Clinical researchers deserve a lot of credit. Bringing a new medical product to market today means navigating a whole host of challenges, from patient recruitment, to shifting regulations, to increasing research and development costs. And the effects are becoming clearer than ever – in just one example, from 2015 to 2016 the number of new drugs approved by the FDA fell by half. In the face of all this, clinical researchers’ ability to remain focused on its goal of fighting disease and saving lives is why we find clinical research so inspiring.
But it also begs the question: What can we do to navigate this growth in complexity and costs in clinical trials, and ensure they don’t impede our ability to deliver life-saving treatments to people in need? How can researchers modify their workflows to adapt to these challenges? The industry has been answering this question in a number of ways – the eSource revolution and the emergence of virtual and hybrid trials are just a couple of them.
Another approach that has been gaining steam: novel clinical trial designs. Any organization seeking ways to keep their research moving forward in the face of rising costs and complexity should be evaluating whether new approaches to clinical trial design can accelerate their progress toward their goals. Here are a few designs researchers are experimenting with, and how they can maximize speed and cost efficiency in clinical research.
1. Adaptive clinical trial designs
Quite possibly the marquee name among innovations in clinical trial design, adaptive designs make trials more efficient by making them iterative. The design allows researchers to make use of insights gained during the course of a study, and incorporate those insights into the same study instead of banking them for subsequent ones. The benefits are truly game changing: by incorporating insights in real time, adaptive designs can make individual trials more successful and reduce the number of trials necessary for regulatory submission.
Just ask Jim Nance of PharPoint Research, a CRO and Medrio customer, who spoke with Medrio about his adaptive design experience on our blog, and whose company provided tips and tricks for successful adaptive design execution in a webinar they hosted with us.
2. Mendelian randomization
What if you could use genetic variations to investigate the causal relationship between potentially modifiable risk factors and health outcomes in observational data? The precision medicine revolution has drawn widespread attention to the use of biomarkers to predict health outcomes and better tailor treatments, and Mendelian randomization is a part of this. By using genetic variants, Mendelian randomization can provide more reliable estimates of the likely underlying causal relationship of a biomarker with risk of disease.
3. Surrogate endpoints
Long-term indicators, such as prolonged survival or improved quality of life, are not necessarily the only useful endpoints for determining the success of a treatment in a clinical trial. Certain clinical trials can also benefit from surrogate endpoints, like lab measurements or radiology images, that can be measured much more quickly and deliver key insights in a clinical trial setting much sooner. The speed possible through surrogate endpoints can have massive results for clinical researchers, with the potential to accelerate regulatory submission and even approval. And the incorporation of surrogate endpoints into trial design is making a dent: between 2009 and 2014, 66% of FDA-approved oncology treatments used surrogate endpoints.
4. Master protocols
Even in clinical research, multitasking is an important skill. Master protocols involve study protocols designed with multiple sub studies. This allows investigators to evaluate multiple products in multiple disease subtypes within the same master trial structure. The results range from reduced cost and risk to the ability to arrive at key insights faster.
Clinical research is changing – and organizations are modifying their workflows to keep up and succeed in their trials. Novel clinical trial designs are a major part of this shift. At this pivotal moment for our industry, it’s a good idea to look into these new designs, and to evaluate whether the eClinical resources you’re using are equipped to take advantage of them.