By setting mandatory requirements, paediatric regulations implemented in the EU and the US have allowed the development of – and improved access to – high-quality drugs for the paediatric population. It is acknowledged that paediatric development remains a challenge...

The conference, organized by TOPRA in collaboration with the European Commission (EC), was the opportunity to hear about the EC’s 10-year review report to the European Parliament and the Council. The report provides an account of the paediatric regulation’s...

While individually rare, orphan diseases collectively are actually quite common, with an estimated 350 million sufferers worldwide. Since the introduction of the Orphan Drug Act in the US more than 30 years ago, followed by legislation in Japan in the 1990s, and the...

Advanced therapy medicinal products (ATMPs) belong to a rapidly developing, state-of-the-art field of science and technology in the EU. Due to the complex nature of ATMPs involving gene and cell therapy products, product development can be challenging. Regulatory...

Early access programs (EAP) provide unauthorized medicinal products prior to marketing authorization and commercial launch for life-threatening, chronically or seriously debilitating diseases, which can not be treated satisfactorily by an authorized medicinal...

This article aims to share some key considerations when defining the strategy for the preparation of a risk management plan (RMP). The RMP is required to be submitted when applying for an EU marketing authorisation application (MAA). Advice is offered to applicants...

This article describes the regulatory frameworks and incentives associated with orphan drug designation (ODD) in the EU and the US, as well as capturing key strategic considerations needed to achieve successful orphan drug product development and registration in...

This article introduces the roles of HTA bodies within the EU member states healthcare system and provides key elements to consider for manufacturers, which would like to enter such geographical market, on key elements to consider. In addition solutions to maximize...

Developing the relevant set of potency assays for cell-based medicinal products comes with numerous challenges due to the complex nature of the products. This paper highlights several approaches to consider when designing the potency assay strategy in the framework...

This article, the second in a two part paper highlights the main challenges faced by marketing authorisation applicants for the conversion of a dossier from one region to another, and deciding between a sequential and a parallel preparation.

Nathalie Boeglin...

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