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1rem

How today’s SSO reduces site burden and spearheads clinical system interoperability.

The Latest Advancements in Single Sign-On Technology

With close collaboration between sponsors, CROs, and regulators, this emerging technology has the potential to greatly improve the lives of patients.

Clinical Strategies to Optimize Software as a Medical Device to Treat Mental Health

Examining the implications of the recent US executive orders concerning diversity, equity, and inclusion pursuits—and the key questions each clinical trial stakeholder must now navigate.




The Impact of DEI Ban On Clinical Research Ecosystem 

Recent actions by the FDA and NIH in response to the current political climate have left key stakeholders in clinical trials with limited resources to conduct equitable research.

Clinical Research, DEI Initiatives Caught in Political Headwinds Amidst Funding Freeze

The key considerations for sponsors in deciding whether to claim the credit. 


The Federal Research Credit: Is It Worth All the Trouble?   

In this video interview, Rob Jones, product manager, TMF practice area, Pharmalex, shares his key takeaways from Cencora’s recent TMF Leadership Summit and touches on the importance of oversight when working with outside stakeholders. 

The Importance of Clinical Trial Oversight when Outsourcing

, Rob Jones, product manager, TMF practice area, Pharmalex, discussed challenges in the trial master file (TMF) space, highlighting outdated regulations such as ICH E6(R3). When it comes to innovation and compliance in the space, he stressed the importance of a risk-based approach, focusing on critical records and leveraging artificial intelligence for big data analysis. Moving forward, Jones believes the TMF community's collaborative nature can be viewed as a strength in navigating regulatory changes and technological advancements.

ACT: Cencora’s TMF Leadership Summit was just a few weeks ago. What were some key discussion points or trends that emerged from conversations there?

It was really good. Again, this is going back to my point about everyone being open and sharing, this was the nice thing. It was a group of us just being brutally honest about the struggles that we all face. There was one word that came up time and time again, and that word is oversight. It's something that has been in our industry for a long time. It's something that the regulation is now putting more pressure on, and it's also a word that I love, because, like many words in the English language, it has two meanings. One meaning is to oversee something, make sure it goes well, and the other is you miss something, and something bad happens. It produces this fantastic sentence that, again, in a lot of my talks, I like to bring it up on the screen, which is, “Without oversight, you risk an oversight.” It really says a lot that, again, in the past, oversight was just this idea of it's a tick-boxing exercise, but we're outsourcing more and more now. We're outsourcing to vendors, to partners, to just third-party groups in general. It's not always cost effective to do everything yourself in-house, and you don't always have the experience in-house, you can't have an A-team for everything you do. This idea of outsourcing is becoming bigger and bigger now the current regulations have really set that up as to what they expect, and in essence, what they're saying is just because you're not behind the wheel of the car at that moment doesn't mean that you should not be in control. You should be in control. You should be ready to correct when things go wrong. What we're having to do is really look at that support, really looking at making sure that it doesn't matter if it is, and this was part of what came up. It doesn't matter if it's a live trial that you're running today, and you're halfway through it, you've got loads of metrics, you still need oversight. It doesn't matter if you are getting to the end of the trial and you're about to receive it from a CRO partner. It doesn't matter if it's a trial that you never did, but your company has acquired that trial and that product, you need to prove oversight, and it's a real delicate balance, going too far one way, you're basically just doing the job that you outsourced, you're checking on everything, you're chasing people up, you're making sure that everything goes exactly as you think it should, and there was no point in outsourcing it, you've overstepped it. On the other side, what you don't want to do is just ignore it, and then when disaster does strike, if you're lucky, you know about it, but a lot of the time you don't even know that disaster struck you. You're oblivious to it, and unfortunately, regulators won't accept innocence. They will accuse you of ignorance at that point, so getting that balance and what we say a lot, is that idea of trust but verify, so when you're thinking about oversight, really think about what sort of checks you're doing. You don't want to do all of them, but how about a sample? You want to set your vendors up for success. You want to share ideas with them, and if you see something going wrong, don't wait for them to tell you, go to them, so have that two-way communication. There is no us in them, everybody's working towards the same goal, and yeah, that ultimately, over those two days that we had at our leadership summit, every talk, at some point, touched on oversight, and again, didn't matter what it was, oversight was still the thing that came in, and I think that's an area that again, as we move forward, as we think about what's happening and what's changing, it's going to become pivotal, especially when you're sat in front of that inspector and they say, “So, what did you do to make sure that this went well?”

