Competencies Consulting Services Drug Development

Orphan designation

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Let us help you to accelerate your orphan drug designation application in the EU and the US both

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.

Ask Accelsiors' Clinical Trial Regulatory Experts

Do you need a clarification for your drug development dilemmas, or you have just got a question that you would like to clarify?

Please contact us and we will schedule a full hour complimentary drug development consultancy.

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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.
Let us support your team’s knowledge and success!

One of the primary objectives for the authorities is to support the development of new treatments for rare diseases and conditions with no available treatment. Both EMA and FDA are able to grant Orphan-Drug Designation (ODD) to a medicine or biological compound to prevent, diagnose or treat unmet diseases. When receiving ODD, you are eligible for protocol assistance at a reduced charge, market exclusivity after approval, fee reductions, tax credits for qualified clinical trials, and so on.

Here at Accelsiors, we can support you during the whole process of preparing and submitting your ODD application.

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,
  • and smoothly navigating you trough the network of complex and interconnected pathways and processes of the designation application procedure.
Two decades 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.

Let us accelerate your clinical drug development program!

Are you interested in learning more about how Accelsiors can create solutions to help you accelerate your clinical trials? Our team is ready to answer your questions. Get in touch with our experienced clinical drug development experts!