Karen Ooms, Executive Vice President and Head of Statistics, Quanticate
02.04.22
Today more and more pharmaceutical companies are turning to real-world evidence (RWE) to understand the real-life effectiveness of their drug products.
For many decades now, a core part of the drug development cycle has been the randomized clinical trial.
Nevertheless, they have limits in terms of the information they are able to supply, which means it is not possible to gain a complete understanding of the real-life effectiveness of new therapies from clinical trials alone. For a growing number of pharmaceutical companies, the answer may well be RWE.
The problem with clinical trials
A key drawback with clinical trials is that they necessarily have narrow inclusion requirements, which mean they must exclude patients with organ dysfunctions and comorbidities who may be taking other treatments. They may also exclude those who are outside a certain age limit.
This is necessary to eliminate confounding factors that could negatively impact on the quality of the trial results. However, in the real world, many patients taking the drug will be elderly or will be receiving concomitant treatments. Failing to include these means we never have a full understanding of the drug’s performance.
It can also be challenging to find a sufficient number of patients to achieve full representation of all the patients who may benefit from the drug being trialed. This is especially the case for demographics with ethical concerns, such as children, the elderly or pregnant people, or for studies of therapies for rare diseases.
Varying perceptions among healthcare professionals (HCPs) and patients of what constitutes a meaningful impact on symptoms and quality of life also cannot be accounted for using clinical trials on their own.
Finally, poor patient adherence cannot be reflected in clinical trials. Study participants are often more compliant with instructions about treatments than patients taking medications at home. In real life, patients may miss a dose or take it at different times of the day.
RWE is the answer
With these drawbacks in mind, it is no surprise that pharma companies are turning to real-world evidence (RWE) to fill the gaps in their understanding of their treatments.
RWE consists of real-world data (RWD), which can come from an array of sources. These include health insurance claims, HCP health records, as well as data from patients’ own wearables or medical devices.
This information can be harnessed to create unique observational longitudinal databases of de-identified patient records, which can be used by clinical research organizations (CROs) to generate in-depth insight into the real-life efficacy of a drug product . Such databases can include information going back several years on more than 100 million people—providing significantly more data than that provide by clinical trials alone.
This vast well of information can allow long-term, in-depth analysis of the evolving condition of patients with rare diseases as they pursue a new course of treatment. It makes it possible to track treatment pattern changes over time, and to understand long-term treatment performance in patients taking concomitant treatments.
Taking this into account, it is no surprise that regulatory authorities are beginning to appreciate the insights offered by RWE. In fact, in a first for the industry, the US Food & Drug Administration ( FDA ) approved Pfizer’s Ibrance treatment in 2019 using information based principally on RWD.
Is RWE the future?
It is still early days for RWE as a source of vital pharma data, which means there are still disadvantages that must be dealt with. For instance, researchers cannot randomly assign patients to a particular therapy, or collect information on all characteristics of interest.
In addition, there is currently little standardization across databases. For instance, databases designed to be sent to insurance companies for billing of private medical care are clean and consistent, making them easy to study. Nevertheless, they only provide the information needed by the insurance company, limiting the insight they offer.
On the other hand, electronic medical records (EMR)—made up of patient records from across different healthcare facilities—may be disorganized and incomplete. However, they offer incredibly rich insight into patient behavior and personal attributes that can impact their response to a therapy, such as pain scores or lung volume.
With all of this in mind, working with an expert CRO with experience capturing, collating and studying RWD is crucial if pharmaceutical companies want to harness this source of insight to its fullest potential.
Benefiting from RWE expertise
RWE offers potential to deepen our understanding of newly commercialized treatments.
Collaborating with an expert CRO, RWE can be used to complement the data from a randomized clinical trial, providing a complete picture of the performance of a new treatment, enabling us to deliver more effective treatments in future.
Karen Ooms is executive vice president and head of statistics at Quanticate. She is responsible for overseeing the statistics department at the company. She is a Chartered Fellow of the Royal Statistical Society and has a background in biostatistics spanning more than 25 years. Prior to joining Quanticate in 1999 (Statwood), she was a senior statistician at Unilever.