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.
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.
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.
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.
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.