Objectives
To evaluate the public health 5-year impact of a new lipid-lowering drug on cardiovascular morbidity and mortality in France
To estimate the number of non-fatal and fatal CV events that could be prevented, and corresponding number of patients needed to treat (NNT) with ezetimibe to prevent one CV event over 5 years.
To evaluate the long-term public health impact of the drug in the real life, under a pricing agreement with the French HTA.
Methods & solution
Bridging concept combined real-world data collection with long-term outcome modelling :
the Model-Observation Bridging Study
The model accounted for the drivers of effectiveness and their dynamics over time.
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Non-interventional 48-month follow-up cohort conducted in hypercholesterolemic patients starting on the drug < 3 months at study entry, either as monotherapy or combined with statins.
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Prediction modeling using discrete event simulation with calibrated Framingham CV risk equations was applied to data from pivotal clinical trials on the drug and real-life data derived from the cohort.
Results
Predictions in Real World were validated by a small prospective study, leading to securing prices in France
Strong model performance and confirmed predictions in real-world, allowed to maintain prices in France.
LDL-C decreased in the first 12 months in LLT (lipid-lowering therapy) initiators, switchers (monotherapy), and combination therapy with a statin.
The corresponding predicted rate reductions of CV events (non-fatal and fatal) compared to no treatment or to a statin (combination therapy) were respectively 8, 2, and 12 per 1000 patients treated over 5 years, with a global NNT of 143 patients over 5 years.
Impact
Positive impact on sales volumes and prices in other countries
- These results, accounting for observed CV event rates, risk factors evolution over time and adherence to treatment in real life, were consistent with those from clinical trials.
- Significant positive impact on sales volumes and prices was observed in multiple other countries.