3rd EHS Task Force Public Debate

Rare Diseases, Advanced Therapies - Gene Therapy
8 July 2021

Play Video

Michel Goldman

President, I3h Institute, ULB - Moderator

“Now that the pandemic is more or less under control, it is the time for addressing major public health issues and especially rare disease research.”

Irene Norstedt

Director, People Directorate DG RTD, European Commission

“Rare disease is a very challenging area. We must collaborate with all sectors at both EU and global level. The EU has already invested quite heavily & will continue to fund research.”

Simone Boselli

Director of Public Affairs at EURORDIS

“We are preparing our patients through training to give input that is timely and effective. Meaningful patient input and engagement at all levels are important for clinical trials.”

Dirk Vander Mijnsbrugge

Vice President, Medical Affairs Lead Rare Diseases, Pfizer

“The Covid-19 pandemic created a sense of urgency which led to an unprecedent level of collaboration, openness & willingness to implement innovative thinking & transformative procedures. Hopefully we will take these lessons learned and apply them to gene therapies.”

Kaja Kantorska

Policy Officer Pharmaceuticals, DG SANTE, European Commission

We are reforming the big pharmaceutical regulation according to three main goals: ensuring timely approuval, enhancing and boosting the development of innovative products and providing better medicine access to patients.”

Brieuc Van Damme

Director-General Healthcare, RIZIV-INAMI

“It is important to facilitate early dialogue between industry, payers & regulators where all actors share their expectations about what they are willing to pay. Contractual pacts can be used to that end.”

Prof. Hildegard Büning

President, European Society of Gene and Cell Therapy

“We have to address three issues: access of patients to the therapies, improving the current therapies and dealing with manufacturing issues.”