Landmark Digital Medicine Trial: Patient-Generated Data Improve Cancer Survival

Over the past several years, there has been speculation as to whether digital tracking by patients via their mobile devices could change clinical outcomes. In fact, when I read a paper presented at the American Society of Clinical Oncology (ASCO) annual meeting in June 2016, I thought it might be too good to be true.[1]

In that study, also covered in the Wall Street Journal,[2] Fabrice Denis, MD, PhD, and colleagues at the Institut Inter-régional de Cancérologie Jean Bernard in Le Mans, France, randomly assigned 133 patients with advanced lung cancer to either receive standard care or use a smartphone app (MoovCare™; Sivan Innovation) tracking 12 symptoms. For the patient self-tracking strategy, an algorithm alerted the doctor.

In this small trial, there was a significant improvement in survival of a median of 7 months for the intervention. The 1-year survival increased from 49% in the standard-care group to 75% in the self-tracking arm. Furthermore, 50% fewer scans were performed, and it took only 15 minutes per week for oncologists to follow the 60 patients in the latter group. The trial was stopped prematurely because of these exceptional results.

I never expected that the validation for patient-generated data in digital medicine would be manifest in oncology with survival as the primary outcome.

My interpretation was that this was very interesting, but the glowing outcomes were unlikely to be replicated in a larger trial. And I never expected that the validation for patient-generated data in digital medicine would be manifest in oncology with survival as the primary outcome. But I was wrong on all counts.

In a large trial from Memorial Sloan Kettering that was presented at the recent ASCO 2017 meeting and simultaneously published in JAMA,[3] 766 patients with diverse metastatic cancers underwent a 2:1 randomization of patient-tracking of 12 symptoms or usual care. As with the French trial, an email alert was triggered by significant worsening of symptoms, but it initially went to a nurse. The patient-reported outcomes group had a 5.2-month median survival improvement (31.2 vs 26.0 months). There were also improvements in the secondary outcomes of 1-year quality-adjusted survival and tolerance of a longer duration of chemotherapy.

Why is this trial so important? First, it is not a stand-alone result but the replication of one with concordant results, now with a fivefold increase in sample size and long-term follow-up (instead of premature discontinuation).

Second, the improvement in survival of > 5 months compares quite favorably with the median survival improvement in major oncology drug trials, such as those seen with the exciting results of immunotherapy. Many of these trials have shown a 3-month median survival improvement for new drugs, leading to commercial approval and costing at least $100,000 per treatment.

But unlike the drug trials, there was little promotion or coverage. The only major newspaper that reported on the trial was the Washington Post.[4] This might have been anticipated; there is no sponsor. The ASCO meeting is characterized by the reporting of hundreds of drug trials, but it turned out that a digital medicine trial had striking results that largely outperformed most of the expensive and heavily promoted biopharma interventions. The digital tracking strategy costs very little: an app, an algorithm, and a strategy for clinician response. But that represents a de minimis amount considering the outcome of improved survival—or the new drug cost of $100,000 per patient.

Most important, this trial emphasizes the role of engaging patients in tracking their symptoms and of generating their own data. Patients have long been neglected for having a more active role in their care, when in fact, given the appropriate tools, they represent true "blockbuster" potential for improving their outcomes.[5]

Eric Topol, MD
Editor-in-Chief, Medscape


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