In 2024, many authors shared their insights on clinical trials in our journal. Their articles published with us have received very well feedback in the field and stimulate a lot of discussions and new insights among the peers.

Hereby, we would like to highlight some of our outstanding authors who have been making immense efforts in their research fields, with a brief interview of their unique perspectives and insightful views as authors.

Outstanding Authors (2024)

Toyoaki Hida, Lung Cancer Center, Central Japan International Medical Center, Japan

Zhonghua Sun, Curtin Medical School, Curtin University, Australia

Savvas Lampridis, Imperial College London, UK

Joseph Dux, Laniado Hospital, Israel

Takehiro Uemura, Graduate School of Medical Sciences, Nagoya City University, Japan

Hirohisa Kato, Okitama Public General Hospital, Japan

Igor Gómez-Randulfe, The Christie NHS Foundation Trust, UK

Sebastian Stintzing, Charité - Universitaetsmedizin Berlin, Germany

Christine Pocha, University of South Dakota, USA

Jiaxin Niu, Banner MD Anderson Cancer Center, USA

Outstanding Author

Toyoaki Hida

Dr. Toyoaki Hida, MD, PhD, is the Director of Lung Cancer Center at Central Japan International Medical Center, Japan. He spent 2 years in the Biomarkers and Prevention Research Branch of the National Cancer Institute, Maryland. Thereafter, he joined Aichi Cancer Center’s Thoracic Oncology unit in 1996. At Aichi Cancer Center, he was Director of Thoracic Oncology from 2005, and vice president from 2018.

His primary interests are translational medicine in the field of non-small cell lung cancer. In the first period of his career, his work focused on the studies on lung cancer pathogenesis, and then on the personalized cancer therapy using molecular target agents, immune-checkpoint inhibitors, and antibody-drug conjugates.

Dr. Hida emphasized that clinical trials play a crucial role in determining the clinical value of expected treatments. Good clinical trials may need the key elements such as appropriate trial population, adequate sample size, adherence to allocated trial intervention, trustworthy capture of data, monitoring emerging information on benefits and toxicities, thorough follow-up, and appropriate statistical analysis.

When discussing the interpretation of clinical trial results, Dr. Hida provided an in-depth example from the field of non-small cell lung cancer (NSCLC). Durvalumab sequentially administered after platinum-based CRT is the established, global standard of care for the treatment of unresectable, stage III NSCLC based on the results of the PACIFIC trial. The PACIFIC-2 trial was initiated to evaluate concurrent durvalumab administration with CRT, with the aim of addressing patients who progress or discontinue treatment during CRT and are therefore ineligible for the PACIFIC regimen. The PACIFIC-2 trial for durvalumab concurrently administered with CRT did not achieve statistical significance for the primary endpoint of PFS versus CRT alone. While the PACIFIC-2 trial, like the NRG-LUOO5 study of atezolizumab, did not show favorable results, the PACIFIC regimen remains the standard of care for patients with unresectable, stage III NSCLC. These outcomes should guide future research on combining immunotherapy with radiation therapy.

Dr. Hida also introduced some aspects of clinical trials that he is currently following. He mentioned that investigators frequently use analyses of subgroups to extract as much information as possible. These analyses may provide useful information. However, subgroup analyses sometimes may lead to overstated and misleading results. Randomized trials on drugs with positive results obtained more citations than those with neutral results.

In Dr. Hida's view, one aspect of clinical trials that has received insufficient attention is the analysis of late effects of adverse events (such as adverse cardiovascular effects) associated with immune checkpoint inhibitors, molecular targeted agents, or anticancer agents. A prospective study of long-term serial cardiac monitoring after ICI therapy, molecular targeted therapy, or anticancer therapy may be needed.

Conducting clinical trials is not without its challenges, and Dr. Hida has also encountered several in his work. One of key barriers that impede progress in clinical research was the lack of financial resources. Dr. Hida faced a challenge when recruiting participants within the required timeline, and to address this challenge, he proactively established hospital networks to enter potential participants. With this strategy, he successfully enrolled the required number of participants within the scheduled timeline and planned funding limits. 

