Digital health technologies could be key to the search for treatments that can slow, stop or reverse Parkinson’s. We find out how the Parkinson’s community is working together to develop digital tools to use in clinical trials.
Parkinson’s is a progressive condition. Brain cells get damaged slowly over time, and this leads to gradually worsening symptoms that get harder to manage with current treatments.
The search is on for therapies that can slow or stop the progression of Parkinson’s rather than just address symptoms.
These kinds of therapies are called ‘disease modifying’ and there are many promising new drugs and therapies currently in development.
Most scientists believe these therapies will be most effective if they are given early, when there are still plenty of working brain cells left to protect. That’s why clinical trials testing these new therapies often focus on recruiting people recently diagnosed with Parkinson’s to give them the best chance of success.
However, a major challenge in these clinical trials is finding the right way to measure success. With a slowly progressing condition like Parkinson’s, which has symptoms that can vary widely from day-to-day and person-to-person, it is very hard to measure whether the condition is being slowed or stopped.
Now the research community is turning to digital tools to help crack this crucial problem. And importantly, companies, charities and academics are working together to do this through an international consortium called the Critical Path for Parkinson’s.
We’re joined on the blog by Josh Cosman, Director of Digital Health Strategy at the pharmaceutical company Abbvie, to tell us more about this vital work.
What’s the problem with how Parkinson’s is currently measured in clinical trials?
Through a number of recent trials we’ve realised current methods we use to measure symptoms just aren’t cut out for measuring severity of Parkinson’s — especially in the early stages.
The main assessment that is used in Parkinson’s studies today is called the MDS-UPDRS (which stands for the Movement Disorders Society — Unified Parkinson’s Disease Rating Scale).
It’s actually a pretty good scale for capturing the motor symptoms. But, it’s not very good at measuring subtle symptoms or picking up small changes which is what really matters in early Parkinson’s and for trials of disease-modifying therapies. It simply wasn’t designed for this purpose.
And while the MDS-UPDRS does provide some measure of the non-motor aspects of Parkinson’s it’s limited compared to the assessment of motor function. Despite the fact that non-motor symptoms often have a high burden to people with Parkinson’s.
Coupled to that, in a trial it’s only possible to perform assessments like the MDS-UPDRS (which need to be done by a trained assessor in a clinic setting) periodically — say every few months. So this means these kinds of assessments only provide a snapshot of how an individual’s Parkinson’s was on a particular day at a particular time. They cannot capture changes that happen from day to day, week to week or month to month.
If we can’t measure severity accurately and reliably, that makes it really difficult for us to tell whether the new treatments we’re testing work or not.
How could digital health technologies solve this problem?
Digital technologies are now incredibly powerful. Smart watches can collect information about everything from walking and balance, to sleep. While simple mobile phone apps can gather patient reported feedback on things like mood and pain.
These capabilities make these new digital tools an exciting prospect for developing much better ways to monitor Parkinson’s — in real time and in real life rather than assessments in the clinic — which could be much more sensitive and meaningful than our current approaches.
However, while digital technologies hold enormous potential they also come with their own challenges. And so far, the impact of digital health tools on therapeutic development in Parkinson’s has been limited. So why is that?
- Development of these tools has been siloed. Different groups — both in academia and industry — have created their own tools, when actually what we need is to work together to develop a unified approach to using digital tools in Parkinson’s trials.
- It sounds simple but it isn’t. It may seem easy to strap a wearable device on someone or ask them to use a mobile app but we need to be sure that the data gathered is consistent, high quality and that we understand what it really means. To date, these kinds of tools have been used in a number of trials but the data and impact has been disappointing.
- Issues with data standards, security and privacy. When you’re gathering this quantity of data about individuals it is crucial to have robust procedures to ensure that this data is safe and used in the right way.
For the reasons above, regulatory authorities — the national and international agencies that approve new medicines for use — will not currently accept evidence gathered using digital tools as sufficient evidence to approve new Parkinson’s treatments. If we want to use digital tools as the main outcome measures in clinical trials, we need to work together and with these agencies to develop the right approach.
How is the research community working together to drive forward digital tools for trials?
