As if the small number of patients is not enough of a hurdle, snapshot and episodic collection methods can make it impossible to amass the data needed to convince regulators and reimbursement agencies that therapies for rare diseases are effective. Wearables can help, says Elin Haf Davies, founder and CEO of aparito.
Long experience as a paediatric nurse specialising in rare disease drug development made Elin Haf Davies only too aware of the shortcomings of traditional hospital-based data collection methods, to say nothing of the impact of often-invasive testing on young patients.
Current techniques are not only unsatisfactory from a regulatory perspective and burdensome for patients, they also fail to reflect outcomes that are important to patients and their carers.
“I thought there must be some technology-enabled solution that would allow it to be done better”, Davies said.
The frustrations and challenges spurred Davies to found her company aparito to develop wearable monitoring devices and the means to analyse their outputs in a way that makes the data digestible and meaningful to clinicians.
“It is hard enough to take the measurements needed for regulators in the clinic, let alone to reflect what matters to patients, and wearables certainly help”, said Davies.
But more than that, the technology allows the 24-hour collection of objective data on fundamental concerns in paediatric drug development, such as walking and sleeping.
The watch-like wearable, which is soon to be registered as a medical device class I(m), records data in real time and uploads it by Bluetooth, while a smartphone app collects information from the patient perspective, allowing them to give direct feedback and report adverse events. All the information is analysed and displayed on a dashboard, enabling authorised clinical staff and researchers to review individual or cohort data in real time.
While some factors are constant, others vary by disease and, in each case, the wearable app is made disease-specific.
aparito’s technology complies with Computer System Integration standards, meaning it is very easily integrated into existing systems at clinical trial sites.
From its foundation at the start of 2015, aparito has gone on to attract funding from patients’ groups and companies and to deploy its technology in five studies in three countries.
While none of the studies have reported full results as yet, Davies said the value of real-time monitoring is already evident.
For example, the system supports medication adherence, giving prompts if drugs are not taken, providing patients with evidence that a drug is working if they stop taking it because they perceive no effect, and alerting clinicians if patients stop because they find the side-effects are too bad.
“This helps improve adherence, and if there is low adherence you get a better view of the reasons why”, said Davies.
At the same time, having standardised measuring and constant monitoring overcomes the problem of site variation in global clinical studies, Davies noted. “Truly universal monitoring smooths it out”, she said.
The five aparito-monitored studies underway include paediatric and adult patients, and in each case there is great data capture and good patient adherence.
That makes it possible to build a patient profile based on baseline data, which can then be used to alert in case of disease progression, and eventually as a predictive of future course of disease.
This type of insight is very important in rare diseases, where in many cases the natural history is not understood, and great heterogeneity is seen across the disease spectrum.
Davies also believes wearables will make it possible to provide objective measurements of improvements that matter to patients. “There is a huge difference between endpoints selected to define efficacy in clinical trials and outcomes that relate to effectiveness in the real world; we can show what matters to patients and their carers”, she said.
As an example of how this may translate through to improve the development of orphan drugs, aparito technology is being used in a US National Institutes of Health-sponsored study that aims to fill gaps in understanding of the natural history of Tay-Sachs disease, a fatal inherited lipid deficiency.
“We want to identify what matters to patients and their parents and carers, and will feed this into the design of an imminent pivotal drug study, and included in an IND application to the FDA to ensure regulatory buy-in for its use in a clinical trial of a potential new treatment”, Davies said.