Leveraging RWE early is critical for emerging biopharma companies
Barbara Arone, vice president, real world solutions, IQVIA, examines the ways biotech and emerging biopharma companies can effectively apply real world evidence (RWE) early in the product lifecycle while staying within their resource limits.
Key insights:
- With the rise in precision medicine, RWE is a 'must have' that helps to contextualise clinical research and aid in the understanding of treatments being developed.
- RWE can help tell a story, by identifying the target patient population, establishing a baseline of their disease state and providing insight into sites and treatment centres that would be a fit for the trial.
- A way biopharma and biotech companies can address the downsides of each type of research is to combine both primary and secondary data on the same patients.
The need for real world evidence (RWE) is rapidly growing in the product lifecycle, especially in drug development for rare diseases. Using RWE allows biotech and emerging biopharma organisations to understand the patient population on a more granular level and provide compelling evidence for stakeholders such as regulators, payers, patients and even the investor communities. With the rise in precision medicine, RWE is no longer a 'nice to have' but a 'must have' that helps to contextualise clinical research and aid in the understanding of treatments being developed.
Adoption of RWE early in the product lifecycle isn’t isolated to specific regions, but rather has become a global trend. In recent years, European regulators began encouraging the use of real world data (RWD) for regulatory decision making. The European Medicines Agency’s big data task force recognised the benefits from the rational use of RWD for approval and monitoring of drug efficacy and safety.
The power of storytelling
When it comes to engaging with investors, delving into the technical aspects of pharmaceutical development isn’t always the best way to gain support. As in most industries, especially where human health and safety is concerned, the ability to tell a story can be the most powerful tool. With the amount of RWD that exists, and the emergence of technology to utilise it, biotech and emerging biopharma companies are in a great position to tell a compelling story. The key to refining that story is understanding what information is available and where its use will be the most impactful.
Clinical design strategy using RWE
One of the critical roles RWE can play in the biopharma industry is to help tell the disease story early in the study design phase. For example, when conducting natural history studies, RWE can help organisations build a foundation of information during the trial development phase. It can help identify the target patient population, establish a baseline of their disease state, provide insight into sites and treatment centres that would be a fit for the trial and help identify specific biomarkers and endpoints of interest.
The use of RWE has also been successful in label expansion studies, by allowing researchers to prove safety and efficacy of off-label drug use in specific populations when justified, without the need for a randomised clinical trial. Each organisation has unique priorities and resource capabilities, so for emerging biopharma companies to successfully utilise RWE early in the product lifestyle they will have to think strategically about the resources they have available.
How to address resource challenges
Technology to maximise the benefits of RWE has become more affordable and accessible, but the reality is that many biotech and emerging biopharma companies will have constraints on the amount of time and money they have available. While secondary data collection introduces the challenge of missing critical data because it isn’t routinely collected, primary data comes with a higher resource burden.
A way these companies can address the downsides of each is to combine both primary and secondary data on the same patients. Early in the research design process, emerging biopharma companies can pull whatever secondary data information that is applicable and identify the areas where primary data collection will be necessary. This will help reduce the amount of time and money that is needed for primary data collection.
Next steps
In order to be successful in the biotech and emerging biopharmaceutical industry, utilising RWE is increasingly becoming a necessity, especially early in the product lifecycle. Being able to articulate a compelling patient story is critical to secure stakeholder buy-in and doing so without RWE leaves a big gap in the narrative. Even among biotech and emerging biopharma companies with resource restrictions, strategic planning will allow the use of RWE in a way that makes sense for their research goals.