Global Mitochondrial Disease Patient Registry
A patient-driven independent registry, where data are entered, stored and managed by the patients themselves but are also linked to the clinician-driven global registry.
The Global Mitochondrial Disease Patient Registry is part of the GENOMIT project.
Work Package 2
The Registry is being created through GENOMIT's Work Package 2 group. WP2 is coordinated by IMP in close cooperation with Mitocon in Italy, The Lily Foundation in the UK, AMMi in France and DGM in Germany.
Why do we need a global patient registry?
Even the most accredited research centers often only have a few dozen, sometimes even less, patients suffering from rare or very rare syndromes. These numbers do not allow for reliable data to be produced regarding specific pathology.
A shared database, on the other hand, can collect and concentrate a wealth of information and knowledge and represents an indispensable tool for any development in the field of medical research.
The Patient Registry will collect information on quality of life, as well as having information on the needs and requirements that the daily management of mitochondrial pathologies requires of relatives and caregivers.
This information will help to:
Identify/characterise patients - it will become an invaluable repository of natural history, quality of life and clinical data
Recruit for clinical trials
To gain as much knowledge and understanding of mito patients and their families and become the authority on understanding the burden of these diseases
Engage and inform the community - recruit new patients/patient associations into IMP - particularly in regions where patients don’t have access to patient organisations, such as Russia, China, Africa, South America, etc.
Who is the Registry for?
This patient registry will be an important tools for mito patients, as well as the data being invaluable to stakeholders, such as patient associations, clinicians, researchers, politicians and public health decision-makers and of course the pharmaceutical industry.
The EMA have stated that patient registries are invaluable data sources on diseases and their treatments.
We are aiming to create…
A global platform where patients from around the world can register and insert their own data about their disease, their needs and their quality of life
A transparent and robust set of rules for data use
A connection with the clinical registry – so that data can be shared with doctors, and PROMs can be validated with patients and clinicians
Proposed Features
The Patient Registry will be implemented as a separate, independent sub-study within the global clinical registry platform.
It will be managed and completed by patients. Patients can log in independently from doctors and insert their own data (PROMs and QoL or other surveys).
Multilingual function: the register will be available in different languages and patients will be able to use their own language to enter the requested data.
For defined projects, stored information can be shared between sub-studies (global registry/patient registry) and/or the user-groups (clinician/patient).
We will have a Policy of Conduct for data sharing inside the GENOMIT Consortium and outside - ie with pharma industry and other research groups.
Privacy is critical. Patients will sign consent forms prior to data being entered.
For more information about the Global Mitochondrial Disease Patient Registry, please contact us.