As recently as a few years ago real-world evidence (RWE) was not on the radar screen of many pharma executives. According to a new study this has dramatically changed over the last few years driven by an increased requirement to demonstrate the value treatments deliver to patients. In parallel, technologies, such as machine learning algorithms, have matured and are now capable of collecting, storing and analyzing health care data from large patient groups.
The same study showed that the overwhelming majority (90%) of the respondents from the pharmaceutical industry are either already investing in RWE initiatives or are planning on doing so to support the value claims of their products, for value-based contracting and regulatory submissions as well as to improve the design and execution of clinical trials. And more than twice the number of executives believe, that RWE will be extremely important by 2020 that considered it extremely important by 2018.
Real-World Data (RWD) and Real-World Evidence (RWE)
RWD and RWE are not the same things, although they are closely related. The FDA defines RWD as“(…) data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.”
Those sources are mainly electronic health records (EHRs), claims and billing activities, product and disease registries, patient-generated data including in home-use settings as well as data gathered from other sources that can inform on health status, such as mobile health-tracking devices.
According to the FDA, RWE is “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.”
RWE Across the Life Cycle
RWE is impacting applications across the entire life cycle of a drug from preclinical research to development and commercialization and will increasingly be a topic Medical Science Liaisons (MSLs) will be asked about and will need to discuss with healthcare professionals in general and key opinion leaders (KOLs) in particular.
RWE is becoming ever more relevant and essential as both healthcare providers, and pharmaceutical companies try to gain more insights into fundamental questions such as:
- What subpopulations of patients will benefit from a treatment?
- How does patient heterogeneity impact treatment effects?
- How can we better monitor patient safety?
- How do we better understand the disease burden?
In short, RWE can help to answer the central questions of personalized medicine: which treatment works for whom, when and in what does?
RWE and Business Considerations
In addition to providing an unprecedented amount of data to move personalized medicine forward and achieve better outcomes for patients, pharmaceutical companies are also looking to collect RWD and generate RWE for business reasons. RWE can help companies streamline some of the most time-consuming and expensive parts of drug development. RWE can
- help optimize clinical trial design
- support clinical trial recruiting
- support regulatory submissions
- reduce the cost of post-market studies
- help generates all the information needed for both regulatory submission and payers’ requirements.
Besides, RWE can be used to obtain label expansions, primarily to expand indications of an approved drug when the studied evidence is similar to the approved ones.
RWE in the Age of Value-Based Outcomes
Rising healthcare cost are driving the paradigm shift from healthcare as a fee-for-service business to one that focuses onvalue-based outcomes. In that context pharmaceutical companies increasingly harness RWE to demonstrate value. RWE has become critical when seeking to enter into value-based contracts.
RWE in the Medical Device Space
RWE can be especially beneficial in the medical device space, where the cost of post-approval studies is rising fast. RWE generated from an already approved version of a medical device is very valuable when the company seeks approval of a newer version. Systematically collecting RWE can lessen the burden of post-market research.
RWE is here to stay with pharmaceutical and medical device companies investing or planning to invest heavily into RWE over the next few years. For the Medical Affairs departments, this means that they need to be prepared to generate and/or support RWE activities in the field and the MSL or HEOR/Payer Liaison teams are the logical groups to take on that task.