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Articles

Will the new pediatric and ethics initiatives receive enough funding to make them truly work?

Pediatrics and Ethics Predominating in 2005

A challenge that intersects the global community

Communication and Clinical Trials

Navigating a complex drug development landscape, sponsors must rethink their approach to risk in study design and execution.

Clinical Trials as a Competitive Edge: Strategic Considerations 

Overcoming the illusion of knowledge in clinical research can enhance decision-making, training, and project success—helping ensure trials run smoothly and effectively.

What We Think We Know: How Overconfidence Derails Clinical Trials

An increase in protocol complexity is paving the way for machine learning models to optimize trial design.

Clinical Trial Failure: A Data-Driven Perspective on Risk Mitigation

The future of clinical trial design involves incorporating the patient experience into strategic asset planning.

Tapping the Patient Voice as a Strategic Differentiator

Anticipating divestment, reallocation of investments, and retrenchment.

The Hard Truth About Rare Disease and Gene Therapy Drug Development

In this video interview, Matt Robinson, PhD, senior director, oncology research program, National Comprehensive Cancer Network (NCCN), shares his perspective on how clinical operations teams can evolve through patient-centered trial design, multi-disciplinary support, and enhanced education to address persistent barriers in cancer trial enrollment over the next five years.

How Clinical Operations Teams Are Adapting to Oncology Trial Challenges

, Matt Robinson, PhD, senior director, oncology research program, National Comprehensive Cancer Network (NCCN), discussed the current state and challenges of cancer patient participation in clinical trials. Barriers to participation can include lack of understanding, economic burdens, and implicit biases. Robinson highlighted solutions such as increased education, legislation like the Clinical Trials Modernization Act, and engaging patients early in trial design. He also touched on the use of technology, such as biomarker testing and wearable devices, which can also enhance recruitment and reduce burdens.

ACT: How do you envision the role of clinical operations evolving to meet the challenges of cancer trial enrollment in the next five years?

I think we've discussed a number of different areas in which clinical operations can work or evolve to meet these challenges. I think recognition or incorporation of the patient voice is going to be important. As I said, companies are already starting to do that, ensuring measurements are meaningful to patients, getting patients perspectives on what they're willing to put up with. I think increased participation by navigators and multi-disciplinary teams is going to be important. As I've said, trials are complex, even having clinicians and scientists keep up with the changing landscape is difficult. Expecting patients to be able to do that, I don't think is fair. Having teams that are able to help them navigate that complex system is going to be really important, and then continued education through plain language resources is also a way that I think we're going to evolve.

Videos

In this video interview, Krinx Kong, chief commercial officer, Cognivia, discusses how trial design and timelines could be impacted by a potential mandate for placebo use across all vaccine trials.

How Vaccine Trials Could be Affected by Potential Policy Changes

, Krinx Kong, chief commercial officer, Cognivia, discussed the potential impacts of policy shifts on vaccine trial design and patient perceptions. Stricter regulations could increase trial complexity, affecting recruitment and retention. Kong emphasized behavioral insights as crucial to understanding patient motivation and mitigating dropouts. Looking forward with the potential for policy changes in vaccine trials, clinical operations professionals must focus on flexibility, behavioral visibility, and effective communication to ensure success.

ACT: US Health and Human Services Secretary Robert F. Kennedy Jr. recently 

 on supporting placebo-controlled trials for all new vaccines. How could a potential policy shift in this area affect trial design and timelines?

First, it's important to clarify that placebo-controlled trials are already the gold standard in vaccine development, and are required by both the FDA and EMA when no approved alternatives exist. Therefore, the notion that these aren't already in use is misleading, or at least lacks context. Now, if a policy shift were to mandate placebo use in all vaccine trials, even where active comparators might be more appropriate, we'd likely see significant impacts on both design complexity and timelines. First, ethics boards may push back. If an effective vaccine already exists, withholding it in favor of placebo could be viewed as unethical. This leads to delays, extra review cycles, and potential public concern. Next, placebo-controlled designs often require longer follow ups, which can prolong timelines by months or even years. Lastly, these designs are frequently more difficult to recruit for, patients and parents may be reluctant to participate if there's a genuine chance of receiving no active protection. If policy does shift, having tools that can adapt trial design without compromising data integrity or patient trust will be essential.

In this video interview, C.K. Wang, MD, chief medical officer, COTA, highlights cost and treatment sequencing as some of oncology’s largest hurdles. 

Challenges in Bringing Effective Therapies to Cancer Patients

, C.K. Wang, MD, chief medical officer, COTA, discussed a recent 

 he led on leveraging real-world data (RWD) to improve oncology reporting. Additionally, Wang highlighted recent trends and challenges in the oncology research space involving the use of RWD as well as real-world evidence (RWE).