(by Ronnie Wu)

Zhonghua Sun

Dr. Sun is a John Curtin Distinguished Professor in medical imaging at Curtin Medical School, Curtin University, Australia. His research interests include 3D image processing and visualization in cardiovascular disease, mainly focusing on the use of 3D printing, virtual reality and mixed reality, and AI technologies in enhancing the diagnosis of coronary artery disease. His research projects involve the development of Generative AI tools in improving diagnostic assessment of calcified coronary plaques, and the development of personalized 3D printed models for medical education and clinical training. Learn more about him here.

A good clinical trial design, according to Dr. Sun, includes innovative ideas, feasible study design, and reasonable sample size that allows for robust conclusions. This foundation is essential for translating positive results into clinical practice, which directly benefits patient care and outcomes. Instead, negative results often stem from challenges in recruiting patients and following up on clinical outcomes, which can hinder the progress of a trial.

Dr. Sun is actively engaged in the field of clinical trials, with a particular focus on the innovative use of AI models in assisting the diagnosis of coronary artery disease This cutting-edge application of artificial intelligence has the potential to revolutionize how medical professionals approach cardiovascular diagnostics. However, Dr Sun also noted that some areas have not received enough attention in clinical trials, such as 3D printing, virtual reality and mixed reality, as well as AI on patient care and management.

Clinical trials are not without challenges, and Dr. Sun also encountered them during clinical trials, including ethics approval from HREC, and clinical support. “Well prepared for these challenges with sufficient time allocated to work on them,” says Dr. Sun.

(By Ronnie Wu)

Savvas Lampridis

Dr. Savvas Lampridis is a cardiothoracic surgeon at the 424 General Military Hospital in Greece, where he serves as a Major in the Army Medical Corps, and is also a Researcher at Imperial College London, UK. His clinical and research interests include minimally invasive surgery, thoracic trauma, translational cardiovascular medicine, surgical education, and health policy. He trained in Greece and the UK, including at the Royal Brompton Hospital and University College London Hospitals, and worked at Guy’s Hospital and Hammersmith Hospital in London. Dr. Lampridis completed master’s degrees in Translational Cardiovascular Medicine and Health Policy and Planning, both with Distinction. He is a member of the Royal College of Surgeons of Edinburgh and the European Association for Cardio-Thoracic Surgery, where he serves on the Solitary Pulmonary Nodules Task Force. He holds positions on editorial boards of several peer-reviewed journals, has guest-edited multiple special issues, and has reviewed more than 1,000 manuscripts for over 200 biomedical journals. Dr Lampridis is also active in medical education, authoring teaching materials and organizing surgical courses. Learn more about him here and on ORCID, and connect with him on LinkedIn.

According to Dr. Lampridis, it's crucial to understand the basic components of a well-designed clinical trial. Dr. Lampridis thinks it starts with being very clear about what question the researchers are trying to answer and who exactly should be included in the study. Proper randomization and the right sample size ensure that the results are genuinely meaningful. When possible, blinding helps prevent bias from creeping in, and strict adherence to ethical standards and regulations keeps patient welfare at the forefront. Ultimately, the goal is to create a setup where the data generated can be trusted—both by the researchers and by the clinicians who may use the results to guide patient care.

In terms of the positive and negative results of a clinical trial, Dr. Lampridis thinks it’s important to recognize that both positive and negative findings have real value. Positive results, such as a lung cancer therapy shown to improve survival in a randomized trial, can reshape clinical guidelines and influence patient care. At the same time, negative results are not failures; instead, they tell us what doesn’t work. For example, if a much-anticipated treatment shows no meaningful improvement, that result can save time, resources, and patients from unnecessary interventions. In other words, both positive and negative data points help refine our understanding of what genuinely benefits patients, ensuring that future research efforts are focused and productive.

In addition, Dr. Lampridis shares some of the clinical trials that he is following. He has been following several key aspects of clinical trials, especially those related to surgical approaches and perioperative systemic therapies for lung cancer. For example, he pays close attention to how trials incorporate new endpoints—like patient-reported outcomes—and how they determine who qualifies for more limited resections, such as segmentectomy or wedge resection, instead of standard lobectomy. Dr. Lampridis is also interested in trials exploring the integration of immunotherapy before and after surgery, as well as how these studies are adapting to evolving regulatory guidelines and ethical standards. By keeping track of these various elements, Dr. Lampridis gains a clearer picture of how clinical trials can influence real-world care.

