In May 2018, Tools4Patient (T4P) presented a webinar entitled “Characterization of Individual Patient Placebo Response: Impact on the Clinical Study Power”. This webinar aimed to explain the challenge of the placebo effect, introduce the application of Placebell©™ technology and describe how it may improve interpretation of clinical trial results. A recorded session is archived and available here. During this webinar, questions from the participants were addressed; the main ones are compiled below.

What is the Regulatory Status of the Placebell©TM Approach?
FDA: Tools4Patient (T4P) interacted with FDA at the Critical Path Innovation Meeting (CPIM) in April 2016 and presented Placebell©™ (association of proprietary questionnaire and a covariate). There is no FDA pathway/guidance applicable to the review of Placebell©™ technology. As the technology provides a covariate, the FDA has recommended to submit the use of the Placebell PlaceboCovariate (like any covariate) in the context of individual drug development programs and individual divisions, until broad application across therapeutic areas.

EMA: T4P interacted with the EMA at a meeting with the Innovation Task Force in June, 2018. T4P presented the generalized approach of validating Placebell©™ in each therapeutic indication, in combination with the data from validation studies in peripheral neuropathic pain as a case study. Based on the information shared, ITF has recommended that T4P pursue submission of Placebell©™ for a Qualification Advice.

Many of the Areas in Which Placebell©™ is Being Developed are in Neuroscience; Can This Technology be Utilized in Other Therapeutic Areas?
The initial concept of the Placebo-Covariate was tested and confirmed in peripheral neuropathic pain by the conduct of stand-alone, placebo only trials. Clinical studies in several other indications are currently ongoing. Beyond this, the Placebell©™ technology can be applied to virtually any indication where the placebo effect is known to confound clinical evaluation of efficacy. In these situations, relevant disease-specific modifications to Placebell©™ will be identified by an initial clinical study within the clinical development plan prior to its use in subsequent trials.

How is the Data Required by Placebell©TM Generated?
In the Placebell©™ approach, the placebo-covariate is calculated on a per-patient basis via a mathematical model combined with patient data. Patient data includes disease characteristics, demographics, and responses to a specific personality questionnaire. From its inception, the team behind Placebell©™ worked to ensure that its inclusion in a clinical study would be as non-disruptive to trial operations as possible. As such, patients only need to complete the Placebell©™ questionnaire once, before the first dose administration.