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Vendor Strategy & Collaboration

In this video interview, Rob Jones, product manager, TMF practice area, Pharmalex, discusses how risk-based approaches must be prioritized amidst the increased demand for quality and compliance. 

Embracing Risk-Based Approaches with Clinical Trial Oversight

ACT: What should leaders in the TMF space be focused on the most in navigating the increased demand for quality and compliance?

There is a quote that anyone who's ever listened to any of my talks or webinars or blogs or anything. I love this quote, and it's in ICH E8(R1), I've got it written down here. “Perfection in every aspect of activity is rarely achievable, or can only be achieved by use of resources that are out of proportion to the benefit obtained.” Sounds like a Taylor Swift lyric, but realistically, what it's saying is, in order to get perfection, you're going to have to put in far more effort than it's worth, so focus where the focus needs to be. Not all records in your TMF are equal. They're not. A protocol amendment changing the fundamental part of your study because of an endpoint is far more important than the routine chit chat that you have and meeting minutes you kicked out. There's more risk at a high enrolling site that's got 1,000 patients, than there are at another site that's got one, so focusing on where the risk is and really embracing this idea of a risk-based approach, I think that's where we need to focus on. That's where we can really get to this new demand for quality and compliance and being able to say the rest is good enough. It does exactly what we need it to do. It's fit for purpose. The other thing with risk, though, we always need to remember is, I've seen people in the past use risk-based as a way to justify doing less. I think the thing we need to remember is, risk goes both ways. There's some times where the risk drops, and you can say, “Brilliant. Don't need to do as much work as I was doing yesterday. All my metrics are green. I've got everything that I need to. I've just had a call with the rest of the study team. Everything's flying through. Brilliant.” There's other times, though, when you have a bad week and the number's aren’t looking great, and at those times, you have to do more work than you thought, but balancing that being available and being able to pivot where needed, that's really what the leaders in this space need to do. I think again, going back to the AI, this idea of there's people who can really have a first mover advantage. Going to be the same here. There's going to be people who really embrace this and really make a difference to their organization, and there's going to be other people who say, “It's too risky. I don’t want to, and I'm going to check everything twice, just to be safe.” That, to me, is where really the leadership is going to be focused, and again, where quality, compliance, cost, everything that comes into it can really be tackled head on.

In this video interview, Rob Jones, product manager, TMF practice area, Pharmalex, highlights recent ICH guidelines and how risk should be an area of focus as trial designs continue to get more complex.

Understanding the True Meaning of Risk in Clinical Trial Operations

 I mentioned, not only have we got these new regulations, but things are changing at a rate that we've never seen before, we can't keep up with. There was a time back in the 2010s where it felt like there was new things happening all the time with, again, bringing in a real foundational level smartphones and tablets and suddenly, internet everywhere, but we're even past that now, it's moving even faster. I think the thing is, if you look at things like ICH E8(R1) that was released the other year, that was almost like a foundation for ICH E6(R3), and it really talked about quality by design. It really talked about thinking about the holistic and bigger picture. They talk in there that if you're thinking about going to certain countries, don't wait until further on down the line, just incorporate those countries into the trials you're doing. That means that trials are going to become more complicated. Complication brings cost, and fast and complicated bring risk. There's no getting around that. Risk is a big, big thing, but again, ICH E8(R1) really built in the idea of quality by design and risk being a big part of that. ICH E6(R3) also talks a lot about risk. I think the thing that we should be focusing on from a leadership point of view, is really understanding that word, risk-based has been a buzzword for as long as I've been around in the scene. We've talked about risk-based, we've talked about these different approaches, but I think it's time for us to really think what that means. Risk inherently means the potential of something bad happening. It's our job to understand that and do what we can to minimize it. In the past, terms like fit-for-purpose, good enough, they're almost dirty words. They were sins. “Good enough isn't enough.” Actually, it is now, that is enough.

In this video interview, Rob Jones product manager, TMF practice area, Pharmalex, discusses challenges the TMF space is currently facing and how it can utilize collaboration in making advancements. 