Nevertheless, Dr. Lampridis points out that not all aspects of clinical trials receive the attention they deserve. A few examples include patient experience and long-term quality-of-life measures. Researchers spend a lot of time focusing on short-term clinical endpoints, but understanding how treatments affect daily life, mental well-being, and overall health over the long haul can be just as important. Also, issues like patient diversity and broader real-world applicability sometimes feel underexplored. Making sure trials incorporate these elements from the start could help generate results that speak more directly to the varied patient populations we actually treat.

Conducting clinical trials is not without its challenges, and Dr. Lampridis has also encountered several in his work. “One of the biggest challenges is ensuring consistent patient enrollment, especially when dealing with complex eligibility criteria or rare conditions. To address this, I’ve found it helpful to work closely with referring physicians, invest time in clear patient communication, and, when possible, collaborate with multiple centers. Another issue can be protocol adherence—making sure everyone involved follows the study plan precisely. Regular training sessions, open communication channels, and having a dedicated coordinator can keep everyone aligned. These measures help maintain the integrity of the trial and ultimately lead to more reliable results”, says Dr. Lampridis.

(By Ronnie Wu)

Joseph Dux

Dr. Joseph Dux specializes in breast surgery and breast cancer patient care, with a particular focus on oncoplastic surgery and tailored surgical approaches for patients. His clinical expertise spans all aspects of breast cancer care, including surgery and collaboration with medical and radiation oncology teams to provide comprehensive, patient-centered treatments. On the research front, Dr. Dux is dedicated to improving diagnostic imaging and advancing methodologies to enhance the precision and effectiveness of breast cancer diagnostics and therapies. He is also actively pioneering innovations in breast cancer surgery and exploring novel surgical approaches to improve outcomes for a broad range of patients. Learn more about him on LinkedIn.

A good clinical trial, according to Dr. Dux, starts with identifying the real, practical questions that clinicians face every day. These questions should be relevant and address challenges that impact patient care. It’s also crucial to include a diverse patient population, covering all ages and races. For instance, breast cancer studies often focus on older-postmenopausal patients, which can leave younger populations underrepresented. A more inclusive approach ensures that the findings apply broadly and benefit everyone.

In evaluating the positive and negative results of a clinical trial, Dr. Dux thinks both positive and negative results are valuable. Positive results are encouraging, but it’s important to analyze them critically to fully understand their implications and ensure they are applied appropriately. For example, the recent publication in the New England Journal of Medicine about avoiding lymph node surgery in breast cancer patients offers promising insights, but it requires careful consideration before being widely adopted. Negative results, on the other hand, often lead to progress by showing us what doesn’t work and guiding us toward better solutions. A good example is the Alliance 1071 trial, which revealed higher false-negative rates in certain lymph node surgeries. While disappointing at first, these findings led to the development of targeted axillary surgery, which has greatly improved patient care.

Furthermore, Dr. Dux introduced some clinical trials he is following. He is currently involved in clinical trials that aim to improve breast cancer care, particularly through tailored surgical approaches and new technologies. One area he’s focusing on is how artificial intelligence can enhance mammogram interpretation by integrating patient-specific factors. This kind of innovation has the potential to make diagnostics more accurate and personalized, bridging the gap between technology and clinical care.

However, Dr. Dux points out that some aspects of clinical trials are not receiving the attention they deserve. One area that needs more focus is the integration of new technologies, like artificial intelligence, into clinical trials. AI can help streamline trial processes and improve data analysis, but it’s still underutilized. Another challenge is demonstrating meaningful outcomes in early-stage breast cancer trials. With high survival rates and low recurrence risks, it’s hard to show significant differences in treatment approaches without huge patient populations, which are often difficult to recruit. We need to explore alternative trial designs and statistical methods to address this issue and still get valuable insights.

Conducting clinical trials is not without its challenges. “Recruiting both patients and staff for clinical trials has been a significant challenge. With the heavy clinical workload, it can be hard to find the time and resources to dedicate to research. Another difficulty is securing enough support for study design and execution, like getting help from statisticians or research assistants. What has helped us overcome these challenges is the enthusiasm of the team. Everyone shares a strong commitment to improving patient care, and that passion motivates us to push through even when things get tough,” says Dr. Dux.

