The Value of Data Beyond the Trial

By Brian Nye

Clinical researchers strive to hit trial endpoints as quickly and cost-effectively as possible. Efficacy and safety are, of course, critical to the approval and commercialization of the molecule. The strength of those data points is obviously dependent on a number of factors, including patient adherence throughout the duration of the trial.  But there can be far more to the story that can optimize the commercial potential of the molecule once approval is granted. Once commercialized, clinicians want to know not only that the medication works on the disease and is safe to use. They also want to know if it will improve the patient’s signs and symptoms that are impacting their health, and quality of life, which drove them to seek medical help in the first place. This makes the quality of the data in the trial phase, and the insights that data can provide, critical to the commercial success of the molecule.

This may be especially true for drugs that don’t produce particularly strong endpoint results. Endpoints may be met with statistical significance, but if strong deltas are not demonstrated it may leave the provider questioning the true value of the product to their patients. Even with strong deltas, there are opportunities to demonstrate ways that it improves the patient’s health and quality of life beyond just the primary/secondary endpoints. For example, while a study medication may not demonstrate a full curative effect, or the endpoint delta was numerically small, a high percentage of patients may still report a reduction in specific symptoms, fewer days of missed work, an improved sense of well-being, or improved overall functioning. In other words, any data that provides insights into how a therapy improves patient quality of life will resonate with clinicians.

In this way, research and development impact the commercial success of a new therapy beyond the endpoint data. Information specific to adherence in specific study populations, information gleaned from patient-reported outcomes (e.g. diaries), and digital biomarkers are all examples of the insights that can add to the quality of the data and, therefore, the commercial value of the medication.

Adherence Data – Importance Beyond Efficacy and Safety

Patient medication adherence is extremely important for ensuring the statistical significance of data throughout the course of a clinical trial. AiCure’s customers rely on our adherence solutions through the Patient Connect app to collect dosing data, but the value doesn’t end there. Not only does the app, via it’s reminders and easy to follow dosing guidance, help to increase patient adherence, it also provides 24/7 visibility for the study team, giving them clear insights into what’s happening with their patients in between clinic visits.

This round-the-clock data collection produces other benefits. Through our proprietary AI, we offer sponsors predictive analytics that can be shared with potential customers following the study. These analytics can show a range of potential patient benefits, such as:

  • Performance of the therapy over time
  • Likelihood of side-effects

Digital Biomarkers – Real Insights from Real Patients

Our Digital Biomarkers use facial and voice recognition, collected with a smartphone or similar device, to help flag symptom changes – many of which can be extremely subtle. Ranging from the obvious, like detecting jaundice, to nearly imperceptible changes in facial or voice affect, characteristics picked up by the app tell us a lot about how patients are being affected by the therapy. By utilizing these biomarkers, data is collected that can show (among other things):

  • Improvements in pain
  • Changes in mobility
  • Occurrence and changes to tremors and other neurological responses

Tracking these types of data can give sponsors clear tools for differentiating, marketing and selling their therapy. 

Tracking the Patient’s Journey

Total cure or remission is wonderful, but not always possible. Many therapies exist to extend life, reduce painful and/or unpleasant symptoms, and to improve the patients’ ability to lead as close to a healthy life as possible for as long as possible. 

Through the ePro features and functionality within the Patient Connect app, AiCure makes it simple for patients to chronicle their symptoms, providing insight beyond adherence alone. Through this data, sponsors have a repository of information that can be used to help market their therapy. For instance, the ePro data can show that patients are feeling better day to day, missing fewer days of work, or enjoying more quality time with their loved ones. It’s hard to quantify some of this kind of information in a clinical trial endpoint, but it can be powerful to share with clinicians who are looking for new ways to help their patients.

As you are building your endpoints and identifying which results will help to prove efficacy and safety, it’s important to also give thought to what kinds of data can help you set your therapy apart once it’s on the market. Clinicians care deeply about their patients. While they want to know how the therapy performed relative to study endpoints, they also want to know if the therapy can make their patients feel healthier, have less pain, have a more normal social experience, or allow them to spend more time with their family. If you can use data collected during the clinical study to prove that your therapy makes patients healthier and improves the quality of life, you greatly enhance your chance for successful commercialization.