Day 1 Orphan 14th Nov

Panel discussion

Plenary discussion: ORPH-VAL Using best practice European principles in assessing the value of orphan medicines: Correcting the discrepancy between countries

  • How do these principles compare to the criteria that current countries use?
  • Opportunity for different European payers/HTAs to openly suggest what is realistic and why
  • Creating a harmonised and sustainable model adapted to rare diseases
  • How is it acceptable that each country has a different post marketing criteria?
  • What are the interests of payers and HTA bodies when considering how to improve access to rare disease therapies?
Panel discussion

Current state of the art in multi-stakeholder and international collaborative processes: The importance of early dialogue initiatives & expedited regulatory pathways

  • Encouraging bigger and more innovative collaboration approaches between pharma, patient and academic organisations
  • Good practices to generate pre and post-marketing data – the value of shared governance of registries
  • Possible solutions and guidance to the regulatory requirements and standardisation of registry data

Networking refreshment break



Covering a number of hot topics and delivered by patient groups, industry and leading solution providers, choose which two roundtable discussions you would like to join, for ‘off the beaten track’ learning and information you can’t find online. Each session will last 40 minutes so you can join two discussions in the allotted time. The same roundtables will be run twice back to back.

Round 1: Bringing clinical trials directly to patients: Solutions to reduce the burden of clinical trial participation
Round 2: Rare disease patients as consumers:  Latest strategies to engage and include patients in the clinical development ecosystem
Scott Schliebner, VP, Scientific Affairs, PRA Health Sciences 
Innovative program design: Strategic program consideration, using observational research, and post-marketing
Dr Michael Murphy, Chief Medical & Scientific Offiicer, Worldwide Clinical Trial 

Integrating patient advocacy groups in the drug development process in rare disease
Lynne Hughes, VP and Global Head of Therapeutic Strategy, Neurology,  QuintilesIMS
Marie Trad, Executive Medical Director, QuintilesIMS
Alan Nolan, Associate Director, Site and Patient Networks (EMEA), QuintilesIMS

A global approach to shared governance of registries across multi-stakeholders including patients
Vanessa Boulanger, Drector of Research Programs, NORD
Jan Span, Member of the Cross-Committee Task Force on Registries and Senior Clinical Assessor, Medicines Evaluation Board, The Netherlands
Jim Green, President, International Niemann-Pick Disease Alliance, UK
Dr Heide Stirnadel-Farrant, Director Epidemiology, Real World Evidence and Epidemiology, R&D Projects Clinical Platforms and Sciences, GSK
David A Pearce, President, Sanford Health and Senior Scientist, Children’s Health Research Center and Professor, Department of Pediatrics, Sanford School of Medicine of the University of South Dakota
Having a multi-stakeholder approach early to maximise your chances in a new launch – Overcoming clinical and market challenges
Rob Walton, Managing Partner, Cello Health Public Affairs

Moving the needle from anguish to hope: How the OMP regualtion impacts lives of rare disease patients
Ivana Cattaneo, Public Affairs Director, Novartis Oncology Europe
How European Reference Networks (ERNs) could become part of the solution/ enablers of quality data generation?
Maryna Kolochavina, Project Director, RWLP Operations, Europe and Asia Pacific, INC Research/inVentiv Health

Strategies to provide patient-centric services to speed up development timelines 
Gail Adinamis​, CEO, GlobalCare Clinical Trials
Effective strategies for setting drug prices and achieving reimbursement of orphan drugs
Matthias Schönermark, Chief Executive Officer, SKC Beratungsgesellschaft
Real-world patient experience: A patient-led, peer reviewed survey
Julie Walters, Founder, Raremark
Alan Thomas, Founder, Atacsia a Fi - Ataxia and Me

How certain should we be before we make medicines available?
Dr Paul Robinson, Executive Director, Medicine & Patient Perspectives, MSD
Using new technologies to predict the outcome of approval evaluations
Dr Pedro Franco, Director of Europe for Global Regulatory & Scientific Policy (GRASP), Merck KGaA

Value based agreements & innovative contracting with orphan drugs
Omar Ali, Visiting Lecturer, University of Portsmouth
& Former Adviser for NICE Adoption & Impact Programme Reference Panel
Addressing the elephant in the room: Next steps towards long-term affordability & sustainability problems with RD drug development and access
Dr Carina Schey, Researcher, University of Groningen

Networking Lunch & Poster Session

Innovative Clinical Trial Partnerships & Designs

The Value of Orphan Drugs

Advancing Operations and the Place for Partnerships

The Business of Rare Disease


Ex vivo gene therapy for monogenic bone marrow disorders

  • Sharing GSK’s journey in treating ADA-SCID through gene therapy: perspectives on regulation, facing challenging communities and the reputable high price

Early access to orphan medicines – a glimpse into the future

  • Current market trends and key predictions over next 5 years in relation to how pharma and biotech companies will plan for and enable successful early access that compliments and forms part of their market access strategy

Patient Engagement and Patient Data


Networking refreshment break



At what price? Addressing the most difficult question in rare diseases

  • 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

Preclinical & Clinical Advancements


Incorporating the Patient Voice into the Rare Disease Therapeutic Development Process

  • Significant attention has been given to the concept of patient engagement in the drug development process in the past few years and rightly so
  • This presentation will examine specific touchpoints during the rare disease therapeutic development process where the patient voice can be invaluable
  • Providing patients and their families, patient advocates, and sponsor companies an overview how and when the patient/caregiver perspective can shape drug development for a specific therapeutic

Assessing the Value of Immunotherapy (IO): Challenges and Opportunities

  • How this has affected future plans with increasingly more treatments tailored for smaller populations

Discussion: Examining the challenges of valuation from payers and HTAs

  • Pricing and evaluating orphan drugs - present and future
  • What are the interests of payers and HTA bodies when considering how to improve access to rare disease therapies?
  • How will the increase of cancer, biosimilars and gene therapies change the pricing and access?

The future of gene therapies for rare disease: Managing the rapid progression of gene therapies

  • Review of industry trends and recent collaborative development programmes
  • What can we expect moving forward into 2018?

Chair’s closing remarks


Networking Drinks

last published: 10/Nov/17 16:05 GMT