Turning challenges into opportunities for people living with cyclic vomiting syndrome

The following story is part of an ongoing series celebrating Rare Disease Day 2022. For more information, click here.

At the start of the COVID-19 pandemic, many people learned for the first time what it was like to be confined at home, unable to make plans and worried about what tomorrow would bring. For people living with cyclic vomiting syndrome (CVS), this was already part of their daily reality.

CVS is a chronic, disabling disorder that causes recurrent cycles of nausea and vomiting lasting for hours or even days, with symptom-free intervals in between.¹ The epidemiology of CVS is not well established, and the disorder is underrecognized in the medical community given challenges to diagnose and manage it.

There’s never been an approved treatment for CVS, largely because the unpredictable nature of these vomiting episodes challenges traditional practices of clinical trials. But in 2020, we identified an opportunity to bring the clinical trial to the patient and change how we conduct certain studies.

With the help of a data collection platform offered by Cambridge Cognition and wrist-worn electronic devices, we conducted a fully decentralized e-source non-interventional trial designed to identify biomarkers that could reliably predict the onset of nausea and vomiting. The data collected would help researchers better understand the course of the disorder and when to introduce therapeutic interventions.²

The convenience of a decentralized trial allowed participants to stay home and use wearable devices to monitor their symptoms in a fully remote manner.²All data was collected electronically with no paper source data used.

It was a trial of firsts for Takeda²:

First trial to use a single-site and single-principal investigator (the individual responsible for conducting objective research in a clinical trial) to enroll patients in multiple states across the country
First trial to use electronic consent forms (or eConsent, an electronic format to aid the consenting process) for every participant
First trial in research and development to use a combination of smart watch and smart phone to collect daily electronic patient reported outcomes (or ePROs, a health outcome directly based on patient self-reporting and a key aspect of clinical trials and treatments for data collection methods)

Today, the learnings from this trial are helping to drive other remote studies, proving that thinking about rare diseases in a non-traditional manner can impact medicine on a much larger scale. CVS may only occur in a small percentage of the population, but many other patients around the world can benefit from decentralized, technology-based clinical trials and treatments.³

At Takeda, we’re committed to harnessing cutting-edge science and digital health technology to push the boundaries of what is possible and exploring other innovative, out-of-the-box solutions that help meet patients where they are.

References

Cyclic Vomiting Syndrome. National Organization for Rare Disorders. https://rarediseases.org/rare-diseases/cyclic-vomiting-syndrome/ Accessed January 2022.
Data on File. Takeda Pharmaceuticals.
Galvin, Gaby. Clinical Trials Are Slow and Expensive. Industry Leaders Want That to Change in 2022. https://morningconsult.com/2021/12/21/clinical-trials-poll-2022/ Accessed January 2022.