Introduction Over the past few years economic stagnation
has made the challenge for sustainability an even more
complicated issue for public healthcare systems, also
affecting pharmaceutical pricing. The objective of this
study is to delve into the impact on decision-making
of the adoption of different effectiveness measures,
with a specific interest for the introduction of new drug
treatments into the market – including a significant
number of immunotherapies.
Methods The study has examined molecules for first-line
treatment of metastatic cancer, approved in Italy for public
reimbursement over the last ten years. Through statistical
regression, the analysis has researched the relation between
treatment cost, clinical benefits, potential market size
(expressed in terms of patients eligible for treatment) and
the year in which reimbursement was approved. Clinical
benefits have been expressed in terms of absolute and
relative increase of average and median OS and PFS.
Results Analysis has brought to light significant pricing
differences, both related to the endpoint taken into consideration
and to the measurement methods. The “confounding” effect of
the market size seems limited. The variable inherent to the year
of approval seems, on the contrary, significant.
Discussion This analysis suggests that it is worthwhile
carry out an in-depth reflection on the choice of the benefit
measures considered in the negotiation process: especially
considering the different capabilities of the measurements
themselves in grasping different mechanisms of action
of the drugs employed, that might induce different types
of patterns in patient responsiveness. The impact of the
chosen endpoint is very relevant for the “new generation” of
immunotherapy treatments
