Real-world evidence comes of age for pharma: What to learn and how to earn

Understanding how medicines perform in the real world has the potential to transform the pharma business — from driving increased efficiency and cost savings in drug development, to helping identify new patient populations for marketed drugs. This can translate into higher profitability and shareholder returns, as we explain in our Next in pharma series on driving value growth.

The big challenge for pharma leaders now is learning how to leverage real-world data and evidence (RWE) as a sustainable competitive advantage in a landscape that has changed dramatically over the past few years.

Here are some examples of positive RWE-related outcomes that have recently been reported in the literature.

  • Faster drug trials: Use of RWE led to a 40% reduction in the planned sample size of a pivotal phase III program, which translated into six months of time savings.1
  • Inclusivity: A study of real-world data using artificial intelligence (AI) suggested the pool of patients eligible to take and benefit from cancer drugs could be doubled.2
  • Expanded approvals: The US Food and Drug Administration has shown its acceptance of RWE from well-designed observational trials in approving new indications for marketed drugs.3

Let’s review three rapidly evolving drivers of the RWE market, based on PwC research and recent interviews with RWE specialists working in pharma or in a vendor capacity with pharma.

Driver #1: The volume of enhanced data and advanced analytics is large and growing fast.

The amount and types of available health data continue to grow at an incredible rate with the compounded annual growth rate (CAGR) of health data expected to reach 36% by 2028.4 At the same time, longitudinal data is becoming more prevalent — in some cases extending to several years of information on a patient’s health.

Available data is now more thorough due to the integration of multiple data sets. A fuller picture may now be drawn from electronic health records (EHRs) across provider organizations and sites of care, associated pharmacy claims data, genomics testing data, and patient demographic data. Several RWE vendors are also exploring ways to create differentiated value such as computer-assisted mining of clinical notes and use of derived/synthetic data to further enrich existing data sets.

Driven by this rapid evolution in data and related technologies, PwC anticipates spending on data products to increase at an 8% to 10% CAGR through 2028 with integrated, linked data sets leading the data segment with a CAGR of approximately 14%.5

Separately, the RWE analytics market is expected to grow at a 15% CAGR through 2029 as vendors face increasing competition and pricing pressures on data products and seek competitive advantages through sophisticated analytical platforms designed to generate insights across the drug development, care delivery and commercial life cycles.13

To take advantage of these improved RWE offerings, pharma organizations should consider the use of RWE to inform aspects of their product life-cycle management, including clinical development, product launches and efforts to stay competitive in the market. 

Pharma should be looking to hire personnel — from medical professionals to data scientists to business leaders — who have experience in using RWE insights to improve business decisions. There is also an opportunity to leverage the rising number of advisory service offerings from RWE vendors for specific areas where in-house talent may be lacking.

Pharma companies can leverage multiple data types and predictive analytics on their own to improve clinical trials and post-marketing studies — however, RWE vendors may be able to deliver with greater efficiency and effectiveness.

Driver #2: Real-world evidence companies are becoming more specialized.

As the RWE ecosystem has matured, oncology and rare diseases have become the largest and fastest growing therapeutic areas for application due to their complexity and a relatively high unmet need in curative treatment.17

In order to capitalize on these opportunities and build solutions tailored to specific disease areas (and their underlying indications), RWE companies are investing in both technical and clinical experience as well as sophisticated analytics platforms with AI and natural language processing (NLP) technologies that are better able to aggregate data, fill gaps and harness the power of enhanced data sets.

Competition and specialization within the RWE market is producing more novel applications — such as external comparators, pragmatic trials and enriched studies — and it’s spurring more strategic relationships with topic and application specialists. This can translate into greater insights and improved outcomes for pharma pipelines and commercialized assets. Pharma companies should identify vendors by therapeutic area focus and actively manage a broader portfolio of RWE providers rather than selecting a few when starting an RWE program.

Driver #3: Regulatory bodies are providing more support for real-world evidence.

Dedicated RWE task forces, new data standards and guidance from regulatory bodies such as the US Food and Drug Administration (FDA) and the UK’s National Institute for Health and Care Excellence (NICE) are encouraging the use of RWE for a growing number of applications, including the evaluation of safety and effectiveness as well as use in supporting submissions and approvals. The European Medicines Agency’s (EMA) Data Analysis and Real World Interrogation Network (DARWIN EU) project has been gathering real-world data through a range of studies and is expanding.

