Like most industries, the biopharmaceutical space is undergoing a radical evolution. The sector, for quite some time now, has been embracing – and necessitating – the use of big data and sophisticated analytics to navigate the increasingly complex and demanding clinical trial process.
The pressure to adapt is coming from every direction: regulators, sponsors, patients and the market itself. Combined with reimbursement hurdles, patient recruitment challenges and therapeutic area nuances, drug and medical device developers are finding it vital to evolve alongside these changing winds where data has become so critical.
When Clinical Trial Data Just Isn’t Enough
According to leaders at PAREXEL, one of the largest biopharmaceutical services providers globally, there are two megatrends happening right now in the drug development space that are affecting how clinical trials are being carried out.
The first is a sector-wide push for real-world data (RWD) to support clinical trial findings. Gone are the days when data collected during efficacy trials was enough to convince payers or national agencies of a drug candidate’s value. It is becoming increasingly necessary to demonstrate the value of the therapy in patient populations that mirror the expected recipients.
“As healthcare costs have continued to go up and as the cost of specialty pharmaceuticals – in particular – have gone up, there has been a lot of pressure in the system to manage those costs,” said Josh Schultz, Senior Vice President & Worldwide Head of PAREXEL Access.
“The most effective way for pharma companies to advocate for reimbursement with more expensive products is to demonstrate and de-risk for payers that the compound will have the expected value in a clearly targeted group of patients," said Schultz.
The FDA officially defines RWD as data coming from sources outside of traditional clinical studies, which can include information from Electronic Medical Records (EMRs), administrative claims databases, patient registries – and even wearable medical devices. This data provides researchers with insights directly from patients’ clinical experiences, and RWD is being requested more and more to complement information gathered during normal clinical trials.[1]
According to Schultz, the industry has come to appreciate how data from even a carefully-designed trial is not sufficient enough to prove a drug candidate’s value. While randomized controlled trials (RCTs) have been known as a sort of gold standard for demonstrating how effective a drug candidate will be, the data gathered during efficacy studies is drawn from an idealized environment – and only gives us information about a limited population within a highly-managed setting. There are many factors that come into play once the confines of an efficacy trial are removed, including differences among practice settings, comorbidities, concurrent treatment regimens, and provider decision making.[2]
In conventional clinical studies, these factors would be seen as noise or errors that need to be minimized to reduce variation. In day-to-day medical care, however, these variations are directly relevant to the practical decisions that patients, their doctors, and providers are making in the real world. They are necessary to gain real-life insights as to how a drug will work in a given patient population, and they are also fundamental for stakeholders to be aware of during every stage of the development journey.
The good news is that, while the demand for more evidence from more data sources is growing, the amount of data available is growing right alongside it. This brings us to the second major trend – the amount and availability of RWD has skyrocketed in recent years. Everything from wearable blood-glucose monitoring devices to smartphone apps have rapidly accelerated how much RWD is out there for drug developers to take advantage of.
It’s Not How Much Data You Have; It’s the Insights You Gain From That Data
So, we’re swimming in data – more information than has ever been available before from a growing number of sources. According to Xavier Flinois, President of PAREXEL Informatics, this presents both an opportunity and a challenge. Massive pools of data are nearly meaningless without having a way to capture it, aggregate it, and architect it in a way that can benefit stakeholders’ decision making.
“In the past, the problem was that we didn’t have the data. Now, we have piles of data but there is now the need to apply sophisticated informatics and statistical approaches to get value from the data,” said Flinois.
Moreover, data gathered from traditional clinical studies have historically been siloed – separated from each other in isolated stacks, sitting behind firewalls or stored in filing cabinets of pharmaceutical companies.[3] Siloed away, safety databases and trial results were just meant for a drug maker’s staff to work with – but if you were an academic, a payer, or a national health organization, your ability to engage with that data was limited.
Of equal importance is data on sites and investigators. The pool of available investigators is large, dispersed globally and constantly changing. Many investigators become involved in one study only and never do another so a key success factor is identifying who the experienced investigators are and how they perform.
“We know that just because an investigator has had previous success doesn’t mean he/she will be successful in the next study they do because protocols are different,” said Paul Evans, Corporate Vice President of Global Site Solutions at PAREXEL. “But we do know that investigators that have figured out how to address the challenges of patient recruitment and understand how to deliver successfully on a study are a much lower risk for future studies, continued Evans. “This is a business where good experience counts.”
According to Evans, the challenge is that the data on investigator performance that will make a difference in decision making is not easily available and is tightly held. PAREXEL has overcome this by creating a knowledge repository called SIMS (Site Intelligence Management System) that collects data on investigator performance from numerous different sources. PAREXEL has a rich source of clinical data collected over decades from sources including lab data, RTSM data, EDC data and IRB data amongst others. This information is then mastered to clean duplicates for example, and ensure consistency, increasing both the breadth and depth of PAREXEL’s knowledge of investigators.
Thankfully, the days of disjointed and unorganized drug development data are numbered. PAREXEL has been working to aggregate vast swaths of data and construct a connected architecture that guides stakeholders through the increasingly data-driven clinical trial process to bring drugs to market faster than ever before. This connected architecture, combined with world-class clinical, regulatory and technology domain expertise enables targeted solutions and insights that yield higher efficiency, cost savings and quality.
“Over the years we’ve been at the frontline of innovation, investing heavily in technologies that help optimize the drug development process. For sponsors, this means that whenever new data assets and real world tools come to light, our ability to incorporate them into our workflows is far easier thus increasing the speed of the drug development process,” said Flinois.
The company partners with some of the most successful pharmaceutical researchers in the industry through every step of the drug development journey, from pre-clinical all the way to post-commercialization. Through its decades supporting many of the most widely-sold drugs in the world, PAREXEL has been building and creating the industry’s largest environment of connected and collaborative technology, processes and expertise – a purpose-built ecosystem called the Connected Journey™.
“The Connected Journey™ enables our customers to do their jobs better. We believe this approach is of high value because it enables them to make better decisions with real-time performance data,” said Sean Gallimore, Corporate Vice President and Chief Marketing Officer.
“That, coupled with domain and practice expertise – harnessed in a single offering – really makes it easier to simplify their development journey,” concluded Gallimore.
According to PAREXEL’s leaders, the impetus behind the Connected Journey™ is to simplify the drug development process by giving stakeholders access to clean, secure and high-quality data in real-time – powered by PAREXEL® Analytics, a robust multi-dimensional business intelligence layer that aggregates internal and open source data in real-time. The ecosystem was designed so that data from multiple sources “talk to each other,” allowing biopharmaceutical researchers to make timely, actionable decisions – and bring their product candidates to the patients who need them quickly.
The Connected Journey™ is an integrated range of solutions that sweeps across the entire drug development process, allowing a variety of stakeholders to engage with the ecosystem. Not only does the Connected Journey™ help researchers make critical decisions quickly, the insights are delivered as user-friendly visualizations that allow stakeholders, at any level, to engage with the data. More importantly, the system ensures that the right data is analyzed in the right way – and finds itself in the hands of the right people for smarter decisions at every step of the drug development journey.
[1] https://blogs.fda.gov/fdavoice/index.php/tag/real-world-evidence/