Insight
Discover insight from our renowned speakers and Advisory Board members ahead of the inaugural Real-World Evidence 2022: Rare Diseases and Innovative Therapies meeting. Access exclusive interviews, editorials, journal pieces and more!
(Please note, some of this content was published ahead of the originally planned RWE 2020)
Interviews:
Meet 
the speakers: 5 minutes with Alastair Kent
“Until relatively recently, ‘rare’ was synonymous with ‘not many affected, so not much could be done’, and therefore ‘probably not very important’.”
“…by bringing together families with rare diseases, we have realized that they represent a huge health issue, not just for those affected, but for the health service as a whole. Building that critical mass of affected individuals has both enabled research and facilitated the development of services.”
Read the full interview on The Evidence Base® now>>
Meet the speakers: 5 minutes with Paolo Morgese
“Real-world evidence, the infrastructure and methods to generate, collect and use it, represent the present and the future of innovative healthcare. Without a robust and reliable real-world evidence ecosystem, timely access to advanced therapies will be very challenging and will impose a high burden on society.”
“Without real-world evidence, the choice for access bodies will be between taking ill-informed decisions and not taking decisions, which are practically equivalent.”
Read the full interview on The Evidence Base® now>>
Meet the speakers: 5 minutes with Maya Zlatanova 
“When designing a clinical trial, one must go through protocol development; I have personally witnessed clinical trial protocols structured in such a way that it means that practically no eligible patient exists for trial participation – when we combine indication with eligibility criteria – or that the clinical trial schedule is absolutely impossible to comply with if you are a patient with pain, living far from the trial site, working, have kids etc. This calls for urgent patient and caregiver engagement in clinical trial design.”
“I am excited to see people finally sit together and really try to overcome challenges together because they understand that if we don’t do it together, then it won’t ever happen.”
Read the full interview on The Evidence Base® now>>
Articles published in the Journal of Comparative Effectiveness Research:
- A new modeling approach allowing prediction and comparison of the long-term outcomes of treatments for hemophilia B
Christopher Knight, senior study author - As health technology assessment evolves so must its approach to patient involvement
Karen Facey, author - Bringing regenerative medicines to the clinic: the future for regulation and reimbursement
Panos Kefalas, contributing study author - Building trust in real-world evidence and comparative effectiveness research: the need for transparency
Richard White, author - Can real-world evidence save pharma US$1 billion per year? A framework for an integrated evidence generation strategy
Melvin (Skip) Olson, author - Long-term cost–effectiveness of Oncotype DX® versus current clinical practice from a Dutch cost perspective
Lotte Steuten, senior study author - Policy perspectives on alternative models for pharmaceutical rebates: a report from the Institute for Clinical and Economic Review Policy Summit
Amanda Cole, contributing study author