We launched a project called 3DT in 2018 to bring together industry, academia and patient organisations to work openly and collaboratively to advance digital health tools for Parkinson’s clinical trials. A crucial aspect of this project is working with regulatory agencies to make sure the tools we generate meet their requirements and standards, and can therefore be adopted as quickly as possible.
This international initiative is part of the Critical Path for Parkinson’s — a global consortium founded and led by Parkinson’s UK and the Critical Path Institute — to develop better tools for conducting clinical trials in Parkinson’s through collaboration.
The Critical Path for Parkinson’s launched in 2015 and now brings together 11 pharmaceutical companies, 5 leading universities and 5 patient organisations to work with regulatory agencies.
In this digital workstream, we’re working together to focus on getting full utility out of digital tools. We all see the promise but we also see the gaps. So we’re coming together as a larger group to share the risks, to share learnings and share costs to really move this field forward in a way that wouldn’t be possible if we worked alone.
We are working with the Watch-PD study — which is funded by two companies Biogen and Takeda — to develop and understand digital tools in early Parkinson’s.
This US study, led by investigators at the University of Rochester, aims to involve 100 people with early-stage Parkinson’s.
Over the course of 12 months, participants will have 6 assessments in the clinic using special sensors that are placed on the body. This will help us see whether the digital sensors can provide a better picture of the motor symptoms than is captured by the traditional MDS-UPDRS assessment.
Participants will also wear a smartwatch for 7 days after each clinic visit to gather movement data, and use a mobile application to capture information about motor and non-motor symptoms twice a month. Gathering information while participants are at home will help us understand how data captured in this way could supplement and strengthen in clinic assessments.
All the data collected in the study will then be compared back to patient perceptions of their symptoms, ability to carry out daily activities and quality of life — this is crucial to ensuring that the digital tools and measurements being developed capture what is truly meaningful to people with Parkinson’s.
How are you working with regulatory agencies?
Before the Watch-PD study started we approached regulatory agencies to help us understand what evidence they need to approve these new digital tools to measure Parkinson’s in clinical trials.
The regulatory agencies really welcomed us coming to them for input early as a team and provided detailed and constructive feedback which we have built into the study design for Watch-PD to ensure it will have maximum impact.
One especially important example was that both the US and the European agencies — the Food and Drugs Administration (FDA) in the US and European Medicines Agency (EMA) — highlighted the importance of collecting data from a comparison group of healthy, age-matched participants.
As a result of this clear feedback we changed the study to add 50 non-Parkinson’s participants and divided the extra costs equally among the 3DT project members.
This really demonstrates that by working together we can really speed things up. This collaborative approach is the only way we’re going to move these technologies and better clinical trials forward.
How are you working with people with Parkinson’s?
Engaging with the Parkinson’s community is crucial to develop digital tools that capture the right information in the right way.
People with Parkinson’s played a vital part in designing the Watch-PD study and the technologies we’re testing in it are as user-friendly as possible.
We will also be asking the people who participate in the study to give us feedback on the digital tools we’re using in this project. We want to know whether they feel the tools are easy to use, capture what’s meaningful to them and are not too burdensome to use regularly in everyday life. This feedback is so important because ultimately, if digital tools are going to work in clinical trials, they need to work for people with the condition.
I want to thank people affected by Parkinson’s for participating in every step of the process to make this work as impactful as it can be.
What are the next steps?
We’ve already made really unprecedented progress in this project and that’s really testament to the approach and the leadership of the Critical Path Institute. I’ve been involved in many of these kinds of consortia in the past and I’ve never seen this level of progress so quickly.
We’re continuing to engage with both the FDA and EMA to move things forwards. This is the beginning of the journey to regulatory endorsement of such digital tools and we know that collaboration and data sharing will help us get there faster. We’re under no illusions that this is going to be quick or easy. The Watch-PD data alone will likely not be enough.
All the data from the Watch-PD study will be shared with the 3DT team for analysis. We’re also looking to use data from 3 other studies in early Parkinson’s to supplement the data from Watch-PD.
Our ultimate goal is to develop digital technologies as true quantitative tools that can really help in therapeutic development in Parkinson’s — not just in the early stages of the condition but across the spectrum.
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