Battling Outdated Regulations in the Trial Master File Space

ACT: What are some challenges the TMF space is currently facing? How do you think they can be addressed?

It's a nice, broad question. I think really the biggest challenge is keeping up. I think right now we've got new regulations coming in, things like ICH E6(R3) have finally come in this year, after being delayed for a little bit because of other things that were going on in the early 2020s. We've got new proposed standards from the reference model with V4 and all of this while trying to balance that desire for innovation, cost effective solutions, just an ever-changing landscape. I think one of the things that we have to look at and admit is regulations have been a little out of step for a while. We were expecting ICH E6(R3) months, if not years ago, but again, due to things that got delayed. That's fine, but a lot of things have already passed it by. The EMA has already put in new guidelines around their perception of the TMF. They had a brilliant new guideline around computerized system validation, which included things like the electronic TMF. The MHRA have been giving us more and more feedback on their current public forums and Q&As and things like that. It's kind of a little too late in some ways, ICH E6(R2) came out in 2016, and even then it was only a small look there in the grand scheme of things, realistically, it was written back in 1996. I don't need to tell you how much has changed since 1996. My marker for a lot of these things when I talk with people, is the first iPhone came out in 2007, so anything post-2007, you have had constant satellite communication world in your pocket. We're an industry that's built on stability, order, organization, we're managing clinical trial documents, but this new world hasn't been that for a long time, it's messy in a lot of ways.

The challenge really is, what do we do when the regulations are a little bit too old and outdated? How do we manage when other industries, especially outside of clinical trials, are just flying by, and what can we do to keep up without jeopardizing people's comfort, without jeopardizing all of the hard work that's gone into it? I think the nice thing really, with the TMF, and this shows every single time I go to a conference. It is one of the very few parts of clinical trials where it's an open book. There is a real community behind everything. Everyone's talking to each other. You go and talk with people, and they will tell you that TMF stands for “too much fun.” They'll tell you that TMF stands for “too many friends.” There is really this idea of everyone's in it together. I think the nice thing with that is that's been built from the beginning. The reference model when it came up was sponsors, CROs, vendors, consultants, all working together, sharing what they were doing. Nothing was secret. Nothing was hidden behind closed doors. I think, really, that is the biggest strength, and that's how we can address this ever-changing landscape, if we all come to get together and make a bit of a collective decision and say, “We as a group are going to make an interpretation of this, and this is how we're going to take it forwards.” There's a little bit where perception becomes reality, and if we're all pushing in that same direction, that really helps with how we can address all of this. I think, again, just for the TMF world, that's really how we're going to get by with these things changing at such a rate. It's one of the biggest strengths of our little, niche part of the industry.

In this video interview, Rob Jones, product manager, TMF practice area, Pharmalex, discusses challenges the TMF space is currently facing and how it can utilize collaboration in making advancements. 

In this video interview, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, highlights the Rally for Rare event and how it succeeded in providing the community a space for collaboration and innovation.

2025 Rally for Rare: Bringing the Rare Disease Community Together

, 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: Could you tell our audience a bit about the Rally for Rare event and its importance in raising awareness around rare disease research?

Our Rally for Rare event was a last-minute event, I will say. We were very much looking forward to attending the combined NIH-FDA, Rare Disease Day event in Bethesda that was postponed, and that event is so important, one, because it is an avenue for both patients, caregivers, those affected in the rare disease community to collaborate directly with researchers and physicians at NIH, at FDA, and other various scientific consortium and it really sets the precedent for global engagement in rare disease research. It's quite a remarkable event, so the fact that the event was postponed, really represented a gap for that collaboration. The work that these institutions do for rare disease research is critical and having that platform to discuss all of the things that I've mentioned thus far, a commitment around global awareness and engagement, commitment on endpoints, and removing those barriers and removing the red tape to drive rare disease research is really the pinnacle of that event, so Rally for Rare was our response to continuing that conversation, allowing those who otherwise would have been at that event to come and share in that space the same platform discussion and innovation they would have had at that event. It was held on Rare Disease Day, February 28, and we welcomed everybody in the rare disease community, patients, caregivers, advocacy groups, researchers, those of us in the industry to again, to continue that conversation. We use the term “rally for rare” to again, represent just how important this day is, Rare Disease Day, but also just because the event was being postponed to a later date in the year, that it was still important to have that conversation on that day for all the various reasons that I've mentioned.

