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Real-World Evidence (RWE) And Its Use In HEOR

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The healthcare sector is evolving rapidly. The importance of Health Economics and Outcomes Research (HEOR) is increasing, and its objective is to fill a knowledge gap in the field.

HEOR helps generate real-world evidence (RWE) that proves to be a solid foundation to analyze and study how medical treatments work and remain effective and how pharmaceutical companies, healthcare providers, and payers should improve health systems and patient outcomes.

Healthcare Decision-Making and the Use of Real-World Data

The healthcare community uses real-world data (RWD) and RWE to make better decisions and develop guidelines for clinical practice. For instance, the US Food and Drug Administration (FDA) uses RWD and RWE to monitor safety and adverse events in order to make regulatory decisions; the 21st Century Cures Act of 2016 places an additional focus on these types of data that influences drug approval.

It’s no wonder that data collection and the use of RWE in HEOR are happening now as a consequence of technology, that is, the use of mobile phones, wearables, and other devices that can generate health-related data and electronic health records. The combination of this additional information with data mining techniques can improve the drug development process of biopharmaceutical companies, patient care, and healthcare systems.

The Use of RWE in HEOR: Choosing Value

One of the potential benefits of the use of RWE and data in healthcare is real-time decision-making. HEOR is helping healthcare to transition to patient-choice systems that focus on outcomes and value. Environmental and social influences, along with genomics and behavior, are also critical factors that can deliver value.

However, challenges are also present when determining health outcomes, such as:

  • Integration of data from multiple sources.
  • Collaboration with stakeholders holding data.
  • Achieving medical-level accuracy.
  • Removing confounders.
  • Correlating biomarkers with outcomes.
  • Complying with regulatory requirements.

Data sets can complement the knowledge gained from traditional clinical trials. RWD traditionally comes from four sources:

  • Clinical data;
  • Administrative/claims data;
  • Patient-generated/reported data; and
  • Emerging data sources.

Where Does Real-World Data Come From?

This data environment is maturing rapidly, especially in developed countries where data from hundreds of millions of patients are accumulated. This is why processing this data is of the utmost importance and is attracting interest from corporations, not-for-profit organizations, and public sector organizations.

The following list is an example of high-value data pools accumulated by developed countries, according to McKinsey & Company sources:

  • Japan. National claims database. 126 million lives covered.
  • United States. Medicaid/Medicare claims. 120 million lives covered.
  • France. National claims database and national hospital claims database. 60 million and 60 million lives covered, respectively.
  • United Kingdom. Electronic medical record (EMR) data from 10% of general practitioners and English hospital EMR database. 53 million and 15 million lives covered, respectively.

The Possibilities Of RWD In HEOR

Today, we are subject to the endless possibilities and growth of big data in many industries including healthcare. Much of the advance is happening in clinical research; linking genomics data to outcomes allows experts to discover why some patients are more prone to specific health conditions. Yet, the field of epidemiology and HEOR is still in its conception pertaining to RWD.

RWD is fragmented since it includes only a healthcare sector or location. It is also incomplete because key outcomes are missing or are not collected routinely. In addition, time stamps for significant health events are subject to reporting bias or are not present at all.

Therefore, how can RWD application and utilization be improved? Here are some of the following key concepts:

  1. Collaboration of stakeholders. The cooperation of multiple stakeholders leads to the development of standards with more complex and robust approaches. A typical data model should be one of the main focuses of this increased collaboration.
  2. Production of clinically-relevant outputs. The drug development process has become multi-disciplinary, and it now incorporates aspects such as market access as part of multi-functional brand teams. This increased visibility leads to RWE generation and overcoming the stigma of observational research as being lower in the scientific evidence hierarchy.
  3. Faster research outputs. The slowness of data access has a negative impact on research quality; some real-world analyses are outdated by the time they are public. This leads to obstacles in market access and data availability delays. Therefore, researchers can use technology and automation to speed up data management and analysis.

The Role Of The Stakeholders In RWE And RWD

Stakeholders are present across the healthcare value chain using RWD and evidence to guide their decisions:

Physicians and Providers

Physicians and providers rely on electronic medical records (EMR) for clinical research led by physicians. Health system administrators, conversely, use the same data to monitor the care quality delivered across the system. Physicians can now access the same data of a larger number of patients and institutions, which has contributed to research innovation.

For example, in the United States, the consolidation of hospitals and systems has resulted in a larger scale of operations, focusing more on value due to risk-bearing contracts. On the other hand, the National Health Services (NHS) of the United Kingdom is already imposing value-based pricing for some therapies.

Pharmaceutical Companies

Pharmaceutical companies had limited use of RWE at the beginning of the 2010s and were heavily focused on safety and post-market surveillance. The growth of RWE use occurred approximately from 2011 to 2015 across the end-to-end product lifecycle.


Regulators use RWE to monitor the safety of marketed products through traditional pharmacovigilance tools. They also monitor newer digital aids with a post-market active safety surveillance system. 

Still, regulators could use it even more broadly, such as in rare diseases, oncology, and pediatric conditions, when randomized controlled trials are unethical or impossible to conduct. The FDA is making more efforts to integrate data collected from EMR, claims, and registries to create a unified system.


Payers focus on improving the affordability of healthcare for members. They frequently integrate claims with EMR data to generate insights regarding the value and effectiveness of providers or protocols. Outcomes-based contracts with providers are now more widely used in the US, with an estimated 80% of physicians and 100% of hospitals having at least one of these contracts.

However, payers still rely on traditional levers when it comes to pharmacy costs. All in all, companies are starting to enter into value-based partnerships that link a drug’s net price to expected outcomes.

The Future Of Real-World Evidence And Data In HEOR

As we have seen, and despite challenges, the use of RWE and data in HEOR and the healthcare field is growing steadily. RWE is now commonly used by pharmaceutical companies, providers, and payers to provide more value and make better cost-effectiveness and comparative efficacy decisions.

The next steps should be for stakeholders to increase understanding and communication of RWE value drivers, create an operating model to integrate and adopt RWE, and build platforms at scale to manage and analyze data.