Head of Cell & Gene Therapy @ ICON plc |Collaboration Expert| Strategic Innovator| Change Agent| Entrepreneur
For patient’s sake - we must keep reminding ourselves that ‘approval’ doesn’t equal ‘patient access...’ Yes, it is of course a crucial step in the development pathway & yes, it is a milestone worth celebrating for all the ‘blood, sweat & tears’ that go into the journey that far but there is still much ground which exists between approval and access that needs to be bridged to enable patients to benefit! Lately, we’ve seen a breakthrough in the # genetherapy space with the recent approvals of Lyfgenia and Casgevy by #Bluebirdbio and #Vertex, respectively, for the treatment of sickle cell disease (SCD)… #patientaccess will now be driven by the ability of each company being able to successfully negotiate deals with payors and insurers globally. I hope that they are successful for the sake of the many sickle cell patients worldwide that will benefit from these marvelous innovations! #patientaccess #sicklecelldisease #raredisease #genetherapy #iconplc
Frances Sassaman
9mo
Thank you for sharing this Emily. A really important and sobering reminder
Senior Director @ ICON plc | Clinical Trials, Pediatric Strategy
8mo💯 So important to remember this! (Also funnily enough I used the Mind the Gap logo in a slide deck the other day, but not sure my audience picked up on it 😅)