(By Ronnie Wu)

Takehiro Uemura

Dr. Takehiro Uemura, a lecturer in the Department of Respiratory Medicine, Allergy, and Clinical Immunology at the Graduate School of Medical Sciences, Nagoya City University, Japan. Dr. Uemura earned his M.D. in 2003 and his Ph.D. in 2010, both from Nagoya City University. His research primarily focuses on drug sensitivity and resistance in lung cancer therapy. Additionally, he is actively engaged in the design, proposal, and participation in clinical trials exploring innovative therapeutic strategies for lung cancer treatment.

A good clinical trial design, as Dr. Uemura points out, should be tailored to address the clinical question relevant to the new therapeutic intervention. It should ensure the reliability, validity, and ethical soundness of the study while enabling the generation of actionable data to inform clinical practice.

In the view of positive and negative outcomes in clinical trials, Dr. Uemura provides some examples. Positive results from clinical trials are pivotal in driving transformative changes in clinical practice. For example, trials demonstrating the efficacy of immune checkpoint inhibitors revolutionized the treatment of advanced lung cancer. However, it is equally important to critically assess such results to ensure their robustness and reproducibility. Negative results, while disappointing, are not without value; they provide an opportunity to investigate the reasons behind the failure and explore modifications to improve the likelihood of success in future studies.

Additionally, Dr. Uemura shares some of the clinical trials that he is following. Currently, he is closely monitoring trials investigating novel approaches in lung cancer treatment, including the use of antibody-drug conjugates (ADCs) and bispecific antibodies. These innovative strategies have the potential to address unmet clinical needs and significantly improve patient outcomes.

However, it is evident that not all aspects of clinical trials receive the attention they deserve. One area that Dr. Uemura believes requires more attention is the integration of real-world evidence (RWE) into clinical trials. While randomized controlled trials (RCTs) remain the gold standard, incorporating RWE can provide complementary insights into the efficacy and safety of interventions in broader, more diverse patient populations. Additionally, the long-term follow-up and quality-of-life assessments in trial design often remain underemphasized, despite their critical importance for patient-centered care.

Conducting clinical trials is not without its challenges. “Proposing new clinical trials can be challenging, particularly when working independently. Collaborating with experienced clinicians and biostatisticians is essential to overcome these hurdles. Their expertise can help refine trial designs, address logistical issues, and ensure statistical rigor, enabling the successful execution of complex studies,” says Dr. Uemura.

(By Ronnie Wu)

Hirohisa Kato

Dr. Hirohisa Kato received his M.D. and Ph.D. degrees from the Faculty of Medicine, Yamagata University, Japan. After his residency in general surgery, cardiovascular surgery, and thoracic surgery, he was an assistant professor of the Department of Surgery at Yamagata University, Faculty of Medicine. He also learned various surgical techniques at the Department of Thoracic Surgery, University of Hospital, Strasbourg, France, with a fellowship from The Japanese Association for Thoracic Surgery. He has worked at the Department of Thoracic Surgery, Okitama Public General Hospital, since June 2020. His research interests include minimally invasive surgery, thoracoscopic sublobar resections, and thoracoscopic segmentectomy. Learn more about him on ResearchGate and LinkedIn.

A good design of clinical trials consists of some key elements. Dr. Kato shared that his team has had various clinical questions while treating patients, although some clinical evidence might have already been confirmed. To him, it is natural that such clinical questions arise, and clinical trials are planned for those questions. Therefore, Dr. Kato thinks that trend, simplicity, and low cost are crucial elements in implementing clinical trials. To apply this trend, it would be necessary to promptly plan an appropriate protocol and gather a number of patients. The planned protocol therefore needs to be simple and clear to promptly conduct the clinical trial. If an appropriate clinical trial can be conducted at a low cost, it is considered to be more feasible.

From Dr. Kato’s perspective, any positive results would be applicable to their routine medical treatments or examinations. He added that even if a clinical trial showed any negative results, the trial can be useful as the data collected may have some potential to bring new insight.

