Jan-Inge Henter, MD, PhD Professor of Pediatrics Karolinska University Hospital Stockholm, Sweden

Linking Academic Discoveries and Industry Product Development Activities at the Academic Research Centers:Identifying Present Activities and Future Opportunities Jan-Inge Henter, MD, PhD Professor of Pediatrics Karolinska University Hospital Stockholm, Sweden

Activities at Academic Research Centers • THE CLINICAL RESEARCHER • Identifies clinical syndromes • Develops diagnostics (essential for proper therapy)! • Improves the value of existing drugs • Runs clinical trials • Finds new indications for old drugs • Improves patient and disease monitoring

Activities at Academic Research Centers • Discovery (!!) • Research (!) • Biology • Chemistry • Pharmacology • Drug Development • Animal science • Clinical science • Efficacy • Safety

Needs at Academic Research Centers • Innovation centers • Identifying value of potential products • Patent assistance • Investor contacts • Drug development • Contacts with pharmaceutical companies • Governmental support • Would be valuable in bridging the financial gap

Collaboration Academia and Patients/Parents on Rare Diseases • Collaboration Academia – Patients/Parents can be very valuable Supporting clinical studies Supporting research grants Supporting administrative duties = Access to physicians, new treatments and research data • International Clinical Studies can be performed in Rare Diseases • To learn - step by step, stone by stone, patient by patient

How to get Academia into ICORDMake ICORD a Large Rare Disease Forum! • Maybe family organisations can arrange Scientific Society Annual Meetings at ICORD! • Provides access to physicians, new therapy and research • Provides support for academic clinical trials • In addition maybe one common day for all Societies • Statistical lectures • Clinical trials support, ethical applications, FDA/COMP • Industry will get access to many meetings in one • Regulators get close to researchers and industry • Family organisations can meet and teach each other

Future Opportunities Make ICORD a Large Rare Disease Forum!

Jan-Inge Henter, MD, PhD Professor of Pediatrics Karolinska University Hospital Stockholm, Sweden

Jan-Inge Henter, MD, PhD Professor of Pediatrics Karolinska University Hospital Stockholm, Sweden

Presentation Transcript

Nicola Longo MD PhD FACMG Professor and Chief Division of Medical Genetics, Department of Pediatrics, University of Utah

Niklas Nielsen, MD, PhD, EDIC, DEAA Helsingborg Hospital Lund University Sweden

Dale T. Umetsu , MD, PhD The Prince Turki al Saud Professor of Pediatrics

Piotr Richter Professor of Surgery MD PhD The new University Hospital in Krakow

Fernando Mendoza, MD, MPH Professor of Pediatrics Stanford University

Dan Greitz Dept. of Neuroradiology Karolinska University Hospital Stockholm, Sweden

Robert N. Baldassano, MD Colman Professor of Pediatrics

Cathy Jordan, PhD Associate Professor of Pediatrics

Professor .Orhan CANBOLAT ; MD , PhD

Richard C. “Mort” Wasserman, MD, MPH Professor of Pediatrics, University of Vermont

G. deVeber MD, MHS Associate Professor, Pediatrics, University of Toronto

Participating Institutions: Aarhus University Karolinska Institute Stockholm

R van Vollenhoven Karolinska University Hospital, Stockholm, Sweden

Jean-François Lemay, MD FRCPC Professor of Pediatrics Dept of Pediatrics

Valentina Intagliata, MD Assistant Professor of Pediatrics UVa Children ’ s Hospital

Kenneth Haller, MD Associate Professor of Pediatrics

Karolinska University Hospital

Christopher Boys, PhD, LP Associate Professor of Pediatrics

Cathy Jordan, PhD Associate Professor of Pediatrics

Karolinska University Hospital