Day 1 Day 2

DAY ONE – Wednesday 29 June 2022

08:15 09:00 Registration, breakfast, coffee and exhibition
09:00 09:05 Welcome
Laura Dormer, Future Science Group
09:05 09:15 Opening remarks
Conference chair
09:20 09:50 Opening keynote fireside chat: understanding the human angle – the impact of rare diseases: my story

Jean-Christophe Novelli

09:50 10:35 Regulator keynote
2025 vision: collaborating to enable use and establish value

  • Delivering future visions through multi-stakeholder collaboration
  • The future of RWE policy
  • Latest updates from Europe, DARWIN EU and more
10:30 11:05 Morning coffee, speed networking and exhibition
COLLABORATION AND MASTERY ZONE DEBATE AND DIALOGUE ZONE
Understanding reimbursement
11:05 11:35 Gaining clarity on RWE and payer models across Europe: managing the uncertainty of approving and reimbursing drugs

Lotte Steuten, Office of Health Economics 

Two parallel sessions:
Fishbowl session – “moving panel” with participants stepping in and out to keep conversation going and fresh ideas flowingHow has access to RWD informed care pathways for patients?

Roundtable conversation – kickstarted with opening remarks, this concurrent conversation is designed to encourage debate and collaboration

Using RWE to support cell and gene therapies

  • Reimbursement and outcome-based agreements to provide cell and gene therapies with the necessary RWD
11:35 12:05 Understanding the lifecycle approach to determining the value of treatments

  • The need for a standardized, iterative scientific process for valuing treatments to support HTA and payers

Amr Makady, Janssen

12:05 12:35 RWE for HTA payer decisions: the rare diseases perspective

  • Overcoming the challenges of reimbursement decisions in rare diseases
Fishbowl session: TBA

Roundtable conversation: Outcomes-based payment models 

12:35 13:05 Economic modelling in rare diseases: how should we assess value? 

Christian Hill, MAP BioPharma

13:05 14:10 Lunch and exhibition
COLLABORATION AND MASTERY ZONE DEBATE AND DIALOGUE ZONE
Achieving patient focus
14:10 14:30 Listen and learn: partnering with patient advocates and groups

 

Fishbowl session: Developing patient centrality in RWE

Roundtable conversation: Using RWD to identify patients eligible for clinical trials

Moderator: Tim Williams, CPRD, MHRA

14:30 15:00 How do you share real-world patient data?

Andreas Hager, Patient Data Lawyer

15:00 16:30 Afternoon tea and exhibition
Registries: curse or cure?
15:10 15:40 Should you trust Trusted Research Environments? Why? Why not?

  • How to collect RWD while remaining GDPR-compliant
  • Leveraging primary care data for planning and research

Susheel Varma, HDR UK

Fishbowl session: Untapped opportunities in registries

  • Treatment vs disease registries
  • How does industry get access to registries?
  • How do we achieve collaboration?

Roundtable conversation: Developments in trial methodology

  • Investigating the different hybrid study designs
  • Determining the efficacy remote and decentralized trials?
16:10 16:40 How are registries learning and changing?

Sofie Terwel, European Blood and Marrow Transplant registry

16:40 16:55 Closing questions
17:00 18:30 Drinks reception

DAY TWO – Thursday 30 June 2022

08:15 09:00 Registration, breakfast, coffee and exhibition
09:00 09:20 Welcome address
Laura Dormer, Future Science Group
09:20 09:50 Harnessing partnerships to unlock data and creative collaborative learning systems
09:50 10:35 Accelerated regulatory approval in 2022 and beyond: managing uncertainty with the impact of Brexit and COVID

  • How have regulators handled areas of unmet need
  • Determining what is reasonable when approving and reimbursing drugs in times of Brexit and COVID

Panelists: Alexander Shacht, Veramed

Leslie Galloway, Ethical Medicines Industry Group

10:35 11:05 Morning coffee, speed networking and exhibition
COLLABORATION AND MASTERY ZONE DEBATE AND DIALOGUE ZONE
Agility and collaboration
11:05 11:35 Achieving transparency in the generation and use of RWE

Richard White, Oxford PharmaGenesis

Fishbowl session: Untapped opportunities in registries

  • Treatment over disease registries
  • Ensuring the industry can get access to registries
  • Determining the steps needed to achieve collaboration

Roundtable conversation: TBA

11:35 12:05 HTA-quality real world data – what quality is required?

  • Clearly defining “regulatory-grade RWD”
  • Ensuring that RWD submitted to regulators can meet expectations
  • The benefits of collaboration in creating regulatory-grade RWD

Catia Proenca, Alira Health

12:05 12:35 Is it fair? Understanding the healthcare disparities and fair access to treatment Fishbowl session: TBA

Roundtable conversation: Data and the patient: are there novel ways of generating patient-centric data?
12:35 13:05 Opportunity for a sponsored feature, if you’re interested in filling this slot please contact our Business Development Director, Ian Law, at [email protected]
13:05 14:10 Lunch and exhibition
COLLABORATION AND MASTERY ZONE DEBATE AND DIALOGUE ZONE
Impact of data quality and artificial intelligence
14:10 14:40 Title TBC: Leveraging EHRs for trial recruitment and management

Tim Williams, CPRD, MHRA

Fishbowl session: TBA

Roundtable conversation: Who owns the data? How much are patients, the public and governments willing to share?

  • Data access and data confidence
14:40 15:10 Leveraging artificial intelligence and machine learning for data quality

Akrivia Health

Speakers TBC

Digital health, cognition and decentralized and virtual trials

  • What we can learn from digital health data and how to best analyse it
  • Teaching from COVID about digital health, cognition and decentralized / virtual trials
  • Current digital health projects underway (e.g., Duchenne Group)
15:10 15:40 Afternoon tea, speed networking and exhibition
15:40 16:10 How much uncertainty in the changing regulatory environment: what is the risk and benefit balance? Fishbowl session: TBA

Roundtable conversation: The challenges of data collection 
16:10 16:40 Closing keynote fireside chat
Topic: TBA
16:40 17:00 Closing keynote remarks
Next steps in RWE and rare diseases