Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases - Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?

Autor:	Horgan D; European Alliance for Personalised Medicine, Brussels, Belgium., Moss B; Europacolon (UK), London, United Kingdom.; Bowel Cancer UK, London, United Kingdom., Boccia S; Sezione di Igiene, Dipartimento Universitario Scienze della Vita e Sanità Pubblica, Università Cattolica del Sacro Cuore, Rome, Italy.; Department of Woman and Child Health and Public Health - Public Health Area, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy., Genuardi M; European Society of Human Genetics, Vienna, Austria.; Complex Operational Unit, Medical Genetics, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy., Gajewski M; Alexion, Zurich, Switzerland., Capurso G; PancreatoBiliary Endoscopy and EUS Division, Pancreas Translational and Clinical Research Center, San Raffaele Scientific Institute IRCCS, Milan, Italy., Fenaux P; Service d'hématologie seniors, Hôpital St Louis / Université Paris 7, Paris, France.; ERN-EuroBloodNet, the European Reference Network on Rare Hematological Disease, Paris, France., Gulbis B; ERN-EuroBloodNet, the European Reference Network on Rare Hematological Disease, Paris, France.; Ht. ERASME-CUB, Brussels, LHUB-ULB, Brussels, Belgium., Pellegrini M; ERN-EuroBloodNet, the European Reference Network on Rare Hematological Disease, Paris, France.; Assistance Publique-Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France., Mañú Pereira MDM; ERN-EuroBloodNet, the European Reference Network on Rare Hematological Disease, Paris, France.; University Hospital Vall d'Hebron - Vall d'Hebron Research Institute, Barcelona, Spain., Gutiérrez Valle V; ERN-EuroBloodNet, the European Reference Network on Rare Hematological Disease, Paris, France.; University Hospital Vall d'Hebron - Vall d'Hebron Research Institute, Barcelona, Spain., Gutiérrez Ibarluzea I; EuroScan International Network, Cologne, Germany.; BIOEF, Basque Foundation for Health Innovation and Research, Barakaldo, Basque Country, Spain., Kent A; Independent Patient Advocate, London, United Kingdom., Cattaneo I; Novartis Pharma SpA, Origgio, Italy., Jagielska B; Clinic of Oncological Diagnosis and Cardio-oncology, Maria Skłodowska-Curie Institute of Oncology, Warsaw, Poland., Belina I; Coalition of Healthcare Association, Zagreb, Croatia., Tumiene B; Vilnius University Hospital Santaros Clinics, Vilnius, Lithuania., Ward A; F. Hoffmann-La Roche Ltd, Basel, Switzerland., Papaluca M; Imperial College London, London, United Kingdom.
Jazyk:	angličtina
Zdroj:	Biomedicine hub [Biomed Hub] 2020 Jul 17; Vol. 5 (2), pp. 1-11. Date of Electronic Publication: 2020 Jul 17 (Print Publication: 2020).
DOI:	10.1159/000509272
Abstrakt:	Since developments are global in the healthcare arena, more should be done to align EU and other big markets' regulatory practices for rare disease patients. Notwithstanding efforts and cooperation between the US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulatory criteria and procedures to gain the designation, terms and classifications should be still harmonised. Aligning the criteria of prevalence and support to orphan medicines in the various jurisdictions internationally, would facilitate patient recruitment eventually at global level, so as to gain the data and the biological insights required to identify biomarkers and appropriate endpoints needed for progressing clinical development. A conducive regulatory environment can further support the development of medicines to treat rare diseases. Overall there is a need for joined-up regulatory process coordination. Better integration of regulatory pathways and better integration of regulatory systems, such as scientific tools and methods to generate evidence, would be helpful. There is a need to revise and agree the current frameworks to be improved which will take into account the considerations and challenges to diagnose and treat different rare diseases and improve quality of life. Deliberative processes with multi-stakeholders' involvement for reimbursement should be considered. This paper explores the successes and limitation of both the regulation and its implementation mechanisms in the current regulatory context, and suggests some improvements that could maximise its benefits and boost rare disease research even further. Competing Interests: All authors declare that they have no competing interests. Ivana Cattaneo is a Novartis employee and has stock options in Novartis. (Copyright © 2020 by S. Karger AG, Basel.)
Databáze:	MEDLINE
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