AGENDA OVERVIEW

 

The 8th World Orphan Drug Congress is taking place 13 - 15 November, Barcelona and is not an event to be missed this year. Now recognised as the largest and most established European orphan drug event, we are once again proud to deliver another fantastic speaker line up and content filled agenda that represents the whole orphan drug scene. The 8th annual event to address the strategic and commercial aspects of bringing new treatments to patients who suffer from rare diseases. You’ll notice that the Patient Advocacy Track is no longer here, it’s because we’ve integrated the patient voice in every part of the whole agenda.

Through strategic keynote plenaries, themed tracks and dedicated networking sessions, you have the chance to really tailor the event to the needs of your day-to-day role.

Attend the Pre-Congress Workshop on the 13th November to learn from interactive presentations and make new contacts during the networking breaks.

 

Barcelona, 13 - 15 November 2017

Schedule

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Nov 1309:00
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Morning Pre-congress Workshop Monday 13 November

Keynotes
Pricing, Reimbursement and Market Access Challenges for Advanced Therapy Medicinal Product / Orphan Drugs in the EU – to be updated
 
Major hurdles exist even in the EU health systems delaying and preventing patient access to Orphan Drugs (ODs) approved by the EMA. If possible, such hurdles are even greater for ODs that are also Advanced Therapy Medicinal Products (ATMPs) - Cell therapies, gene therapies and hybrid cell-gene therapies.
 
In this workshop we will review the actual market access (MA) status and timing in the main EU countries of ODs approved by the EMA in the recent past and will then discuss the additional issues for ATMP/ODs.
 
Real-world experience will be presented and discussed, with the testimony of a medium-size European Company with a specific focus on ODs and, in particular, ATMP/ODs.
Finally, potential solutions will be discussed, to address key pricing, reimbursement and MA challenges for ATMP/ODs in the EU context.
Gain from a unique combination of 1) manufacturer, customer, economic, and clinical perspectives combined with 2) solid academic theory from attending this workshop.
 
Dr Renato Dellamano, President, MME Europe
Tiziana Magni, Global Pricing & Market Access Specialist, CHIESI FARMACEUTICI SPA
Davide Finocchiaro, Global Pricing & Market Access Specialist, CHIESI FARMACEUTICI SPA

 
Expertise shared from MME:
  • In addition to work in oncology and orphan markets, we have helped developers and marketers of >40 ultra-orphan drugs and several ATMPs to identify, frame, communicate and capture the value of their therapies.
  • Unique combination of manufacturer and customer perspectives combined with solid academic theory
  • Strategy development and tactical execution to support informed decision making
  • Assessment and planning of opportunities and competitive situations at every stage of the product life cycle
  • Our methods are designed to develop an understanding of the value perceptions of all meaningful stakeholders
 
What are the benefits of attending this workshop?
  • The orphan markets have examples of products that have exceeded expectations as well as some that have yet to achieve initial forecasts: pricing and reimbursement is a factor that can work in either direction.
  • During this workshop we will look at the general issues and challenges for ODs and at the additional particular challenges for ODs that are also ATMPs.
  • In this particularly innovative field, understanding the current orphan drug environment and the potential changes in the near future include sustainability of orphan drug pricing plus the additional key issues related to pricing and reimbursement of cell and gene therapies.
 
12.30 Networking Lunch followed by afternoon workshop
 
Nov 1313:10
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Afternoon Pre-congress Workshop Monday 13 November

Keynotes
Assuring success from Regulatory to Global Market Access of Rare Disease Treatments
 
13.30 – 17.00:
Partnering with patients for clinical success
  • This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them for the successful design and execution of clinical studies. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation.
  • Participants will then learn how insights are analysed and applied in order to:
    • Design studies that appeal to the targeted patients
    • Determine effective and motivating recruitment methods
    • Engage and communicate with patients and caregivers
    • Identify retention methods that meet the needs of a specific patient population
    • Develop materials that resonate with patients and caregivers
Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group
 
An introduction to the science of endpoint design in the current regulatory and HTA environment in the US and Europe
  • Regulatory strategies for early scientific advice: FDA & EMA scientific advice, PRIME & adaptive pathways link to scientific advice
  • The creation, categorization and hierarchy of endpoints and requirements of different stakeholder groups, including: regulatory agencies, clinicians, HTA bodies, payers, increasingly active patient groups
  • Real-life examples of endpoints used in selected therapeutic area & techniques for the development and validation of new endpoints
  • Highlighting guidance and publications that will be of use to newcomers to the area
  • Solving the non-alignment between different agencies and geographical  differences in approach
Rory Graham, Senior Director, EU Regulatory Services, Mapi Group
Martine Zimmermann, Global Head of Regulatory Affairs, Alexion pharma GmbH
 
Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies
  • How is expanded access used to provide treatment for patients with rare diseases?
  • Compassionate Use Programs in the window between submission and commercialization
  • What are the benefits of conducting clinical studies and registries for rare diseases?
  • What are the types of studies conducted during different phases of orphan drug development?
  • How are patient registries used to generate real-world data for rare diseases and orphan drugs?
  • Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
  • What are the unique challenges of conducting rare disease/orphan drug registries?
  • How can proactive study management strategies successfully address these challenges?
  • Potential risk mitigation strategies, and alternative registration strategies will be discussed.  
Dr Will Maier, Chief Scientific Officer and Head of Patient-Centered Sciences, Mapi Group
 
17.00 End of Workshop Followed by Networking Drinks Hosted by Mapi Group

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Nov 1408:50
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Nov 1409:00
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Plenary discussion: ORPH-VAL Using best practice European principles in assessing the value of orphan medicines: Correcting the discrepancy between countries

Keynotes
  • 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?
More panellists to be confirmed shortly:
Senior representative HTA/payer, Italy
Senior representative HTA/payer, France
 
Nov 1409:50
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Current state of the art in multi-stakeholder and international collaborative processes: The importance of early dialogue initiatives & expedited regulatory pathways

Keynotes
  • 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
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 Aliance
Nov 1411:30
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INTERACTIVE ROUNDTABLES

Keynotes
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.

DRUG DEVELOPMENT roundtables:

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, CMO, 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
 
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


ACCESS & MARKET CHALLENGES roundtables
 
Effective strategies for setting drug prices and achieving reimbursement of orphan drugs
Matthias Schönermark, Chief Executive Officer, SKC Beratungsgesellschaft
 
Case study: Supporting market access through patient experience – How early patient engagement will support the value of new treatments
Julie Walters, Founder, Raremark
Patient group representative – TBC

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


How should we value repurposed drugs?
Sheela Upadhyaya, Associate Director Highly Specialised Technologies, NICE
 
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
 
Nov 1414:15
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Chair opening remarks

SCIENCE & STRATEGY
Nov 1414:20
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A joint Innovative clinical development and commercialization collaboration for TAK-935 in rare paediatric epilepsies

CLINICAL DEVELOPMENT
Emiliangelo Ratti, Senior Vice President and Head of C.N.S. Therapeutic Area Unit, Takeda Pharmaceuticals International Ltd
Nov 1414:20
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How will the new Highly specialised technology (HST) methods from NICE change the way you prepare?

APPROVAL & ACCESS
Nov 1414:20
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Advancing innovation and the development of gene therapies through the maintenance of a collaborative approach

GENE THERAPY
  • Sharing GSK’s journey in treating ADA-SCID through gene therapy: perspectives on regulation, facing challenging communities and the reputable high price
Nov 1414:20
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A sustainable approach to repurposing generic drugs for rare diseases: Proving a social impact bond concept

SCIENCE & STRATEGY
Nov 1414:45
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Nov 1414:45
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Making sure your patients understand: A review of translation methods for consistency of patient-facing documents for rare disease research in international trials

APPROVAL & ACCESS
Senior representative, Mapi Group
Nov 1414:45
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The evolving orphan drug development model: how the business case to pursue a disease changes over time

SCIENCE & STRATEGY
Nov 1415:15
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Tailoring drug development to meet an evolving orphan drug approval landscape

CLINICAL DEVELOPMENT
Senior representative, INC Research
Nov 1415:15
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Early access to orphan medicines – a glimpse into the future

APPROVAL & ACCESS
  • 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
Nov 1415:15
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Successfully managing and implementing drug development programs for domestic and international commercialisation of orphan drugs

GENE THERAPY
Nov 1415:15
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Global regulatory landscape for rare disease therapeutics: implications on approval and access

SCIENCE & STRATEGY
Nov 1415:40
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Embracing real-world evidence as a tool to demonstrate effectiveness beyond the clinical trial

APPROVAL & ACCESS
Nov 1415:40
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Innovative strategies for clinical development for genetic rare disease

CLINICAL DEVELOPMENT
  •          Advancing of development of technologies and platforms supporting product development for genetic rare diseases
  •          Novel protocol design to approach clinical development for genetic rare disease: umbrella protocols and basket studies
  •          Gene therapy clinical development design supported by RWE to improve management of post marketing requirements and to facilitate price negotiation
Nov 1415:40
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Resource management: Strategies enabling the efficient development of multiple promising gene therapy programs – even as a smaller company

GENE THERAPY
  • Clinical trial updates
  • The importance of education and communication for accelerated program progression
Nov 1415:40
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Do we need to bring the patient together with the science and what is the role of patient engagement in research and development?

