The wide-flung empire of the health care ecosystem encompasses care delivery, biopharma, the public health sector and far more. The Digital Medicine Society (DiMe) is an interdisciplinary professional organization that sits at the intersection of global health care and technology. DiMe aims to elevate the exponentially crowded field of digital health tools by developing shared standards of evidence, frameworks and best practices that can help ensure rigorous vetting and appropriate use of digital medicine products, such as sensors that track falls, interactive software supporting a therapeutic intervention and continuous glucose monitors paired with companion apps designed to feed data to a health care team.
Jennifer Goldsack, MChem, MA, MBA, is the executive director of the Digital Medicine Society and a strong advocate of a unifying foundation for digital clinical measures, set out in the DiMe Playbook. She answers our questions about the current status of digital endpoints in clinical trials and strategies to drive the field forward.
Interview condensed and edited for clarity
How are digital endpoints being used in industry clinical trials, including in pivotal trials? Can you describe a successful example or two?
After receiving this question many, many times from our members and the field, we developed a crowdsourced library of digital endpoints being used in medical product development. Our initial findings, if I remember correctly, were 38 unique digital endpoints being used by 18 different sponsors in industry trials. We thought, oh gosh, this is fabulous. One year on, there are 52 medical product developers using 166 different unique endpoints in industry-sponsored trials: unbelievable growth! They're being used primarily in drug trials, but also in biologic and device trials. Nearly 50% of those measures are being used in phase two and phase three trials. Nearly a third are being used as primary endpoints.
Now, who’s had a drug or medical product approved using a digital endpoint? No one. Yet. I know anecdotally that many sponsors featured in our crowdsourced library are having ongoing discussions with our regulatory colleagues. But we have not yet had a digital endpoint used in an approval.
Nor, in the US, do we have a qualified digital endpoint. This gets down in the weeds, but it’s important. Rather than work within the context of a single trial and a single medical product approval, sponsors seeking a qualified digital endpoint take a different path to establish that a particular digital measurement is fit for purpose. Oftentimes, consortia or nonprofits pursue this path. A qualified endpoint can be used by anyone in a given use context — i.e., a therapeutic area, patient population or trial design. The one digital endpoint qualified in Europe is the 95th centile of stride velocity in the Duchenne muscular dystrophy population.
For patient-facing or consumer-facing digital tools, what is emerging as the gold standard for evidence? Does it differ if the purchaser is the consumer, a payer or a health system?
The evidence needed differs depending on whether you want to sell your product in a big-box store, be recognized as a medical product and be reimbursed or use your digital product in drug development. For example, the quality of information and level of certainty we need is different to support a decision to buy a watch that helps you train for a 5K versus to prescribe a medical product — let’s say, a smartwatch that could diagnose you with AFib based on your data, or treat you through app-based cognitive behavioral therapy. But the questions we ask are the same.
Depending on the use case, we have different levels of expectation around how a product should perform. You can take a commercial product and bring it into the medical sphere. For example, you might choose to repurpose an off-the-shelf watch with a heart rate monitor or a step counter. And as a drug developer, you may choose to evaluate the sensor and develop an algorithm that says, “from this heart rate, calculate heart rate variability.” You may choose to make the case that this is important to your population of interest. You can still buy the watch at Best Buy, but you have given a level of evidence to it yourself because of the way you want to use it. So, we can't just think about what a digital product is sold for. We have to think about the function in a given context.
The phrase "digital health" encompasses such a sprawling variety of tools for consumers, care providers, biotech/pharma and even public health. What’s needed to drive the field forward and help best practices emerge?
First, it's really important that we distinguish between digital health products, digital medicine products and digital therapeutics. That's not because we're the language police; it's because we need to understand what the product is to understand how to evaluate its potential risks and benefits. For us, digital health is big tent: it’s all comers. That includes things like record-keeping technologies like EHR, communications technologies we've been using for telehealth, wellness products you might use to support a healthy lifestyle independent of clinical care, right? Those things don't require the same rigor in terms of evidence for their performance.
When we talk about digital medicine products, we talk about high-quality hardware and software tools that measure and/or intervene in health — say, for example, an algorithm that might support clinical decisions, or a sensor tool that might help you derive an endpoint like glucose monitors and AFib detectors. For digital therapeutics, the software itself is the intervention. For example, there are some cognitive behavioral therapy tools where software running on an iPad or smartphone can be used to reduce anxiety and support patients with PTSD. By contrast, an insulin pump involves software, but in the absence of insulin it has no therapeutic effect.
I can sum up in one word what’s needed to drive the field forward: trust. Trust in quality, trust in fit-for-purpose evidence. Trust that this could work in practice, operationally. Trust in regulatory pathways and that a high-quality product could in the future potentially be reimbursed. Trust by clinicians that it’s not going to add even more administrative burden to their workload, and by patients that their data is not going to be used to harm them. Right now, every single initiative we undertake at DiMe and every limiting factor for the industry comes down to trust.