Although Dr. Kato previously conducted a clinical trial regarding long-term outcomes of limited resection for small-sized lung cancer and reported the results last year, he is regrettably not conducting a clinical trial now. However, he is still interested in the clinical trials following the long-term survival of limited resection for small-sized lung cancer and the technical aspects of limited resection for small-sized lung cancer and follows them closely. In particular, recent clinical trials such as JCOG0802/WJOG4067 and CALGB140503 have demonstrated the feasibility of sublobar resection for small-sized lung cancer. It is expected that the long-term outcomes of the patients enrolled in these clinical trials would also be feasible. Due to the surgical trend of changing to the sublobar resection approach for treating lung cancer, Dr. Kato believes that the outcomes and techniques of this approach will be an increasingly important topic of discussion for thoracic surgeons.

When asked about the challenges he has encountered in conducting clinical trials and how he overcame them, Dr. Kato shared, “Before planning any clinical trials, it is most important to set the appropriate criteria for conducting the clinical trial. Furthermore, it is also important for the principal investigator to discuss the main theme and the appropriate method and get a consensus from the co-authors. By evaluating a lot of previous reports and discussing with the co-authors, it would be desired to create some new evidence from the clinical trials.”

(By Ronnie Wu)

Igor Gómez-Randulfe

Dr. Igor Gómez-Randulfe is a medical oncologist and clinical researcher specializing in lung cancer, immunotherapy, and targeted therapies. He completed his specialist training in Medical Oncology at the University Hospital of A Coruña (Galicia) in 2022. He also holds a master's degree in medical Oncology from the Spanish Society of Medical Oncology and has successfully passed the ESMO Examination. In addition to his formal qualifications, Dr. Gómez-Randulfe has pursued further training in clinical trial design, drug development, and lung cancer. He has authored and co-authored peer-reviewed publications and presented at international conferences. He currently serves as a Locum Consultant in Medical Oncology at The Christie NHS Foundation Trust in Manchester, United Kingdom. Learn more about him here.

Dr. Gómez-Randulfe's expertise is not only reflected in his academic and professional achievements but also in his insights into clinical trials. In his view, a robust trial design begins with a well-defined research question supported by a strong scientific rationale. The methodology should include clear objectives, appropriate patient selection criteria, and relevant endpoints. Equally important are randomization and blinding strategies (when feasible) to minimize bias, as well as appropriate post-protocol therapies.

According to him, both positive and negative results from clinical trials are valuable. When a trial is properly designed and conducted, positive results can lead to more effective treatments. For example, in lung cancer, the demonstration of survival benefits from immunotherapy has revolutionized the treatment landscape. However, negative or inconclusive results are equally—if not more—valuable. Publishing these findings is crucial to inform the scientific community, avoid duplicating similar trials, and advance our overall knowledge base. For instance, the negative results from trials that tested adding immunotherapy to chemoradiotherapy at the start of treatment, rather than sequentially, in stage III non–small cell lung cancer (NSCLC) have provided valuable insights and will guide the design of future trials. Both positive and negative outcomes contribute to the growing body of evidence that informs and refines research going forward.

Currently, Dr. Gómez-Randulfe is closely monitoring several clinical trials, with a primary focus on translational and clinical research in thoracic oncology. He is particularly interested in trials exploring the role of immunotherapy in both early-stage and advanced NSCLC. This includes investigating novel immune checkpoint inhibitors, bispecific antibodies, and personalized vaccine approaches. He also pays close attention to studies on minimal residual disease (MRD) monitoring—using circulating tumor DNA (ctDNA) to track tumor evolution over time and identify early signs of relapse. These trials will hopefully provide a more tailored treatment for patients diagnosed with NSCLC, as long as the design and conduct are appropriate.

However, Dr. Gómez-Randulfe notes that not all aspects of clinical trials receive the attention they deserve. One often overlooked aspect when analyzing trial results is the appropriateness of the control arm treatment, especially the post-protocol therapies. If control-arm patients do not receive the best available standard of care during the trial, any perceived superiority of the experimental treatment should be interpreted with caution. In addition, the quality of life (QoL) component and patient-reported outcomes (PROs) remain underemphasized. These factors are critical for truly personalized cancer care: by capturing how patients feel and function throughout treatment, we can better align therapeutic decisions with individual patient goals. Furthermore, more effort is needed to design trials that specifically address underrepresented populations—such as older adults, those with comorbidities, or individuals with rare molecular subtypes—to ensure that findings are broadly applicable in real-world settings.

