Global Registry Enquiries
The TREAT-NMD Global Registry Network is a federated network of individual, independent patient registries that collect data on neuromuscular patients. The network is recognised as a leading source of data for supporting research, trial planning and trial recruitment at an international level and can be used to support other activities to improve patient care, such as the assessment of care standards in different countries. For information on how the network is composed, managed and governed please see here.
The TREAT-NMD Global Registry Network has been active for over a decade so far and has facilitated academic and industry partnered projects that have a goal to facilitate translational and clinical research. TGDOC has worked with many of the pharmaceutical companies in the neuromuscular field to complete feasibility enquiries for clinical programmes and clinical trial planning as well as recruitment for clinical trials and a multinational study focusing on health economics. All of this work is undertaken with the aim of ensuring that promising new treatments can be brought to patients as quickly as possible.
What is a Global Registry Enquiry?
Pharmaceutical companies, academic researchers, patient organisations and anyone with an interest in neuromuscular patient data are able to request information from the TREAT-NMD Global Registry Network. TGDOC Data Enquiries provide aggregated and deidentified data reports in a secure fashion, including breakdowns of data from individual national registries. These often include general data on patient numbers within particular national registries, and numbers of patients that meet certain criteria, such as specific genetic mutation, age range, ambulation status, etc. These are often requested in the planning phases of a clinical trial. TREAT-NMD is able to provide individual registry data in addition to a master global datasheet, and a summary report to the enquirer, and offer a follow-up call with the TGDOC Chairs to discuss any queries or follow-up questions the enquirer may have based on the report.
Another service TREAT-NMD can provide is recruitment support for clinical trials through TGDOC Recruitment Enquiries, where information on specific clinical trials is sent only to patients likely to be eligible to participate. Patients can be contacted through their national registry if they meet the clinical trial inclusion criteria and are then asked to contact their local trial site. Patients not seen at a particular site, and who wouldn’t otherwise be known to staff at that site, can also be informed about the trial Patients’ details are never disclosed directly to enquirers, but companies can ensure their trial information reaches their target audience and optimise their recruitment potential.
Please contact us for information on how TREAT-NMD can support your organisation through the Registry Enquiry process.
What kind of data is available?
All TGDOC Core Member Registries collect the mandatory data items included in the disease-specific TREAT-NMD Core Datasets, created via consensus-building projects with the registry curators, PIs and academic experts. This ensures confidence that all Core Member Registries are collecting at least all mandatory data items in a disease dataset this allows specific questions to be asked of the registries in the network based on the specific dataset items and collate the results into a report. TREAT-NMD acts as the intermediary between academics, pharma, patient organisations etc. and the network of registries, and only shares anonymised and aggregated data in order to answer a specific research question. Global Registry Enquiries can request data breakdowns on any of the items in these datasets, and can also include additional non-dataset enquiries as many registries collect additional information outside of these Core Datasets.
TREAT-NMD is currently only able to provide aggregate data, however it is anticipated that the Global Registries Platform will be able to support the use of de-identified patient-level data for Post Marketing Surveillance (PMS) projects in the near future
One of the key criteria for joining the TREAT-NMD Global Registry Network as a Core Member Registry is confirming that the registry collects all the mandatory data items included in the most recent TREAT-NMD Core Dataset for each specific disease area. All TGDOC Member Registries also agree to abide by the TGDOC Charter and SOPs in order to guarantee high quality data. Please see the ‘Becoming a TGDOC Member Registry’ page for detailed information on membership requirements.
Process and Timelines
Any third party interested in running a Global Registry Enquiry should contact the TREAT-NMD Global Registry Enquiries Manager, who will work closely with the enquiring organisation directly to develop a Scope of Work (SOW) and a draft data spreadsheet with fields for the individual registries to enter their aggregate data into. Once the SOW and datasheet are developed, they are approved by the TGDOC Chairs, a representative from the TREAT-NMD Ethics Board, and the Subgroup Leads and Patient Representative for the disease area in question.
Once approved internally, there is one final stage – the enquiry vote. As all patient data is owned by the registries themselves, all Core Member Registries for the specified disease area vote on whether they agree the enquiry is a good use of the network, based on the details in the SOW. This is mainly a formality given the preparatory work involved in developing the SOW but is important for the governance of the network. In order for the enquiry to proceed, at least 2/3 of all relevant registries must submit a vote, and at least 2/3 of the received votes must be positive.
Once the enquiry vote has passed, and the contract is executed between TREAT-NMD and the enquiring third party, the enquiry goes live, with the enquiry report usually delivered to the enquiring organisation 6-8 weeks later. TREAT-NMD will always endeavour to meet specific deadlines whenever possible, but it is strongly encouraged to get in touch at the earliest opportunity to allow plenty of time for the planning stages of an enquiry.
TGDOC has developed a costing matrix which calculates a price for each Global Registry Enquiry based on several factors, including but not limited to the number of mandatory and non-mandatory dataset items in the enquiry, the complexity level and the type and size of the enquiring organisation.
A cost-recovery rate is available to academics and patient organisations where necessary and at the discretion of the TGDOC Executive Board, however enquirers are encouraged to apply for funding to support Global Registry Enquiries if possible. TREAT-NMD is a not-for-profit organisation and any surplus funds generated from enquiries is reinvested into the Global Registry Network through funding networking meetings and supporting struggling registries with bursaries, for example.
The basic process for requesting data from the TREAT-NMD Global Registry Network is outlined in the chart below: