World Orphan Drug Congress 2018 - Agenda Day 1

Day 1, Nov 7



Chair’s opening remarks

Dr Segolene Ayme

European Reference Networks (ERNs) – What are they expected to deliver?

  • Why ERNs were established, how do they work and what kinds of ERNs are already in place for rare diseases?
  • Creating a clear governance structure for knowledge sharing and coordination across the EU to improve access to diagnosis, treatment and care
  • What resources do ERNs have to reach their goals, at a European and Member State level
Dr Sander Van Deventer

Keynote discussion: Executive industry insights - Why are we pursuing the same diseases?

  • What is it that companies need in order explore a new orphan indication or therapy area?
  • How to approach developing and launching treatments in as-yet untreated diseases that are not well-characterized
  • Insights into individual pipelines – what have we learnt that will make orphan drug research and development sustainable?

Morning Networking Refreshment Break



Day 1 ROUNDTABLESCovering a number of hot topics and delivered by patient groups, sponsors 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 45 minutes so you can join two discussions in the allotted time.

Accelerating the review and approval of orphan drugs in LATAM

Meriem Bouslouk

Challenges and opportunities in the G-BA benefit assessment of new orphan drugs

Tamsyn Frost

Common early mistakes in orphan drug development and how to avoid them

Tamsyn Frost, CEO & Co-owner, IDEA Regulatory
Dr Mary Wang

Developing and improving access and data sharing in rare disease registries and biobanks worldwide

Prof Nathalie Goemans, Field Of StudyPediatric Neurology, Rehabilitation Medicine, University Hospitals Leuven
Pawel Wozniak

Developments in Eastern Europe: Unique challenges and opportunities

Evaluating population newborn screening practices for rare diseases

Lisa Dilworth

From Endpoints to Endgame: Incorporating the diagnostic odyssey and patient journey in your clinical development plan

Annie Hubert

Gene therapies : are HTA agencies and healthcare systems in Europe ready for their market adoption?

Dennis Akkaya

How can expanded access combined with real-world data to enhance drug development

  • Key challenges of recruiting patients for drug development trials
  • Overcoming failure to treat and the failure to learn
  • Combining expanded access with Real-world data.

How to measure the full economic impact treatments have on patients through their lifetime?

M Ken Kengatharan

Increasing availability of novel drugs for more prevalent diseases thru “orphanization”

Alastair Kent Obe

Innovative ways patient groups can contribute to the drug development process

Reto Schaberl

Market entry strategies for orphan drugs in Central Eastern Europe

Jasmin Barman

Patient empowerment in access negotiations: The case of “afamelanotide” for the treatment of the ultra-rare light intolerance erythropoietic protoporphyria

Jasmin Barman, Scientific Advisor, SGP
Cinzia Dorigo

Regulatory & operational implementation of the direct to patient trial management model

Dorica Dan

Status of access and power of advocacy in more difficult markets: Quality of life and collaborations with neighbouring countries

Denis Horgan

The current European precision medicine landscape and how the Innovation Medicines Initiative is advancing innovation in the field, through public private partnerships

Scott Schliebner

The virtuality of rare disease clinical trials: Where are we heading?


Networking lunch & poster session

Clinical Development

Chair’s opening remarks

Approval & Access

Chair’s opening remarks

Precision Medicine

Chair’s opening remarks

Pitch & Partner

Chair’s opening remarks

Clinical Development

Clinical Trial Development & Design

Approval & Access

The Value of Orphan Drugs

Cell & Gene Therapy

Clinical Advancements in Cell & Gene Therapy

Precision Medicine

Precision Medicine

Pitch & Partner

Funding & Showcases

Approval & Access

A one stop shop for HTA? The commission proposal and impact on rare diseases

  • The future of healthcare technology assessment in Europe
Speaker:European CommissionFollowed by panel:
  • The likelihood of the proposal materializing and perspectives from the commission, HTAs and industry
  • Are we working collaboratively enough?
Senior representative, EUnetHTA WP4 (pharmaceutical Joint Assessments)
Pitch & Partner

Investor panel: Hear how different investors decide on who and what they invest in, and why

  • Compare and contrast different VC methodologies
  • What other types of innovative funding is there?
  • Unorthodox ways to get money
  • What do pharma look for when in-licensing?
More co-speakers to be confirmed shortly
Clinical Development

Strategies for clinical development in genetic rare disease: Study designs, operational challenges, advanced therapies & Patient finding

  • The definition of genetic rare disease and relatedness
  • Study design considerations :Inclusion and exclusion criteria &Siblings and other relatives
  • Operational:Genetic confirmation- historical proof, timing of assays,Central labs: specialty labs and academic labs &Special populations
  • Advanced therapies:Consent, communication,Long-term follow-up,Logistics
  • Finding patients:Screening studies,Patient registry
Pitch & Partner

Multi-perspective discussion: How to partner with public/private organisations

  • How to approach big pharma/government organisations for support
  • How can companies correctly identify a fit and interest?
  • Can partnering prevent biotech start-ups from getting stuck before Ph1?
  • Do and don’ts with partnerships
More co-speakers to be confirmed shortly

Networking refreshment break

Precision Medicine

Gene editing revolution: Hope for rare diseases, but should there be limits?

Professor María Casado, Director, University of Barcelona
Clinical Development

Debate: Tailoring drug development to meet an evolving orphan drug approval landscape

How does the small clinical trial populations, different types and quantities of evidence, accelerated approval pathway and use of surrogate outcomes influence development plans and assessment of effectiveness?
Precision Medicine

Treating patients suffering from rare and ultra-rare diseases caused by Stop Termination Codon nonsense mutations

Precision Medicine

Should rare disease oncology be considered true orphans?

  • Is it too early to classify the majority of orphan cancers by molecular characteristics?
  • Assessing the value of immuno-oncology treatments
Pitch & Partner

Pitch & Partner 5


Chair’s closing remarks

Pitch & Partner

Pitch & Partner 6

Networking Drinks & Party

last published: 15/Oct/18 14:15 GMT



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