In recent years, technological advances have both expanded the amount and availability of real-world data (RWD), and improved our ability to analyze it. Biopharma and other industry stakeholders have taken notice, and they are honing strategies to incorporate real-world evidence (RWE) into their processes and decision-making.
Specifically, global health technology assessment (HTA) bodies are exploring ways to harness RWE, albeit at varying levels of acceptance and in varying stages of guidance development.
To assess how receptive HTAs are to RWE today, and how they’re evolving to incorporate RWE in the future, Aetion assessed leading HTA agencies’ methods for evaluating health technologies, and reviewed any of their publications regarding RWE. Read on for a summary of the findings.
HTA bodies’ current use of RWE
To date, HTAs have varied in their acceptance of RWE, ranging from strict evidence hierarchies that value randomized controlled trial (RCT) data over RWE, such as in Germany’s IQWiG/G-BA benefit assessments, to Brazil’s CONITEC, which requires RWE in submissions for certain “expensive drugs.”
Most agencies state in their methodological guidelines that they accept all evidence of a drug’s efficacy and effectiveness, which implies that they accept RWE. However, evidence hierarchies are pervasive; six out of 13 agencies limit use of RWE due to that framework.
Both ICER in the United States and AEMPS in Spain request RWE to provide evidence of effectiveness in the real world, but they are often limited by the breadth of evidence—or lack thereof—that is submitted by the manufacturer, or what is published in the literature.
For those agencies, like CADTH in Canada, that evaluate cost-effectiveness, RWE plays a more substantial role. RWE is often requested for use in cost-effectiveness models, but use is limited to prevalence and incidence, costs, and resource use. In Sweden, the Netherlands, and Italy, which often use market entry agreements, RWE is used more widely—and sometimes is explicitly required to continue reimbursement.
Trends toward future openness to RWE
The tide is changing as most HTA agencies become more open to RWE. In one of the more surprising moves, Germany’s IQWiG has announced that it will accept high quality RWE generated from registry data in their benefit assessments. However, IQWiG is still unwilling to accept RWE generated from claims or EHR data, claiming that data from these sources are unsuitable for benefit assessments. Despite being a limited opportunity for RWE, this is a substantial step forward in Germany.
Other major advances come from ICER in the United States and NICE in the United Kingdom.
ICER announced that it will generate new RWE for its 2020-2023 assessments, instead of relying solely on published data. In addition, RWE will play an integral role in ICER’s forthcoming value reassessments. In a pilot program, ICER will revisit assessments 24 months after their initial publication for drugs that received accelerated approval. ICER’s goal is to determine how the drug is performing in the real-world, and if changes should be made to the cost-effectiveness model.
NICE has signaled that it will use additional evidence, including RWE, in its guidance development, including technology assessments and clinical guideline creation. It is seeking partnerships with leading RWE researchers and experts to assess how best to incorporate RWE in their guidance. NICE plans to release methodological guidelines in 2020.
Implications for biopharma
As RWE continues to gain traction, HTAs are increasingly requesting that biopharma employ it to supplement evidence packages for assessments. Biopharma should prepare their RWE strategy accordingly as agencies shift away from the traditional evidence hierarchy and embrace RWE’s role in bridging the efficacy-effectiveness gap.
There is an opportunity for biopharma to build their RWE infrastructure for the identification, collection, and analysis of RWD. For HTA purposes, focus should be on identifying opportunities to reduce clinical uncertainty inherent in some pivotal RCTs (e.g., in cases of accelerated regulatory approvals), support clinical and economic assumptions, and improve cost-effectiveness model inputs. These RWD and RWE protocols should be integrated into the brand’s strategic plan early in the development process.
Manufacturers should also facilitate discussions with HTA agencies regarding how RWE will be incorporated in the submission, and how the HTA body will use it in their assessment. By increasing collaboration and transparency with HTAs, biopharma can help facilitate the development of RWE guidance, and eventually enable the use of RWE in decision-making.