment

Partners4Access has announced the appointment of Alison Kneen as Vice President of International Operations to lead its rapidly expanding Market Access Orphan Drug Specialist team. Alison brings extensive international market access experience built over the last 27 years from previous roles with IQVIA; Principal and Head of Market Access Strategic Consulting, as well as during…