Background: TREAT-NMD is an international network of excellence facilitating collaborative research in neuromuscular disease (NMD). Having developed a range of infrastructures, the network aims to accelerate drug development, provide new therapies to patients swiftly and improve access to relevant information on standards of diagnosis and care. One of the key infrastructures is the Global Registry Network which supports individual registries to collect standardised data items from the TREAT-NMD Core Datasets from consenting individuals. These NMD patient registries are valuable resources for facilitating and accelerating research and clinical trials. The Global Registry Network has been created in collaboration with clinicians and patient organisations, and collects key information required for clinical trial feasibility purposes or to establish clinical trial eligibility. Aggregated data reports and other information can be requested securely through the Global Registry Enquiries process.
Objectives: Global Registry Enquiries aim to support stakeholders with data provision or information distribution to primarily facilitate clinical trial planning or recruitment, and to further academic research.
Method: Global Registry Enquiries are reviewed by the TREAT-NMD Global Data systems Oversight Committee (TGDOC) who govern the Global Registry Network. Enquiries can be categorised as data enquiries, providing stakeholders with de-identified, aggregated data from the network, or recruitment enquiries, where clinical trial information is distributed to eligible NMD patients via those registries.
Results: Since 2009, there have been approximately 86 initial enquiries made from academics, clinicians, industry, contract research organisations, regulatory agencies and patient organisations as part of the Global Registry Enquiries process. Of these, 41 (48%) have resulted in successful registry enquiries that have been conducted. The majority (78%) of which have supported data or recruitment enquiries specifically for industry.
Conclusion: TREAT-NMD is still positioned to support high quality registry enquiries from a wide range of stakeholders. As new therapies are approved there will be an increasing demand for provision of de-identified patient-level data to align with Post Marketing Surveillance commitments from pharmaceutical regulatory agencies. The development of the TREAT-NMD Global Registries Platform will help support this new stream of work.