Conducting clinical trials comes with its own set of challenges. “Recruitment is one of the biggest hurdles, particularly in trials that require tissue biopsies or rapid enrollment of patients with aggressive disease. In advanced NSCLC, timing is paramount, as many patients may progress significantly before they can be enrolled”, Dr. Gómez-Randulfe shares. “To address this, we’ve strengthened communication with referring clinicians, streamlined recruitment procedures, and prioritized efficient patient screening. Another challenge is the complexity of constantly evolving regulatory requirements, which can slow trial activation. By establishing dedicated clinical research teams and leveraging robust electronic platforms, we have expedited approvals and data management, ultimately accelerating the initiation of clinical trials,” he added.

(By Ronnie Wu)

Sebastian Stintzing

Dr. Sebastian Stintzing, MD, is a full Professor of Medicine and Head of the Department of Hematology, Oncology, and Cancer Immunology (CCM) of Charité - Universitaetsmedizin Berlin. His research focuses on biomarkers in gastrointestinal cancer. After getting his medical degree from the Friedrich-Alexander University of Erlangen-Nuremberg, he started at the Department of Gastroenterology at the University Hospital in Erlangen. He specialized in Hematology and Oncology at the Department of Hematology and Oncology at the Ludwig-Maximilians University of Munich (Prof. W. Hiddemann), where he joined the research group “Oncology” (“FIRE-group”) (Prof. V. Heinemann). He received an award from the German Cancer Aid enabling him to work at the USC Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA (Prof H.-J. Lenz).

Dr. Stintzing is member of several national and international cancer associations (ESMO, ASCO, and DGHO). Since 2014, he is member of the S3-guideline committee for colorectal cancer in Germany. Learn more about him here and on X and LinkedIn.

According to Dr. Stintzing, a good design of clinical trials is characterized by key elements such as the clinical relevance of the investigated question, easily met inclusion and exclusion criteria, and endpoints that matter for patients. These elements are so crucial that the opinion of patient advocates needs to be combined with the clinical excellence of the scientific lead of the respective study. These elements not only ensure the success of a trial but also influence how we interpret its outcomes.

To Dt. Stintzing, both positive and negative results of clinical trials hold significant value in the medical field. Statistically positive results of clinical trials need to be discussed in the light of the clinical meaning of the results. While positive results open new treatment possibilities and have the potential to change the way doctors treat patients, negative results play an equally important role. Negative studies are needed to get a deeper understanding of the disease and to stop developments of substances without impact on patients.

In the realm of clinical trials, Dr. Stintzing’s current focus lies on the developments and clinical trials of bispecific antibodies and ADCs (antibody drug conjugates) in gastrointestinal (GI) oncology. He is particularly interested in the evaluation of liquid biopsies in the decision-making process of colorectal cancer (CRC) and gastric cancer (GC). These advancements hold promise for more targeted and effective cancer therapies.

Despite the progress in various aspects of clinical trials, there are still areas that receive insufficient attention. Especially in trials dealing with immune-checkpoint inhibitors, the quest for biomarkers is too unambitious. Tumor specimens have been investigated, but the immune system (e.g. MHC classes) of the treated patient and changes in PBMC (Peripheral Blood Mononuclear Cells) during treatment need to be further analyzed.

One significant challenge Dr. Stintzing has faced is securing funding for investigator-initiated trials (IITs). To overcome this hurdle, he has found that combining financial support from industry partners with funding from competitive agencies is effective. This collaborative approach not only enables the conduct of trials but also allows for thorough biomaterial analysis afterward, ensuring that valuable insights are gleaned from every study.

