Regeneron on the Digital Age of Clinical Trials

Clinical trials are difficult. Speed, bottlenecks, costs, and the ability to enroll patients are all major challenges. The COVID-pandemic demonstrated that clinical trials could be accelerated, but how do we repeat that success in the post-pandemic era? The industry must come together to discuss the matter, says Bari Kowal, Regeneron’s head of development operations and portfolio management.

Enticed into the industry by the pipeline, the foundational science, the entrepreneurial spirit, and a reputation for innovation, Kowal has worked in both small biotech and big pharma. Following a four-year spell as operations lead at Pfizer, she joined Regeneron to establish a global development organization that could expand and improve clinical trial management. Here, she offers her view on how clinical trials will continue to evolve in the digital age and beyond.

What geographical trends are you seeing in clinical trials?
 

Trials are being driven now by where the patients are, rather than where anyone else would like them to be! At Regeneron, for example, we spend a lot of time early on looking at epidemiology and the prevalence and incidence of where diseases primarily show up. We look for populations large enough to study and we try to go there. This isn’t necessarily the large, established markets; emerging markets are a key trend right now.

How does regulation affect clinical trials in emerging markets?
 

There’s a greater need for harmonization across markets. Regulatory bodies have been working on this for years, but the industry needs more streamlined efforts to make it easier to get clinical studies underway. I already mentioned that you need to look at where the patients are when planning studies, but you also need to look at which locations make sense – and where you can easily manage your molecule through the process. We first look at where patients are, then see if it makes sense from a regulatory perspective – how long it takes and how willing the regulators in various countries are to work with us in streamlining processes. 

How can industry continue with conversations on promoting harmonization?
 

As a member of the Board of Directors for the TransCelerate consortium, we’re often invited by regulatory bodies to provide guidance input, so there is a great deal of opportunity to help regulators with understanding the nuances of executing clinical trials. There is a lot of openness from regulators, but we still have a lot of work to do in demonstrating the translation of guidance and regulations to the practicalities of running clinical trials. 

Over the years, regulators have continued to greatly encourage the use of risk-based monitoring and data science, and yet companies are reluctant, tending to overachieve when it comes to how much monitoring to perform – even though analytics of study output on data cleanliness suggest there is a very small difference achieved by significant monitoring efforts. Companies have to adapt to the opportunities to review risk-based data in order to confirm quality and streamline our efforts. 

What trends are you seeing in decentralized trials?
 

There are many conversations happening in the industry about these kinds of trials. We want to create an environment where clinical research can be a care option for patients  – something that becomes possible when trials are decentralized or hybrid in nature. I find that patients like relationships – a human touch – as well, so the idea that everything will be decentralized doesn’t necessarily fit. Many trials involve testing and procedures that cannot be done in a patient’s home, but we are looking at opportunities to meet patients where they are and reduce the burden of being in a clinical trial. A broader reach into communities is also needed so that we can connect and raise awareness with the patients who don’t normally have access to clinical trials.

In the future, digital biomarkers and more digital endpoints will hopefully give patients more flexibility and allow us to measure physiology in new ways. We want to use wearable sensors and find new ways to manage large volumes of data while patients are living their lives, rather than expecting them to fill in extensive questionnaires. Hybrid trials are where a lot of the industry is heading – but it’s not a perfect approach. 

Does gamification fit into the future of clinical trials?
 

We explored the concept of gamification in one of our pediatric clinical trials several years ago. Instead of typical questionnaires or just inputting patient reported outcomes in a tablet, we reconfigured it into a game to help children who were scared about being in a trial feel more comfortable. The study participants were even able to create and 3D print their own avatar as part of it. This enabled us to get the data we needed, and it became a fun game for them. It improved retention and compliance too, so I think gamification can be a great opportunity. 

How else can clinical trials be improved?
 

Today, the speed of getting medicines to patients is not where it should be. As an industry, we accelerated things during COVID-19, but this was because the whole world cooperated during that time frame; now, we need to figure out how to repeat that success. 

Technology, collaboration, information, and awareness are all key to improving clinical trials. During COVID-19, the world started to understand more about clinical trials. I want to make sure we keep that momentum going, so that people understand that trials are essential for producing medicines. We enhanced our clinical trials website during that time to make sure we had readily available information for anyone who wanted to access it.

How is Regeneron preparing for the future of clinical trials?
 

We’re really lucky to have been able to bring in talent from some of the best technology companies. Our industry is reliant on data – and using different perspectives and experiences is the ethos of Regeneron. An idea can come from anywhere, but bringing in those different backgrounds helps us see the world differently – and helps us look at the data differently.

I hope that we can use the data that’s out there to reduce the sample sizes of trials. It’s a lofty goal, but if we don’t need to enroll as many patients, trials will be much easier. We also need to collaborate more across the industry. Challenges are not solved by one company or a single individual; we need to bring in regulators and vendors who can work on data processing and management devices so that we can understand how to bring something viable and valuable to clinical trial design.