Budget impact analyses for the treatment of rare diseases are especially important for the sustainability of health systems due to high treatment costs and uncertainties in target population estimates. The objective of this work is to analyze the elements that influence discrepancies between predicted and observed budget impacts for enzyme replacement therapies for rare diseases in Brazil’s public health system.

All enzyme replacement therapies for rare diseases evaluated by the National Committee for Health Technology Incorporation in the Brazilian Public Health System (Conitec) and with at least one year of use were included. For each technology, the following were identified: number of patients, median patient weight, annual quantity of medication, unit price, and budget impact. The attributes were compared between previous estimates and real-world observation after use. The data sources were publicly accessible administrative databases and Conitec technical reports.

Five technologies were selected: elosulfase alfa, alglucosidase alfa, idursulfase, laronidase, and galsulfase. In the first year, the difference between the estimated and the observed number of patients treated was up to 15 percent lower or higher for four technologies, but with monthly fluctuation throughout the year. The median weight of users was between 23 percent and 468 percent higher for three technologies. The observed price was as expected, with variations between three percent lower and 14 percent higher. The quantity of medicines used was lower (between 39% and 46%) than expected for all technologies. The observed budget impact was 37 percent to 47 percent lower than estimated.

Real-world budget impact was lower than expected for all technologies. The main cause of discrepancies was the estimate of the annual amount of medication, which did not consider gradual adherence and discontinuation of treatment. This highlights the need to review the budget impact methodology for rare diseases, forecasting monthly market share and treatment discontinuation rate.

Psoriasis is an immune-mediated chronic inflammatory disease that can affect the skin, joints, and nails. The treatment of the disease is offered by the Brazilian Public Health System (SUS) in accordance with the guidelines of the Ministry of Health. The aim of this study was to analyze real-world data (RWD) on the implementation and use of monoclonal antibodies (mAbs) for the treatment of psoriasis in the SUS.

This is a descriptive study that used national administrative data on drug dispensing from the Open Room for Health Intelligence Situation (SABEIS-SUS) from October 2019 to December 2021. Adult individuals (≥18 years) with vulgar (L40.0), generalized pustular (L40.1), gutata (L40.4) and other (L40.8) psoriasis who used the mAbs adalimumab, etanercept, risankizumab, secukinumab and ustekinumab were included.

The year of implementation of mAbs for the treatment of psoriasis in the SUS was October 2019 (adalimumab, etanercept and secukinumab) and May 2020 (ustekinumab). Risankizumab was implemented in April 2022. The number of individuals using mAb grew from 366 in 2019 to 10,146 in 2021. In 2019, 2020 and 2021, the proportion of individuals using each mAbs was 62.3 percent, 46.2 percent and 35.4 percent (adalimumab), 7.9 percent, 3.3 percent and 2.7 percent (etanercept), 29.8 percent, 33.8 percent and 30.5 percent (secukinumab), 0 percent, 16.7 percent and 31.4 percent (ustekinumab), respectively.

The number of mAbs users has greatly increased from 2019 to 2021, which may indicate a successful implementation of the psoriasis treatment in SUS. Most individuals used adalimumab in the year of the first implementation. However, the proportion of users of this mAb has greatly decreased after the implementation of ustekinumab. This reduction should not be so expressive since adalimumab and ustekinumab are recommended in different lines of treatment. The low proportion of etanercept use may be due the fact the medication is indicated for individuals up 18 years of age. This study provides important real-word evidence for monitoring the implementation of mAbs for psoriasis treatment in Brazil.

In Brazil, the incorporation or disinvestment of health technologies into the Unified Health System (SUS) are advised by the National Committee for Health Technology Incorporation (Conitec). Despite the thorough evaluation carried out by Conitec, the results measured after implementation do not always reflect the economic and clinical impact expected from the incorporation. Thus, real-world evidence (RWE) is essential for monitoring health technologies. The aim of this study was to report how Brazil is using the RWE to obtain additional information about the incorporated technologies.

Actions related to the use of RWE for monitoring of technologies incorporated into the SUS were described. The period evaluated was between 2012 and 2022.

The first Conitec recommendation in which the use of real-life data in the decision-making process was evidenced occurred in 2016. Administrative data from a cohort of patients identified that beta-interferons for Multiple Sclerosis were less effective than the other drugs used in the Brazilian public system. A further eight reports have been published assessing the performance of technologies using administrative data.

Another strategy for RWE generation was through the funding of primary studies, highlighting a study with 21 rare diseases and another one to evaluate Zolgensma gene therapy, acquired through court for Spinal Muscular Atrophy. Both studies are ongoing and aim to evaluate the effectiveness, safety, adherence, and cost of medications in the evaluated diseases. Conitec is also following studies in RWE financed by pharmaceutical companies to evaluate effectiveness for incorporated technologies subject to reassessment. Additionally, managed access arrangements have been promoted for generating RWE when there is uncertainty about outcomes.

Real-world evidence from administrative data and clinical research allows monitoring after the implementation of technologies in the Unified Health System in Brazil. This makes it possible to reallocate resources in health and contribute for the system sustainability, in addition to generating data that reduce the uncertainties assumed at the time of incorporation.