Agenda

9th - 10th March 2017

CHAIR: Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
CO-CHAIR: Segolene Ayme, Director of Research, INSERM

  • Challenges raised by the perception of unjustified high prices and by budget constraints
  • New trends in assessment and appraisal
  • New trends in data collection and data protection
  • Opportunities to generate data demonstrating the added-value of new therapies

Segolene Ayme, Director of Research, INSERM

  • Should rare disease patients have a Macmillan type support service?
  • Could telemedicine and virtual support be a lifeline for rare disease patients?
  • What do rare disease patients want?

Kay Parkinson LLB (Hons), Chief Executive, Cambridge Rare Disease Network

  • What are the challenges HCPs face
  • Why are they different from other therapy areas
  • Using behavioural science to improve patient diagnosis and management

Jane Blyth, Scientific Director, Complete HealthVizion
Angela Ward, Senior Client Service Director, Complete HealthVizion

  • Criteria for Designation
  • Regulatory Review Process
  • Expedited Reviews & Vouchers

Susan Carter, MS, Senior Regulatory Science Specialist, Cote Orphan LLC

  • What contribution have the big pharma companies to guarantee growing development of orphan drugs?
  • Manufacturers collaboration with governments, payers and patients to continue fulfilling unmet needs?
  • How are small biotech start-ups contributing to the orphan drugs market?
  • How can developers demonstrate value for orphan drugs?
  • What is left to be done to increase growth in shortest possible time?

Moderator: Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
Panelist: Steven Zimmer, Chief Executive Officer, MytoDys Therapeutics
Panelist: Dr. med. Jan Geldmacher, Physician and Member, AKDÄ
Panelist: Kay Parkinson LLB (Hons), Chief Executive, Cambridge Rare Disease Network
Panelist: Owen Marks, Country Lead Rare Diseases, Pfizer

  • How to navigate the current regulations
  • How are these regulations influence time to market for orphan drugs?
  • Regulatory obstacles faced by manufacturers during the registration process
  • Designing best strategies for a successful regulatory approval

Andrew Storey, Vice President, Global Regulatory Strategy, AbbVie

  • Identify key aspects of rare disease patients’ mindset and views on logistical elements of clinical trial participation
  • Recognize potential implications of rare disease patients’ views on clinical trial recruitment, retention & compliance
  • Preview more advanced patient survey designs with the aim of further enhancing rare or orphan disease clinical trials

Rolando Gutierrez-Esteinou MD,VP, Therapeutic Area Head Neuroscience Medical & Scientific Services, Covance Inc

  • Rationale for industry
  • Rationale for patient’s organizations
  • Real-life experience
  • Lessons from experience

Christian Girard, Founder, Orphan Drugs Industry Advisory & Intelligence

  • What can stakeholder do to make orphan drugs more affordable to patients?
  • Stakeholders strategies to encourage progress on orphan drugs policies
  • How are government collaborating with stakeholders to make orphan drugs readily available to patient?
  • Stakeholder cooperation to overcome challenges in orphan medicine development

Michael Zaiac, Head of Medical Affairs Haematology/Oncology, EMEA, Celgene

Example of how Alexion works in partnership at different stages of development to increase chances of bringing forward transformative therapies:

  • Follow the biology and apply the best modality
  • Embed research in rare disease hospital
  • Partner with patient foundation and clinician to serve patients as early as possible

Christelle Perros-Huguet, Head of Internal Research, Alexion Pharmaceuticals

  • How can manufacturers work with regulators in understanding regulatory issues and challenges to minimise risks during development?
  • How should the R&D and commercial leaders in pharma anticipate and sort out the public policy risks and benefits of adopting an orphan drug development and commercialization strategy?
  • The future of approving drugs thru orphan drugs designation pathway particularly in near and long term changes that are coming in the future with respect to orphan drugs approval process

Moderator: Sjef De Kimpe, former VP Early Drug Development, BioMarin
Panelist: Susan Carter, MS, Senior Regulatory Science Specialist, Cote Orphan LLC
Panelist: Michael Zaiac, Head of Medical Affairs Haematology/Oncology, EMEA, Celgene
Panelist: Christian Girard, Founder, Orphan Drugs Industry Advisory & Intelligence

