Europe Tackles Digital Endpoints in Drug Development

As digital technology continues to sweep increasingly across the world of healthcare, attention within the clinical trials community is now turning with new intensity to the adoption of digital endpoints in medicine development. The European Federation of Pharmaceutical Industries and Associations (EFPIA), Europe’s principal drug industry association, has recently established a specific workstream to explore and assess the current European landscape affecting the speed of adoption of digital health technology (DHT) derived endpoints.

This is a wide field that is becoming wider. It includes, but is not limited to, endpoints and methodologies based on digital technologies in the development of medicines. The range extends from sensors such as ingestibles and implantables to mobile health tools such as the wearable devices patients carry to measure certain health related parameters, or for remote patient monitoring. It can provide measurements in pharmacodynamics, efficacy, and safety. And it has applications in a wide range of therapeutic areas, including hematology, immunology, neuroscience, respiratory, cardiovascular, oncology, and ophthalmology.

Some guidance already exists on the subject, including European Medicines Agency (EMA) notes on computerized systems and electronic data in clinical trials, or qualification of novel methodologies for drug development, and FDA guidance on digital health technologies for remote data acquisition in clinical investigations. But even the terminology diverges between EMA, FDA, and other organizations. And the US-based Clinical Trials Transformation Initiative has issued recommendations titled "Developing Novel Endpoints Generated by Digital Health Technology for Use in Clinical Trials" with a checklist that urges sponsors to include digitally-derived endpoints in early phase clinical trials and observational cohort studies to demonstrate they are fit-for-purpose, and to select the technology after selecting an outcome.

The subject of DHT is going to be explored in greater depth in a virtual workshop in December titled “Enhancing Patient-centric Outcome Measures and Clinical Trials Using Digital Health Technologies."1 The aim is to advance the thinking on how to optimize the development of DHD, the generation of evidence for validation and qualification, and regulatory pathways. Topics slated for the agenda include expectations and initiatives from regulators in the EU and the US and the perspectives of ethics committees, patients, academia, health technology assessment agencies, and technology developers. The workshop also promises to explore use of DHTs beyond endpoints—for prognosis, patient selection, and diagnosis.

The hopeful output of the workshop is agreement to build a roadmap for enabling the use of DHTs in drug development in Europe and beyond. In preparation, EFPIA is conducting a survey seeking information about experience with digital endpoints intended for use in medicine development.2 It wants to collect views on the current status and experiences in the current environment, so as to better understand what the barriers and potential solutions might be.

The survey is open to academia; companies in the biotech, pharmaceutical, and medical device industries; patient groups; contract research organizations (CROs); and technology vendors. It inquires into where the trials in question were conducted and what form of involvement responders had—ranging from technology development to regulatory, and from clinical trials to analytical validation.

Respondents are also asked to rank the barriers they have encountered specifically in the European environment, from a list that ranges from regulatory and data privacy barriers to lack of perceived value or lack of clarity on development steps, and including challenges related to clinical interpretation or in implementing good clinical practice in relation to data integrity or missing data.