Which Digital Health Clinical Trial Endpoint?
Increasingly, drug and device companies are incorporating digital health products into clinical trials. In addition, digital health companies are more and more challenged to demonstrate not just technical validation and verification, but clinical safety and effectiveness, if not cost-effectiveness, before commercial launch.
What it is you are trying to demonstrate, your end point, is key to the success or failure of a clinical trial and should be specifically stated and will depend on regulatory and marketing requirements.
The medical device sector, and by extension, the digital health market, is governed by regulations that take a risk-based approach to dictating the level of scrutiny each particular product faces. Here, are defined common terms sponsors may encounter when assessing digital health devices. Most of these apply to clinical endpoints.
Consumer-grade: Devices in this category are generally considered Class I devices and are not FDA-cleared devices. Consumer grade devices tend to be appealing in style, functionality and price point to consumers and many have a consumer-facing app paired with the device. Data collected from these devices can typically be used as exploratory endpoints in clinical trials.
Class I: These devices are generally defined as “low-risk” as they are not designed to treat potentially fatal conditions and likely will not cause life-threatening harm if misused. Devices in this class do not have to be approved by the FDA and are generally available over the counter.
Clinical-grade: Clinical-grade devices deliver data that are validated and can be used as a primary or secondary endpoint in regulatory filings. While some clinical-grade devices offer consumer-friendly designs and paired app, not all clinical-grade devices provide these benefits, and they can be more costly.
Class II: Most clinical-grade devices are considered Class II devices. The exceptions are those that sustain or support life, are implanted or present potential unreasonable risk of illness or injury (Class III devices), and those that present minimal potential for harm to the user (Class I devices).
510(k): A premarket submission made to FDA to demonstrate that the device to be marketed is at least as safe and effective or substantially equivalent, that is, to a legally marketed device that is not subject to premarket approval. FDA-cleared: Medical devices in this category have been determined by the FDA to be substantially equivalent to another legally marketed device. A premarket notification, referred to as a 510(k), must be submitted to FDA and approved for clearance.
Class III: Class III devices typically pose the highest risk and are therefore subject to the highest level of regulatory control. The FDA must approve these devices before they are marketed.
Premarket approval: This approval process is generally reserved for high-risk Class III medical devices and involves a more rigorous premarket scientific and regulatory review to evaluate the safety and effectiveness than the 510(k) pathway.
FDA-approved: The FDA has approved a premarket approval (PMA) application prior to marketing the device. This approval process is more intense than the 510(k) pathway and is generally reserved for high-risk medical devices.
Clinical endpoints, however, are not the only part of a digital health value proposition: Others are:
Financial endpoints: What business problem is the digital health product designed to solve? Raise revenue? Cut costs? Increase market share?
UX/UI endpoints: How does the product improve the doctor or patient care experience?
Workflow endpoints: How does the product save time or increase efficiency or productivity or minimize non-reimbursed activity? Does it reduce telephone traffic to the doctor office? Reduce the need for follow-up appointments? Reduce unnecessary prior authorizations? Reduce the need to order prescriptions like pain medicines?
If you are developing a digital health product, be sure to design your trial so that, with statistical significance, you can use the trial results to justify the conclusion that your product achieved the desired clinical, financial, experience or workflow endpoints.
Arlen Meyers, MD, MBA is the President and CEO of the Society of Physician Entrepreneurs.