SCIENCE & STRATEGY
Nov 1416:35
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Making sure your patients understand: A review of translation methods for consistency of patient-facing documents for rare disease research in international trials

CLINICAL DEVELOPMENT
Nov 1416:35
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At what price? Addressing the most difficult question in rare diseases

APPROVAL & ACCESS
 
  • 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
Nov 1416:35
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Are novel pricing & reimbursement mechanisms for gene therapy products necessary?

GENE THERAPY
Nov 1416:35
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The need for patient engagement throughout the life cycle of rare disease drug development

SCIENCE & STRATEGY
Speaker 1
Nov 1417:00
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Incorporating the Patient Voice into the Rare Disease Therapeutic Development Process

CLINICAL DEVELOPMENT
  • 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
Nov 1417:00
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Assessing the Value of Immunotherapy (IO): Challenges and Opportunities

APPROVAL & ACCESS
 
  • How this has affected future plans with increasingly more treatments tailored for smaller populations
Nov 1417:00
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Preclinical and clinical development of tolerogenic nanoparticles to mitigate immunogenicity against enzyme and gene therapies for orphan diseases

GENE THERAPY
Nov 1417:00
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The need for patient engagement throughout the life cycle of rare disease drug development

SCIENCE & STRATEGY
Speaker 2
Nov 1417:25
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Discussion: Examining the challenges of valuation from payers and HTAs

APPROVAL & ACCESS
 
  • 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?
Nov 1417:25
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MPS I and MPS II: Evaluating and selecting the appropriate route of administration for gene therapy

GENE THERAPY
Nov 1417:25
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The role of patient registries, biobanks and clinical bioinformatics within rare disease programmes

SCIENCE & STRATEGY
Nov 1417:50
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Overcoming patient recruitment & trial design challenges to accelerate clinical progression for gene therapies

CLINICAL DEVELOPMENT
Nov 1417:50
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The future of gene therapies for rare disease: Managing the rapid progression of gene therapies

GENE THERAPY
  • Review of industry trends and recent collaborative development programmes
  • What can we expect moving forward into 2018?
Nov 1417:50
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Integrating rare disease patient registries into electronic health records

SCIENCE & STRATEGY

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Nov 1508:30
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Nov 1508:35
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Executive industry discussion: Intentions of pharmaceutical companies developing treatments for rare disease patients – What do they hope to achieve?

Keynotes
 
  • The evolving role of pharma and multi-stakeholders
  • What are the challenges?
  • What are the options moving forward?
  • How do we work together to improve this?
Nov 1509:30
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Case study: Moving Mountains: how one parent led charity raised and spent millions, accelerating research and impacting the landscape

Keynotes
  • Developing innovative and holistic funding model to accelerate trials in rare disease
  • Leveraging investment model to bridge the “valley of death”
  • How we did it
Emily Crossley, Co-founder, Duchenne U.K.
Nov 1509:55
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Introducing a new industrial player emerging from a patient organisation, for the industrialisation of cell & gene therapies to be commercialised at a fair price

Keynotes
 
  • Materializing research outcomes into products
  • The role of a patient group, AFM-Telethon from innovation to production
  • Ability to provide production to pharma and biotech
  • Using new models to give a fair opportunity to all stakeholders
Nov 1511:20
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INTERACTIVE ROUNDTABLES

Keynotes
Covering a number of hot topics and delivered by patient groups, industry and leading solution providers, choose which ONE roundtable discussion you would like to join, for ‘off the beaten track’ learning and information you can’t find online. The session will last 45:


REGIONAL PROVISIONS FOR ORPHAN DRUGS roundtables

Developments in Eastern Europe: Unique challenges and opportunities
Pawel Wozniak, Managing Director, Komtur Polska

A landscape analysis of Rare Diseases and Orphan Drugs availability in South Eastern Europe

Anja Kladar, Rare Diseases Croatia, Executive Director

The future of a fully integrated multi-stake holder model – what can we learn from USA advocate perspective?

Debbie Drell, Director of Membership, NORD

Differences in OD development and registration in the Nordic markets

Einar Andreassen, Senior Adviser, Norwegian Medicines Agency
Dr Douglas Lundin, Chief Economist, TLV, Dental and Pharmaceutical Benefits Agency, Sweden


Linking registries and clinical trials through ERNs:  The paradigm shift in meeting Marketing Authorization requirements
George Reynolds, COO, Vitro Software Int

PATIENT ENGAGEMENT roundtables

ADCY5-related Dyskinesia case study: how research and clinical advancements occur through engagement of the rare disease patient community

Christina Waters, CEO and Founder, RARE Science

How we can ensure rapid development of gene therapies for rare diseases by working with patients and patient organisations?