CHAIR: Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
CO-CHAIR: Segolene Ayme, Director of Research, INSERM

  • Patients are critical stakeholders of the development paradigm yet engaging them objectively in the clinical development program is a recent focus for pharma
  • Key engagement milestones for Pharma with patients and patient communities
  • Developing a framework for patient feedback and regulatory authority data submission

May Orfali, Patient Avocacy and Policy Lead, Rare Diseases, Pfizer

  • Development of a novel first in class antibody by a small Biotech
  • Overview of OPN-305 development in MDS including Clinical strategy, Engagement of Regulatory, Clinical data in second line MDS population & Lessons learnt

Mary Reilly, VP Pharmaceutical Development & Operations, Opsona Therapeutics

  • Principles of substrate reduction therapy
  • Preclinical data on lucerastat
  • Data on lucerastat in healthy subjects
  • First study with lucerastat in subjects with Fabry disease: safety, tolerability, pharmacodynamics, and pharmacokinetics

Jasper Dingemanse, Vice-President, Head Clinical Pharmacology, Actelion

  • Introducing Raremark, an online service for people living with rare disease
  • Why patient input in drug development? And why now?
  • Attitudes towards data donation among the rare disease patient community
  • Patients’ perceptions towards industry-sponsored medical research
  • Why feedback to patients on results of research participation is key

Pete Chan, Head of Research & Analysis, Raremark

  • Overcoming issues with finding a suitable site and qualified investigators
  • What are the outcome measures and researches supporting clinical development
  • How to create a good working relationships between researchers and patient advocacy

Moderator: Jonathan Appleby PhD, MDL and Chief Scientific Officer, Rare Diseases Gene Therapy, GSK
Panelist: Rolando Gutierrez-Esteinou MD,VP, Therapeutic Area Head Neuroscience Medical & Scientific Services, Covance Inc
Panelist: Jasper Dingemanse, Vice-President, Head Clinical Pharmacology, Actelion
Panelist: Dr. Terence Eagleton, MB BS (Hons), Senior Medical Director, Medpace
Panelist: Pete Chan, Head of Research & Analysis, Raremark
Panelist: Anthony Hall, Therapeutic Area Head Orphan Drugs, Mereo BioPharma

  • Osteogenesis imperfecta; a classical orphan disease
  • Potential treatments for OI
  • Approaches to registration

Anthony Hall, Therapeutic Area Head Orphan Drugs, Mereo BioPharma

  • Ex vivo Gene Therapy – why go to all the trouble?
  • Strimvelis, the first approved ex vivo stem cell gene therapy
  • What’s next

Jonathan Appleby PhD, MDL and Chief Scientific Officer, Rare Diseases Gene Therapy, GSK

    • Background to muscular dystrophy: a paradigm for orphan drug development
    • State-of-the-art in antisense and gene therapies
    • Advantages and obstacles ; Orphan drugs in the pipeline

Dr. George Dickson, Chair of Molecular Cell Biology, Royal Holloway, University of London

  • Understanding how to build the patient forecast based on available epidemiological information
  • Other considerations for pricing and access

Joseph B. Musumeci, Senior Advisor, BluePrint Orphan

  • The complexity of pricing in rare diseases
  • Factors that affect pricing decisions
  • Implications for pricing practices
  • How to ensure a sustainable orphan drug business model for all stakeholders

Adam Hutchings, Managing Director, Dolon

  • Early access to treatment
  • Tailored clinical trials
  • How technology can help patients and improve their lives

Nicholas Adlard, Europe Patient Access Manager, Rare Diseases, Novartis Pharma

  • What can patients and families contribute to the determination of research questions?
  • How patient and family engagement can improve the clinical development pathway
  • Why do we need to incorporate the patient and family perspective in the process for creating innovative therapies?
  • How can we persuade the Regulators and HTA agencies to listen to the end user perspective?

Alastair Kent OBE, Director, Genetic Alliance UK