Day 2 Congress – 6th March 2019

Matthias P. Schönermark, Managing Director, SKC Beratungsgesellschaft mbH
Strategies in patient recruitment and retention for a successful clinical trial in rare diseases
How can you prepare healthcare providers to know about the therapy and identify the right patients for your therapy?
How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?
What Are the factors and steps to be taken to ensure success of the drug development?

Tijana Bugarcic, Managing Director, Rarebase, Evaluator, New Therapies for Rare Diseases, European Commission
Thomas Ogorka, Chief Executive Officer, Orphan Reach Ltd
Alain Rinaldi, CEO & Founder, MEDExpansion, CMO & Co-founder, Overlord Pharmaceuticals
Pedro Franco, Director of Europe, Global Regulatory & Scientific Policy, Merck KGaA
Femida Gwadry‐Sridhar, Founder and CEO, Pulse Infoframe Inc.

  • 1968 lift off! The BBS Charity established
  • Disease versus Condition
  • lets talk about language
  • rare bone cousins and our extended family
  • Progress and goals in our healthcare timeline

Patricia Osborne, CEO, Brittle Bone Society

  • What does the Rare Disease Landscape look like today?
  • Strategies for overcoming challenges faced in Rare Disease Study Management
  • Key considerations in setting up Rare Disease Program or study
  • Innovations that enhance Rare Disease Drug Development
  • Applying Patient centric solutions to manage Rare Disease studies

Zizi Imatorbhebhe, Sr Director, Strategic Dev't, Head of PAREXEL’s Rare Disease Center of Excellence, PAREXEL International

  • Muscular dystrophy as paradigm for orphan drug development
  • Promising gene therapy strategies for the treatment of muscular dystrophy
  • Future perspectives; orphan drugs in the pipeline

Alberto Malerba, Research Officer and Project Manager, Royal Holloway, University of London

Emily Culme-Seymour, Associate Director, Clinical Sciences, Sangamo Therapeutics, Inc.

  • Improved clinical outcomes require more than just clinical development
  • Understanding the patient journey in rare diseases is critical to successful clinical development
  • Innovative communication strategies, patient journey insights and behavioural science techniques can all contribute to improved patient
    outcomes

Reserve for COMPLETE HEALTHVIZION

  • Scaling up a patient group for Alkaptonuria (AKU)vb,
  • Building the scientific community around the disease
  • Organising clinical trials of a promising treatment

Nicolas Sireau, CEO and Chair of Trustees, AKU Society

  • What regulatory mechanisms for pricing can be put in place to ensure fast access to needed medicines for patients with rare diseases in the region?
  • How to close the gap between regulatory approval and funding agreement?
  • How to overcome struggles to meet the strict cost-effectiveness threshold for new therapeutics?
  • What is the argument behind undertaking of assessments if it is known in advance that the drug will not be costs effective? How can
    this be changed?
  • How the key HTA and payers operate?

Moderator:
Josie Godfrey, Director, JG Zebra Consulting, former Associate Director, NICE

Panellist:
Julian Trimming, EMEA Regional Commercial Director, Aegerion
Markus Peters, Consultant, former Chief Commercial Officer, Agilis Biotherapeutics
Matthias P. Schönermark, Managing Director, SKC Beratungsgesellschaft mbH
Pedro Franco, Director of Europe, Global Regulatory & Scientific Policy, Merck KGaA

  • How can manufacturers meet global patient demand for access to medicines prior to approval?
  • How can governments and orphan drug companies help support each other’s initiative to provide market access to patients?
  • What are some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the
    market?
  • What R&D strategies can be implemented to accelerate the market access for orphan drugs?

Richard Porter, Chief Operating Officer, Therachon

  • How to assess value of orphan drugs.
  • What are the key challenges with ultra orphan drugs, from a payers perspective.
  • How to deal with the uncertainty in the value assessments

Kristian Samdal, Senior Adviser, Norwegian Medicines Agency

  • The changing environment for ultra orphan drugs
  • High hurdles with little data: examples of creative data generation to support value

Julian Trimming, EMEA Regional Commercial Director, Aegerion

  • 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

Panelist:
Josie Godfrey, Director, JG Zebra Consulting, former Associate Director, NICE
Pedro Franco, Director of Europe, Global Regulatory & Scientific Policy, Merck KGaA

Matthias P. Schönermark, Managing Director, SKC Beratungsgesellschaft mbH