Innovation Bootcamp in Rare Diseases  2019 Program

Monday November, 4th

From 8AM onwards  Registration

08.00-09.00               Welcome coffee

09.00-09.15                Opening 
                                     Karen Vanhoorelbeke – KU Leuven



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