Agenda at a Glance

Orphan Drugs Conference Day 2

Day 2: Wednesday 21st October 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

 

Panellists:

  • Manolo Bellotto, General Manager, Gain Therapeutics SA
  • Dr David A Walker, Professor, Paediatric Oncology, University of Nottingham, Chair, CBTDDC
  • Samantha Oliver, Joint Managing Director, OPEN VIE

- 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In the past 4 decades only 5 drugs have been licensed for brain tumour use, only 3 for children, of which one has been withdrawn.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, Paediatric Oncology, University of Nottingham, Chair, Children’s Brain Tumour Drug Delivery Consortium

Dr Femida Gwadry-Sridhar, Chief Executive Officer & Founder, Pulse Infoframe

 

> How MAPs fit with an orphan launch strategy

> How to design them effectively taking all stakeholders into account

> Best practice in implementation and management

> When does a MAP end?

 

Robert Donnell, Head of Business Development, Durbin PLC, part of uniphar group

David Gillen, Vice President, International Medical Affairs, Vertex

  • There are numerous infectious diseases that are rare and qualified for orphan designation
  • A number of bacterial infections that are unmet medical needs and orphaned
  • An overview of orphan opportunities in ID and the clinical development of targeted anti-bacterial monoclonal antibodies will be provided

Vu Truong, Chief Executive Officer, Aridis Pharmaceutical

Case Study

Accelerating development and lowering treatment for rare disease

Assessing the commercial roadblocks for growth of repurposed drugs

Table 1: Overcoming recruitment challenges in rare disease clinical research

Karen Matthews, Business Development Manager, NIHR

 

Table 2: Patient-centric Drug Development in Rare Diseases: The Ying & Yang

Ricardo Hermosilla, Rare Disease Consultant, formerly, Translational Medicine Leader, F. Hoffmann-La Roche Ltd

 

Table 3: The use of AI in drug discovery & development

Anthony Hall, Co-Founder and Trustee, Findacure

 

Table 4: Improving healthcare communications in rare disease and orphan drugs

Benjamin James, Trustee, DMD pathfinders

 

Table 5: The challenges of implementing mobile research nursing in rare disease trials

Helen Springford, Chief Operating Officer, Illingworth Research Group

-   Case Study

-   Accelerating development and lowering treatment for rare disease

-   Assessing the commercial roadblocks for growth of repurposed drugs

Moderator:

Anthony Hall, Co-Founder & Trustee, Findacure

Panellist:

  • Cesare Spadoni, Co-founder & Chief Operating Officer, Oncoheroes Biosciences Inc.
  • Dr. Didier Caizergues, Head, Regulatory Affairs Department, GENETHON

-   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

  • 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

> International comparison of appraisal processes

> Optimizing deliberative processes to determine value

> Post appraisal determination of value

 

Karen Facey, Senior Research Fellow, Impact HTA - Co-Lead Investigator, WP10 Appraisal of Orphan Medicinal Products

-   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

 

Panellist:

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

-   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

 

Panellists:

  • Dr. Fatima Sulaiman, Head of Research and Services, Scleroderma & Raynaud's UK
  • Bob Stevens, Group CEO, MPS Society & MPS Commercial

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