survival of ovarian cancer (OC) in the EU. The inputs were fed into the DICE model and prevalence over time was evaluated and compared with EMA submissions. Results: A stable OC prevalence of 4.1/10,000 was estimated for the EU and compared with the varying rates submitted to EMA (2.3-4.9/10,000). Multiple scenarios were tested. For example, if incidence was increasing by 2% per year post-2002, prevalence would reach the 5/10,000 cutoff in 2012. Conclusions: The DICE epidemiological model can produce a robust prevalence estimate by synthesizing available data from different sources and allowing for multiple scenarios to be tested. This model can assist sponsors and the EMA in their review by introducing a transparent and flexible tool which can be used as a standard for orphan designation submissions across multiple diseases.
Introduction:
Primary immune thrombocytopenia is a rare autoimmune disease characterised by a decreased platelet count resulting in an increased risk of bleeding events and even life-threatening haemorrhages. Thrombopoietin receptor agonists (TPO-RAs) are the standard of care second-line therapy for adult patients with chronic immune thrombocytopenia. The first TPO-RAs approved and reimbursed in Italy, eltrombopag and romiplostim, while effective, pose some issues in terms of safety (e.g., hepatotoxicity) or general management (e.g., dietary restrictions). Avatrombopag, an effective and well-tolerated TPO-RA, was recently granted reimbursement.
Methods:
A 3-year (2023–2025) budget impact analysis (BIA) was conducted to estimate its impact on the Italian National Health Service (NHS). Two scenarios were compared, of which one represents the current situation, without avatrombopag, and the other provides for an increasing market share of avatrombopag (up to 26.6%).
Results:
BIA shows that the increase in the use of avatrombopag correlates with savings for NHS: in the first year, saving would be €1,300,564, increasing to €2,774,210 in the third year, for a total of €6,083,231 over the 3-year period. The sensitivity analysis confirmed these savings in the scenario with avatrombopag.
Conclusions:
Based on this BIA, the introduction and reimbursement of avatrombopag is an efficient and advantageous choice for the Italian NHS.
European country can vary. Nivolumab has full reimbursement in accordance with the Marketing Authorisation in 4 out of 8 indications in England, and 2 in Scotland and France. In comparison, pembrolizumab is reimbursed with restrictions in England in all indications examined, full reimbursement in 1 indication in Scotland and 2 in France. Scotland also reimburses pembrolizumab for patients with Hodgkin lymphoma whereas access is denied in England and France; conversely England is the only country examined that has approved funding for patients with urothelial carcinoma. Furthermore, access to newer therapies such as atezolizumab is only currently available in England. CONCLUSIONS: Despite PD-1/PD-L1 inhibitors conferring a potentially significant improvement in patient outcomes this study highlights that patient access to these treatments is highly variable across Europe.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.