(By Ronnie Wu, Jin Ye Yeo)

Christine Pocha

Dr. Christine Pocha is an internationally recognized and esteemed transplant hepatologist, researcher, and educator. During medical school and postgraduate training at Friedrich-Schiller-University in Germany, she obtained a PhD degree in Clinical Pharmacology, focusing on drug metabolism in liver disease. Subsequently, she obtained a Master of Public Health in Epidemiology from the University of Massachusetts. After working at various liver transplant centers in the U.S. and around the world, including the University of Berne, Switzerland, and King’s College London, UK, she settled at Avera Medical Center and University Hospital in Sioux Falls, SD, to lead the new liver transplant program. She was recognised as a fellow of the AASLD (American Association for the Study of Liver Diseases). She is a full professor at Sanford School of Medicine, University of South Dakota. Her research and publications focus on clinical trials, liver cancer, metabolic-associated liver disease, and population health. She is a principal site investigator for the newly approved Alfapump to treat ascites. Learn more about her here and on LinkedIn.

According to Dr. Pocha, a good clinical trial includes certain fundamentals, such as a clear and well-defined objective, reliable methods, randomization or best, stratified randomization, blinding, a control group receiving a placebo or standard of care (SOC), credible and accurate data management, and analysis to minimize bias and construct reproducible results.

Normally, clinical trials have two outcomes: positive and negative results. A "positive" result means that the new treatment shows a statistically significant benefit or a positive effect on the primary endpoint being studied compared to placebo. For example, the "IMbrave050" trial is considered a landmark study in hepatocellular cancer (HCC) treatment as it demonstrates an improved survival for high-risk patients who received atezolizumab plus bevacizumab as an adjuvant treatment after surgery. A "negative" result means that there is no significant difference or benefit from the experimental or new treatment. Several trials, including those investigating everolimus or brivanib for HCC treatment, failed to show a significant improvement in overall survival or progression-free survival when compared to the standard of care and, therefore, these trials are referred to as “negative” trials.

Currently, Dr. Pocha closely follows multiple crucial aspects of clinical trials, which are fraught with challenges. Ideally, they should be randomized controlled clinical trials (RCT) and rigorously follow the “Good Clinical Practice” standards. However, they are expensive, time and resource-consuming, and can be burdensome for patients. There are multiple factors that can lead to the failure of clinical trials. For instance, potentially efficacious drugs may still fail if the study design has major flaws, statistical endpoints are poorly chosen, and the study is underpowered with a sample size too small to reject the null hypothesis. Moreover, safety concerns are equally as important as efficacy and may only become apparent when larger populations are being studied, for example, in phase 3 trials or post-marketing.

Dr. Pocha points out that one aspect of clinical trials that receives insufficient attention is publication bias. It is observed that research showing positive results is more likely to be published compared to negative trials as there is a misconception that negative results are less impactful. Publication bias is a consequence of the non-reporting of negative findings or trial results, which leads to a distorted perception of drug efficacy. Reasons for non-reporting of negative findings could be multifactorial and may include concerns about funding or that negative results do not make one famous. This has a negative influence on scientific progress.

In conducting clinical trials, Dr. Pocha has faced several challenges. “During my last clinical trial, the Poseidon Study, enrolment of patients posed some challenges. It is especially important to “toot your horn”, letting your colleagues, institutions, and community know to help with referral of potential study candidates,” Dr. Pocha says.

(By Ronnie Wu, Jin Ye Yeo)

Jiaxin Niu

Dr. Jiaxin Niu, MD, PhD, earned his medical degree at China Medical University and a doctorate degree in molecular pharmacology at the University of Illinois-Chicago. Dr. Niu is triple board - certified in internal medicine, hematology, and medical oncology. He joined Banner MD Anderson Cancer Center (BMDACC) in 2016 and has since been focusing on clinical practice and research in lung, head & neck cancers. He currently serves as co-director of lung cancer program and lead medical oncologist for head and neck cancer program. Dr. Niu has 30 years of experience in both basic and clinical research. His passion is early drug development in oncology. Dr. Niu is well published and was actively involved in the development of several new FDA-approved oncology drugs including Palbociclib, Cemiplimab, Mobocertinib. He is the principal investigator for over 10 ongoing clinical trials at BMDACC.

ACTR: What are the key elements that constitute a good design of clinical trials?

