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?
Access
Biomarkers
Biosimilars
Challenges
Citizens
Data
Diagnostics
Empowerment
European Commission
Incentives
Inequality
Member states
Orphan regulation
Patient
Patients
Personalised healthcare
Rare disease
Regulatory
Reimbursement
Treatment
Ultra-rare disease
Unmet need
Journal
Biomedicine hub
ISSN: 2296-6870
Titre abrégé: Biomed Hub
Pays: Switzerland
ID NLM: 101692630
Informations de publication
Date de publication:
Historique:
received:
04
05
2020
accepted:
09
06
2020
entrez:
10
2
2021
pubmed:
11
2
2021
medline:
11
2
2021
Statut:
epublish
Résumé
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.
Identifiants
pubmed: 33564657
doi: 10.1159/000509272
pii: bmh-0005-0001
pmc: PMC7841717
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1-11Informations de copyright
Copyright © 2020 by S. Karger AG, Basel.
Déclaration de conflit d'intérêts
All authors declare that they have no competing interests. Ivana Cattaneo is a Novartis employee and has stock options in Novartis.
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