CAPTURING PATIENT EXPERIENCES WITH OFF-LABEL USE OF TANGANIL
TREND Community is launching the Tanganil Health Initiative in collaboration with NTSAD (National Tay-Sachs and Allied Disease) and the Cure Tay-Sachs Foundation (CTSF) to document GM1 & GM2 patient experiences with off label use of Tanganil, available without a prescription from Europe and indicated for the treatment of positional vertigo (dizziness). This observational study will be designed to better understand the benefits and risks experienced by patients taking Tanganil to treat symptoms associated with GM1 & GM2 Disease. TREND Community will compile the data contributed by Health Initiative participants and produce a report of deidentified data that they can share with their medical teams, family members, and others in their community.
GM1 & GM2 patients and caregivers who are using or planning to use Tanganil will be invited to participate in this TREND Health Initiative through the TREND platform. The participants will join a private, online community only for these participants for support and sharing. Participants will be asked to answer regular survey questions and keep an online journal to track their experiences with the drug. TREND Community will host a kick-off webinar to make introductions, explain the process and answer questions. The time investment will be kept minimal, and participants are reminded to consult with their physicians regarding medical questions.
The information shared within the private discussion space, along with the data collected from targeted questionnaires and participant journals, will be analyzed periodically by the TREND Community Data Team and the output will be shared back with the Health Initiative participants, NTSAD/CTSF and other key stakeholders. All such data shall be kept anonymous.
HOW IS THIS HEALTH INITIATIVE LIKE A REGISTRY?
All participants will be required to sign an electronic consent form affirming that they understand the purpose of the Health Initiative and agree to share their data (with TREND Community, NTSAD/CTSF, and back with the particpants’ communities).
Participants Remain Anonymous
All data contributed will be deidentified meaning it cannot be linked back to individual participants.
Data Informs Research
Like data from registries, TREND Health Initiative data can be used by researchers and pharmaceutical companies to understand disease burden, generate hypotheses for future research and design better clinical trials. TREND Community knows the drug development process and is working closely with FDA to establish guidelines for the consideration of anecdotal data to inform the FDA review process.
HOW IS THIS HEALTH INITIATIVE DIFFERENT FROM A REGISTRY?
Whereas registries generally track a broad range of information, during the Tanganil Health Initiative participants will record data specific to their experiences using Tanganil. Unlike a registry, TREND Community offers participants an exclusive and private discussion space where they can communicate with others who are also on this journey. A Community Manager will facilitate these discussions and will make every effort to help participants find answers to specific questions. Participants will be encouraged to share their experiences and provide one another with support. The information shared within this discussion space will also be analyzed. Using natural language processing and machine learning techniques, TREND Community can turn these conversations into data.
Additionally, TREND Community shares data back with Health Initiative participants. This information can be used by participants to have better and more informed conversations with their physicians.
Become a Citizen Scientist by sharing your experiences with Tanganil on TREND Community.
We will help you turn your anecdotes into evidence that can benefit your entire community.
How it works:
- Join the GM1 & GM2 Tanganil Health Initiative on the TREND Community platform (participation is free). Go to trend.community, scroll to the bottom and request an invite.
- After receiving the email invite, follow the link to set up an account.
- Sign an electronic informed consent agreeing to share your data in order to participate.
- Join an exclusive and private discussion group within TREND Community for the GM1 & GM2 Tanganil Health Initiative participants to share and support.
- Answer regular survey questions.
- Keep an online journal. You can review these entries over time to discover your own trends.
- Receive TREND Community updates and reports on the Health Initiative progress and trends.
- Share with family members, your medical team and researchers to help provide both you and your child with a potentially better quality of life.