Innovation Bootcamp in Rare Diseases

04 nov
4 november 2019 08:00 – 5 november 2019 18:00Toevoegen aan kalender 2019-11-04 07:00:00 2019-11-05 17:00:00 Title Description Location Domus Medica Europe/Brussels public
Andere organisatie

Faculty Club
Groot Begijnhof 14
3000 Leuven

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What are the key drivers and resistors in the development of diagnostics and therapeutics in rare diseases in 2019? Get economy insights in the healthcare ecosystem of rare diseases. Your update on ATMPs and innovative diagnostics. How to raise awareness on rare diseases? What about access and affordabilityof orphan drugs? Do not miss out on this unique event and join us in Leuven for IBRD 2019. Looking forward to welcome you all in Leuven prof. dr. Karen Vanhoorelbeke (coordinator) and the entire PROFILE team. The PROFILE project is funded by the HORIZON 2020 programme of the European Union.


Monday November, 4th

  • From 8AM onwards Registration
  • 08.00-09.00 Welcome coffee
  • 09.00-09.15 Opening - Karen Vanhoorelbeke – KU Leuven / TBA / TBA
  • 09.15-10.30 Session 1 Rare diseases and orphan drug landscape in 2019
    • 09.15-09.30 Eva Schoeters – RADIORG: Stronger together. The added value of uniting rare disease patients.
    • 09.30-10.00 Chris Van Hul – Onafhankelijk Ziekenfonds: Solidarity as central concept in healthcare organization and insurance for rare disease patients in Belgium
    • 10.00-10.30 Antonine Wyffels – RIZIV INAMI: Update on new developments in RIZV/INAMI conventions and multi-level collaboration incentives on rare diseases
    • 10.30-10.45 Marianne Vanden Bussche – Vandekerckhove – Eye Hope Foundation: The diagnostic and therapeutic oddysey seen from the patient perspective
  • 10.45-11.15 coffee break
  • 11.15-13.30 Session 2 Innovations in RD diagnostics and therapy
    • 11.15-12.00 Erik Tambuyzer – BioPontis Alliance for Rare Diseases: Opportunities and challenges in orphan drug development: policies and technology platforms
    • 12.00-12.30 Hanspeter Rottensteiner – Takeda: Advancements and bottlenecks in gene therapy for hemophilia
    • 12.30-13.00 Rudy Van Coster – UZ Gent: Implementation of next generation sequencing in the diagnosis of neuromuscular disorders
    • 13.00-13.30 Petra Kaufmann – AveXis: Gene therapy for rare diseases
  • 13.30-14.30 lunch break
  • 14.30-16.30 Session 3 Innovative entrepreneurship & health economics in rare diseases
    • 14.30-15.15 Aidan Hollis – University of Calgary: Sustainable funding of innovation in rare diseases and orphan drug development: a economist’s perspective
    • 15.15-16.30 Kris Vander Velpen – Flanders Business School: Strategy thinking, opportunity creation and value proposition in the field of rare diseases
  • 16.30-17.00 coffee break
  • 17.00-18.00 Session 4 Interdisciplinarity and paradigm rethinking I
    • 17.00-17.20 Guy T’Sjoen – UZ Gent: Healthcare organization in transgender medicine
    • 17.20-17.40 Marie-Françoise Vincent – Cliniques Universitaires St Luc: Organization of a rare disease center: experiences of a clinical biologist
    • 17.40-18.00 Bruce Poppe – UZ Gent: Clinical implementation of a multidisciplinary diagnostic approach for rare diseases
  • 18.00-18.15 Short break
  • 18.15-19.15 Session 5 Keynote lecture
    • Timothy Cox – Cambridge University: The truth about rare
  • 19.15 networking reception

Tuesday November, 5th

  • 08.30-10.00 Session 6 Innovations in diagnosis and treatment of rare types of cancer
    • 08.30-09.00 Peter Vandenberghe – LKI/UZ Leuven: CAR T cell therapy in the clinic: lessons learned and the path ahead
    • 09.00-09.30 Marc Peeters – UZA: Tackling the challenges in diagnosis, therapy and healthcare organisation of rare cancers
    • 09.30-10.00 Jan Bogaerts – EORTC: Rational design of clinical trials in rare types of cancer
  • 10.00-10.30 coffee break
  • 10.30-12.00 Session 7 Perceptions on rare diseases and treatments
    • 10.30-10.55 Patrick Tabouring – Université de Luxembourg: Dealing with rare diseases in first-line healthcare: experiences from a GP
    • 10.55-11.20 Rene Westhovens – UZ Leuven: Challenges in implementing optimal care in rare diseases
    • 11.20-11.35 Beatrice Gulbis – ULB Hôpital Erasme: EuroBloodNet: providing specialized care for patients with rare hematological diseases across Europe
    • 11.35-12.00 Marc Dooms – UZ Leuven: Dealing with rare diseases by community and hospital pharmacists: prevention and treatment
  • 12.00-13.00 lunch break
  • 13.00-15.00 Session 8 Interdisciplinarity and paradigm rethinking II
    • 13.00-13.30 Rik Schrijvers – UZ Leuven: Meeting the needs for patients with primary immunodeficiencies
    • 13.30-14.00 Dirk Vander Mijnsbrugge – Pfizer: Hone your Real World Evidence generation strategy in rare diseases
    • 14.00-14.30 David Cassiman – UZ Leuven: Responsible multi-stakeholder management of the orphan drug pipeline
    • 14.30-15.00 Bart Leroy – UZ Gent / Philadelphia Children’s Hospital: Leaving no stone unturned in getting gene therapy to patients with inherited retinal dystrophies
  • 15.00-15.30 coffee break
  • 15.30-16.30 Session 9 Development of orphan medicines
    • 15.30-15.50 Gerben van ‘t Klooster – Galapagos: Orphan drug development from target to clinical program
    • 15.50-16.10 Beatriz Pujol – Alexion: Development of innovative drugs for rare diseases: highs and lows from three decades of frontline research
    • 16.10-16.30 Filip Callewaert – Sanofi: Bringing Cablivi to aTTP patients: from bench to bedside
  • 16.30-16.45 short break
  • 16.45-18.00 Session 10 Orphan drug access and affordability
    • 16.45-17.10 Spring Gombé – Drugs for Neglected Diseases initiative: Beyond the silos: implications of the common features of neglected and rare diseases
    • 17.10-17.35 Ana Palma – SOBI: Access to orphan drugs – how may the current environment impact it in the future
    • 17.35-18.00 Olga Solomon – European Commission: Outcomes from the evaluation of the legislation on medicines for children and rare diseases
  • 18.00 closing

Venue: Faculty Club, Groot Begijnhof 14, 3000 Leuven, Belgium Please click here to get there by car. Please click here to get there by public transport. Directions for public transport to LeuvenAccomodation in Leuven (not included in registration fee)

Rates full registration – two days:

  • Junior academic: € 295 (PhD students and residents in training)
  • Senior academic and other: € 420
  • Industry: € 550

Registration includes: access to event, catering during the event and networking reception on Monday 4 November 2019 and event bag including program book.

Rates day ticket, either Monday November 4th or Tuesday November 5th:

  • Junior academic: € 180 (PhD students and residents in training)
  • Senior academic and other: € 250
  • Industry: € 330

Registration includes: access to event, catering during the event and networking reception on Monday 4 November 2019 and event bag including program book. Patient organisation representatives please contact prior to registration.

Registration and information: