CRO/Sponsor

Clinical Trial Supply Chain

Decentralized Clinical Trials

Early Phase Development

Investigative Sites

Protocol Design

Risk-Based Monitoring

Study Start-Up

Trial Design

Clinical Operations & Strategy

Diversity Action Plans

Industry Initiatives

Recruitment

Analytics

Electronic Data Capture

Metrics & Benchmarks

Real World Evidence

Data Management

CRAACO

Enrollment

Integrated Research

Patient Participation

Institutional Review Board & Ethics Committee

Regulatory

Artificial Intelligence/Machine Learning

eClinical

mHealth

Technology

News

Academy

Publications

All Publications

E-Books

Media

Editorial Podcasts

Editorial Videos

Peer Exchange

Practice Academy

Conferences

Conference Coverage

Conference Listing

Columns

All Columns

A Closing Thought

Clinical Trial Insights

View from Brussels

View from Washington

Resources

Advertise

Blogs

Events

Front & Center

Partner Perspectives

White Papers

Webcasts

Subscribe

Latest solutions, case studies, interviews, practical articles for clinical trials operations, data management, technology, investigative sites and services.

About

Contact Us

Editorial Contacts

Do Not Sell My Personal Information

Privacy Policy

Terms and Conditions

Applied Clinical Trials Online

LinkedIN

Instagram

Facebook

Youtube

Soundcloud

Threads

Spotlight

DE&I

Topic

1rem

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

COVID-19 not only advanced scientific boundaries, but also transformed research methodologies and accelerated adaptive clinical trial design.

COVID-19 Five Years Later: The Impact on Drug Discovery 

Teams must work together with technology solutions and optimize integration to unlock their full potential.

Leveraging Technology for Efficient Trial Design

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.

Videos

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.

In this video interview, Dipanwita Das, CEO & co-founder at Sorcero, highlights how artificial intelligence, real-time monitoring, and historical data can aid in optimizing trial design. 

Technology’s Role in Trial Design Optimization

, Dipanwita Das, CEO & co-founder at Sorcero, discussed advancements and future trends in clinical trials. Key challenges include high costs, long timelines, lack of diversity, and rigid protocols. Das highlighted technological advancements such as AI, real-time monitoring, and historical data analysis, which are all expected to optimize trial design and patient recruitment in 2025 and beyond.

ACT: What types of technology do you think clinical research experts can benefit from the most in their goal of improving patient outcomes?

 Technology, of course, is a huge role in transforming all of 

, artificial intelligence (AI) certainly, but I think in the next one or two years, trial design optimization is where I'm excited to see the use of more historical data at scale, particularly to optimize inclusion/exclusion criteria, use of AI, and better data science and EHR analysis for patient recruitment, making sure they're actually covering all of the patient populations that need to be represented. Better real-time monitoring: real-time monitoring is just really, really difficult right now, so making sure that there is a certain degree of automated detection of data quality issues, etc., and of course, using AI for literature review protocol writing, as well as automating documentation. In the medium to long term, there are lots of other spaces in which AI and technology overall will play a huge role, from synthetic control arms to adaptive trial designs as well as digital biomarker developments, or even the idea of in silico trials, so there's a lot of exciting things coming up, but I think in the next one to two years, optimizing design, recruiting patients, and real-time monitoring is going to go a long way.

In this video interview, Sujay Jadhav, CEO of Verana Health, talks external control arms and how they can increase study feasibility and inclusivity. 

Accelerating Drug Development With External Control Arms

, Sujay Jadhav, CEO of Verana Health, discussed potential advancements the clinical research industry could see in 2025. Jadhav highlighted how unstructured data like physician notes and imaging data can be leveraged to improve disease identification and progression tracking. He also talked synthetic control arms and how they can replace expensive non-treatment arms, enhancing trial efficiency.

ACT: How can external control arms (ECAs) accelerate drug development?

There's a number of areas in terms of how external control arms can accelerate drug development. As I mentioned, the biggest advantage is when you're looking at the non-treatment arms and then the costs associated with recruiting patients is pretty sizable, and that's really where you can plug in real-world data to help be a better proxy, or help bolster that part to allow you to compare and contrast. Some of the areas where control arms can accelerate drug development, one is addressing ethical dilemmas. When you look at ECAs, they eliminate the need to randomize patients into non-interventional groups, particularly in studies where a placebo or a standard of care may be ethically questionable.

Another area is utilizing existing data, so what we find is in rare diseases with small patient populations, external control arms allow researchers to use historical, real-world data to make studies more feasible and more inclusive. As you can imagine, without external control arms, going through and recruiting enough patients, which can be a good comparison there as well in rare disease areas, is very, very difficult to do, and that's where real-world data can shine quite well.

There's a quality element in terms of how external control arms can help, so providing high quality insights by creating reliable comparison groups that can really help reduce the reliance on traditional non-interventional groups, or maintaining robust scientific validity there as well. What we are seeing, again, is the theme of going after the unstructured data to help improve these high-quality insights. With technology we have today with helping ingest data in a more efficient fashion than historically has been as well, that allows you to bring in a lot of this unstructured data and provide insights in a very timely fashion, which historically has been very difficult to do. Fundamentally, I think when you look at external control arms, they accelerate the overall therapy development, and these methods help streamline the clinical trial process, enabling faster development and effective therapies without actually compromising scientific integrity.

Trial Design

Latest News