About the economic interest of the compassionate use of innovative treatments

Italian researchers and a Roche laboratory representative jointly analyzed the economic impact of compassionate use protocols for some innovative health products:

  • two of the eleven protocols studied concerned infantile spinal muscular atrophy (SMA) and risdiplam in particular, the others were linked to cancer and multiple sclerosis,
  • the SMA protocols respectively concerned children with type 1 (28 patients) and the other with type 2 (31 patients),
  • taking into account the expenses falling to the manufacturer (the drug itself) but also the indirect costs induced, they conclude that all the protocols prove to be profitable from the point of view of the payer, the overall saving representing approximately 50 millions of euros.


The economic impact of compassionate use of medicines. Jommi C, Pantellini F, Stagi L, et al. BMC Health Serv Res. 2021 Dec 4;21(1):1303.