Clinical data management
inefficient workflows, lingering paper processes, and technology that does not integrate”. And that is where Florence steps in.
“At Florence,” Bastek explains, “we have the goal of giving sites their day back from paperwork, so they can do the important work of advancing clinical trials. We offer eRegulatory and eConsent products to reduce paper and create efficient electronic workflows that also benefit the sponsor by making remote access possible and easy.” It sounds like a simple solution – but in the regulated world of clinical trials, Florence’s work is critical. “It does sound so ridiculously simple,” Bastek agrees, “but the clinical trial space is just so highly regulated, it’s so far behind the rest of the medical field.”
Change of pace
The introduction of technology like this into the clinical trial space has been growing gradually over the past ten years – but in the past two, the pace of change has visibly gained momentum. “I would say the rate of change from ten to five years was very slow; and from five years to one year was not much faster. But then Covid-19 really accelerated everything. Covid was such a catalyst for clinical trials. People would never have done the things they’ve done in the last two years had it not been for a global pandemic.”
The development of advanced technology, coupled with the circumstances of a global pandemic, has had measurable impact on clinical trials. Now, thanks to companies like Florence, workflow is beginning to be streamlined, so that physicians and investigators can spend more time on the things that really matter: patient recruitment and communication. Yet, as Bastek acknowledges, while efficiency gains are critical, they simply aren’t enough on their own to offset the innate difficulty of conducting a trial for a low-prevalence disease. “In rare disease trials, all of these challenges are amplified” she explains. “There are fewer specialised physicians for each rare disease, which means less possible clinical trial sites; there are less potential subjects to recruit and they are likely [to be] more geographically distributed, which means many won’t be close to the small number of clinical trials sites.” Increased geographic distribution means that there are more regulations to be considered, both on a national and regional basis. The limitations of studies that serve such a small population are numerous, and include sponsors being less inclined to fund them, as well as recruitment often taking place over a long time frame in order to have a statistically significant data set. With all of these challenges to face, rare disease trials need more than a technological band-aid.
Clinical Trials Insight /
www.worldpharmaceuticals.net
“One of the real challenges in rare diseases is that it is expensive to run the trials and that translates forward into expensive therapies that the health system is reluctant to adopt.”
Mark Caulfield Reality check
In the UK, genomic sequencing is becoming a reality. In November 2021, the ‘100,000 Genomes Pilot on Rare-Disease Diagnosis in Health Care – Preliminary Report’ was published – and the first findings are already revealing just how transformative genomic sequencing could be in rare disease diagnosis and treatment. “One of the real challenges in rare diseases,” explains professor Sir Mark Caulfield, a primary investigator on the pilot, “is that it is expensive to run the trials and that translates forward into expensive therapies that the health system is reluctant to adopt. So, we need to improve the opportunity cost of these trials. One way of doing that, which we’ve set out to do in the 100,000 Genomes project, is to create a resource where, based on informed consent, you longitudinally live-course follow people, right up until after their death. You have the ability to concentrate all their electronic health data, alongside their genome, so that you can research on the disease of interest, but also on other things that turn up in that data.” In other words, genomic sequencing is far more than just the next technological add-on; this research really does have the potential to transform the rare disease clinical trial as we know it, at a structural level. “What if,” Caulfield asks, “from a national genomic medicine service, you could have your entire
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As Bastek recently observed in an article for MedCity News: “Technology can help rare disease clinical trials by making it easier for sites to find patients, share data, and run studies. But technology alone won’t overcome the challenges […] We need to embrace a variety of strategies to serve the millions of people waiting for life- changing rare disease treatments.” One such strategy Bastek suggests as a potential game changer in the rare disease trial space is genomic sequencing. “When people think of advanced technology,” she says, “they’re often thinking of AI. And, you know, if you can do genomic matching – that sort of advanced technology really will help when you’re talking about low-prevalence rare diseases.”
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