SCOPE 2023: Adapting to the Changing Landscape of Clinical Trials and the Biopharmaceutical Industry

The 2023 SCOPE Summit brought together industry leaders to discuss the challenges and opportunities in clinical trials. The conference covered various topics, including patient diversity, shifts in the biopharmaceutical industry, clinical trial quality, and decentralized clinical trials. This report will summarize some key topics discussed during the conference.

FDA: Clinical Trial Patient Diversity is Expanding, but Inclusion is Not

Jamie Brewer, Clinical Team Lead at FDA CDER, discussed diversity in clinical trials and recent FDA recommendations. Brewer indicated that due to the biopharmaceutical industry focusing on shortened timelines for drug development, the biopharmaceutical industry has gone global to recruit patients; however, there is a trade-off between time to characterize the safety profile of drugs before approval and time to enroll representative clinical trial populations. As a result, clinical trial populations have become less representative of regional ethnic minorities due to this globalization. Brewer encourages biopharmaceutical enterprises to focus on local patient populations to ensure that the population is representative of the country of regulatory submission; for example, enrolling more patients in the US to represent the US population during FDA regulatory submissions.

Brewer then went into restrictive protocol eligibility criteria, which fail to identify populations representative of the disease population, and there have been efforts to expand eligibility requirements. For example, the FDA issued guidance on this topic, including clinical trial eligibility for cancer patients; however, the industry needs more inclusive and expansive eligibility requirements for more comprehensive patient representation. Sources such as FDA Snapshot quantify clinical trial diversity, but subtle data representation must be considered, especially since certain racial and ethnic groups are less likely to be represented in global clinical trials. Brewer also brought up a concern that bias may be introduced if clinical trials focus solely on academic medical research institutions since only a small proportion of patients receive clinical care in academic centers conducting clinical trials; this could suggest that FDA wants the industry to expand clinical research to underserved communities to expand local patient population representation.

Shifts in Clinical Trials and The Biopharmaceutical Industry

Ken Getz, Executive Director of The Tufts Center for the Study of Drug Development, discussed how the pharmaceutical industry's rise of personalized medicine, rare and ultra-rare diseases, and biomarkers stresses the biopharmaceutical industry to undergo rapid change and increasing complexity. Moreover, due to the pandemic and the subsequent shifts in workforce dynamics and clinical trial execution, the industry faces additional obstacles.

The first obstacle is extreme complexity and personalization, which makes it difficult for the pharmaceutical industry to manage the growing number of options and stakeholders' resistance to change. This presents a challenge in accommodating all of the possibilities and opportunities in the present environment, as the industry has never before encountered such extreme customization.

The second challenge is difficulties with the workforce, exacerbated by the pandemic. The pandemic has disrupted industry operations, such as clinical trial execution and patient engagement. Stakeholders are concerned about their ability to evaluate and analyze new solutions and approaches with the same degree of discretion and deliberation as in the past.

The third difficulty is the pressure to maximize the value of each potential drug candidate. Over the past 15 years, the return on investment for new commercial drugs has decreased by approximately one-third, putting increasing pressure on the industry to balance the cost of development with the value of the drug candidate to ensure that the investment is worthwhile.

In response to these challenges, the pharmaceutical industry is examining novel approaches to manage complexity and customization, adapt to a shifting environment, and maximize the value of drug candidates. This includes leveraging technology and data to streamline the execution of clinical trials, adopting patient-centric approaches, and rethinking the drug development process to make it more efficient and effective. In addition, in a rapidly changing environment, the industry is seeking new ways to innovate and provide patients with value.

Clinical Trial Quality Update

Panels discussed the necessity of quality tolerance limits (QTLs) and whether removing QTLs from regulations impacts using QTLs. The panel agreed that while regulations are important, the quality of the study is the ultimate goal, and QTLs can help achieve this. They also discussed the importance of evaluating quality during the study and how it can facilitate risk mitigation plans. There is some concern about the language used to replace quality tolerance limits, but companies have invested a lot of time and effort into implementing them, and they are a useful tool for understanding what is going on in a study. Overall, the panel believes that, despite its removal from regulations, QTLs are still important even if they are no longer mandated.
Another presentation by Esther Huffman O’Keefe, Director of Adaptive Monitoring Excellence at Takeda Pharmaceuticals, suggests adopting a quality-by-design (QbD) approach, which promotes critical thinking and dialogue about quality, rather than relying on traditional monitoring methods that use checklists and tools. She emphasized the need to build quality into all levels of study planning and conduct and periodically review critical quality factors. O’Keefe argues that traditional monitoring approaches may not achieve quality goals and suggests a risk-based, data-driven monitoring approach involving all roles in the trial and that quality and monitoring strategies should be a top priority in the industry.

Decentralized Clinical Trials

Patrick Floody, Executive Director of Global Clinical Trial Services, Regeneron, explained that DCT is a conversation about technology and services enabling remote interactions. His group aims to simplify the process and make it accessible to patients, which could increase enrollment and retention. The group has been surveying patients to understand what they think about DCT and how it can be made more accessible. Floody said that around 70% of patients live two or more hours away from clinical trial sites, making it challenging for them to participate, and patients may be more likely to engage with clinical trials by providing remote options.

His group has identified four models on the DCT spectrum: traditional, where everything is done on-site; partial decentralized, where some elements are done remotely; fully decentralized, where everything is done remotely; and centralized virtual, where some elements are done remotely and some are done on-site.

To make DCTs more accessible to patients, the group is considering several options, including mobile testing centers, phone apps, commercial labs, radiology centers, and shipping optimization. Floody emphasized enhancing the patient experience and gaining user feedback to understand their perceptions.

Summary

In summary, some of the discussions at SCOPE centered around the need for increased local patient diversity and inclusion in clinical trials, the challenges posed by the rise of personalized medicine and rare diseases, the importance of quality in clinical trial execution, and the potential benefits of decentralized clinical trials and the need to make them more accessible to patients. Overall, the summit highlighted the industry's efforts to adapt to a rapidly changing environment and innovate new approaches to ensure that clinical trials continue producing safe and effective treatments for patients.

Moe Alsumidaie, MBA, MSF, is a thought leader and expert in the application of business analytics toward clinical trials, and regular contributor to Applied Clinical Trials.