LAUSR

The International Niemann-Pick Disease Registry (INPDR) is a single, rare disease-specific registry collating global Niemann-Pick Disease data. Created by professionals and patients for worldwide use, it collects clinical and patient reported data with separate datasets for ASMD and NP-C. It will replace the need for multiple registries and offer a single, effective and robustly regulated data resource for NPD. The registry is currently established in 6 countries - UK, Czech Republic, Spain, Italy, Ireland and partly in Germany. Countries currently progressing R and D applications include Germany, USA, Argentina, Brazil, Turkey, Netherlands, France and Australia. Additionally discussions have started with Israel, Taiwan and China. The way forward for the INPDR as well as other LSD and rare disorder registries requires international collaboration. Our experience to date in establishing the INPDR is that there exist major challenges in meeting regulatory requirements, which vary between centres, countries and supranational organisations, such as the European Union. We will be looking at the regulatory requirements of all centres currently undergoing the approval process to compare requirements and identify potential approval barriers, initially starting with those that are a particular challenge for various reasons: USA, France and Germany. In each of these jurisdictions, there are high standards for research compliance, which are not aligned. For example, in the United States, all human research must comply with regulations administered by the Department of Health and Human Services, specifically 45 CFR 46, administered by the Office of Human Research Protections (OHRP), where as in the European Union human research may be subject to the General Data Projection Regulation. We will compare and contrast, in detail, the regulatory requirements that apply to the establishment of an international rare disease registry.