Shifting Expectations for Clinical Development

Jeffrey Spaeder, MD

COVID-19 has reemphasized the importance of clinical research on individual patients and families and society as a whole. It helped crystalize how clinical trials can address medical needs, especially on population and societal levels, and catalyzed the development of new technologies. Researching novel and unproven therapies demands caution and humility and gives deference to precedent. So, while there was interest in innovation to mitigate the increasing expense and complexity of drug development, there were modest enhancements in conducting these studies. However, the pandemic accelerated the pace of development and number of innovations. In aiming to develop and provide COVID-19 vaccines, we saw a united urgency within the biopharma industry to speed up decision-making, set-up and execute trials and ensure those most affected are represented in the research.

Direct-to-patient approaches have historically been utilized tepidly and with skepticism. Impacting routine methods of patient care, COVID-19 made stakeholders more responsive to virtual trial solutions and other tech-enabled approaches. For example, pre-pandemic, study investigators in the US expressed interest in the use of digital tools/technologies (62%), remote monitoring methods (52%) and telehealth (25%) in study processes. After the outbreak, interest increased: use of digital tools/technologies (83%); remote monitoring methods (76%) and telehealth (73%).

Sponsors may increasingly accept, if not expect, the incorporation of direct-to-patient approaches as:

1. Future outbreaks due to coronavirus or other highly infectious pathogens are likely, making planning for clinical studies with limited access to traditional research sites important.
2. Growing societal acceptance of remote and digital interaction impacts expectations of study subjects regarding remote participation.
3. These approaches enable patients who have limited or no access to studies to participate—enhancing diversity within the research.

Sponsors starting clinical programs will need to determine how virtual they can go and in some cases, may look to hybrid approaches—mixing traditional design with virtual elements. The industry’s ability to design, execute and complete COVID-19 vaccine trials regionally or globally within a year led to the development of several highly efficacious vaccines. It exemplifies how virtual studies can increase trial speed and efficiency when scaled to meet the needs within individual therapeutic spaces.

The urgency to increase trial efficiencies and timelines is an industry constant. The longer trials take, the longer patients may be waiting for a viable option for debilitating and potentially life-threatening conditions. But, traditionally, trial activities and processes have worked sequentially, taking up time in order to de-risk decisions, focus and investments.

To rapidly develop COVID-19 treatments and vaccines, trial teams stepped outside the norm of traditional sequential trial activities. In some cases, critical trial steps occurred in parallel of another, including more at-risk ways of working.

Given the unknowns around COVID-19 and the urgency for resolution, sponsors made study design and protocol details public—creating information transparency among stakeholders like never before.

Governments were involved as stakeholders in the design and execution of COVID-19 vaccine trials, closely collaborating with sponsors. Budding collaborations among pharmaceutical companies and governments may happen more often as public health needs arise and as we continue seeing positive gains of working together in fighting COVID-19.

The industry is increasingly accelerating strategies to ensure underrepresented communities, including those of color. Through targeted and intentional outreach, collaboration and awareness, there is a renewed emphasis on traditionally underrepresented communities to expand participation in clinical research and access to healthcare. In COVID-19 research, sponsors such as Moderna showed their willingness to modulate trial enrollment and prioritize engagement to ensure adequate participation of communities disproportionately impacted by COVID-19.

Industry stakeholders must continue to be agile in strategy and ready to adapt. But, as we navigate through our biggest global healthcare challenge to date, we are collecting numerous learnings to further improve future clinical trials. That much we do know.

Jeffrey Spaeder, MD, Chief Medical and Scientific Officer, IQVIA