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HTA outcomes for an in-development Orphan Drug

Client Challenge

Identifying and mitigating key gaps in evidence from an EU HTA perspective.

A biotech were about to enter into a phase 3 trial for an OD targeting particular benign giant cell tumours that are severely debilitating.

While regulatory feedback had been sought for the pivotal trial design, it was unclear how the evidence package would be received by payers.

P4A Solution

P4A advised a parallel approach to evidence acquisition using both formal and informal consultations.

P4A conducted analogue analysis and generated a hypotheses using the evidence shared by the client. The recommended consultations then took place:

  • Informal payer advice - P4A’s Orphan Oncology
  • Formal scientific advice - G-BA

P4A then created a mitigation strategy and action plan.

The Successful Outcome

  • Altered hierarchy of endpoints with more emphasis on physical functioning and symptomology improvement
  • PRO plan was streamlined to 2 payer relevant instruments (from 6 instruments)
  • Amended the stop criteria in the trial to focus on functioning
P4A showed our team that quality over quantity is a better approach to evidence gathering.
Global Program Leader
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We are award winning global experts in access for orphan drugs and Cell and Gene Therapies.

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