ACT: As an oncologist, what are some of your greatest challenges in bringing the most effective therapies to patients?

This is an excellent question. There are definitely many challenges that oncologists face day to day when it comes to treating cancer patients, but I would say that two come to mind, as we speak. First and foremost is cost and financial toxicity. Now I will say that this has been a constant through time, but it has definitely become a larger issue with all of our novel therapies. As I think we all know, the majority of these novel therapies are very expensive. This cost is almost always passed on to the patient, and in more cases than not, these costs are just too high for most.

I would say that a second challenge, which has resulted from the rapid explosion of the novel therapies that we see in oncology is the understanding of what is the best sequence to use these new treatments. Our clinical trials are really not the best at helping us with this question as they are conducted only in very specific treatment settings that the sponsor is looking at, and many times they're even compared to therapies that are no longer considered to be the standard of care in that field. Now I will say though that this problem, it is a good problem to have, because in a sense, it shows, and it is the direct result of the vast number of treatments that we have in the field, but to be honest, this is a scenario where too much of a good thing too soon, or in too short of a time period can be a true challenge when it comes to translating all that into real-world everyday clinical practice.

In this video interview, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discusses the most prominent challenges he is seeing in the space right now including competing research priorities and finding endpoints.

Unique Challenges in Rare Disease Research

, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discussed the unique challenges in rare disease R&D, including competing priorities among researchers, difficulty in diagnosing and treating patients, and defining endpoints due to varying symptoms. Ansel also touched on the potential of technology and artificial intelligence to address some of these challenges and improve rare disease research.

ACT: What are some unique R&D challenges to the rare disease space that the industry is currently facing?

It's a great question, Andy. There's a lot of challenges that the rare disease space is facing right now, differences in competing priorities, whether that's between researchers, advocacy organizations, biotech companies, and even regulators, because rare disease is difficult to diagnose and treat these patients, there's sometimes differences in how do we do that? How do we handle their care? You can imagine the downstream effect that has and every other aspect of a patient's life with a rare disease. Also, finding endpoints is becoming a challenge. It has always been a challenge. A lot of the rare diseases we treat today don't have the same symptoms. We don't see the same severity of symptoms in all patients, so when you go and create a clinical program to prove that your drug is efficacious or effective and safe, regulators really look for defining data, defining criteria that prove that your drug, of course, works, and that can be really challenging when your patients have a spectrum of symptoms that go across every organ. It's hard to find a measurable change and maybe one symptom that not everyone in the patient population experiences, so I would always say that endpoints are particularly challenging in rare disease. Probably last but not least is funding. Rare is difficult because, collectively, rare is not rare, but as you start to narrow down the rare umbrella into very precision-based medicine, or grouping subpatient populations by genotype, your numbers go from quite large to very, very small and very narrow, so as you start to cut the pie, if you will, of funding to each of those groups, there's only so much to go around. I think rare disease is always faced with financial restrictions and considerations when it comes to R&D.

In this video interview, Thierry Escudier, portfolio lead, Pistoia Alliance, discusses how an emphasis on patient centricity can encourage more sustainable trial designs. 

Sustainability by Design

, Theirry Escudier, Portfolio Lead, Pistoia Alliance, discussed the alliance’s newest initiative to assess the carbon footprint of clinical trials. The initiative aims to answer whether decentralized clinical trials positively or negatively impact carbon footprints. Escudier also discussed how clinical research stakeholders are slowly beginning to adopt more sustainability practices, focusing on reducing waste and costs.

ACT: In terms of sustainability, how have clinical research stakeholders been responding to challenges such as operational costs?

 If you start to think differently and looking to what do I need, really, in my trial? Once again, it's also part of the overall new concept, but it's not anymore really in the EU now on the patient centricity and patient engagement is, if we think, in the place of the patient, what matters for them, and it's one of the reasons we have so much concern in terms of patient recruitment. It's also well known that in most of the trials there is difficulty to recruit patients and/or to keep them in a trial, because sometimes there is too much burden. If you start to think differently, thinking more on the on the patient side, then you also start to think on the way you can design your trial. Maybe you can reduce some of the visits. Do you really need to have all those patient visits in the flow chart? Like maybe we can keep the visit, but we could have, I would say, a phone call, instead of asking the patient to cross the city, to go to the hospital, all those things, and in particular, with the technology, because now you could even have those Zoom calls, Teams calls that you can even put in place. You could also have all those new tools that you can give to the patient on those apps that will remind what they have to do, when they have to come, what type of data they have to record, so there is maybe some new ways of thinking on designing your trial, so that's what we call now sustainability by design. Instead of doing what we have been doing forever, can we think differently in maybe designing the trial somewhat differently, of course, keeping the most important, that is to say, ensuring that the safety of the patient and being able to assess the efficacy, because that's the rationale of doing those clinical trials, but maybe if you do that a little bit differently, you could act on two sides, reducing the cost in a way, reducing the carbon footprint, and at the end, it's a win-win situation for everyone.