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

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, highlights the need for maintaining data privacy in a highly regulated clinical research industry.

Maintaining Regulatory Adherence with Clinical Technology

, 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: What would you say to stakeholders who are still unsure of implementing more clinical technology?

I think it's very much beyond, I would say, the technology itself because, as we can see we are able to speak to each other. I think we have, maybe the Atlantic Ocean between the two of us, and we are still able to interact as we were in the same room, so the technology is there. It's more the question of data privacy in particular, I would say, for patients in using those technologies, we have to ensure that the data privacy is respected. In particular, that for the pharmaceutical sponsor, they shouldn't know anything about the patient identity that should be disclosed and things like this, so there is some of the regulatory, ethics, also difference between, I would say, all countries. Even I would say we have the global ICH E6 that is defining the way clinical studies should be run, but each country may have this their own ethics, let's say mindset, as well as data privacy and therefore, of course, there is sometimes also that fear from the pharmaceutical sponsor side is that, if I'm doing something brand new, is it going to be accepted by the regulatory when I will submit my dossier? That's why, very often the technology is very much, I would say, on line, with the technology of daily life, like even you can look to your bank account, I would say, using the web and so on, and you can interact with friends, but then, as you know, clinical trials are so regulated for the sake of protecting the patient and also the quality of data, that sometimes pharma, I think, is a little bit anxious to move towards those technologies until there is maybe some new guidance. As everyone had to deal with the pandemic crisis, with COVID, of course, there was an increasing speed of trying to adopt some technology, because no one was able to travel anymore, even the patients, they were, I would say, less accepting to travel to the to the sites. The monitors were not able to go anymore to the to the sites to review the source documents, so there was some attempt to try to look to the source documents, but depending on the countries, it might be very difficult. Now that we are back in what I would call a normal situation, after, indeed the very high increase of using technology during the pandemic, there was almost not a shift, but okay, let's come back to what we know, what we master, and let's see how the regulatory will help us, so that's why some of the recent guidance, the one from the EMA and the FDA regarding decentralized components is helping very much the pharmaceutical industry, as well as the CRO service provider and the tech provider to be a little bit more comfortable in the way they can think on implementing those new technologies, but yes, clinical research is a specific word, with a lot of concern for protecting patient data and making sure that you have no deviation, that you have a harmony between standardization across all sides, and of course, depending on the country, it might be very different.

In this video interview, Thierry Escudier, portfolio lead, Pistoia Alliance, talks the current landscape of sustainability in clinical trials and how stakeholders are beginning to pay more attention to carbon footprint amid increasing protocol complexity. 

A New Era for Sustainability in Clinical Research

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

Except a few, let's say, front-edge companies, that have already looked to that domain for a couple of years, I guess most of the clinical leaders have never really considered the sustainability, because I think no one has really assessed in the past, in fact, the impact of the clinical trial implementation. Everyone is focused for decades about ethics, about safety, about regulatory compliance, keeping I would say those trials within the budget, within the timelines, make sure that patients will, I would say, accept to be part of those clinical trials, having good data, good quality, so all those things with an increasing complexity of the trials, this has been proven many times by those studies from Tufts, it's clear that the protocols are becoming more and more complex. There is more and more, I would say, demand on both the investigator side and on both the patient and everyone was focused on that, and it's only in the recent days that there was a new mindset that is appearing to say also, well, clinical trials are not equal to zero in terms of carbon footprint, and because this is an overall action and implementation, can we reduce that impact that now people are also looking and a couple of comments is, is not to oppose, I would say, the sustainability versus the cost, because, if you think also in a different manner, one of the other issues in clinical trials is the waste, waste of packaging, waste of blood samples that are not used and even they are sent to the site, some waste of the drugs even now with all the IRT system, you can really more control, I would say, the number of supplies you are sending, but still, there is a lot of waste and this waste is purely, I would say, carbon footprint, but also it's a waste of money.

In this video interview, Thierry Escudier, portfolio lead, Pistoia Alliance, highlights where regulatory agencies currently stand on sustainability in clinical trials and how pharma companies are looking to reduce their carbon footprint.