Still, navigating the separate guidelines and data standards on a global scale can also increase the complexity of bringing a new product to market. As a result, RWE companies have sought to build international data sets and local regulatory knowledge via serial acquisitions and strategic collaborations. The acceleration of development of new regulatory guidelines has also brought an increased focus on regulatory-grade data sets for clinical trial design and other critical drug development activities.23

Evaluating your needs and opportunities

The rapid expansion of the RWE ecosystem is broadening what is achievable with real-world data while simultaneously increasing the complexity, investment and experience required for successful deployment across the value chain.

Pharmaceutical companies should keep their RWE strategies up to date and collaborate with leading companies to complement in-house capabilities and improve value capture across drug development and care delivery life cycles.

Here are five questions to consider when evaluating and updating your strategy.

How do your business objectives and unique asset portfolio influence your RWE needs?

  • Asset portfolio and product mix include the volume, risk profile, performance and stage of pipeline and in-market assets as well as therapeutic areas/indications and therapy classes of focus.
  • Business objectives include speed to market, market share, revenue maximization, cost savings, market access and generation of evidence on safety and efficacy to support label expansion.

In which specific areas can RWE provide your organization with a meaningful return on investment today versus in the near future?

  • Today: Mature applications of RWE may represent immediate opportunities for value capture based on more established use cases (e.g., outcomes studies, patient cohort design, post-market safety studies).
  • Near future: Emerging applications of RWE (advanced clinical protocol design, development of synthetic control arms in clinical trials, optimization of commercial activities) have the potential to deliver greater value and competitive advantages for those organizations who are able to get it right.

How should your RWE operations be structured to improve value capture?

  • Centralized, scaled function is likely in larger organizations to enable greater utilization and return from RWE across the entire life cycle of multiple assets over extended periods of time.
  • Core, embedded function (e.g., R&D, commercial) is likely in organizations with smaller budgets and more targeted point-in-time needs and RWE resources.

What value can an RWE vendor offer compared to internally developed capabilities, assets and experience?

  • In-house RWE capability can be an important differentiator for pharma while allowing greater control in key areas (such as quality and regulatory governance), but it requires significant investment to innovate and maintain in a rapidly evolving RWE ecosystem.
  • Specialized vendors often focus on innovation and depth in targeted use cases, data types, diseases, regions and patient populations. 
  • Scaled vendors often offer greater data volume, geographic reach, integrated and longitudinal assets via longer-term investments and working relationships (health systems, aggregators, molecular testing companies).

Which RWE vendors can effectively address your strategy, operating model and unique needs?

  • Analyze your answers to questions 1-4 to determine your organization’s unique RWE requirements and narrow the broad spectrum of RWE companies to those most likely to meet your specific needs, then construct a network of vendors to help your organization capture the value of RWE across the drug development and care delivery life cycle.
  • Base your network decisions on vendor selection criteria to assess the ideal fit according to your organization’s specific strategy, portfolio, operating structure and objectives. Keep in mind that no single organization or vendor can address every RWE need across the entire life cycle of an asset. 

With knowledge and experience that spans the pharma value chain and the RWE ecosystem, PwC can help guide you through the development of your RWE strategy and vendor selection process. Please reach out to one of our experienced professionals for additional information.

