Perspectives

A global biopharmaceutical company wanted to understand how influential stakeholders within the online and offline landscape for a specific type of cancer are currently engaging and influencing the wider community. Specifically, the team sought to identify who is driving discussions on- and off-line, and what those discussions look like – in order to inform scientific story evolution and optimize communications programming through engagements and activations.

Drug development is changing, and guidance around the evaluation and approval of new treatments must change with it. The drive to develop medicines for conditions with the most severe unmet need – often rare and with no established or effective standard of care – frequently requires manufacturers to launch technologies based on evidence characterized by non-randomized trial designs with small sample sizes and limited follow-up. Of course, such evidence leads to regulatory and payer decision makers facing a high level of uncertainty when assessing the magnitude of the novel technology’s relative treatment benefit. If we are to accelerate access to transformative therapies, then we must acknowledge that greater flexibility in assessment and payment models is needed to address this uncertainty.