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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?
Article in Biomedicine hub. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed.
- How to evaluate rare disease policy effectiveness based on policy modeling consistency (PMC) index model: A quantitative assessment of policy implementation in China.Intractable & rare diseases research · 2026Article
- How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases.Orphanet journal of rare diseases · 2025Article
- Reforming the innovation system to deliver affordable medicines: a conceptual framework of pharmaceutical innovation as a complex adaptive system (forest) and theory of change.Journal of pharmaceutical policy and practice · 2025Article
- Project SATURN- a real-world evidence data collaboration with existing European datasets in Osteogenesis Imperfecta to support future therapies.Orphanet journal of rare diseases · 2024Article
- Pull me - push you? The disparate financing mechanisms of drug research in global health.Globalization and health · 2024Article
- Understanding the multifaceted role of miRNAs in Alzheimer's disease pathology.Metabolic brain disease · 2024Review
- Unmet medical needs definition and incentives: stakeholders perspectives on the reform of the EU pharmaceutical legislation.Frontiers in medicine · 2024Article
- Health inequity in genomic personalized medicine in underrepresented populations: a look at the current evidence.Functional & integrative genomics · 2023Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Tackling Mantle Cell Lymphoma in Europe.Healthcare (Basel, Switzerland) · 2022Article
- Making Sure That Orphan Incentives Tip the Right Way in Europe.Healthcare (Basel, Switzerland) · 2022Article
- Towards Better Pharmaceutical Provision in Europe-Who Decides the Future?Healthcare (Basel, Switzerland) · 2022Article
- Meeting the Need for a Discussion of Unmet Medical Need.Healthcare (Basel, Switzerland) · 2022Article
- Clinical outcomes and temporal trends of immunological and non-immunological rare diseases in adult kidney transplant.BMC nephrology · 2021Article
Corrections and comments
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Authors and funding
19 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.