Let us help you to accelerate your orphan designation
Developing drugs and finding effective treatments for patients with rare diseases present one of the most scientifically complicated challenges for the medical world. Millions of people suffer from a rare disease but only a few percentage of the known, over 6 000 such diseases have any approved treatment. For us at Accelsiors, this huge medical need is something that we fully embrace as a part of paving the way for future research.
We provide a comprehensive solution to properly handle your orphan drug development program from professional guidance on orphan designation application preparations to successful clinical study conduct at any stage of clinical research.
Professional support to reach an access to the benefits of orphan designation
However, the designation application procedure consists of a network of complex and interconnected pathways and processes Sponsors of designated orphan medicines authorized by EMA and/or FDA can benefit from numerous incentives both in the EU and the US both, such as
- granted 7 (US) / 10 (EU) years market protection and exclusivity,
- special scientific advice and assistance on study protocol,
- various fee reductions,
- or access to the centralized authorization procedure in the EU.
Do you need more professional help?
Feel free to ask Accelsiors’ Orphan Designation experts! Please, contact us:
Write Us
Call Us: +36 1 299 0091
Request for Proposal
and we will schedule a meeting around your calendar!
Let us support your team’s knowledge and success! Accelsiors’ experienced orphan drug development team will help you find the shortest and most efficient way and advise you how to prepare and conduct successfully the application process. Our experts have significant experience in dealing with all the challenges of orphan designation applications. We provide
- an expert analysis and advice on how to overcome challenges arising from orphan designation application set-up,
- a proper scientific justification and data supporting the intended use is submitted at any stage of development,
- we will guide you through the current regulatory framework both in the EU and in the United States, or support also for EU legal representation,
- and help you in collating all the necessary documentation to support the submission, validation, and ongoing updates.
17 years of experience with clinical trials in rare diseases
Accelsiors is an expert in planning and conducting clinical trials in orphan medicines for a wide range of therapeutic areas and indications, such as Acromegaly, Cushing Syndrome, Genetic Diseases, Cystic Fibrosis and Growth Hormone Disorders, etc. both in pediatric and adult population. We have an excellent understanding of study endpoints, scientific and medical aspects, deep experience in assay validation and additionally, a close relationship with a network of Key Opinion Leaders in these fields to help reduce costs of your study.
Dedicated rare disease study team to tailor and lead your development
Do you need a specialist CRO partner to support and successfully navigate your development challenges with a rare disease? Our team can help you to accelerate your program. We not only monitor a study! We gain access to our multidisciplinary team of experts with in-depth knowledge of the therapeutic area. The members of our StrongCore ScientificTM group are involved in harmony with your special development needs, who understand your needs, interpret data in its real context, tailor the clinical development strategy and advise you concerning the optimal strategic direction at any stage of the clinical trials.
