OBJECTIVES

To evaluate the budget impact of ruxolitinib incorporation for patients with primary or secondary myelofibrosis (MF), classified as intermediate-2 or high risk, according to the International Prognostic Scoring System (IPSS), and ineligible for hematopoietic stem cell transplantation in the Brazilian public healthcare system (SUS).

METHODS

A five-year BIA was developed to estimate incremental cost of ruxolitinib incorporation in SUS. A prevalence-based model was considered to estimate MF population, calculated by retrospective data from a Brazilian reference institution (Hematology Department of Hospital de Base do Distrito Federal), considering collected data of treated MF patients between 2014 and 2018 applied to total population in the covered region (Distrito Federal - DF) per year. For extrapolation to total Brazilian population, the resulting DF prevalence of 0.69/100,000 inhabitants was applied to population projection in Brazil for the next five years. Additionally, 49% patients were assumed to have intermediate-2 and high risk IPSS (Cervantes, 2009), and 11% were assumed to be ineligible to ruxolitinib due to platelet counts lower than 50x10^9/L (Devos, 2015). For the scenario without ruxolitinib, 100% of patients were assumed to use reimbursement procedures for MF therapy in SUS. Market-share of ruxolitinib inclusion ranged from 30% (year 1) to 50% (year 5). Cost of treatment consisted of official Brazilian maximum government sales price and DATASUS database.

RESULTS

Approximately 640 patients per year were estimated to be eligible to MF treatment in SUS. In terms of cost, ruxolitinib incorporation resulted in an incremental cost of BRL45.78 million in the first year and BRL78.34 million by the end of the fifth year.

CONCLUSIONS

Eligible patient population is restricted due to MF low prevalence. For this reason, incorporation of ruxolitinib leads to a predictable and limited budget impact to SUS, especially in the context of absence of alternative therapies with benefits in quality of life and overall survival.