P&MA Assessment to Support BD&L Decision-Making

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Trinity completed a P&MA assessment for a gene therapy in Leber hereditary optic neuropathy (LHON) to support BD&L decision-making.

Geographic Scope: US Flag

Client Situation

The client wanted to understand the current and future market landscape and the resulting pricing and access potential for a gene therapy asset in Leber hereditary optic neuropathy to support their BD&L decision-making.

Trinity’s Solution

Trinity conducted in-depth interview (IDI) discussions with payers and key opinion leaders (KOLs) to evaluate current treatment practices and price thresholds for LHON, and understand likely payer restrictions as a function of price.


  • Landscape assessment and analogue assessment across key markets
  • Payer & KOL moderator guides and research stimuli
  • Interim research findings summary
  • Detailed final research findings summary
  • Strategic business development and licensing (BD&L) recommendations and differentiation opportunities

Project Outcomes & Impact

The strategic guidance that Trinity delivered informed the client’s decision not to move forward with the acquisition of the LHON gene therapy asset.

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