Digital technologies for medicines: shaping a framework for success

Cerreta, Francesca; Ritzhaupt, Armin; Metcalfe, Thomas; Askin, Scott; Duarte, João; Berntgen, Michael; Vamvakas, Spiros

doi:10.1038/d41573-020-00080-6

Cited by 39 publications

(37 citation statements)

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“…Some of these limitations will be addressed in the follow-up work that the Mobilise-D consortium is undertaking, according to the staged qualification approach described in the “Qualification Process” section above. Such a stepwise approach was recently praised in a paper co-authored by EMA scientific officers [ 59 ]. In a second request for EMA qualification advice, which is currently in preparation, we plan to use the same approach described here for the PD to seek qualification for the use of DMOs as monitoring biomarkers in MS, COPD, and PFF.…”

Section: Discussionmentioning

confidence: 99%

Toward a Regulatory Qualification of Real-World Mobility Performance Biomarkers in Parkinson’s Patients Using Digital Mobility Outcomes

Viceconti

Penna

Dartee

et al. 2020

Sensors

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Wearable inertial sensors can be used to monitor mobility in real-world settings over extended periods. Although these technologies are widely used in human movement research, they have not yet been qualified by drug regulatory agencies for their use in regulatory drug trials. This is because the first generation of these sensors was unreliable when used on slow-walking subjects. However, intense research in this area is now offering a new generation of algorithms to quantify Digital Mobility Outcomes so accurate they may be considered as biomarkers in regulatory drug trials. This perspective paper summarises the work in the Mobilise-D consortium around the regulatory qualification of the use of wearable sensors to quantify real-world mobility performance in patients affected by Parkinson’s Disease. The paper describes the qualification strategy and both the technical and clinical validation plans, which have recently received highly supportive qualification advice from the European Medicines Agency. The scope is to provide detailed guidance for the preparation of similar qualification submissions to broaden the use of real-world mobility assessment in regulatory drug trials.

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Section: Discussionmentioning

confidence: 99%

Toward a Regulatory Qualification of Real-World Mobility Performance Biomarkers in Parkinson’s Patients Using Digital Mobility Outcomes

Viceconti

Penna

Dartee

et al. 2020

Sensors

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“…More recently, the EMA has outlined a framework for success in reference to deployment of DHT in clinical trials [51]. Recommendations include the following: (1) early interaction with the EMA to maximize knowledge and align across multidisciplinary teams;…”

Section: European Medicines Agencymentioning

confidence: 99%

Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science

et al. 2020

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Innovative tools are urgently needed to accelerate the evaluation and subsequent approval of novel treatments that may slow, halt, or reverse the relentless progression of Parkinson disease (PD). Therapies that intervene early in the disease continuum are a priority for the many candidates in the drug development pipeline. There is a paucity of sensitive and objective, yet clinically interpretable, measures that can capture meaningful aspects of the disease. This poses a major challenge for the development of new therapies and is compounded by the considerable heterogeneity in clinical manifestations across patients and the fluctuating nature of many signs and symptoms of PD. Digital health technologies (DHT), such as smartphone applications, wearable sensors, and digital diaries, have the potential to address many of these gaps by enabling the objective, remote, and frequent measurement of PD signs and symptoms in natural living environments. The current climate of the COVID-19 pandemic creates a heightened sense of urgency for effective implementation of such strategies. In order for these technologies to be adopted in drug development studies, a regulatory-aligned consensus on best practices in implementing appropriate technologies, including the collection, processing, and interpretation of digital sensor data, is required. A growing number of collaborative initiatives are being launched to identify effective ways to advance the use of DHT in PD clinical trials. The Critical Path for Parkinson’s Consortium of the Critical Path Institute is highlighted as a case example where stakeholders collectively engaged regulatory agencies on the effective use of DHT in PD clinical trials. Global regulatory agencies, including the US Food and Drug Administration and the European Medicines Agency, are encouraging the efficiencies of data-driven engagements through multistakeholder consortia. To this end, we review how the advancement of DHT can be most effectively achieved by aligning knowledge, expertise, and data sharing in ways that maximize efficiencies.

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“…These could be predefined by the study team and should be accepted by regulatory bodies. The very positive signals and recommendations of the EMA 39 and the FDA 23 to include performance instruments on an exploratory level in the assessment panels of clinical trials support this proposal by producing data sets that can be analyzed accordingly. Still, only one‐third of clinical trials on movement disorders that are actually registered in http://clinicaltrials.gov include such performance instruments in their assessment panels 43 .…”

Section: How To Best Assess Daily Function?mentioning

confidence: 97%

Modernizing Daily Function Assessment in Parkinson's Disease Using Capacity, Perception, and Performance Measures

et al. 2020

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A BS TRACT: Many disease symptoms restrict the quality of life of the affected. This usually occurs indirectly, at least in most neurological diseases. Here, impaired daily function is interposed between the symptoms and the reduced quality of life. This is reflected in the International Classification of Function, Disability and Health model published by the World Health Organization in 2001. This correlation between symptom, daily function, and quality of life makes it clear that to evaluate the success of a therapy and develop new therapies, daily function must also be evaluated as accurately as possible. However, daily function is a complex construct and therefore difficult to quantify. To date, daily function has been measured primarily by capacity (clinical assessments) and perception (surveys and patient-reported outcomes) assessment approaches. Now, daily function can be captured in a new dimension, that is, performance, through new digital technologies that can be used in the home environment of patients. This viewpoint discusses the differences and interdependencies of capacity, perception, and performance assessment types using the example of Parkinson's disease. Options regarding how future study protocols should be designed to get the most comprehensive and validated picture of daily function in patients are presented.

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Digital technologies for medicines: shaping a framework for success

Abstract: Regulatory agencies can provide advice to support developers of digital technologies for medicines use, but what are the best strategies to maximize the chance of a successful regulatory interaction? Here, EMA and industry representatives comment on the experience so far.

Cited by 39 publications

References 0 publications

Toward a Regulatory Qualification of Real-World Mobility Performance Biomarkers in Parkinson’s Patients Using Digital Mobility Outcomes

Toward a Regulatory Qualification of Real-World Mobility Performance Biomarkers in Parkinson’s Patients Using Digital Mobility Outcomes

Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science

Modernizing Daily Function Assessment in Parkinson's Disease Using Capacity, Perception, and Performance Measures

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