Through the presentations, we invited our speakers to illustrate how we could capture real word data in different formats such as video, text, photos, and audio and develop novel digital biomarkers.
Our first speaker, Tanya Collin-Histed, CEO of the International Gaucher Alliance, opened the event with the perspective of a patient advocate and parent, reminding the audience of ‘Aunty Elin’ at Great Ormond Street where the inspiration for Aparito originated. She recalled a difficult time historically when a clinical trial for Gaucher was halted with inconclusive results, reinforcing the toll it takes on families and the opportunity cost in terms of time and the appetite for future participation.
Fast forward to the present day and an IGA powered solution that has been worked on in collaboration with Aparito which advocates for global, federated, patient-led disease registries that support a better understanding of the disease. The aim of combining a global registry with technology is to be able to empower patients and support recruitment for studies, validate new endpoints – clinical and PROs, but also encourage and support the education of health professionals. The model is made sustainable via pharma investment.
Dr. Aimee Donald from the Royal Manchester University Children’s Hospital spoke next and took up the topic of generating meaningful data in her research presentation, titled ‘Measuring disease activity and patient outcomes in Gaucher disease with wearable tech and mobile phone apps.’She noted three major challenges in the research: The scientific challenges of the disease itself; the fact that it is a rare disease and what that means for patient recruitment and the implications for clinical trial design. She spoke about the practical considerations of hospital-based trials for families, often having to undertake extensive travel and the impact on children’s motivation in particular, of multiple tests, in a short time period and their resulting fatigue.
She contrasted that with the results of an Aparito assisted app-based trial using wearables which yielded a rich and meaningful data set. Her conclusions pointed to the importance of patient engagement and motivation and the support she observed for the technology. With these two factors in place, it is possible to both expand data collection and focus on specific areas with enhanced patient feedback.
We next had the privilege to hear from Maddie Collin, a Gaucher Patient and Patient advocate about the experience of participating in clinical trials as a child in conversation with Dr. Aimee Donald. Now in her twenties, Maddie and her mother Tanya have a long experience to draw on which can be illuminating for companies designing trials. Listening to Maddie explaining her burden and attitude to different invasive procedures necessary for clinical practice and clinical trials it was clear that a patient-centric approach is important for success in patient recruitment and engagement.
It is key for Aparito to support access to healthcare in every region of the world, therefore Dr. Karen Fieggen, Head of Medical Genetics at Groote Schuur and Red Cross Children’s War Memorial Hospitals in Cape Town, SA spoke about our collaboration and research into ‘Precision medicine in South African children with refractory epilepsy’, based on the hypothesis that home technology-based monitoring has the potential to improve the management of children with epilepsy in South Africa.Working with the Aparito Atom5 platform she summarised the inherent challenges of working with trial participants living in the townships and rural areas that they needed to overcome, such as the high cost of cellular data and limited cellular access in rural areas. Despite the limitations, she concluded that the technology opens up areas that were previously not accessible.
Taking into consideration the necessary valid tools to develop regulatory and clinical purposes, Dr. Marcus Grobe-Einsler from the German Centre for Neurodegenerative Diseases (DZNE) at University Hospital in Bonn spoke about SARAhome a new patient related outcome measure for ataxia. Using the Aparito platform patients can video themselves performing a series of tests that would normally take place in clinic. The images are downloaded and assessed by experts remotely. In the future, AI based assessment could be used to automate this process further. The use of technology will reduce patient hospital visits and with the help of AI in interpreting the data, medical doctors will have more time for patient evaluation.
Also looking to the future, Dr. Alberto E. Tozzi, Chief Innovation Officer at Bambino Gesu hospital in Rome spoke about the eHealth Hub initiative to identify new technololgy based innovations. From forty innovations, Aparito was the idea that was selected to support a project in children with shunts in cases of hydrocephalus. Dr. Andrea Carai from the Oncological Neurosurgery Unit presented the issues of monitoring these children which he characterized as:
- Inappropriate ER referrals; Avoidable diagnostic tests;
- Delayed referrals; Clinical risk
- Costs: Financial, Biological and Human
Aparito is joining forces with Bambino Gesu hospital to develop a platform for better monitoring and quicker interventions addressing the unmet needs of patients and health care professionals in cases of hydrocephalus.
Aparito also invests in research: Grace Bailey from Cardiff University presented her PhD research on Use of technological platforms to enhance phenotypic understanding in dystonia. Working with Aparito as a research partner, Grace is currently recruiting patients nationwide for her study. She is focusing on sleep as one of the areas where remote wearables can help remove the difficulties of subjective patient description. She pointed out that sleep is not normally included in symptomatic clinical review and there is now the opportunity to objectively assess sleep quality in “real-life” environments.
The last word was given over to finance, specifically Aparito’s first investor, the impact investment firm Bethnal Green Ventures. Managing Partner, Melanie Hayes spoke about scale, and their mission to back ambitious entrepreneurs using technology to tackle big issues with the potential of radically improving millions of lives. Melanie pointed out that impact investors proactively seek out diverse management teams (which includes female founders) because they have been proven to lead to better impact and superior returns.
That’s one thesis we’re taking at face value.