Real-world evidence (RWE) generation continues to be the driver for decision-making by regulators, payers, and providers, and has assumed a progressively greater importance over time to the market access landscape. Agencies are using RWE not only to make reimbursement decisions, but also to monitor post-market safety and develop guidelines and decision support tools. RWE has become a key element in demonstrating value for therapeutics—especially in rare diseases where evidence gaps are common due to the very low number of patients affected. Planning for RWE is best early in the pre-launch period as it can allow for the unified integration of health economics and outcomes research (HEOR) to support the value of a product and market access strategy.

The RWE path:

• A strong aspirational Value Story can help inform evidence generation strategy—including the need for RWE—systematic literature review (SLR) can be used to determine whether support for aspirational messages exist or if evidence generation is needed to fill key data gaps
• Outcomes research using RWE may inform the choice of endpoints and improve trial design
• Patient reported outcomes or medical chart review may help clarify the unmet need in the population
• Comparative clinical efficacy in rare disease can be difficult and may be supported by the development of a synthetic control arm for treatment comparison

IMAC has considerable experience with comprehensive RWE strategy to bring together innovative ideas and technology solutions to identify, generate, and communicate the efficacy/safety and cost-effectiveness evidence and successfully bring your product to market.