Contact us

Ash Malik

Principal, Strategy& US


Greg Rotz

Pharmaceutical & Life Sciences Advisory Leader, PwC US


Alan Keener

Director, Strategy& US


Additional Strategy& Contributors

Angad Nagwan, Manager, Strategy& USA

Brian Wong, Senior Associate, Strategy& USA

Gage Kaefring, Senior Associate, Strategy& USA

1 Reynaldo Martina, David Jenkins, Sylwia Bujkiewicz, et al. “The inclusion of real world evidence in clinical development planning,” Trials, August 29, 2018, 468, Vol. 19.
2 Ruishan Liu, Shemra Rizzo, Navdeep Pal, et al. “Evaluating eligibility criteria of oncology trials using real-world data and AI,” Nature, April 7, 2021, 629-633, Vol. 592.
3 U.S. Food and Drug Administration. “FDA approves new use of transplant drug based on real-world evidence,” (July 16, 2021), accessed April 18, 2023.
4 Cheryl Coughlin, David Roberts, Kenneth O’Neill, et al. “Looking to tomorrow’s healthcare today: a participatory health perspective,” Internal Medicine Journal, January 2018, 92-96, Vol. 1.
5 PwC analysis informed by paid specialist interviews and purchased market reports from Meticulous Research and Grand View Research.
6Sharing images helps veterans get better health care,” (May 16, 2021), accessed April 21, 2023.
7Clinical genomic data is a game-changer for precision oncology,” (March 8, 2023), accessed April 21, 2023.
8The key to longitudinal patient journeys,”, accessed April 21, 2023.
9 Orien Avatar,, accessed April 21, 2023.
10 Aetion Evidence Platform,, accessed April 21, 2023.
11Using AI to accelerate the discovery of insights and evidence from clinical text,” (June 6, 2022), accessed April 21, 2023.
12Ciox Health acquires biomedical natural language processing pioneer, Medal, Inc.,” (July 18, 2020), accessed April 21, 2023.
13 Meticulous Research. “Real-world evidence analytics market by application, end user – global forecast to 2029,” June 23, 2022.
14Analytics and innovation available to clinicians in drug development,” (March 22, 2022), accessed February 20, 2023.
15Accelerating ADCs’ development pipeline using Simcyp PBPK,” (October 15, 2021), accessed February 20, 2023.
16Finding the right patients and investigators for faster clinical trials”, (August 4, 2015), accessed February 20, 2023.
17 PwC analysis informed by purchased market research report from Meticulous Research.
18For rare diseases with no standardized treatment strategy, insights can help inform next steps in patients’ journeys”, (November 17, 2022), accessed February 20, 2023.
19Realizing the full potential of real-world evidence in oncology,”, accessed April 21, 2023.
20 TriNetX data sets,, accessed April 21, 2023.
21Improved maternal health outcomes begin with MOM,”, accessed April 21, 2023.
22 IQVIA PharMetrics Plus,, accessed April 21, 2023.
23Regulatory and safety: Innovative approaches to stay ahead of regulatory change,”, accessed April 21, 2023.
24 U.S. Food and Drug Administration. “Best practices for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data sets,” (May 2013), accessed April 21, 2023.
25 U.S. Food and Drug Administration. “21st Century Cures Act,” (January 31, 2020), accessed April 21, 2023. 
26 U.S. Food and Drug Administration, “Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s new strategic framework to advance use of real-world evidence to support development of drugs and biologics,” (December 6, 2018), accessed April 21, 2023.
27 U.S. Food and Drug Administration. “Submitting documents using real-world data and real-world evidence to FDA for drug and biological products,” (final guidance September 8, 2022, draft 2019), accessed April 21, 2023.
28 Sarah Karlin-Smith, “Cures 2.0 likely to push US FDA to further embrace real-world evidence,” (December 9, 2021), accessed April 21, 2023.
29 U.S. Food and Drug Administration, “Data standards for drug and biological product submissions,” (October 21, 2021), accessed April 21, 2023.
30 U.S. Food and Drug Administration. “Real-world data: Assessing registries to support regulatory decision-making for drug and biological products guidance for industry,” (November 29, 2021), accessed April 21, 2023.
31 U.S. Food and Drug Administration. “Advancing real-world evidence program,” (October 19, 2022), accessed April 21, 2023.
32 The National Institute for Health and Care Excellence. “NICE real-world evidence framework,” (June 23, 2022), accessed April 21, 2023.
33 European Medicines Agency. “DARWIN EU completed its first studies and is calling for new data partners,” (March 28, 2023), accessed April 21, 2023.

Follow us

Required fields are marked with an asterisk(*)

By submitting your email address, you acknowledge that you have read the Privacy Statement and that you consent to our processing data in accordance with the Privacy Statement (including international transfers). If you change your mind at any time about wishing to receive the information from us, you can send us an email message using the Contact Us page.