Agenda at a Glance

Orphan Drugs Conference Day 2

Day 2: Wednesday 18th March 2020

  • Can big data improve the diagnosis of rare disease?
  • Focus on data quality validation, alternative research designs and how they affect outcome assessment, and aspects of reporting and transparency
  • Use of data and knowledge management to increase efficiency
  • The importance of patient registries to optimize rare disease management and improve patient outcomes
  • International collaboration and data transparency to increase understanding, diagnosis and developing therapies for rare diseases

Moderator: Femida Gwadry-Sridhar, Chief Executive Officer and Founder, Pulse Infoframe

  • Brain tumours in children are differ from adults, arise in the developing brain and need therapies to their type and location.
  • In the past 4 decades only 5 drugs have been licensed for brain tumour use, only 3 for children, of which one has been withdrawn.
  • The diversity of tumour type and growth patterns the detail of which need to be incirporated into drug a delivery system design to ensure that the drug is delivered at effective concentrations to the sites of tumour growth.
  • The talk will identify the challenges facing delivery system designers and the opportunities of repurposing drugs with such systems.  This will be illustrated by the referring to the current trial development of Convection Enhance Drug Delivery of Carboplatin and Sodium Valproate for Diffuse Intrinsic Pontine Glioma of childhood that is in process.

Dr David A Walker, Professor of Paediatric Oncology, Children’s Brain Tumour Research Centre

  • Unpack the meaning and utility of real-world data (RWD) & real-real world evidence (RWE)
  • Explain regulatory and legislative underpinning
  • Identify disparate sources from which RWD is obtained
  • Describe the collaborative ecosystems optimized to enable data

Femida Gwadry-Sridhar, Chief Executive Officer and Founder, Pulse Infoframe

-   Current state of RNAi therapeutics development

-   Overcoming challenges in developmental process of RNAi treatment

-   Addressing the challenges of delivering RNAi to new cell types

-   RNAi therapies in the pipeline

Round table Discussions (2 x 30 mins. Session. with *10 mins. in between to swap tables)


Table 1:

Topic to be Confirmed

Rosa Oliveira, Senior Director, Head Commercial Neurology, EMEA, Lupin Neurosciences


Table 2:

Overcoming recruitment challenges in rare disease clinical research

Karen Matthews, Business Development Manager, NIHR


Table 3:

Topic to be Confirmed

Dr. Rohit Batta, Chief Medical Officer, Vicore Pharma Holding AB


Table 4:

Topic to be Confirmed

Sylvie Gabriel, SVP, Global Market Access & Pricing, Ipsen


Table 5:

Topic to be Confirmed

Kerry Leeson-Beevers, National Development Manager & Project Lead, Alström Syndrome

Case Study

Accelerating development and lowering treatment for rare disease

Assessing the commercial roadblocks for growth of repurposed drugs

-   Need for better research

-   The UK as a Hub

-   Changing the Research Model

-   Delivering better research

Leslie Galloway, Expert Adviser, NICE, Chairman, Ethical Medicines Industry Group

  • Market implication as a result of Brexit
  • Diverse political and economic implications which can affect payers leading to potential restriction in pricing
  • Impact potential if current legislation is changed

  • Means in overcoming issues with “Diagnostic Odyssey“.
  • Scientific advances in rare disease research that can positively impact treatments
  • Strategies to overcome issues the rare disease community experience in accessing medical care, treatments, and supplemental income.
  • Addressing the complete lack of public awareness

Scroll to Top