.@VitalTransform is proud to be supporting the 1st Int Conference on Rare Diseases:
“Building a Pathway from Diagnosis to Access”
1-2 March 2021
Info & Registration -> https://t.co/X5IsY9qSKp
#rarediseasesconference21
Do not miss this - Register and Share!
Advancing patient-driven research in @Share4Rare
Join us in the Virtual Coffee with patient organisations on Tuesday, June 16, 16:00 CEST.
@eurordis@eupatients@eupatientsforum
https://t.co/tKTI2MBbY0
Novartis has sold 100 lots of its gene therapy for spinal muscular atrophy so far this year at $1.75 million a shot. Not much uptake for annual payments. This is a disease with no other treatment. I do not believe these data will apply to #hemophilia. https://t.co/apON9fafNu
On September 7, I bring my light for World Duchenne Awareness Day to raise awareness on #Duchenne muscular dystrophy. Join @DuchenneDay for #WDAD2019 🎈
https://t.co/ApD7TD4nrH
Delighted to see strong media coverage of our new study across Europe
At a glance, here's how Europe's top 30 medical universities performed on #clinicaltrials transparency
https://t.co/JQJpa5fOcV
Three questions yesterday @EFPIA dinner:
1 What does #EU contribute to #health & care?
2 Are we adding the maximum value to #patients, families, & healthcare workers?
3 Can we do more to lessen the burden on health systems today & in the future?
Answers 👉https://t.co/eItiFQ1roE
#FDA officials see 'missed opportunity' with patient outcomes in clinical trials: FDA has shown an interest in PROs,: The information is seen as a valuable tool for improving clinical trial designs, guiding drug approvals & supporting label decisions.
https://t.co/rKcYkgOkWe
#ppmdconference there was mention about the FDA being prepared to extrapolate from trial populations to wider populations. The EMA seems less so, as well as payers. How can we address this with pharma?
#EMA carried out a total of 925 activities involving #patients and 445 activities involving #HealthcareProfessionals during 2017. Read more in our latest Stakeholder Engagement report: https://t.co/g6x8v4koFs
📣Make sure to register at the best rate! 2 days left to benefit from Early Bird rates for the Joint #EFGCP & @DrugInfoAssn Better Medicines for Children Conference: https://t.co/lc2r1WIbug #Paediatrics2018