Challenges of Advancing Innovation in Clinical Trials

ACT: You were a part of panel discussion on start-up companies. Can you briefly discuss what the panel entailed?

A few innovative startup companies were selected to share their insights on advancing innovation in clinical development and ensuring that pharma sponsors and CROs can effectively leverage these solutions. Additionally, there is a growing need to adopt a more patient-centric approach in clinical trials.

ACT: Startups often face challenges in gaining traction. What has been the biggest hurdle for One2Treat in advancing clinical trial innovation, and how are you overcoming it?

Over the past two years, the FDA and EMA have provided valuable guidance, particularly in patient-focused drug development and optimal project management. While this guidance is helping pharma companies make progress, there remains some uncertainty about how regulators can further support the adoption of concrete methodologies.

At times, sponsors may be hesitant to embrace innovative opportunities, which underscores the need for clearer frameworks and greater confidence in emerging solutions.

In an interview with Applied Clinical Trials Associate Editor Don Tracy, Sebastien Coppe, CEO, One2Treat, talks challenges faced by startups in advancing clinical trial innovation. 

SCOPE Summit 2025: Sebastien Coppe Discusses Advancing Innovation in Clinical Trials

Trial Efficiency

ACT: What types of partnerships—whether with CROs, technology vendors, or patient advocacy groups—do you see as crucial for improving trial efficiency and success?

From my point of view, we principally work with site investigators and patient representatives. At One2Treat, we give tools for sponsors to interact with patient representative groups and site investigators, so they can get feedback on the best way to assess their treatment efficacy. By doing that, we think that it gives more value to the treatment that is being assessed, because now you can really measure what truly matters to these stakeholders.

One2Treat s attending SCOPE to explore innovations in clinical trial operations, with a strong focus on patient-centric trial design. They highlight the event’s diverse range of participants, including companies, vendors, startups, and sponsors, all contributing to discussions on trial advancements.

While decentralized trials, real-world data integration, and AI-driven protocols are major industry trends, the speaker is primarily interested in how patient voices can be effectively integrated into trial design. They note that while the FDA has recently issued guidance on patient inclusion, it lacks detailed implementation strategies. Conferences like SCOPE provide a platform to discuss concrete methods for incorporating patient perspectives into trial strategies.

Regarding partnerships, Salvaggio emphasizes the importance of collaboration with site investigators and patient representatives. These stakeholders play a crucial role in ensuring that trials are designed with patient needs and experiences in mind, ultimately improving trial success and patient engagement.

In an interview with Applied Clinical Trials Associate Editor Don Tracy, Samuel Salvaggio Senior Trial Design Lead, One2Treat, discusses partnerships that remain vital for guaranteeing success in clinical trial efficiency. 

Latest Conference Coverage

SCOPE Summit 2025: Samuel Salvaggio Discusses Crucial Partnerships for Trial Efficiency

One2Treat Trial Design

ACT: What are your primary objectives for attending SCOPE 2025, and what insights are you hoping to gain regarding trial design innovations?

Like most people coming to SCOPE, we came here to see what's driving innovations in clinical trials and operations. At SCOPE, you have hundreds of companies, vendors, and clinical startups like us, and they all present in different tracks that are of interest to us. Specifically, I’m most interested in the patient centric trial design track.

ACT: Are there any specific trends in trial design, such as decentralized trials, real-world data integration, or AI-driven protocols that you anticipate being major discussion points?

Those are all very interesting tracks that are well presented here. But as I mentioned, I'm more interested in patient centric trial design and what it entails. While those methods are used in patient centric trial design, they are not the only one that are being used. The FDA recently promoted regulations and guidance on how to include patients in clinical trial development, but they don't really say how to do it. That’s why we come to these kinds of conferences, to promote new, concrete ways on how can you include patient voices into clinical trial design and strategy.

In an interview with Applied Clinical Trials Associate Editor Don Tracy, Samuel Salvaggio, Senior Trial Design Lead, One2Treat, discusses the company's main goals for attending the SCOPE Summit, including patient centric trial design. 

SCOPE Summit 2025: Samuel Salvaggio Discusses One2Treat's Primary Objectives for Attending SCOPE

CluePoints Risk Detection

ACT: What are CluePoints’ key objectives for participating in SCOPE 2025, and how do you see the conference shaping the conversation around risk-based quality management?

