Looking Ahead: RWD/RWE Opportunities Loom Large
Recent actions by the FDA and the global response to COVID have accelerated interest in real-world data (RWD) and real-world evidence (RWE). Often for use in clinical trial design and execution via distributed clinical trials, this de-identified patient data is quickly becoming an in-demand commodity, for payer and life sciences companies.
Data owners (hospital systems and payers) and data stewards (EHR and analytics vendors) are accelerating the access to said data as a response to the market’s demand. Aggregating these datasets can lead to more rapid targeting of rare, “orphan” diseases, which is an attractive market due to a number of federal incentives. Ultimately, the commoditization of these datasets will occur quickly—within five years.
- Due to government action, the COVID pandemic, and other factors, the opportunities in the patient data market will continue to make it an important sector in healthcare.
- While randomized clinical trials are still the main method of receiving approval for a therapeutic, expect RWE and RWD to play a bigger part in the coming days.
- Despite growing usage and potential of these datasets, the right go-to-market strategy is still up for debate, however, the AI/ML enhancement of analytics tools to harness them shows the most promise.
Creating Value and Boosting Usage
Payer claims data has been used for many years by both payers and life sciences companies. The dominant use case for payers has been measuring the value/efficacy (RWE) of a given therapeutic or medical device, enabling them to negotiate pricing and create clinical pathways; life sciences use the data similarly, to justify pricing based on a given therapeutic’s outcome.
This has led to the creation of health economics and outcomes research (HEOR) from a variety of companies, many of whom are also data stewards. As value-based care models continue to rise, life sciences companies will be under increasing pressure to continue justifying pricing, and HEOR will continue as an important market.
In the landmark 21st Century Cures Act, Congress instructed the FDA to adopt a more flexible approach to RCTS that would lead to safe testing of new therapeutics while accelerating time to market. This led to new guidelines from the FDA, providing clear instruction to life sciences companies on what to expect during study design, clinical trials and importantly, the approval of new therapeutics. In 2019, the breast cancer drug Ibrance was approved for men based only on RWE alone, gathered from off-label use of the drug, marking the first time that RWE replaced RCT to offer a new drug to an underserved population.
Usage of RWE/RWD was further boosted in 2020, when the global pandemic struck. Care protocols and outcomes were widely shared, as well as RWE largely replacing RCT as providers scrambled to find ways to treat the new virus. Lastly, vaccine development required a distributed clinical trial methodology as hospitals became overwhelmed, and the data explosion that resulted was the final push to augment the rapid rise of RWD/RWE.
Surveillance, patient safety, and efficacy of a given therapeutic will continue to be important use cases for RWD/RWE. However, a rising market is the use of said data for drug development and approval, especially “orphan drugs,” or drugs for rare diseases. This can be a way for companies to advance the pace of FDA approval for a therapeutic they want to bring to market.
The value of these datasets can be seen in Roche’s 2018 acquisition of Flatiron Health (also covered in our 2019 Precision Medicine report, now free), a startup with a vast oncology dataset that made the acquisition worth a stunning $1.9B. As one of the leading oncology life sciences companies, Roche saw an opportunity to significantly advance its development pipeline and shorten time to market.
As it becomes clear how valuable these datasets can be, there are also a growing number of companies looking to leverage the ability to provide this kind of data to life sciences companies. Cerner, Health Catalyst, Tempus, TriNetX, and Veradigm are all examples of entities that are active in the clinical data market today. This is only clinical data, not to mention the companies that primarily leverage claims data in a similar way, e.g. Anthem, IBM, IQVIA, and so on.
Although the discussion over usage of patient data has been a hot topic in the past, expect that it will only continue to be at the forefront as more datasets become available to life sciences companies, and public awareness grows. The RCT gold standard will remain for now, but RWD/RWE is quickly becoming a viable and valuable alternative in drug development and approval, especially for rare diseases.
While the market is hot right now for those companies able to leverage de-identified patient data, the increasing number of entities offering clinical datasets to the market will lead to commoditization and subsequent fall in pricing. This will squeeze the margins of companies who want to simply pursue a data monetization strategy.
As the market evolves, the question of the right go-to-market strategy has not been settled. The traditional high-touch services approach is clashing with the self-service model that uses sophisticated analytics tools. We believe that said sophistication combined with the emergence of AI/ML will lead to this approach being the victor by 2030.