By:
Jasmine Begin, M.S., RAC - Director, Regulatory Affairs - Regulatory Professionals, A Division of Premier Research
Daniel Semere, MTOPRA - Senior Regulatory Submissions Manager - Premier Research
“Patient centricity” has long been a guiding principle of clinical trial design and conduct, and it has become even more critical in the COVID era as trial sponsors and regulatory authorities have had to adapt to a continually shifting landscape. Pandemic-related restrictions have prompted substantial changes in how clinical trials are developed and implemented, requiring sponsors to comply with new guidance and processes designed to promote speed and efficiency while remaining mindful of patient safety.
The U.S. Food and Drug Administration (FDA) has issued Emergency Use Authorizations to facilitate the availability of testing kits, treatments, and vaccines; established a Coronavirus Treatment Acceleration Program; and authorized use of reference panel comparative data to enhance diagnostic capabilities. Additionally, the FDA has issued thirteen guidance documents since May 2020, incluing several updates designed to address various aspects of product development and clinical trials (Table 1). The FDA’s recent actions reflect a renewed focus on the patient experience as the pandemic has forced a reimagining of standard practices and protocols.
As a further spur to innovation, the FDA has encouraged using mechanisms such as Q-submissions, pre-investigational new drug application (pre-IND) meetings, and humanitarian device exemptions to expedite review of investigational therapies and devices with potential efficacy and sufficient safety data to enter clinical trials. The FDA is encouraging the use of these mechanisms even when clinical data are not yet available, presumably because of their timeline-shortening potential. Such initiatives are already showing results, with some protocols reviewed within 24 hours and single-patient expanded access requests granted within three hours.
The European Medicines Agency (EMA) has demonstrated a similar inclination to fast-track its review of clinical trial protocols for treatments and vaccines, compressing its standard 40-to-70-day timeline to roughly 20 days. The accelerated timeline includes pediatric investigation plans, for which review timelines previously took up to 120 days. In addition, the EMA has issued six updated guidance documents for clinical trials in the past year (Table 2), most of which emphasize patient-focused considerations that were gaining currency pre-COVID. The EMA is also making greater use of accelerated assessment, rolling review, indication extensions (for already approved medicines), and compassionate use programs to expedite marketing authorization applications and enhance patient access to investigational treatments.
Adapting to a Shifting Landscape
The FDA, EMA, and other national competent authorities have responded and adapted to the shifting landscape, as evidenced by the shortened review timelines and the increase in trial approvals. Similarly, the industry has exhibited heightened creativity and inventiveness in reimagining the patient experience in clinical trials. Such flexibility and resourcefulness will be essential in the near term, as many pandemic-related restrictions will likely remain in place for the foreseeable future. Those restrictions may require trial sponsors and CROs to modify specific trial processes and implement novel methods to engage and retain participants while ensuring patient safety and trial integrity.
We have already seen substantial growth in decentralized clinical trials (DCTs), mobile health (mHealth), virtual visits, electronic consent, and other technological advances that reduce or eliminate the need for many participants to visit physical investigational sites. These trends will likely continue as the pandemic runs its course.
Similarly, participants lacking access to investigational agents may need additional safety monitoring, possibly necessitating protocol amendments and enhanced coordination with institutional review boards (IRBs), ethics committees, and local authorities
Looking Toward the Future of Patient-Focused Trials
The flurry of recent regulatory developments and trial modifications is expected to reinforce and amplify the profile of clinical research participation as a noble and vitally important pursuit in the post-COVID era. Ushering in a new generation of patient-centric trials will likely entail tightening some regulations and processes to enhance patient safety and loosening others to make participation more convenient and accessible, and less burdensome. Empowered by such changes, drug developers will increasingly rely on electronic data collection, remote patient monitoring for DCTs, and other technological advances that allow them to conduct trials faster and more efficiently.
As the updated regulatory guidelines shape the new trial landscape, sponsors and CROs may need to overhaul good clinical practice principles and standard operating procedures (SOPs) to keep pace. In the near term, sites will need to adopt newly mandated safety measures and other strategies to enhance the patient experience. The accelerating shift to mHealth and remote monitoring technologies will likely continue as enrolled patients continue to opt for the flexibility of remote participation.
While shorter approval timelines will likely benefit drug developers and patients alike, sponsors and CROs will need to regularly review their policies and strategies, particularly concerning the recruitment and retention of more diverse patient populations. Moreover, enhancing the patient experience will be a long-term proposition, requiring modification of SOPs to comply with updated regulations and guidance documents that specifically address patient access and ease of participation. Some new regulations may mandate additional documentation and closer coordination with IRBs and ethics committees.
The Speed-And-Safety Balancing Act
The clinical trial environment has always been fiercely competitive, and the pandemic has intensified pressure on trial sponsors to optimize their clinical development strategies and the patient experience. This balancing act places patient safety at the forefront of every trial. As new regulations continue to intensify the competing pressures for speed, efficiency, and safety, sponsors will need to consider patient needs at every step of the clinical trial. Collaborating with a partner with the essential expertise can help even the most agile drug developer navigate an increasingly complex regulatory landscape.
Jasmine Begin, M.S., RAC, is Director of Regulatory Affairs at Regulatory Professionals, A Division of Premier Research. Ms. Begin brings more than 16 years of experience in the industry working on an array of different products, predominantly in small molecule and drug/device combination products, with 11 of those years working within regulations. In her current position, she provides senior strategic regulatory management and advice on nonclinical and clinical programs for investigational and filed products.
Daniel Semere, MTOPRA, is Senior Regulatory Submissions Manager at Premier Research. Mr. Semere is responsible for the global regulatory start-up strategy for each given projects or products in various areas such as endocrinology, pediatrics, rare disease, cardiology, neurology, rheumatology, and oncology. With more than 13 years of experience as regulatory and study start-up manager, he has worked in large pharmaceutical companies and other CROs contributing to many successful approvals.
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