Germany’s first outcomes deal for CAR-T-cell therapy

By Ciaran Cassidy, Analyst Email: ccassidy@partners4access.com Novartis recently announced an innovative reimbursement scheme with the GWQ –  (a body representing a small number of insurance funds in Germany) –  for its CAR-T cell therapy, Kymriah (tisagenlecleucel). Since September 2018, Kymriah has been used to treat patients up to the age of 25 with refractory and…

Sustainable funding of cell and gene therapies: bluebird bio puts the ball in the payer’s court

By Nader Murad, Associate Consultant Email: nmurad@partners4access.com At JPMorgan’s  37th Healthcare Conference in 2019, bluebird bio provided its strategic outlook and plans for the year where it highlighted its pipeline progress and commercial activities. bluebird’s pipeline consists of several gene therapies which have demonstrated in clinical studies a high likelihood of potentially curing severe rare…

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…

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NICE balks at Spinraza price, rejects reimbursement in its draft guidance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

Slow Brexit proceedings frustrates biotech industry

By Aparna Krishnan, Corporate Affairs Lead As the UK Prime Minister’s office is preparing for the cabinet meeting at Chequers this Friday, British businesses including the biotech industry are none the wiser on what lays ahead after March 29, 2019 when the UK exits the European Union. The current discussion is focussed on the Brexit…