Inform the evidence generation plan for a treatment for a chronic progressive disease

photo illustration of man typing on a laptop with colored charts and graphs overlaid

Early Cost-Effectiveness Model for Gene Therapy

VALUE TO CLIENT

Insights from the early cost-effectiveness model helped our client manage internal pricing expectations, identify studies to address key data gaps, and align on a patient-level simulation approach—serving as a through-line to a robust, evidence-based launch CEM.

OPPORTUNITY

Our client asked us to develop an early cost-effectiveness model (CEM) for a gene therapy to assess the target product profile, inform decisions around the target population, identify data gaps and the value of filling them, and provide economically justifiable price estimates for executive leadership for product investment.

CHALLENGE

The therapy was for a complex, genetic, chronic progressive disease with heterogeneous manifestations marked by pain crises, stroke, irreversible multisystem organ damage, and premature mortality. This diagnosis represents substantial effects on quality of life, socioeconomic outcomes, and caregiver burden. A comprehensive modeling approach was required to capture the potentially transformative impact of early treatment with a gene therapy.

OUR APPROACH

  • Our team developed a cohort-based early CEM with a lifetime time horizon reflecting age-based disease-related event, complication, and mortality risks.
  • We estimated the potential EJP of gene therapy in 2 indications, relying on assumptions for the hypothetical impact of gene therapy on events, complications, and mortality.
  • Our experts were able to identify the limitations of cohort-based approaches for this complex heterogeneous disease and recommended studies to generate evidence needed to inform a patient-level simulation approach. This approach would allow us to fully capture evidence of value of the gene therapy at launch and for HTA submissions.