Real World Evidence and Rare Diseases


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By Jeffrey Stewart. Discovered by Player FM and our community — copyright is owned by the publisher, not Player FM, and audio is streamed directly from their servers. Hit the Subscribe button to track updates in Player FM, or paste the feed URL into other podcast apps.

A striking life sciences trend has come into focus the past 12 months. Global regulators—often viewed as taking a conservative approach to transformative technology—have moved into the vanguard in discussions and guidance around real world data (RWD) and real world evidence (RWE).

It is not so much the case with payers. According to new research by Syneos Health on payer perceptions of RWE in rare diseases, many payers are not entirely receptive to RWE-based representations or health economic forecasts in sponsors’ dossiers or related conversations.

What accounts for payers’ hesitation? Michelle Leeds, Vice President, Risk and Reputation Management and Alastair MacDonald, Senior Vice President, Real World Late Phase, join the podcast to examine the issues from several perspectives based on conversations with payers, drug developers, regulatory experts and patient organizations.

For more detail on the Syneos Health research into payer perceptions, download the full report “Real World Value: Advancing Payer Understanding of RWE in Rare Disease.”

To hear more from Alastair MacDonald, check out Episode 031, “Real World Evidence in Europe.”

If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you’ll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision making and investment. You can find it all at

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62 episodes