Future-proofing 2019 plans: P4A’s 6 Orphan Drug, Cell & Gene Therapy trends to watch out for

2019 is expected to a strong year for the orphan drug industry with an expected 10% surge in sales growth. Contributing to this growth will be new treatment launches particularly from cell and gene therapies. As these novel therapies are geared towards commercialization, the industry is witnessing a change in approach by manufacturers, regulators 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…

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).…

NICE balks at Spinraza price, rejects reimbursement in its draft guidance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