for Rare Diseases
Some diseases are so rare that there are only a handful of people affected in Germany or even worldwide. Since these diseases are often underdiagnosed and almost unresearched, the exchange among patients and their relatives is difficult and the development of evidence-based treatments or new therapies for these diseases is enormously inhibited.
Therefore, the establishment of a National Registry for Rare Diseases (NARSE) has been identified by the members of our foundation's think tank as a priority goal in improving the care of patients with rare and ultra-rare diseases.
In order to systematically track patients suffering from specific (ultra)rare diseases provide, our foundation has initiated the implementation of such a registry together with the members of the think tank.
- the consent-based entry of patient data by the treating physicians
- the finding and networking of patients
- Entries in the NARSE register by affected persons themselves or patients organisations
- for the first time a comprehensive overview of patients living in Germany
- the exchange of data on an European and international level
- the collection of data according to data protection regulations.
In the current pilot phase, NARSE will initially record ultra-rare diseases (< 1 : 50,000) for which therapy development may already be possible in the foreseeable future. In collaboration with the Centers for Rare Diseases and research initiatives, NARSE should be able to collect basic data on all rare diseases in the future. In this way, we will create an important basis for effective research efforts and new evidence-based treatment methods. In the accompanying evaluation project FAIR4Rare, funded by the Innovation Fund of The Federal Joint Committee (G-BA), the highest decision-making body of the joint self-government, project partners from health services research, clinics, registry operators and patient organizations are examining NARSE: the extent to which it meets with acceptance among users as well as which further developments are necessary to establish it in such a way that patients with rare diseases are enabled to participate more fairly in our health care system.