Dr. Niu: A well-designed clinical trial ensures scientifically sound, ethical, and practical execution, and generates reliable evidence. Key elements of good clinical trial design include: (1) clear objectives; (2) right study population; (3) appropriate randomization & blinding; (4) sufficient sample size & power; (5) reliable data collection & safety monitoring. These elements are key to generating robust, reliable results while maintaining patient safety and scientific integrity throughout the study.

ACTR: How do you see the positive and negative results of a clinical trial with examples？

Dr. Niu: A well-conducted clinical trial, whether yielding positive or negative results, raises new questions and drives the advancement of medical knowledge. In lung cancer research, particularly in the development of targeted therapies against epidermal growth factor receptor (EGFR), we have gained invaluable insights from both successes and failures. Among the most illustrative examples are the evolution of anti-EGFR therapy.

In the early 2000s, EGFR inhibitors like gefitinib failed in large, unselected non-small cell lung cancer (NSCLC) trials, such as the ISEL trial. The initial assumption was that all lung cancer patients would benefit from EGFR inhibition, but the results showed no significant survival advantage. This failure led to a pivotal discovery—only patients with specific EGFR mutations (e.g., L858R and exon 19 deletions) responded to these drugs, marking a major shift toward precision medicine.

First-generation EGFR tyrosine kinase inhibitors (TKIs) like gefitinib and erlotinib proved highly effective in these selected patients, leading to their approval and widespread use. However, clinical experience quickly revealed two major challenges: tolerance issues with oral agents and the emergence of resistance mutations, such as T790M. These limitations drove the development of third-generation TKIs, like osimertinib, which effectively target resistant mutations and further improve patient outcomes.

The lessons learned from EGFR-targeted therapies have since been widely applied to other targeted treatments in lung cancer and even in the broader field of solid tumor oncology, shaping the future of precision medicine.

ACTR: Could you please introduce any aspects of clinical trials you are following?

Dr. Niu: In recent years, clinical trial designs have increasingly focused on accelerating the traditionally lengthy process of drug development. Adaptive trial designs have gained popularity due to their flexibility, allowing for modifications based on interim data. This adaptability is particularly valuable in oncology, where the rapid evolution of cancer biology and treatment responses necessitates a more dynamic approach.

Additionally, master protocols, including basket and umbrella trials, are being utilized more frequently. Basket trials evaluate a single drug across multiple tumor types with a common molecular alteration, while umbrella trials assess multiple agents within a single cancer type based on distinct genetic profiles. Both designs have significantly expedited drug development by streamlining the evaluation of targeted therapies.

Another increasingly favored approach is the Seamless Phase II / III design with dose optimization, which allows for continuous patient enrollment and analysis while identifying the optimal therapeutic dose. This approach enhances efficiency and ensures that promising treatments reach patients more quickly.

ACTR: Are there any aspects of clinical trials that you think receive insufficient attention?

Dr. Niu: Most clinical trials fail to adequately address the financial burden on participants. Many patients struggle to afford participation due to travel costs, time off work, or the lack of accessible trial sites. These financial barriers can limit trial enrollment and reduce the diversity of study populations, ultimately affecting the generalizability of trial results.

Additionally, many trials focus primarily on short-term outcomes, often neglecting long-term safety monitoring and post-trial follow-up. This is particularly concerning for highly effective targeted therapies, where patients may remain on treatment for years and develop unique, long-term adverse effects. Establishing comprehensive post-trial monitoring would provide crucial insights into the real-world durability of response and help guide future treatment strategies.

ACTR: What kind of challenges have you encountered in conducting clinical trials and how did you overcome them?

Dr. Niu:Conducting clinical trials presents a wide range of challenges, but overcoming them requires a team-based approach. One of the most frustrating obstacles is poor patient accrual. To address this, I am extremely careful in selecting the right trials for my patient population, ensuring that eligibility criteria align with those most likely to benefit. Additionally, securing financial support for participants and keeping referring physicians well-informed are crucial factors in improving enrollment and trial success.

Another ongoing challenge is maintaining a dedicated, well-trained staff to ensure the efficient execution of trials and the safety of patients. Working closely with our research department, we prioritize specialized training for our staff, ensuring they are highly skilled in the complexities of our field. This commitment to expertise and collaboration is essential for conducting high-quality clinical research.

(By Ronnie Wu)