Despite the evolution of mobile technology capable of monitoring and measuring data, it remains uncommon for such novel clinical endpoints to be used in clinical trials, especially those trials that are performed to support regulatory approval for a new therapy or indication. All stakeholders—patients and patient groups, investigators, research sponsors, regulators, and technology developers—would benefit from clarification regarding the processes for developing these novel endpoints in order to realize the benefits of incorporating mobile technology into clinical trials.

Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials (2015–2017), one of four projects within CTTI’s MCT Program.

Describe best practices for identifying, selecting, and developing novel endpoints, generated using mobile technology, for use in regulatory clinical trials.

The results from the MCT Novel Endpoints Project will inform the development of novel endpoints generated from mobile technologies for use in FDA-regulated clinical trials.

CTTI hosted a multi-stakeholder expert meeting to develop four use cases describing the steps involved in developing a novel endpoint from data generated by a mobile device. This process helped CTTI to outline requirements at each step in the process, identify challenges that may occur, and propose solutions to those challenges. The use cases informed the development of more general recommendations and tools that clarify the pathway for developing technology-derived novel endpoints. The MCT Novel Endpoints Project Team has also conducted a systematic review of published clinical studies using novel endpoints in order to learn from existing approaches. This article will be posted here following publication.

CTTI convened a multi-stakeholder expert meeting to draft use cases, each developing a particular novel endpoint. Meeting attendees explored the use of accelerometers to measure treatment benefit in clinical trials of heart failure, Parkinson’s disease, and Duchenne muscular dystrophy, and the use of continuous glucose monitors to measure treatment benefit in clinical trials of diabetes. In addition to the four use cases, a meeting summary is available that highlights themes from these discussions.

A set of recommendations were distilled from the meeting findings and related discussions. The recommendations, which are expected to have short-, medium-, and long-term benefits when applied in clinical trials, are grouped into two main sections. The first, which focuses on optimizing the selection of novel endpoints, identifies the following key recommendations:

1. Patient’s voices must drive the identification of novel endpoints;
2. Select the mobile technology after selecting an outcome assessment; and
3. Use a systematic approach to identify key novel endpoints.

The second section provides recommendations on practical approaches to developing novel endpoints, including:

1. Foster collaboration among key stakeholders;
2. Create technical standards for assessments derived from mobile technology;
3. Engage with regulators; and
4. Include novel endpoints as exploratory endpoints in existing clinical trials and observational studies.

These major recommendations are presented in detail, along with complementary materials that include an interactive tool for choosing among possible novel endpoints for development, a quick reference for interacting with regulatory authorities during the process of novel endpoint development, a summary of required steps in novel endpoint development, a plain-language process summary, and detailed, disease area-specific use cases.

The team has also conducted a systematic review of published clinical studies that have employed novel endpoints. The resulting paper will be posted here following publication.