Educating caregivers of children with a rare neuro disease about a natural history study

Educating caregivers of children with a rare neuro disease about a natural history study
Digital Health Skills
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Cognitant has worked with Azafaros to deliver the first clinical study which incorporates a visually engaging, interactive tool to educate participants and their careers about the study. The natural history study, run by AZAFAROS, is with children who have GM1 and GM2 gangliosidoses, a class of rare inherited disorders that include the fatal childhood disease, Tay-Sachs. Cognitant followed a co-creation approach with families, caregivers and the patient advisory groups Acción y Cura para Tay-Sachs (ACTAYS ), Cure & Action for Tay-Sachs (CATS) Foundation and the National Tay-Sachs & Allied Diseases Association to maximise the usefulness and accessibility of the education for the GM1 and GM2 communities.

The background

The PROspective Neurological Disease TrajectOry study (or PRONTO) is a natural history study that will follow children who have, or are at risk of developing, GM1 and GM2 gangliosidoses, a class of rare inherited disorders that include fatal childhood disease, Tay-Sachs.

The study, run by Azafaros, will follow 60 to 200 patients in Brazil, Germany, and Spain for five years, in collaboration with Acción y Cura para Tay-Sachs (ACTAYS), Cure & Action for Tay-Sachs Foundation (CATS) and the National Tay-Sachs and Allied Diseases Association.

It will follow children at risk of infantile Tay-Sachs disease, a fatal neurological disorder caused by the toxic build-up of GM2 ganglioside in neurons in the brain and spinal cord. Usually detected at three to six months of age, children with infantile Tay-Sachs rarely live beyond early childhood.

The challenge:

Recruiting participants to take part in natural history studies can be challenging, which is particularly true in the rare disease community where there are often lower sample sizes to recruit from. Motivation to participate in observational studies is often further reduced by the lack of effective information materials shared with patients and their families about the implications of participation.

 Azafaros engaged with Cognitant to overcome the challenges of recruiting children with this rare neuro disease for their study.

The solution:

Azafaros with Cognitant created a campaign to explain to the caregivers of  children with a rare neuro disease the different elements involved when participating in a natural history study. Through the utilisation of a co-creation process, ensuring a user-centred design, the group developed a patient-led digital education resource about the upcoming PRONTO study.

The resource includes unique videos that detail what a natural history study is, the benefits of participation to GM1 and GM2 patient communities, and what participation will entail – including information about the study assessments.

As the brain processes visual information 60,000 times faster than text, presenting information in video format promotes accessibility.

By hosting this content on a digital hub, clinicians and researchers will be able to send curated content directly to participants and caregivers, meaning that the videos can be viewed, and shared, away from the clinic and in their own time.

Our approach:

Cognitant brought together key stakeholders, including KOLs, clinicians, patients and caregivers from Europe and the US, and advocacy groups (the Cure & Action for Tay-Sachs (CATS Foundation), Accion y cura para tay-sachs (ACTAYS) and the National Tay-sachs and Alied Diseases Association (NTSAD). The group were included in a series of virtual co-creation workshops to design an educational video series.

Cognitant facilitated stakeholders to discuss the unmet needs and what the curriculum should cover, and prepared concepts for discussion, such as animation styles & colour schemes.  Each group was given time to discuss their opinions about the pros and cons of the different approaches. Through productive discussion, an educational curriculum and the materials required for maximised impact were agreed upon.

Measuring the impact

The PRONTO resource has been designed to educate and empower individuals and their families, and reduce attrition throughout the course of the research. The impact of this digital intervention will be assessed through patient feedback and engagement metrics collected through Cognitant’s Healthinote platform.

Discover more about Cognitant Group:

Cognitant Group is an Oxford-based business that uses immersive technology to provide people with accessible, personalised and trustworthy information about health and treatment. The company works with lifescience companies, patient advocacy groups, the NHS, health insurers and care providers to empower people to manage their health more effectively. Cognitant Group’s data-driven information prescription platform, healthinote, saves clinician time, improves patient reported outcomes and reduces unnecessary complications across many care pathways.


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