Emily Culme-Seymour, External Strategy Manager, Gene Therapy, GSK

GOSgene: enabling innovation through gene discovery. Application of precision medicine within rare disease development

Dr Hywel Williams, Senior Research Associate, The Centre for Translational Omics – GOSgene, Genomics and Systems Medicine, UCL, GOS Institute of Child Health
 

A guide to effective patient submissions for HTA processes
Sandra Nestler-Parr, Director of Rare Access and as Trustee of Alpha-1 UK Support Group
Sheela Upadhyaya, Associate Director Highly Specialised Technologies, NICE

How to develop a platform to unite clinical research on paediatric RDs
Beygonya Nafria Escalera, Patient Advocacy Manager, Sant Joan de Deu Children's Hospital

First-hand experience as a patient representative: Making a difference from trial design to access negotiations in orphan drugs
Dr Jasmin Barman, Scientific Advisor, Swiss Society for Porphyria

Scientific innovation to bring new treatments for rare disease (mAbs, gene therapy, mRNA-based therapeutics, synthetic biology, antisense)

Ensuring that new technologies and platforms are continually incorporated into pre-launch strategies, development and with patients
Nov 1513:35
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Nov 1513:35
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Nov 1513:35
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Nov 1513:35
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Nov 1513:40
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An update on Fondazione Telethon’s multi-stakeholder collaborative model with pharmaceutical/biotech companies and the success story and strategy leading to Strimvelis

PRODUCT DEVELOPMENT
Lucia Faccio, Head of Research Development Office, Fondazione Telethon
Nov 1513:40
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Investor panel: Hear how different investors decide on who and what they invest in, and why

PITCH & PARTNER
 
  • What stage do they invest in biotechs and why?
  • How much clinical data do they need to demonstrate?
  • What terms do VCs look for to invest?
  • What could a biotech demonstrate to investors earlier to gain their support?
Additional panellists to be confirmed...
Nov 1513:40
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What changes need to be made in the public arena to create the right platform for gene therapy?

GENE THERAPY
Nov 1513:40
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Potential impact and contribution of an Orphan Diagnostics Act to drug development for rare diseases in the future

SCIENCE & STRATEGY
Nov 1514:05
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Post-approval challenges for orphan advanced therapies: Where we stand and what lies ahead

GENE THERAPY
Nov 1514:05
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The potential of gene editing therapies for orphan drugs: Harnessing CRISPR technology for rare disease drug development

SCIENCE & STRATEGY
  • The current state of CRISPR gene editing technology  
  • Employing revolutionary gene editing technology to treat monogenic rare diseases
  • The opportunities and the challenges 
Nov 1514:35
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A panel discussion on an innovative and groundbreaking collaborative approach to approaching HTA in rare disease

PRODUCT DEVELOPMENT
Project HERCULES: A platform to share evidence to HTAs – a model for DMD and beyond

Senior pharma representative
Senior HTA representative
 
Emily Crossley, Co-founder, Duchenne U.K.
Fleur Chandler, Therapy Area Head of Respiratory Value Evidence and Outcomes, GlaxoSmithKline Biologicals
Josie Godfrey, Director at JG Zebra Consulting, Former Associate Director of HST, National Institute for Health and Care Excellence
Nov 1514:35
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Pitch & Partner 1: A progress update on small molecule-based causal therapy for FSHD

PITCH & PARTNER
Nov 1514:35
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Overcoming the complex challenges associated with ex vivo gene therapies

GENE THERAPY
Nov 1514:50
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Pitch & Partner 2: Drug repurposing in CNS orphan indications

PITCH & PARTNER

 
Nov 1515:05
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Pitch & Partner 3: BRAINCURES PLATFORM: a tool for intelligent repurposing, drug discovery and personalized medicine

PITCH & PARTNER
Nov 1515:05
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Advances in genetics for rare diseases: Is gene therapy the best way forward?

GENE THERAPY
Nov 1515:05
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Successfully exploiting CAR-T approaches to meet the needs of patients where there are very few treatment advances

SCIENCE & STRATEGY
Nov 1516:05
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Best practices when considering gene therapies: compliantly facilitating PAGs panel

GENE THERAPY
Nov 1516:05
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A cannabinoid-based medicine offering relief and meeting the needs of patients with a rare childhood-onset epilepsy

SCIENCE & STRATEGY
Nov 1516:35
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Paediatric drug development: The interaction between industry, physicians and patient groups to get the drug into the market and subsequent P&R approval

PRODUCT DEVELOPMENT

Senior representative, Novartis
Nov 1516:35
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Galafold’s approval in Europe and the HTA alignment required for its success

SCIENCE & STRATEGY
Francis Pang, VP of Global Market Access, Amicus Therapeutics Inc
last published: 18/Oct/17 14:35 GMT