March 21, 2017 -The first Neurosciences Medicine Digital Health Clinical Trials Symposium was held last month in San Francisco.
The DCRI hosted the inaugural Neurosciences Medicine Digital Health Clinical Trials Symposium in San Francisco this February. The agenda included a wide-ranging discussion of the current state of clinical trials for games, apps, and electronic medical devices to diagnose or alleviate symptoms of psychiatric and neurological disorders.
“There are unique challenges in digital health applications for neurosciences medicine, and the genesis of this meeting was the concept of bringing together experts to identify and develop solutions to move the field forward,” said Andrew Krystal, MD, MS, (right) in opening remarks. Krystal is co-director of Neurosciences Medicine at the DCRI; adjunct professor of Psychiatry and Behavioral Sciences at Duke University School of Medicine; and Ray and Dagmar Dolby distinguished professor of psychiatry and vice chair for research in the Departments of Psychiatry and Neurology, University of California San Francisco.
“The DCRI is active in developing diagnostic and treatment interventions for neuropsychiatric conditions, including studies of digital health applications.” Krystal also gave a talk on designing appropriate control devices for trials.
The keynote presentation, “Neuroscience Meets Technology: A vision of the future of brain health,” was given by Adam Gazzaley, MD, PhD, (left) who is a professor in Neurology, Physiology and Psychiatry at UC San Francisco; founder and executive director of Neuroscape; co-founder and chief science advisor of Akili Interactive Labs; and co-founder and chief scientist of JAZZ Venture Partners. He noted that challenges include the need to better assess and enhance cognition – including elements such as attention, memory, mood, compassion, and empathy – in a meaningful and lasting way.
Other key takeaways from DCRI presentations at the meeting included:
- There are currently no standard endpoints for psychiatry trials in digital health, and the endpoints used for drug trials may not be appropriate; academia, industry, the Food and Drug Administration, and the National Institute of Mental Health should all work together to address this.
- There are unique challenges in designing control interventions for digital health apps such as games, since none have been definitively proven to be effective or ineffective. As a result, careful control design is essential to elucidate effects.
- Many statistical challenges are unique to digital health, and these must be taken into account in innovative trial designs, since “there are no right answers to wrong questions.”
- Computerized testing has potential in standardizing measurements of cognition. Today, these measurements are based on tests developed in the 1980s, often administered by testers with no psychometric experience, which poses a risk of inaccuracy and inconsistency. This can potentially be addressed via computerization.
- Technology is moving forward rapidly, but medicine and research are still catching up, mainly due to regulatory issues and trial design limitations. Enhanced recruitment using the web will help reduce the effort required to collect data and the cost of trials, by allowing brick and mortar sites to be added where the patients are, instead of maintaining non-enrolling sites. The electronic medical record has potential as a viable tool for large-scale clinical research, though integration into clinical trial designs and validation of data flow remain issues. There are opportunities to provide standardization of protocol, decision support and automated data capture within the workflows of ongoing clinical care.
Other DCRI speakers on the agenda were: Scott H. Kollins, PhD, professor and vice chair for research strategy and development, and director of the Duke ADHD Program, Department of Psychiatry & Behavioral Sciences, Duke University School of Medicine, and global lead for ADHD and SUD, Neurosciences Medicine, DCRI, who examined standard and exploratory endpoints for psychiatry trials in digital health; Roseann White, MA, director, Pragmatic Clinical Trial Statistics, who described biostatistical trial design and the need to power studies properly; Richard Keefe, PhD, professor in Psychiatry and Behavioral Sciences, Duke Institute for Brain Sciences, and Global Cognition Sciences Lead, DCRI, who focused on where cognition fits into trial design; and Brad J. Kolls, MD, PhD, of the Department of Neurology, Duke University School of Medicine, and Clinical Informatics Global Trials Lead, DCRI Neurosciences Medicine, whose presentation focused on capturing data from systems and digital interfaces in clinical trials.
The business side of digital health was discussed by a panel including: Eddie Martucci, CEO, Akili Interactive Labs; Henry Mahnke, CEO, Posit Science; Zack Lynch, general partner, JAZZ Venture Partners; Craig Fischer, vice president of Medical Professional Markets, Pearson; Anand Iyer, chief strategy officer, Welldoc; and Carolyn Jasik, medical director, Omada Health.
A panel discussion addressed the question of “What do emerging digital health companies need to know about business decisions regarding clinical trials?” Key takeaways included:
- There is no agreed blueprint for digital health clinical trials. A company must identify the most important question, then think two questions ahead and try to get preliminary answers to those, too.
- Access to patients is a major issue. Many psychiatrists and psychologists are outside of the mainstream health care system.
- Clinical outcomes open the door in discussions with payers, but the challenge is demonstrating what financial impact these outcomes have and what the intervention does from an efficacy standpoint.
- The company must understand whom they are delivering the study results to: the needs may be different for the medical community vs. the psychology community. Potential customers should be asked what they need. It is vital to make sure that the study output includes tools and resources.
- It is essential to understand the value of the product to your customer’s customer. The customer will need to “sell” the product to someone else – such as the government, employers, health care organizations, medical groups, health plans, or patients
- From venture capital viewpoint, clinical trial results are table stakes. Investors are most concerned about reimbursement on a broad scale and how that will happen.
Participants also had a choice of two breakout sessions, on FDA Guidance on Digital Health, and Business Models 101: Direct-to-consumer, workplace wellness, and the payer model.