Contributing Experts: Shaheen Lakhan, M.D., Ph.D., FAAN, Chief Medical Officer, Click Therapeutics; Bob Schafer, Ph.D., CEO, Lumos Labs (Lumosity); John A. Wagner, M.D., Ph.D., Chief Medical Officer, Koneksa Health; and Nicole Latimer, Chief Executive Officer, Medrio
Digital health technologies (DHTs) are gaining traction. As of December 2022, there were approximately 23 digital therapeutics (DTx) products approved or cleared by the FDA for the purpose of preventing, treating, or curing an illness. In addition, there are many other digital health technologies in the market that help patients manage and track their health and wellness-related activities.
Despite innovation and growth in sectors, organizations still face challenges. Recently, DHT experts gathered to discuss the current landscape, strategies for conducting successful clinical trials, and how to generate quality evidence on the journey to approval.
In this article, we share insights from Nicole Latimer’s moderated discussion with Shaheen Lakhan, Bob Schafer and John A. Wagner about:
- Why some companies pursue regulatory approval
- How defining a product’s purpose may determine its regulatory status
- What role digital biomarkers can play in DHT trials
- Why running more trials, but smaller ones, can be a good strategy
- How consulting with regulatory bodies can clear up confusion
You can watch the entire conversation from the original webinar to gain additional insights about how technology affects patients, critical considerations for selecting the right partners, and potential hurdles in DHT trials.
Determine the Need for Regulatory Approval
One of the main concerns amongst DHT companies is whether or not to seek regulatory approval. Regardless of whether you are using digital measures, digital biomarkers, or traditional biomarkers, determining regulatory qualification depends on the use case.
John explains, “For earlier drug development or exploratory objectives, you generally don’t require regulatory attention. If you’re using a primary objective or your goal is general medical consumer use, then you do need qualification and you need that extra evidentiary support. In those cases, qualification may be based on an investigational new drug (IND) pathway, a biomarker qualification pathway, or a clinical outcome assessment (COA) qualification pathway.”
There are several reasons why a DHT company might pursue regulatory approval:
- Proves a product’s value to stakeholders
- Legitimizes health claims
- Acts as a validating data point
- Reinforces credibility in these new forms of therapy
- Helps with reimbursement for physicians
But not all DHT products will automatically meet the criteria requiring regulatory approval. First, it’s important to define a product’s purpose.
Define a Product’s Purpose
A digital health technology doesn’t necessarily need regulatory approval. But a DTx—which, by definition, is used to treat, prevent, or cure medical conditions—will often require regulatory oversight.
In some cases, a product may have been originally designed for a general audience before promising clinical applications became apparent. When this happens, organizations have a decision to make. They must decide if they want to focus their product on a medical condition—which would involve dealing with regulatory bodies for market approval.
“The criteria to think about is ‘Who is this [product] intended for?’”, says Bob. “Is it trying to treat, prevent or cure any medical conditions?” This is an important distinction for organizations to make.
In other cases, a product may be intended for clinical application from the start. The developer may even plan to make a prescription-based product.
Shaheen explains, “When you are designing digital therapeutics for serious illnesses like partially responsive major depressive disorder or schizophrenia, you cannot have unregulated products. These are patients under the management of physicians and it is important for solutions to be prescribed and monitored accordingly.”
Use Endpoints Strategically
Choosing the correct endpoints for clinical trials can help to legitimize a company’s DHT program. According to Shaheen, when choosing endpoints, companies should consider hitting druglike targets. These prototypical scales or inventories:
- Indicate that the therapy is targeting the disease and not just symptoms
- Are essential for clinical adoption
- Harness the power of holistic solutions to measure quality of life
Organizations may need to collect different data points for different audiences. Potential audiences and data types to consider:
- The FDA is looking for an endpoint that they consider critical to granting clearance.
- Clinicians want to see instruments they typically use in their practice
- Patients are most interested in the felt impact of a therapy.
Bob explains, “We evolved into a different way of looking at endpoint selection by understanding who the different audiences are who might be interested in the clinical research.”
Of course, clinical trials are changing and evolving. “There are digital biomarkers being incorporated [intro trials],” says John. Digital biomarkers can accelerate decision-making or a drug development program.
John explains, “Digital biomarkers can speed decision-making by offering access to more precise information because you’re able to do it in a mobile setting and you’re able to do it at home.”
Run More Small Trials
When designing a data collection strategy, consider whether it may make sense to run multiple trials.
For example, a company may choose to simultaneously run:
- A pivotal trial primarily structured around what the FDA needs to see.
- An adjunct study focused on capturing patient benefits, like quality of life and other instruments used in clinical practice.
“Rather than trying to incorporate every single endpoint for all audiences in the same study,” explains Bob, “We chose to split this out.” This approach allows a company to leverage a variety of trial designs, depending on the study’s goal. This approach can also lower the operational costs or effort associated with running a single larger and complex trial.
The world of DHTs is moving fast and offers huge potential to unlock patient benefits. Shaheen noted, “I think that digital technologies are the bridge between what drugs cannot accomplish on their own—functional restoration.” But for developers to convince regulatory bodies, clinicians, and insurers of these products’ value, they’ll need to produce quality evidence.
Consult with Regulatory Bodies
When deciding whether to pursue regulatory approval, a developer should:
- Look at actions taken by any similar DHT products
- Stay updated on evolving regulatory guidance
- Speak directly with the FDA
As Bob pointed out, the FDA knows that this regulatory area is in flux. “Definitions are changing, expectations are changing—but they [the FDA] are open to and want to have conversations to help you understand the clinical path, clinical expectations, and the regulatory path.”
Running Successful Digital Health Technology Clinical Trials
We only covered part of Bob, Shaheen, John, and Nicole’s conversation in this article. Watch the entire conversation and gain additional insights about how technology affects patients and how to select a vendor to fit your needs.