Oncology is recognized as having the largest drug pipeline of any therapeutic area. Estimates based on publicly available information suggest more than 40 percent of all new therapies in development are cancer treatments. Given this hotbed of activity, innovation in the space to drive faster decisions and more efficient trials is intense.
There are many aspects of oncology drug development that are driven by the unique nature of the treatments being developed and the needs of the patients, their caregivers and the doctors that treat them. Continued advances in the understanding of the molecular basis for cancer, coupled with the development of precision medicine have led to the development of trial models that allow for more patient-centric trial designs that have been endorsed by the regulatory agencies globally, and in turn receiving a lot of attention from drug developers, particularly since the start of the pandemic. Changes in what clinical performance data is used to inform the trial design and site selection, new approaches to patient recruitment and the rise of advanced treatments such as cell and gene therapy are rewriting the script.
Key oncology conferences such as the one organized by the European Society for Medical Oncology (ESMO) which took place in October 2023, are some of the best places to keep a finger on the pulse of these innovations.
In this Xtalks Spotlight edition, Xtalks spoke with two oncology clinical research experts from Fortrea, Laura Vidal, MD, Medical Head of Oncology, EU (European Union), and Ken Morrison, PhD, Global Head of Strategic Delivery & Growth Oncology.
Dr. Morrison and Dr. Vidal shared their insights from the 2023 ESMO conference which occurred in Madrid. They discussed the top new trends in oncology trial design that sponsors are either incorporating into clinical trials or need to start planning to include.
Dr. Vidal is a certified medical oncologist with extensive experience in oncology drug development.
Dr. Morrison has over 30 years of oncology preclinical and clinical experience between academia and the industry sector in pharma and clinical research organizations (CROs).
Key Highlights from ESMO 2023
Dr. Vidal emphasized the remarkable quality of work presented at ESMO 2023, particularly in the presidential session, where promising results were shown for challenging conditions such as bladder cancer and non-small cell lung cancers with rare mutations.
“It was also very nice to see a lot of patients and advocacy groups involved in discussions with investigators and regulators to be part of clinical trial designs,” said Dr. Vidal. “It’s very positive and it’s going to help move forward the pathway for new drugs.”
Dr. Morrison remarked that the latest ESMO was unique in its broad discussion scope, touching on various innovative treatments. “There were a range of therapies under discussion, from targeted therapies to advanced antibody-drug conjugates (ADCs) and cell and gene therapies,” he says.
Additionally, Dr. Morrison mentioned that different types of data are being applied in the clinical development space, including the increasing focus and importance of real-world evidence to support clinical data and early registration of assets where there is a significant unmet need.
Evolution in Oncology Clinical Trial Design
Recent discussions at ESMO highlight a shift in how clinical trials are designed and conducted. This shift is driven by the rapid advancements in understanding the biology of specific cancers and the development of targeted therapeutic interventions.
According to Dr. Morrison, trial designs are increasingly focusing on specific patient populations with certain biomarkers, particularly in cancers like non-small cell lung cancer. “A basket study (in which a group of tumors express the same target) or an umbrella study (where the same tumor expresses multiple targets), two types of master protocol trial designs mentioned in the FDA’s Guidance document on March 2022, allow early decision-making regarding which cancer histologies each asset has most promise clinically with the focus on establishing the recommended Phase II dose (RP2D),” he notes.
Dr. Vidal also points out that sometimes these lead to more of the smaller, targeted studies focused on patients with specific biomarkers. This approach emphasizes precision medicine based on individual biological markers and is different from traditional large-scale trial designs, which are based on the staging of the cancer and broad therapeutic Standard of Care options.
Furthermore, Vidal underscores a significant trend: the increasing importance of patient voices in the visit schedules of such trial designs, where often additional blood, tissue samples are collected, together with imaging and Patient Reported Outcomes (PROs). Discussions at ESMO have increasingly revolved around the importance of involving patients in designing studies, understanding outcomes important to them and ensuring that the trials address quality of life issues, together with ensuring that the visit schedules minimize the patient burden associated with trial participation. The involvement of patients, caregivers and patient advocacy groups is seen as a transformative factor in drug development, in addressing patient needs, as well as accelerating the interest and inclusion of more cancer patients in clinical trials as an option at the point of care.
Collaboration in Oncology Clinical Trials
The unique aspect of oncology trials is that there is a strong and unified alignment among all stakeholders to ensure that patients have the opportunity to participate in trials, and that there is a focus on diversity and inclusion ethnically and socioeconomically. For oncology trials to be successful, there must be a strong partnership between the sponsors, the CROs that manage the trial execution and the investigator sites that directly interact with the patients and their caregivers.
“The drug developer needs to think about the patient’s needs in the context of the trial design, the visit schedule [and] the physician obviously in terms of informing the patient and supporting them and their families through the study,” said Dr. Morrison, emphasizing the commitment to supporting patients throughout their trial journey, balancing their contributions to the trial with their valuable time spent with loved ones.
As Dr. Vidal points out, “Innovation is not effortless and requires a lot of strong collaboration among all the stakeholders.” She stressed the importance of direct interactions with investigators to understand on-site challenges and improve trial experiences. She also mentioned the need for dialogue with patients and caregivers to ensure a positive trial experience and willingness to participate.
Reflecting on ESMO: Transforming Oncology Trial Design and Execution
Dr. Morrison and Dr. Vidal shared their insights on how the 2023 ESMO conference has influenced their perspectives on oncology trial design and execution.
Dr. Morrison’s reflections were centered around the pivotal role of real-world data in cancer treatment. A significant discussion at ESMO revolved around the quality and interpretation of data from electronic medical records, highlighting both its potential and limitations.
For Dr. Morrison, the key takeaway was the necessity for the industry to bridge the gap between traditional clinical trial settings and real-world data applications. This involves innovative trial designs and technologies that track patient experiences from the experimental stage to post-approval of a drug. Especially in the realm of cell and gene therapy, long-term follow-up is vital, requiring a reconsideration of endpoints and safety profiles for up to 15 years; thus, efficient solutions to allow this to happen are critical. Fortrea has a number of solutions to support this need.
Dr. Vidal brought attention to a remarkable development in cervical cancer treatment presented at ESMO. A Phase III study showed an impact on overall survival using standard chemotherapy in a novel way. This approach, particularly beneficial for less developed countries due to its cost-effectiveness, could be implemented almost immediately, as evidenced by the enthusiastic response from investigators.
Furthermore, Dr. Vidal highlighted discussions on artificial intelligence (AI) in clinical trials. The challenge lies in applying AI meaningfully while navigating issues around data confidentiality. This suggests a need for regulatory bodies to adapt rapidly to these technological advancements to harness data more effectively and expediently in patient treatment.
Overall, the insights from Dr. Morrison and Dr. Vidal reflect a dynamic landscape in oncology clinical trials, marked by cutting-edge research, patient-centered approaches and innovative treatment modalities. Their observations underscore the rapid advancements in cancer treatment and the importance of integrating diverse data sources to enhance clinical research and patient care.
For more insights into oncology clinical trial trends, be sure to watch the Spotlight feature with Dr. Vidal and Dr Morrison.
This article was created in collaboration with the sponsoring company and the Xtalks editorial team.