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More case studies

MODELLING PRICE POTENTIAL ACROSS MULTIPLE COMMERCIAL SCENARIOS

 

  • Product was a subcutaneously-administered, anti-FXIIa monoclonal antibody, indicated as LTP treatment of HAE
  • As Phase 2 clinical trials for the product were being finalised, client wanted to understand the pharmacoeconomic value of the product, and drivers thereof, to evaluate price potential in cost effectiveness-driven markets
CHALLENGE
  • A flexible early CEM was required to reflect the uncertainty in the data at the time, and to enable exploration of the many possible scenarios arising from the finalisation of the data
  • Clinical data was limited for the very small patient population, so robust assumptions were required where data was missing
  • Two models were available for other drugs in the indication, so the early CEM needed to explore assumptions made in each of these
Action
Respecting the technical:
  • Developed an early cost-effectiveness Markov model using FIECON Model Builder to estimate the cost per QALY of the product versus other LTP treatments from a health system perspective over a lifetime time horizon
  • The model allowed users to explore the drivers of cost-effectiveness by changing a range of model settings, including: structural settings (e.g. treatment sequence, level of treatment usage, discontinuation), population sub-groups, and cost and clinical input source
  • A critical appraisal of comparator models informed the testing of key assumptions for which precedent existed in HTA submissions
Delivering the practical:
  • One-way sensitivity analysis and value of information analyses were conducted in response to the limited data, to provide targeted recommendations around where client resources would be best directed for future evidence generation
  • The model was populated using available clinical data sourced from the client and pivotal trial evidence for key comparators
result
  • A robust early CEM was developed to meet standard HTA requirements and had a flexible design allowing local affiliates to adapt the model with ease to meet local requirements
    A executive report in PPT was prepared which:
    • 1) outlined the main drivers of cost-effectiveness for the product, and;
    • 2) recommended areas for future evidence generation
Acronyms: CEM - cost-effectiveness model; HAE - hereditary angioedema; HTA - health technology appraisal; LTP - long term prophylaxis; PPT - PowerPoint presentation; QALY - quality-adjusted life year