Knowledge generation in the field of drug development for individuals suffering from rare diseases faces difficulties that must be overcome. Digitisation is believed to contribute significant improvements in knowledge generation in this field, and here, the expected improvement from increasing digitalisation is outlined from the perspective of three healthcare institutions: the Federal Institute for Drugs and Medical Devices, the Institute for Quality and Efficiency in Health Care, and the Federal Joint Committee.
The three healthcare institutions argue that:
- Digitisation has a high potential to increase the efficiency of clinical development and regulatory decision making
- Digitisation can be a tool to reduce barriers to the implementation of care-associated, register-based, randomised controlled trials
- High-quality registry studies should start within the approval process to ensure the evidence necessary for therapy decisions is available immediately following approval
The final point emphasised that the improvement of the evidence base through the qualitative improvement of data sources will result in direct benefits to patients, with effective drug provision being ensured by usable evidence that can be generated over longer periods of time. The institutions concur that high-quality indication registers should be developed as independent infrastructures to facilitate the access of high-quality data early in the medicinal development for rare diseases.
Sources:
- Evidenzbasierte Arzneimittelversorgung bei Seltenen Erkrankungen: die Rolle der Digitalisierung. https://www.iqwig.de/presse/pressemitteilungen/pressemitteilungen-detailseite_80709.html. Accessed 1st November 2022.