Clinical data management
models, and vary from place to place. It isn’t one software company that’s going to be able to achieve that – but there are organisations working on it.” The Global Alliance for Genomic Health is one such organisation, aiming to create, as Caulfield explains, “national data assets that can be linked together through electronic methods such as application programming interfaces, [which] connect assets. So, the data asset doesn’t leave the jurisdiction, which means it complies with laws […] but there is information sharing. And that information sharing allows access to data in a way that you don’t get from simply sitting within your own silos.”
Genomic sequencing has the potential to transform the rare disease clinical trial at the structural level.
nation’s rare diseases – which have been through your diagnostic investigations – there with their data (or as many as consent), and that is then your sampling frame?” Not only would this help “address the major block, which is that it’s difficult to enrol people for rare disease trials”, but it may mean that the clinical trial becomes a more prevalent part of life, even from infancy. “In rare disease, if you had such a platform […] in early life, perhaps in the newborn period, where you could detect these diseases when they’re fully penetrant,” Caulfield explains, “then you would be trialling your therapy not simply after someone has got a series of fixed deficits that you can’t recover, but at the earliest possible stage.”
“Country-specific regulations are often outdated, based on paper models, and vary from place to place. It isn’t one software company that’s going to be able to achieve that – but there are organisations working on it.”
Andrea Bastek
Caulfield’s prospective model seems almost utopian, yet it does reveal one of greatest hurdles that the rare disease trial has yet to overcome: for now, a genomic medicine service like this would have to remain a national, rather than global, enterprise. For rare disease trials, some form of international consensus “will be necessary”, Bastek urges. “In order to get the data needed to show the success of new treatments [and to] reach more patients with a rare disease so that enrolment is faster. However, this raises huge regulatory and data privacy challenges. Country-specific regulations are often outdated, based on paper
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Battle it out At the end of the day, these questions will need to be battled out by national and global regulators and lawmakers, not by software developers and scientific researchers. But what is clear, from both Bastek and Caulfield, is that we need to be investing in technological solutions – from something as simple as an e-signature, to something as revolutionary as genomic sequencing – if the future of the rare disease trial is going to improve. Collaboration will be key, as Bastek emphasises, between sites and sponsors, between patients and physicians, between nations and regulators – and between different technologies. “In a utopian word,” Bastek says, “all software works nicely together, we don’t have [workflow] redundancies, and we can really get things done faster, we can reach more people.”
For Caulfield, the future holds the promise of early diagnosis and intervention through advanced national platforms. “What we need to do is to get the earliest diagnosis and earliest intervention, because we’ve got the greatest chance of modifying disability and adverse outcomes if we do that. I also think by having national platforms, [through] which we can access and recall patients for research, then we can do these trials much faster and much more affordably – and this will then make the medicine more affordable [for] the health systems.” What’s more, as Caulfield says, “we are entering an era where – whether it be rare disease, cancer or any other trial – we should longitudinally live-course follow people beyond the end of the trial, because there may be late signatures of harm or signatures of benefit that are not captured within the finite period of the trial.” The key, it seems, to advancing clinical trials for rare diseases, lies in a total shift in perspective – the kind of shift that only technology can bring about. With Florence’s digital software, then, we can start to think beyond the constraints of space and site. While, with genomic sequencing, we might even begin to surpass the limitation of time. ●
Clinical Trials Insight /
www.worldpharmaceuticals.net
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