Case study
How To Expedite Clinical Development Via Early Access Programs
If you are developing a drug for a rare disease in the European Union, you may qualify for an Early…
Case study
A Proactive and Reactive Team in Orphan Drug Development
Are you in need of a proactive and reactive team in orphan drug development? An orphan drug is a designation…
webinar
Regulatory pathway and agency interaction: The EU vs the US
The introduction of a new medicinal product to the US and European market follows a complex process that may extend…
blog
What are the Need-to-Know incentives associated with an Orphan Drug Designation (ODD)?
With a low prevalence, rare diseases affect six to eight percent of the population worldwide and therefore represent a global…
Case study
Multidisciplinary Support for a Successful ODD Application
A US biopharmaceutical client had to withdraw their ODD application during the COMP’s review procedure, due to the lack of…
publication
AMR: Is a Paradigm Shift Needed in Medicines
Antimicrobial resistance (AMR) is the ability developed by bacteria, viruses, parasites or fungi to fight and block the effect of…
publication
Challenges in Conducting Clinical Trials in Rare Diseases
While individually rare, orphan diseases collectively are actually quite common, with an estimated 350 million sufferers worldwide. Since the introduction…
publication
Obtaining Orphan Status – A key milestone within a development strategy
This article describes the regulatory frameworks and incentives associated with orphan drug designation (ODD) in the EU and the US,…
