Real World Data in the Regulatory & Reimbursement process – Implications for value and access

Real World Data in the Regulatory & Reimbursement process – Implications for value and access

From mobile devices to wearables and biosensors, digital innovation has led to an exponential increase in the availability of health-related data. While the healthcare market has traditionally been slow to adopt digital innovations (in 2017 14% of US clinician offices still relied on paper medical records) there are now a multitude of ways healthcare companies are beginning to leverage digital innovation and the real-world evidence (RWE) it generates to better identify, understand and treat disease1. Healthcare companies aren’t alone in their increasing utilization of digital data; In the US, Congress has recognized the potential for RWD to dramatically improve the American healthcare system. With the signing of the 21st Century Cures Act in 2016, Congress created a mandate for the FDA to promote RWE for use in regulatory decision-making2,3. However, the use of RWE in the regulatory approval process remains limited to date. FDA Commissioner Scott Gottlieb signaled that may begin to change this year with a recent announcement that RWE will be a priority for the FDA in 20192.

Across Europe, the EU Network Strategy to 2020 identifies RWE as a key enabler to bring innovative products to patients with unmet medical needs and to support the safe and effective use of medicines4. However, whilst the European Medicines Agency (EMA) already uses RWE sources in some of its evaluations, this is typically on an ‘ad hoc’ basis; and despite the global traction that RWE has gained, its acceptance is by no means uniform and indeed varies considerably between countries.

Such geographic differences in Europe are particularly pronounced in relation to market access. There are some countries, such as the UK, Netherlands and the Nordics that are receptive towards RWE, e.g. are willing to consider RWE derived treatment effects where RCT generated evidence is limited, such as in orphan diseases. For other countries, such as Germany and Spain skepticism over the robustness of RWE for demonstrating treatment effects prevails amongst market access stakeholders.

There are still a great many obstacles to the widespread use of RWE in the regulatory and reimbursement approval process. Questions regarding data quality, interoperability of data collection systems, analytic methods to ensure study validity, patient privacy and ethical considerations around sharing patient data remain to be fully addressed 2,5.

Notwithstanding these issues, RWE is increasingly recognized as a valuable source of evidence for both regulatory and reimbursement (market access) decisions. In 2015 a “Payer RWE voice of the customer project” gained insight into RWE needs and priorities across ten European markets6.

The value and importance placed on RWE in 10 European countries:

There have also been some key successes in the US including the FDA approval of Bavencio in Merkel Cell Carcinoma (MCC) which leveraged electronic medical record (EMR) data to demonstrate the drug’s improved outcomes over the existing standard of care7. In rare indications like MCC where patient recruitment can be a significant obstacle to generating the statistical power required for regulatory approval, RWD has the potential to dramatically alter evidence generation.

Looking beyond rare disease, RWE also has the potential to transform the way in which drugs demonstrate clinical and economic value. One of payers’ biggest concerns when assessing new drugs is the uncertain clinical and economic benefits of the product in the real world. When speaking to payers about clinical data we often hear the question ‘I understand the statistical significance of the data, but what does it mean for my patients?’. Payers want manufacturers to connect clinical trial data, which may rely on surrogate endpoints or small populations in highly controlled environments, to the therapeutic value the product delivers in the real world. Are patients able to live fuller lives? Are they hospitalized less? Do they need to take less medication? Do they live longer? What does the data mean?

There seems to be a consensus that the use of RWE is valuable for the provision of clinical practice evidence on treatment pathways, resource use, long-term natural history, and true effectiveness. The interest in RWE is so great that a survey of payers published in Genentech’s 2018 Oncology Trend Report found that ~59% of US payers expect that within 5 years, drugs that do not support their benefit with RWE will likely lose coverage8.

While it is unlikely that RWE will become a coverage requirement for most drugs in the US within the next 5 years, it will become an increasingly important part of coverage decisions and will certainly support differential value between competing products. Manufacturers that leverage technology to generate RWE earlier in the development cycle will have a clear advantage over competing products.

In the major European markets, we can expect to see access stakeholders’ familiarity with, and acceptance of RWE continue to grow; which could eventually translate into an essential requirement. Moreover, in some smaller European countries, where RWE is being rapidly embraced, it could be ‘the norm’ as part of the submission package to inform their pricing and market access decisions in the future.

  1. Health IT Quick Stats. (n.d.). Retrieved February 12, 2019, from
  2. BipartisianPolicy Center (January 28, 2019), Webcast: “The role of real-world evidence in regulatory and value-based payment decision making.” Retrieved from
  3. Gregory D (September 13, 2017) Clarifying the Real-World Data and Evidence Landscape. Retrieved from
  4. EU Medicines Agencies Network Strategy to 2020 – Working together to improve health (2015) Retrieved from
  5. PrismeForum Technical Meeting (November 17, 2016) Real world evidence generation: preparing for the future. Retrieved from
  6. McKesson (2018) Case Study: Using Real-World Evidence to Accelerate Drug Approvals for Rare Diseases
  7. The 2018 Genentech Oncology Trend Report, 10thedition. Retrieved from