How to Leverage Real-World Evidence for Market Access in Rare Disease

When working in rare diseases, clinical trial design is focused on an exceedingly small population. The clinical evidence in these cases often is based on open label, single arm studies that have a high degree of uncertainty due to the lack of randomization and direct comparison. The resulting data might fail to satisfy payers during price and reimbursement negotiations due to uncertainty about the treatment effectiveness. This is when real-world evidence (RWE) can make a major impact by illustrating disease burden, demonstrating value, defining market size and expectations, and acting as an external control arm to provide context for payers. 

Getting Started 

Base your strategy on understanding your patient population and their needs. Then identify what endpoints and outcomes you expect from the pivotal clinical trial. From there, identify potential gaps and what evidence you will need to mitigate them. Once you know what RWE you need, you can determine how you will obtain or generate it and pull it all together in an integrated evidence plan.

RWE will become a pillar of your overall market access strategy. It can help define your target population and their needs and help with not only price and reimbursement negotiations, but also your commercial strategy.  

Identifying Available Data Sources for Targeted Rare Diseases

A good way to identify the right data source for a targeted rare disease is to perform a RWD source mapping exercise by searching the existing literature to find all available RWD sources in a given geography, such as existing registries or population-based data sources. Combining secondary research with RWD source surveys or interviews, you gain insight into the data captured in the available sources and how well these sources may fit your needs.

Companies frequently need to invest in creating a patient registry to ensure that adequate data is captured with sufficient quality in a timely manner. Designing the registry in collaboration with the regulatory agencies, health technology assessment (HTA) bodies, and patients increases the likelihood that the data will inform regulatory and HTA decision-making.  

Next Steps

You must begin developing an integrated evidence plan early and take into account key stakeholders’ needs as well as evidence gaps. The bottom line is that in rare disease treatments, up-to-date and relevant RWE is fundamental to successful market access.

To learn more about using RWE to estimate targe rare disease populations and generate additional insights post-launch, start reading Navigating Market Access for Rare Diseases: The Potential of Real-World Evidence.