Date: August, 2017
The NBIA Alliance, an umbrella organization for NBIA patient advocacy groups around the world, has received a $122,000 donation to sustain a critical research tool: the International NBIA Patient Registry and Biobank from July 2017 to December 2018.
The gift is from Retrophin Inc., a San Diego-based company that has begun testing a drug for PKAN patients. The registry and biobank will be used to advance NBIA research, enable faster access to worldwide clinical trials and draw greater interest from potential funding sources to bring a drug to trial. It will assist researchers working on these diseases with the natural history studies data, which give insights into the course of a disease.
The data will be made available to other parties, including pharmaceutical companies, once a steering committee made up of researchers, biotech, pharma and patient advocacy representatives that oversees the registry approves.
All information in the registry is entered without any identifying information to protect the privacy of individuals. Only the clinician entering the data has access to the name of the patient. NBIA families participating in natural history studies with clinicians should inquire if they are also participating in the International NBIA Patient Registry to ensure their anonymized data is part of this important resource.
It became possible in March for the Alliance to accept donations after the NBIA Disorders Association agreed to act as the Alliance’s “fiscal sponsor.” This is necessary because the Alliance is not a nonprofit. Gaining nonprofit status is complicated with the multitude of countries involved and more joining in the future, so the fiscal sponsorship is a good solution at this time. We had the assistance of a nonprofit lawyer to prepare the fiscal sponsorship agreement between NBIA Disorders Association and the Alliance, and to establish rules and a charter for the Alliance that was agreed to by all member organizations.
Alliance donations will be put into a restricted fund, with each member country getting one vote to decide which projects to fund and also one vote for all other Alliance matters.
As the fiscal sponsor, NBIA Disorders Association has the responsibility to make sure all Alliance work is in alignment with their mission. Donations will appear on the NBIA Disorders Association’s income tax return with notations of their fiscal sponsorship.
The Alliance accepted its first donation in April from Retrophin for the registry and biobank. These resources were created under the European Union grant TIRCON, which also spearheaded the first international NBIA clinical trial to test the drug deferiprone. TIRCON provided the funding for the registry and the biobank until the grant ended on Oct. 31, 2015.
In January, 2016, eight Alliance member organizations pledged a combined 84,400 euros for 18 months of funding for the registry and biobank. This covered costs through this past June, and now Retrophin’s donation will carry the work through next year, giving Alliance members and the NBIA community time to secure additional funding to sustain these valuable resources.