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Framework for Multistakeholder Patient Registries in the Field of Rare Diseases Schoenmakers, Daphne H.; van den Berg, Sibren; Timmers, Lonneke; Adang, Laura A.; Baumer, Tobias; Bosch, Annet; van de Casteele, Marc; Datema, Mareen R.; Dekker, Hanka; Donnelly, Conan; Driessens, Mariette H. E.; Graessner, Holm; Greger, Valerie; Haddad, Tala; Hoglinger, Gunter U.; van den Hout, Hannerieke; Jonker, Carla; Langeveld, Mirjam; Lambert, Laurie J.; Neacy, Eileen; Nieuwland, Marc; Klockgether, Thomas; van der Knaap, Marjo S.; Papadopoulou, Andri; Plueschke, Kelly; van Rijn, Sanne; Rosenberg, Noa; Saunier-Vivar, Elise F.; Vieira, Bruna dos Santos; Hollak, Carla E. M.; Goettsch, Wim G.; Wolf, Nicole I. Share Save
Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA Cavaller-Bellaubi, Maria; Hughes-Wilson, Wills; Kubinova, Sarka; Van de Casteele, Marc; Van Lente, Evert Jan; Degortes, Emanuele; Ponten, Johan; Eichler, Hans-Georg; Le Cam, Yann; Boselli, Simone; Bucsics, Anna Share Save
Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines Moseley, Jane; Vamvakas, Spiros; Berntgen, Michael; Cave, Alison; Kurz, Xavier; Arlett, Peter; Acha, Virginia; Bennett, Simon; Cohet, Catherine; Corriol-Rohou, Solange; Du Four, Emma; Lamoril, Christelle; Langeneckert, Anja; Koban, Maren; Paste, Muriel; Sandler, Susan; Van Baelen, Karin; Cangini, Agnese; Garcia, Sonia; Obach, Merce; Gimenez Garcia, Emmanuel; Varela Lema, Leonor; Jauhonen, Hanna-Mari; Rannanheimo, Piia; Morrison, Deborah; Van De Casteele, Marc; Stromgren, Anna; Viberg, Anders; Makady, Amr; Guilhaume, Chantal Share Save
The impact of parallel regulatory-health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations Tafuri, Giovanni; Lucas, Ines; Estevao, Steve; Moseley, Jane; d'Andon, Anne; Bruehl, Hannah; Gajraj, Elangovan; Garcia, Sonia; Hedberg, Niklas; Massari, Marco; Molina, Andrea; Obach, Merce; Osipenko, Leeza; Petavy, Frank; Petschulies, Marco; Pontes, Caridad; Russo, Pierluigi; Schiel, Anja; Van de Casteele, Marc; Zebedin-Brandl, Eva-Maria; Rasi, Guido; Vamvakas, Spiros Share Save
How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory-HTA parallel scientific advice Tafuri, Giovanni; Pagnini, Margherita; Moseley, Jane; Massari, Marco; Petavy, Frank; Behring, Antje; Catalan, Arantxa; Gajraj, Elangovan; Hedberg, Niklas; Obach, Merce; Osipenko, Leeza; Russo, Pierluigi; Van De Casteele, Marc; Zebedin, Eva-Maria; Rasi, Guido; Vamvakas, Spiros Share Save
Pdx1 and Ngn3-Cre-Mediated PLAG1 Expression in the Pancreas Leads to Endocrine Hormone Imbalances That Affect Glucose Metabolism Declercq, Jeroen; Kumar, Anujith; Gysemans, Conny; Di Pietro, Caterina; Schraenen, Anica; Chintinne, Marie; Lemaire, Katleen; Van Lommel, Leentje; Van De Casteele, Marc; Heimberg, Harry; Pipeleers, Daniel; Schuit, Frans C.; Mathieu, Chantal; Ectors, Nadine; Van de Ven, Wim J. M.; Verfaillie, Catherine M. Share Save
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