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We discuss the pilot siteless models in clinical trial patient centricity with Gabriel Vargas, an executive at Amgen.
Biopharmaceutical enterprises are starting to pilot siteless models in clinical trials by applying digital wearable technologies in hopes to reduce data variability and enhance data quality. Gabriel Vargas, executive medical director, Digital Health & Neuroscience Early Development Therapeutic Area Head at Amgen, presented an mHealth case study at PanAgora’s Clinical Trials Patient Experience Summit about the use of digital wearables in a migraine study. There will be more discussion on the use of wearables in clinical trials at PanAgora’s Clinical Trials Mobile Innovations Summit.
Moe Alsumidaie: Can you describe the design of the mHealth migraine study?
Gabriel Vargas: The mHealth study has three major objectives. One is to run a siteless and virtual study to enable
Amgen’s vision of using digital and to bring the trial to the patient. We still have a lot of work to get there, but
wewanted to test if we can do a non-interventional study where patients sign up online. The second objective was to test the informed consent form process through an app. We developed an internal app that we put on the Apple app store, which had a built-in informed consent form. When people downloaded the app, they could complete the consent and enter into the trial. The last objective was to try to understand and develop more objective measures for migraine. Migraine is a disease in which data capture is only through subjective rating scales, which could result in a lot of data variability. We are very interested in migraine because we are building a migraine franchise. We have a drug that is going to be filed this year and another Phase II drug, so we would like to understand migraine a little bit better. This study aims to look at how activity correlates with migraine; everybody in the study is given an Apple watch. We use this to measure step-count and ask subjects to fill out a diary. At the end of the study we will be looking at how step-count correlates with migraines.
MA: What observations have you made from conducting this study, and what data points is Amgen collecting in this study?
GV: The trial is in progress and hasn't finished yet. We are collecting step count and heart rates from the Apple Watch. The heart rate is mainly to show whether the person is wearing the watch or not. We are asking subjects to keep headache diaries, so when they get a headache they report details such as time, duration, and severity. By the end of the study, we are hoping to be able to look at the times they had or did not have a headache and the step-count related to it in order to make the correlation. As is the case for any study, there were some people who were extremely compliant and were using the watch as directed across all waking hours and then there are some people (about 30%) who are less compliant. We are still in the early days and getting our first look at the data. The description of how we would look at step count and migraine is too simplistic, but that was our initial approach. We have only looked at the first month of data at this point so we will know more once the study is finished at the end of three months. We will have to be a little more granular in understanding people’s activity patterns, such as changes in the weekdays vs. the weekends. We need to take those factors into account before we can get a good estimate of how activity and migraine go hand in hand. This is our second foray into the digital study arena for my group. We did a healthy subject study looking at Garmin and Vivofit to understand more about data flow and the usability of wearable devices.
MA: What forces drove your team to design and execute this mHealth study?
GV: There is a group, created at Amgen about a year and a half ago, called the Strategy Innovations Group which was pushing towards how we can be more innovative. The rationale behind the creation of this group was that
drug development is very costly and the likelihood of success is low; we hope to improve efficiency by innovating. We are reviewing digital health as a means of gathering higher quantity, and hopefully higher quality data with less variability. We want to run large trials at lower costs early on by doing a lot of the measurements at home instead of at the sites. Every time a patient comes to visit a site, it costs us at least $500-$1,000. If you have a 1,000-person trial and you are eliminating ten site visits by enabling data collection directly from patients, that’s a lot of money saved.
MA: What are Amgen’s plans to bring digital into interventional trials?
GV: We have a four-step plan for the movement of digital. We are envisioning a pilot approach where the first part is a technology assessment in healthy subjects to answer basic questions: Is it usable? Is it a hassle to charge? How would we get the data from the device into our database? If that goes well, we do the Phase 0-I study in collaboration with a CRO. Then we would put it in a real Phase II study, where digital data would be an exploratory endpoint. Then the holy grail is to move digital data collection into a primary or secondary endpoint. As far as what has been done so far, we are going to see digital health becoming a primary endpoint in Parkinson's disease. Measurements have already been done by a variety of teams using digital wearable devices that provide much better data than what you would get from a six-minute walk test conducted by a neurologist.
MA: What about using data collected from digital wearable devices in regulatory submissions?
GV: That is an area of concern, but the discussions that we have had with the FDA is that they are very open to digital health. They are aligned to work with us and our plan is to develop the digital strategy to move it towards clinical endpoints as part of the regulatory submission. We would be sure to have early and frequent meetings with the FDA to talk about the digital strategies and include their input in our trials. Amgen is investing in Akili, a neurotech company, which is developing a videogame as a treatment for ADHD. That is a quintessential example of a digital therapeutic. Akili met early and often with the FDA to chart a path towards what I believe may be the first digital therapeutic ever approved. It is currently collecting the data in Phase III, and this path will lead us to digital health regulatory pathways in the future.
Gabriel Vargas executive medical director, Digital Health & Neuroscience Early Development Therapeutic Area Head at Amgen, presented an mHealth case study at PanAgora’s Clinical Trials Patient Experience Summit about the use of digital wearables in a migraine study.
Moe Alsumidaie, MBA, MSF is Chief Data Scientist at Annex Clinical, and Editorial Advisory Board member for and regular contributor to Applied Clinical Trials.