“Real World Evidence (RWE) Significance in Healthcare and Drug Development”

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“There is no point in stakeholders closing their eyes and pretending nothing is changing. We’re moving in the direction of greater use of RWE.”

                                                                            ­– Dr Pall Jonsson, NICE

What is RWE?

Real-World Data (RWD) is healthcare information that comes from sources like

  • Electronic health records (reports from diagnostic laboratories, discharge summaries, prescriptions, scan reports etc.)
  • Claims and billing data (information from insurance companies that includes amount claimed, type of diseases treated, duration, history of the patient, demographic data)
  • Records on specific diseases and its treatment procedure
  • Digital applications and health monitoring devices

These data sources are diverse and will be of varying quality. Analyzing this data involves a lot of complexities concerning the accessing of data and the subsequent drawing of a conclusion. Real-World Evidence (RWE) is generated by analyzing the above records and has also been proved in multiple instances that this evidence virtually matches the data gained from supplementary clinical trials.

How RWD is turned to RWE? [1]

Vijay's article figures_Edited


Why RWE is exciting to pharmaceutical industry?

Drug approval decisions

Regulatory professionals are considering RWE as a crucial deciding parameter while approving a drug application. Based on the importance of drug under review, accelerated approval is granted on account of the related RWE key role. FDA published guidelines in 2016 for the use of RWE to modernize the drug development and approval process. These guidelines were intended to ensure quicker drug discovery and approval so that the medicine can reach patients much earlier; For instance, on 04th April 2019, the US FDA announced its extension of the indication of Ibrance (palbociclib) in combination with endocrine therapy for metastatic breast cancer in men. The drug is currently marketed for breast cancer in women. This approval for the additional indication is considered as a landmark in pharmaceutical, as it was largely considered on the analysis of RWD and evidence generated from it.[2]This approval will set an example for future applications to include additional indications using RWE, with less or no further clinical trials.

Safety monitoring

Also, real-world evidence provides more information than what a clinical trial can offer, like safety and efficacy from the patient’s daily life. A more effective collection of pharmacovigilance, adverse drug effects data will help to provide more relevant real-time data on the effects of the drug on the general public.

Focused Drug Development

The journey from drug discovery to approval followed by commercialization is a challenging journey, in which billions of dollars and thousands of man-hours are invested. It takes approximately 10 to 15 years for a new drug to reach the market. There have been instances whereby the time a new molecule is approved and marketed; its intended market need would have changed.[3] The availability of relevant RWE would help the R&D team to accelerate the drug development process. This has encouraged pharmaceutical companies to invest in RWE, to utilize its capability through product life cycle starting from development through commercialization and post-approval activities.

Challenges associated with the use of real-world data

The critical test in RWE fetching the much-deserved place in healthcare decision making is the lack of consensus available among the medical fraternity as to what can be considered as relevant acceptable data to arrive at RWE. This can be primarily attributed to the complexity of the data and the wide range of sources available. Collecting a meaningful perception from these data is a serious task.

Possibility of relevant stakeholders picking up those real-world data to emphasis on their desired result and converting those into RWE has been a raging debate in the industry. But with increased investment in analysing RWD, there is a substantial improvement in showcasing how companies are overpowering these challenges and applying RWE.[4]

In a typical clinical trial, metrics are pre-defined and the result is derived upon analysis of the data against metrics, to conclude if a drug is really providing the intended result. But in a real-world data, same procedure cannot be implemented. This is really challenging in cancer drug testing where regulations and approval are highly uncertain. Randomized Clinical Trial has remained the standard procedure followed in oncology drug research since decades and will continue to be followed.

Companies are finding it difficult to source the precise service providers to support in building the RWE platform. Hence companies are partnering to acquire new capabilities for generating and analysing large amounts of data. For instance, Roche paid $1.9B to acquire Flatiron Health, Inc., which owns an oncology electronic medical record platform.[5]

RWE: Way forward

With an increasing emphasis to use RWE throughout the product life cycle, starting from molecule screening until approval and post approval activities, having highly trained cross skilled resources will be the need of the hour. People with strong knowledge in clinical trials bundled with good analytical and writing skills will be required in huge numbers going forward. Companies must invest both in people and in technology to sustain the growing need and importance in RWE.

