Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union

Establishing medical plausibility in the context of orphan medicines designation in the European Union

In the European Union, sponsors have the responsibility to demonstrate the "intention to diagnose, prevent or treat" a serious and rare condition before the Committee of Orphan Medicinal Products (COMP), for a medicinal product to meet the criteria for Orphan Designation. This requirement is commonly referred to as "medical plausibility" and the justification of this intention is assessed on th...

Use of biomarkers in the context of orphan medicines designation in the European Union

The use of biomarkers within the procedures of the Committee of Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) is discussed herein. The applications for Orphan Medicinal Product designation in the EU are evaluated at two stages. At the time of orphan designation application, the file undergoes an assessment to establish whether the proposed condition is a distinct and s...

Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation

Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation

Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation

Reaching Outside the Comfort Zone: Realising the FCTC’s Potential for Public Health Governance and Regulation in the European Union; Comment on “The Legal Strength of International Health Instruments – What It Brings to Global Health Governance?”

In their paper, Nikogosian and Kickbusch show how the effects of the adoption by the World Health Organization (WHO) of the Framework Convention on Tobacco Control (WHO FCTC) and its first Protocol extend beyond tobacco control and contribute to public health governance more broadly, by revealing new processes, institutions and instruments. While there are certainly good reasons to be optimisti...