Applied Clinical Trials
Jeff Lee had the opportunity to be a moderator at the Mobile in Clinical Trials event where he sat down with mobile experts within the pharmaceutical industry. The session allowed for new observations in regards to the value that these mobile technologies have within clinical trials.
As a moderator at the Mobile in Clinical Trials event at DPharm in Boston in early September, I had the opportunity to sit down with some of the pharmaceutical industry’s top mobile experts: Munther Baara, Senior Director, Clinical Paradigm, Pfizer; Jacob LaPorte, Head of Digital Development, Novartis; Jane Myles, Head, Operational Intelligence and Innovation, Genentech/Roche; and Alex Simmonds, Business Partner, Digital Clinical, R&D IT, Bristol-Myers Squibb, to get their perspectives on quantifying and deriving value from mobile technologies, as well as how they get buy-in from study teams and executives to scale the use of these tools.
I started the session by asking the audience, via a text-message polling system, how many of the following mobile technologies they have used in clinical trials: mobile engagement apps (i.e. reminders, education, etc.); BYOD ePRO; eConsent; wearables/sensors; ResearchKit; digital biomarkers; or virtual study solutions. Here’s what they said. 23% had used four of the above, and that was the most popular response. 13% had used none, while the rest of respondents were fairly evenly divided among the remaining choices.
Once we established the mobile technologies up for discussion, we got the session started. Here are some excerpts I want to share:
We started by talking about the value achieved by using mobile solutions.
The panel quickly came to a consensus that many of the well-adopted mobile technologies provide significant value. One panelist described a study that used a mobile app for patient engagement, which showed an 83% decrease in dropout rates among users of the app versus non-users. While that level of retention may not be reproducible in every trial, it was clear that “it makes a difference.”
The conversation shifted to talk about using the right metrics to measure value and the misconception about the value of eConsent. One panelist said: the value is not faster recruitment, it’s reduced protocol deviations. With eConsent you can reduce the number of missing signatures, missing dates, and missing eConsent documents, among others.
I challenged the panelists to take it a step further and discuss how they might alter study design to capitalize on the value of mobile and achieve cost savings.
How can we ensure these mobile technologies are providing a tangible ROI? For example, is it conceivable to lower the enrollment targets on future studies when you use a technology that has been proven to improve retention rates? If we don’t alter study design in a way that reduces costs, then is the solution adding a line-item to the budget, without providing real savings?
One panelist described a clear example of achieving the value of mobile solutions. On a Phase IV heart failure study, one of the measurements was to assess the effect of the intervention on quality of life (QOL). For this study, they chose not to use a paper-based approach for patient-reported outcomes (PRO), but instead chose to use a combination of ePRO, an actigraphy watch (to measure activity patterns as a surrogate for QOL), and an at-home sleep test. Biostatisticians assessed that using a paper-based survey would have required enrolling over 5,000 patients to power the study, but with the mobile solutions and digital tools they were able to lower recruitment targets to under 200 patients. In the words of this panelist: “there is nothing more patient-centric than not requiring the patient to join the trial in the first place.”
While not all the panelists were ready to reduce sample size targets, (even with solutions proven to reduce dropout rates), the sentiment was that keeping patients in the study is valuable enough in itself, even if doing so doesn’t reduce a line item on the budget.
I wanted to hear how the panelists support their study teams in making decisions about which mobile solution to select for a trial.
One of the problems for study teams is that the question about whether to add a mobile technology to a trial often gets asked after the study is designed. It’s critical to think about incorporating mobile innovations from inception and analyzing how it fits within the protocol.
One panelist described building a simple, matrix-like visual tool that their study teams could use in a consultative way to understand which technology is right for the problem they want to solve. Another panelist described providing a white-glove service to study teams so they don’t feel left alone to implement the solution with the vendor. For example, the innovation group implemented a recommendation tool for bring-your-own-device (BYOD) ePRO that collects several study parameters from the study team, assesses complexity and other factors, and generates a recommendation on whether it’s appropriate for the study.
Lastly, we talked about getting internal buy-in to use mobile solutions.
One panelist cautioned that innovation groups need to be careful not to boil the ocean. Don’t ask for millions of dollars for an innovation that might fail. Creativity often starts when you have less budget. Go find the most innovative clinicians and vendors who want to think out of the box and collaborate with them.
On a personal note, I’m inspired by the advances that have been made by these industry leaders and their organizations since I participated in this event last year. The panelists’ remarks clearly indicate mobile technologies are not only on the roadmap at major pharmaceutical companies, but many of these solutions are deemed valuable and well on their way to becoming mainstream. Innovation groups are supporting study teams to design trials with patient-centric mobile solutions in mind. That said, to avoid these technologies from simply being a ‘window dressing’ (representing additional cost without savings), we need to challenge ourselves to rethink study design in a way that achieves tangible value.
Jeff Lee, CEO, mProve Health