An Era of Innovation in Phase 1 Clinical Trials

Clinical research is undergoing a period of rapid transformation. New technology that focuses on greater speed and efficiency is shaking up all aspects of the research process, from Phase 1 clinical trials to post-market surveillance. The growing use of artificial intelligence, eClinical tools, and mHealth is helping researchers to more quickly overcome challenges with patient recruitment and retention, data capture and analysis, and other long-standing issues.

These advancements in speed and efficiency in clinical research are typically seen as the domain of private-sector innovators. But it’s not just industry – regulatory bodies are emphasizing speed and flexibility as well. The FDA and others have not only signaled their embrace of new timesaving tools, but have also announced new guidelines and novel trial designs aimed at further innovating and streamlining trials.

This combination of technology and regulatory measures has wide-reaching implications for the future of the entire clinical research industry. Several of the most recent developments hold particular promise for oncology drugs and biologics in Phase 1 clinical trials.

A New Paradigm

In August 2018, the FDA launched the Complex Innovative Designs Pilot Meeting Program, which allows drug and biologic researchers to meet with agency staff to discuss novel clinical trial designs and to develop more effective strategies to assess the safety and efficacy of medical products. The central goal: adopt techniques that will make clinical trials more cost-efficient and flexible.

The CID Pilot builds on earlier steps the FDA took this year with guidelines on the use of expansion cohorts study designs in Phase 1 clinical trials in oncology. As cancer treatment shifts away from traditional chemotherapeutics to molecularly targeted agents, larger cohorts have the potential to generate data faster, use researchers’ time more efficiently, and assess the safety of new compounds sooner.

Where Technology Fits

Pairing these developments with new technology is particularly compelling. Ethical and safety concerns are especially significant in oncology. Since in Phase 1 the safety profile of a new compound isn’t fully clear, tools that allow real-time data entry like eSource complement CID by detecting safety signals more quickly. Additionally, while EDC can help set up trials and make common Phase 1 mid-study changes without downtime, expansion cohorts permit faster data collection from a broader set of patients – since more will meet inclusion criteria.