I think the very first thing is we're delighted to be here, because there's a need to validate the conversation. 12 years ago, a word that didn’t exist in our vocabulary—nor did AI, to be honest. Now it’s front and center. We’re here really to continue that dialogue, to share and expand, but also transition from the potential to the reality. This is what we're doing today, this is what we’re going to do tomorrow. I think that’s the exciting thing about meeting like this, it’s not a false environment, it's a genuine collaborative environment with so many different conversations, and it's nice to be at the center of it.

ACT: How has CluePoints’ approach to risk detection and mitigation evolved in response to the increasing complexity of global clinical trials?

We’re about to live through the ICH GCP E6(R3) release. In fact, we’re hosting a meeting with the FDA in just about five weeks’ time to go over it. It's going to completely change our approach. I actually think one of the biggest outcomes will be to challenge the use of that word risk. It's a word that confuses, and I wonder whether we might move into more of an informed or a data-driven era where risk isn't a negative word, it's a really positive phrase. But those guidelines are going to change everything from chain of custody to the approach we take, to just embedding a transformational, modern methodology into every decision we make.

At SCOPE this year, the key objective is to advance the conversation around risk-based quality management (RBQM), transitioning it from a theoretical concept to an actionable reality. The conference provides a collaborative platform to discuss the evolution of RBQM, particularly in light of emerging technologies like AI, which were not even part of the industry's vocabulary a decade ago. The goal is to validate and expand discussions, ensuring that RBQM is implemented effectively across clinical trials.

A significant regulatory shift impacting RBQM is the imminent release of the updated ICH GCP E6(R3) guidelines. These changes are expected to transform how sponsors approach RBQM, potentially even redefining the concept of “risk” itself. Rather than being viewed as a negative factor, risk management could shift toward a more data-driven, informed decision-making process. These guidelines will influence aspects such as chain of custody and the integration of modern methodologies into trial management.

The future of RBQM is increasingly centered around data as a comprehensive ecosystem rather than isolated data points. Industry stakeholders are prioritizing different drivers—cost, time, resources, or quality—in their approach to RBQM. While all four factors are important, organizations must determine their primary focus to optimize decision-making. The industry is working toward a model where these priorities can be balanced more effectively, ensuring that clinical trials remain patient-centric while also meeting regulatory and operational goals.

Ultimately, SCOPE serves as a critical venue for shaping the future of RBQM, fostering discussions that drive innovation and efficiency in clinical research.

In an interview with Applied Clinical Trials Associate Editor Don Tracy, Richard Young, chief strategy officer, CluePoints, discusses goals of the SCOPE Summit and CluePoints' evolving approach to risk detection. 

SCOPE Summit 2025: Richard Young on CluePoints' Approach to Risk Detection in Clinical Trials

In this video interview, Michael Andreini, president and CEO of the Multiple Myeloma Research Foundation, highlights the design of Horizon, an adaptative platform trial for the treatment of multiple myeloma. 

Multiple Myeloma Research Foundation’s Horizon Clinical Trial Program

, Michael Andreini, president and CEO of the Multiple Myeloma Research Foundation (MMRF), discussed challenges in multiple myeloma clinical trials, including inefficiency, lack of focus on critical patient questions, and underrepresentation of diverse patient populations. Andreini also highlighted the Horizon clinical trial and how MMRF is working to address unmet need in the myeloma research space.

ACT: Could you tell us about the design of the Horizon clinical trial?

First of all, conducting clinical trials at the MMRF is not new to us. We have a consortium of 14 leading centers in the United States, have conducted nearly 100 clinical trials to date, and Horizon is really the next evolution of our clinical research strategy. What Horizon is, is an adaptive platform trial for patients with relapsed and refractory myeloma, and because it's a platform trial, we can test multiple treatment strategies at once under a single master protocol against a common control. Because this trial is also adaptive in its design, it's evergreen in nature, so we can more efficiently then add new treatment arms to the study as new hypotheses are developed, we can close treatment arms if we feel like this treatment is not serving patients in the best way anymore. Also, we can add new controls to the platform as the standards of care might change over time. Really, the results of this are that it's a much more efficient clinical trial design that can start up more quickly, that can enroll patients more quickly, and can really evolve with the needs of patients and the research community over time. The other thing is that we've built into the design of the trial, a very robust strategy to ensure that we enroll representative patient population as part of the trial, so we've nominated an independent diversity officer to advise on strategy and execution as part of the trial. We've stated mandates in the protocol for representation from specific racial and ethnic groups. We've broadened the eligibility criteria as much as possible. We're including over 30 satellite community sites in diverse locations to ensure we have a diverse catchment area for the trial. Then also, we've built in financial assistance for patients as part of this trial, through a part of our family reach to ensure that we lower as many financial barriers as possible for participation, to cover things like travel costs, lost work days, child care, food, which are real things that can really impact patients abilities to enroll and participate in a clinical trial.

Trial Design

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