Abstract

---

Challenge

A mid-size US-headquartered biotech with a molecule in an orphan indication in Phase 2 sought EU HTA input to Phase 3 design and guidance on HTA submission with a conditional marketing authorisation. Focus areas were level of data uncertainty, validation needs and post-launch evidence requirements.

Solution

Ascenian worked across the client’s functional areas teams to align on strategic approach (e.g., parallel advice vs. advice from individual HTA agencies), facilitating prioritisation of questions, identifying scientific and business risks, aligning evidence and rationale for questions, and developing a briefing book which all client team members approved. Moving to consultation of individual HTA agencies, we facilitated pre-meeting interaction with HTAs and local regulators, and we executed a ‘challenge board’ comprised of former HTA and regulatory experts from Ascenian’s Professional Learning Network, for the client to rehearse and validate the briefing book and questions. The 5-month project allowed regular face-to-face client and Ascenian team interaction.

Result 

The most important result was that the client obtained the feedback needed, managed risks and felt invigorated in moving forward. The noted critical difference was the opportunity to challenge questions before the meeting with the Ascenian Professional Learning network – ‘It reassured us and helped us to speak confidently at the actual meeting. We could fully concentrate during the meeting to get the answers we needed. Thanks!’