The rise of personalised medicine is revolutionising the healthcare landscape, prompting a re-evaluation of traditional randomised, double-blind drug trials in Germany. At the recent German Network for Personalised Medicine (DNPM) congress, Josef Hecken, chairman of the Federal Joint Committee (G-BA), introduced an innovative approach to evidence generation and patient care within this growing field.
Personalised medicine, which targets specific patient subgroups based on genetic, molecular, or cellular characteristics, challenges the feasibility of conventional trial models due to the small and unique populations involved. Hecken emphasised the necessity for new methods of evidence generation. One promising approach is the establishment of patient registries with automated data collection and therapy endpoints predefined by professional associations.
The suggestion to prescribe personalised drugs in specialised centres aims to maintain high diagnostic quality and effectively manage side effects. This centralised strategy could streamline the approval process and facilitate the collection of comprehensive real-world evidence (RWE), which is crucial for demonstrating the value of personalised therapies.
Despite the higher costs associated with personalised medicine, Hecken argued for the ethical imperative to provide these treatments to eligible patients. This perspective indicates that the G-BA and other Health Technology Assessment (HTA) bodies may increasingly prioritise patient outcomes and long-term benefits over initial costs.
Although randomised controlled trials (RCTs) remain the gold standard in Europe, discussions at the DNPM congress highlight the growing need for innovative evidence generation methods. Active engagement with regulatory and HTA bodies will be essential to ensure alignment with evolving requirements and standards.
Implications:
The shift towards personalised medicine in Germany marks a significant departure from the traditional reliance on RCTs. While this development is promising, it is essential to recognise that previous efforts to incorporate RWE into assessments have faced challenges in Germany. If successful, this new approach could facilitate easier access to personalised medicines, benefiting both patients and manufacturers. We eagerly await the criteria for qualification and hope this initiative succeeds where others have not.
Source:
https://www.aerzteblatt.de/nachrichten/152277/Personalisierte-Medizin-Das-Zeitalter-der-klassischen-Arzneimittelstudien-ist-vorbei?rt=4b5d33e5ec126e7cd8ac579c8e93ff6a. Accessed 21st June 2024