Due to the huge volume of data (both structured and unstructured) involved from different sources, involvement of artificial intelligence and machine learning cannot be refuted.[6]

RWE cannot out-date Randomized Clinical Trial (RCT) and are also considered complementary to each other. For instance, if RCT is focused on finding out the effectiveness of the drug, RWE can find out information on safety, pharmacovigilance and treatment cost of the drug.

Advantages and Limitations of RWE when compared with RCT [7]

Advantages Limitations
Reduced cost and effort Requirement of huge data
Considered as step stone for Artificial Intelligence Complexity involved in analysis
Research with data is possible where RCT cannot be performed Data privacy
Detecting less frequent side effects Lack of required talent tool


Graphical Illustration of RWE in Clinical Development [8]


2nd fig.


There is a higher possibility of RWE information generated subsequent to the clinical trial output being influenced by unidentified financial interests. Hence it is important to negate any such credibility issues before making critical decisions, which should always be in the interest of patient safety.

Wider acceptance of RWE information will bridge the existing gap between R&D and healthcare and bring these two worlds together. This will ensure quicker approval of new drugs, adding new indications much easier, all these at a much more accelerated rate and considerably of low cost.

Organizations should implement better and effective working models which can bring in much needed change in the mindset of people who still see outcomes of RWE as futile. It is vital to have the buy-in of concerned professionals on RWE and ensure that they find RWE as a possible alternative to other traditional methods available.


Enhancing the usage of RWE, more particularly, with the supporting data for approval of new indications and post-approval requirements can significantly reduce cost and the associated effort, thereby increasing the company’s revenue and faster access to patients.

Considering the numerous hurdles involved in new drug discovery, RWE can be used as a powerful tool for planning product development, bringing in required changes during the execution process and rescheduling the pipeline to ensure drugs reach patients at the right time. Pharmaceutical companies should be more vigilant to bring in pioneering ideas to maximize the benefits of patients while also increasing financial success.

This will encourage better invest in R&D, as we will have better value for the billions of dollars spent to bring a new molecule to market and importantly more people will come forward to improvise the livelihood of millions of patients.

Organizations should tactically make use of digital technology and artificial intelligence to expand the possibility of RWE generation by maximum utilizing the available data sources. While small and medium level companies cannot be expected to finance all areas of RWE, identifying key areas which require better attention and by finding out correct partners for rest of the activity will only enhance better usage of RWE.

It is also important for insurance companies and the relevant stakeholders to understand the benefits of drug administered to a patient and RWE will help to achieve this by reinforcing the confidence of patients and the treatment’s success.


  1. https://www.researchgate.net/publication/318391159_Deriving_more_value_from_RWE_to_ensure_timely_access_of_medicines_by_patients
  2. https://www.drugs.com/newdrugs/fda-approves-ibrance-palbociclib-men-hr-her2-metastatic-breast-cancer-4943.html
  3. https://www.clinicalleader.com/doc/real-world-evidence-and-the-collision-between-pharma-r-d-and-healthcare-0001.
  4. https://scrip.pharmaintelligence.informa.com/SC100133/Tackling-RWE-Challenges-To-Demonstrate-Healthcare-Value
  1. https://www.biocentury.com/biocentury/strategy/2018-03-09/how-roche-acquisition-accelerates-expansion-flatiron%E2%80%99s-real-world-
  1. https://www2.deloitte.com/us/en/insights/industry/life-sciences/2018-real-world-evidence-benchmarking.html?icid=dcom_promo_featured%7Cglobal;en
  2. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6097073/
  3. https://www.researchgate.net/figure/Graphical-illustration-of-inclusion-of-RWE-in-the-clinical-development-strategy_fig3_327297925

Authors’ Profile

Photo.jpgVijay Sivanandam 

Director, Regulatory Affairs at FMD K&L

A result oriented professional with 14+ years of industry experience in pharma space covering drug, consumer and medical device regulatory affairs, encompassing the US, EU and Rest of the World countries, including strategy & intelligence, life cycle management submissions and other services under regulatory affairs and operations