Regulating advanced therapy medicinal products through the Hospital Exemption: an analysis of regulatory approaches in nine EU countries.

To study regulatory approaches for the implementation and utilization of the Hospital Exemption (HE) in nine EU countries. Using public regulatory documentation and interviews with authorities we characterized the national implementation process of the HE, including national implementation characteristics and two outcomes: national licensing provisions and the amount of license holders. National licensing provisions vary substantially among selected countries as a result of different regulatory considerations that relate to unmet medical needs, benefit/risk balance, and innovation.

Publication rates and reported results in a cohort of gene- and cell-based therapy trials.

We investigated publication rates and reported results for gene- and cell-based therapy trials. In a cohort of Institutional Review Board (IRB)-authorized trials during 2007-2017 in the Netherlands (n = 105), we examine publication rates and reported results in scientific papers and conference abstracts as well as associations with the occurrence of trial characteristics. The publication rate for scientific papers was 27% and 17% for conference abstracts (median survival time: 1050 days).

Access to hepatitis C medicines.

Hepatitis C is a global epidemic. Worldwide, 185 million people are estimated to be infected, most of whom live in low- and middle-income countries. Recent advances in the development of antiviral drugs have produced therapies that are more effective, safer and better tolerated than existing treatments for the disease.