It’s near impossible to overstate the growth of real world evidence (RWE) in the healthcare space – from relatively unknown a decade ago to today, where an internet search for RWE produces 1.5 billion results, almost all relating to healthcare.
It’s fair to say that industry stakeholders – payers, providers and regulators – are getting much more comfortable with the use of real world data (RWD). More and more regulatory guidance is being released across the US, UK and EU, with evidence produced from RWD being viewed and accepted as a complement to randomized controlled trials (RCTs), as well as valuable in its own right.
Underpinning the increasing need for and comfort with RWE, are the trends in drug discovery and development favoring far more complex pharmacological products for very specific niche populations. Trials in these small, sometimes ultra-small, patient populations are challenging, so the desire and interest in further monitoring patients via RWD is high. Using RWD also allows for country-specific monitoring, which can be cost-prohibitive to implement using RCTs alone.
The increased acceptance of RWE is also due to the growing number of sources and tools available to store and analyze healthcare data. Access to de-identified, patient-level data is becoming easier and easier to obtain. New access models, such as those provided by HealthVerity and DataVant, are gaining traction.
Expanded access brings expanded choice. However, expanded choice brings more decisions to be made along the process and thus an increased number of points for potential missteps. We’ve seen a number of questions arise when speaking to people: How do I find the right data source? Who can I turn to for help? And which services and tools can I trust?
Many more types of data becoming available sounds like a total win, but just because there are more data doesn’t mean all sources are suitable – you have to be able to evaluate each source for quality, completeness, relevancy and, most importantly, appropriateness. Does the data cover the right population for the treatment, and is it an accurate representation of the cohort? Some companies offering data and analysis also over-promise on what can actually be delivered using RWD today – it has lots to offer but is not a magic cure-all for all uncertainties around a new healthcare product.
Pharma companies are getting better in terms of their understanding of RWE/D and strategies and tactics to use it. Utilized effectively, RWE can help mitigate risk and uncertainty in the R&D environment, the pipeline stage where a new treatment faces the most jeopardy.
However, there is arguably a gap in understanding at the level of regulation, particularly when working in different geographies. The US and the EU have different regulatory processes when it comes to data protection: HIPAA and the GDPR, the main regulation for each geography for healthcare data, have fundamentally different purposes and cannot be treated as equal. We’ve seen that many pharma companies fail to take into account the differences across geographies – data access requests in the EU often take much longer to be granted than in the US, but some companies fail to account for this in their launch strategy, leading to avoidable delays and uncertainty.
We’re aware that many pharma companies are still looking for a one-stop shop when it comes to looking for partners and vendors to support a new product. However, not all vendors out there are equipped to support all along the product pipeline, especially when it comes to RWE. Companies looking for a partner need to find a Data Luminary, someone who:
- Asks the right questions. A Data Luminary is someone who sheds light on different aspects of RWD. Someone who asks the right questions and who understands primary uses for various types of secondary data, e.g., How are the data meant to be used? What is the underlying population? What is the generalizability of findings, and to which populations?
- Knows when and with whom to partner. No one individual can be an expert in everything. A critical Data Luminary skill is knowing when and how to consult and work with other experts and academic and clinical networks.
- Watches the whole product lifecycle. Recognizing that RWE/D has value across the entire product lifecycle, pharma should push partners to think broadly, beyond any individual study.
- Understands and embraces regulation. Data Luminaries must possess strong understanding of local privacy regulations, particularly in Western Europe. There are local dynamics in the US (e.g., California’s new privacy laws) that could impact privacy regulations across the country, moving US policy closer to the EU’s GDPR requirements. Keeping abreast of these regulatory activities is critical to maintaining data access and to understanding how international patient-level data can be used in other markets.
- Acts with integrity. It seems obvious, but given all the things that could go awry in the complex RWE/D space, a pharma organization must have full trust in its partners: trust that patient data will be protected, trust that the data selection process will be completely unbiased, trust that both the strengths and, more importantly, the weaknesses and limitations of any source of RWD will be understood and communicated, trust that scientific rigor will be applied to all decision-making throughout the study execution, and that there will be full transparency along the way.
Data expertise paired with research acumen and clinical depth is the winning combination
The need for RWE is growing but pairing the right partner(s) at the right time is crucial to the success of the study and the understanding of the data. It is vital to have an expert who understands the landscape and the key considerations for complex decisions that need to be made. The Data Luminary is a critical part of the RWE/D team, but the best-in-breed service providers in this space will be those who bring a scientific approach across three dimensions: data, research methodological considerations specific to the use case (e.g., outcomes, safety, trial design), and clinical/medical expertise. A unicorn well worth pursuing!