Pharmaceutical companies and their research partners have come to discover real-world evidence (RWE) offers myriad benefits in the drug development process. Leanne Larson, senior vice president of RWE and access with Parexel, recently connected with Outsourcing-Pharma to discuss the number of ways in which RWE can streamline patient recruitment, conserve costs, and benefit in a number of other ways.
OSP: Could you please talk about some of the ways in which the use and understanding of RWE have evolved in drug development in recent years?
LL: We recognize that RWE today is becoming truly central to drug development and ultimately to patient access to important new therapies - it’s no longer an innovation, or a ‘nice to have’. The newer approaches today, along with the evolving guidance, now allow us to harness the power of RWE throughout the product development and lifecycle – today, we can ensure that we’re bringing the very best study design and approach to every research question and doing so in the most cost-effective way possible.
To realize this potential, today we can consider whether the data needed to answer a key research or stakeholder question already exist and can be essentially ‘repurposed’ for this new application, or whether we need to collect new data through a site-based study or even utilize both in a hybrid model. We have data-collection options today that didn’t exist in a meaningful way ten, or even five years ago.
Over the past two decades, we’ve evolved not just our access to data – with the ability today to even gather data directly from EHR systems – but also have advanced our ability to analyze these data more deeply, addressing bias and confounding more effectively and lending more strength to the findings.
RWE is helping researchers better understand challenges in caring for and treating patients, and in the early stages of product development, this is allowing for more effective trial design. RWE helps ensure the right patients are targeted for trials and helps fill the information gaps that may exist.
In the end, it can allow us to bring important new therapies to patients even more quickly – the real reason we do what we do, each and every day.
OSP: Could you please talk about its potential to help pharma firms and their research partners conserve costs?
LL: This ability to utilize the best data source to answer a key question means that we can drive toward the most cost-effective model possible for any given study. We quickly evaluate in the earliest study stages whether we can access existing data, or whether we need to go directly to sites and follow patients longitudinally – and go forward confident that we’ve chosen the best approach.
It’s important to note that existing/secondary data isn’t always cheaper, faster, and easier, though; it’s critical to assess upfront the costs of data identification, acquisition, aggregation, and transformation – and then compare those to alternative approaches. Beyond this, even our study designs have evolved; today we often consider whether an external control arm (ECA) is appropriate for a study - particularly in rare or serious conditions – and can cut the study timelines and costs considerably.
OSP: What other benefits might harnessing the power of RWE offer?
LL: The newer guidance reflects, in fact, the important role RWE can play in supporting and even accelerating drug development and commercialization. External Control Arms, by avoiding randomization to a placebo control, can drive more-effective study recruitment; patients, particularly those with serious or rare illnesses, truly appreciate not risking randomization, and be assured they’ll receive an important new treatment option. Hope matters, and both studies and patients benefit.
OSP: Please talk about the sources that RWE can be drawn from—and feel free to mention sources that might be less common or untapped.
LL: It’s critical to note that RWE is derived from both site-based (primary) and existing (secondary) data sources – it simply means that the data were gathered in routine care settings, outside a clinical trial environment. The new FDA guidance defines RWD and RWE as:
- RWD are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. Examples include – as described by FDA:
- Existing/secondary data: Data derived from electronic health records (EHRs); medical claims and billing data; data from product and disease registries; and data gathered from other sources that can inform on health status, such as mobile devices.
- Primary (de novo) data: Observational studies; pragmatic trials; patient-generated data including patient-reported outcomes.
- RWE is the clinical evidence about the usage and potential benefits or risks of a medical 36 product derived from an analysis of RWD.
OSP: Please talk about how RWE can inform current industry pricing.
LL: RWE, through its unique ability to describe the natural history of disease and burden of illness, can indeed help inform pricing. RWE can help elucidate the costs of treatment with a new product, and a comparison to the costs associated with standard of care, helping to inform pricing and reimbursement decisions.
OSP: Then, how can RWE be useful in answering long-term safety questions post-approval?
LL: RWE has long been a valuable approach to long-term safety surveillance, offering the ability to track product outcomes and impacts on larger patient populations over longer time periods. It’s perhaps even more important today, as so many new products – for instance, cell and gene therapies - require 10-15 years of follow-up to assess the treatment's true effects, and risks, over time.
As you can imagine, doing this in the context of a typical clinical trial can just be prohibitively complex and expensive – if even possible. A real-world approach, though, may offer the flexibility to follow patients more effectively throughout this extended time period.
OSP: What should a study team know about RWE before putting it to work in their operations?
LL: Most importantly, conducting a successful RWE study requires the same focus and attention as a clinical trial. We still need to ensure we are developing a scientifically strong study design based on epidemiologic principles and the most effective analytic and operational plan.
Critically, though, implementing and managing a real-world study requires an understanding of its very-different setting. Whether it’s a site-based study following patients in routine care, or a study accessing existing/secondary data for analysis, or both.
Study teams need to understand how best to identify and enroll sites and patients – and keep them engaged throughout a typically longer study. They need to understand how to analyze real-world data that may include non-comparable study cohorts, or that has some degree of missing data. And if utilizing secondary data, they need to understand how to ingest and aggregate disparate data and transform those data into a dataset that can deliver the needed insights and meaningful evidence that the study demands.
OSP: Are there any useful technological tools that can help with RWE?
LL: Absolutely, we’ve seen important technological advances that are helping to drive important new insights and applications within RWE. A few exciting advances include:
- Tokenization – the ability to identify and match patients across multiple datasets allows us to piece together a patient’s care path more fully, and better understand their treatments and course of illness. As more and more patients are tokenized across various care settings and providers, we will be able to look more often and more effectively to existing data for the insights and evidence we need to support our research questions
- EHR extraction technologies – we’re seeing options emerging today allowing us to extract data directly from EHRs essentially into EDC platforms, creating one of the most important linkages today in our ability to gather the most complete data possible.
- Decentralized trial platforms – there are newer technologies that allow for direct-to-patient studies, providing the ability to identify and consent patients, as well as provide patients with an interface to enter quality-of-life questionnaires and/or link with the patients’ medical record.
OSP: Specifically, what resources can Parexel offer with RWE strategies and implementation?
LL: Parexel brings a team with deep and longstanding experience and expertise in RWE strategy, science, and operations – ensuring we are able to generate the most scientifically strong evidence, targeted toward the most critical stakeholder questions and needs. Importantly, we have RWE scientists and strategists each with decades of experience in designing and conducting these important studies, who’ve been central to the development of today’s RWE standards, best practices, and guidance. And these RWE experts collaborate with Parexel’s leading Regulatory and Access teams – ensuring that our RWE is informed by experts who understand what regulators, payers, and clinicians demand from RWE today.
Our studies are informed by these insights and our experts work closely with stakeholders to inform them on what to best expect from this important new model. This expert team works alongside the industry’s longest-standing operational team – Parexel has been conducting real-world research for over twenty years, having completed close to 1000 studies across all designs, globally. We understand how to run these studies, and we have the teams in place to ensure they’re completed successfully.
Parexel is exhibiting at this year's Summit for Clinical Ops Executives (SCOPE), taking place February 7-10 in Orlando, Florida, and online. Company representatives also are participating in a number of sessions. For more information about the event, visit www.scopesummit.com.