Being Prepared for Regulatory Sustainability Requirements in Clinical Research

ACT: How will this initiative aid in regulators’ increased demand for sustainability and transparency?

In a way, it's beyond, I would say, the regulatory requirement, because, as such, regulatory agencies, that is to say those health regulatory agencies looking for new drug submissions are not yet asking for any data on sustainability itself. This is more of a high-level request from in particular Europe, we have some very ambitious goals to reduce the carbon footprint in order to have less impact on the climate change. Some of the goals have been set up by the European Commission, the European Parliament, the National Parliament, and the national government, so there is, I would say, an overall trend within the industry, not specifically in the pharmaceutical world. Therefore, currently, I don't think there is any requirement from specific regulatory agencies such as the EMA, MHRA, or the FDA. It may come one day or the other, so that's why we have to be prepared, but it's more the voluntary approach from the pharmaceutical industry to consider that they want to also behave even beyond the fact that, as you know, the pharmaceutical industry is working to provide new drugs for saving lives, I would say, reducing the pain, maintaining people in good health, and at the same time, we know that our processes, whatever they are coming from R&D or from the industry, are producing carbon footprint, therefore may have an impact on the climate change, so therefore it's even more important that as an industry as a whole, the pharmaceutical companies are looking to how to reduce the production of carbon in order that in fact, not to offset the benefit of the new drugs, or the drugs we are currently, I would say offering to the to the patient.

In this video interview, Craig Lipset, co-chair, Decentralized Trials & Research Alliance (DTRA), discusses numerous themes that clin ops can focus on to keep its trials running efficiently and effectively.  

Areas of Focus for Clinical Operations Leaders in the Current Research Climate

, Craig Lipset, co-chair, Decentralized Trials & Research Alliance (DTRA), discussed the impact of recent regulatory decisions on clinical research. Funding cuts by the NIH will affect intramural research and shared infrastructure. Additionally, the politicization of diversity initiatives will affect the enforcement of action plans. Lipset also highlighted the industry’s response to these actions and what it can expect moving forward.

ACT: From a clinical operations point of view, what does the industry need to focus on to ensure trials remain efficient and effective in light of some of the recent government actions affecting it?

One theme I would say is we do need to save our sites, but in the year 2025 our attention has to be on US academic research. We can't abandon them now. We have to be a little patient with them right now, so we shouldn't start reallocating our research portfolios away from academic sites and shifting studies, in earnest, more towards private, free-standing sites, which are great ways to mix a study, for sure, but our academic sites are going to face a resourcing challenge. Many of them have hiring freezes. They're having to reshuffle a lot of their own budgets right now. It's a challenging time for them, and not a time for industry to turn away. We need them to be sustainable, and we are going to have to be an important part of that sustainability, so be patient and stay by the side of America's academic research infrastructure.

Diversity and representation investments; hopefully we've learned enough and we can continue to share enough in this space that we can be efficient in realizing representation. It still will take extra effort in planning, but hopefully we can sustain our work there, because we could do it in smarter and more efficient ways.

Certainly for clin ops friends, the regulatory environment will remain somewhat unpredictable. There are regular changes happening on websites, on nights and on weekends, and we don't have email communications or newsletters coming out that are summarizing those yet. There is a certain level of unpredictability, and that unpredictability will continue with turnover and leadership. Will turnover in leadership with the inspection community in FDA bring consequences in terms of what inspection readiness looks like? We just we just don't know yet. I would say a last call for folks will be being mindful of possible changes to Medicare and Medicaid coverage, and realizing that that has consequences for research participation. When we do coverage determinations in our protocols, and we have decided that certain procedures in the site's study budget are not going to be paid for by the sponsor, but are expected to be covered by the patient's insurance, we may have more patients who are being exposed to cost sharing or who may have coverage gaps as a result of some policy changes this year, so this is a very important year for us to make sure that we understand ways that we as sponsors can help to cover these costs, so that we are not neglecting uninsured or underinsured patients and preventing them from being able to participate in trials simply because we've done a coverage determination where we thought an expensive CT scan, MRI, or routine care chemo was supposed to be covered. We need to make sure that these patients are being kept whole, and there are some very smart and innovative folks out there that have been figuring out ways to do so.

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