Budget impact analysis of medicines: estimated values versus real-world evidence and the implications

Abstract

Objectives: Budget Impact Analyses (BIA) of medicines helps managers in promoting health systems' sustainability when assessing the role and value of new medicines. However, it is not clear whether BIAs typically underestimate or overestimate the impact on real-world budgets. This retroactive analysis seeks to compare estimated values obtained by a BIA and Real-World Evidence (RWE) to guide discussions. Methods: The estimated values were obtained through a BIA concerning the incorporation of adalimumab for the treatment of Rheumatoid Arthritis into the Brazilian Unified Health System (SUS) carried out retroactively and per international guidelines. RWE data was extracted from SUS computerized systems. We subsequently compared the number of treatments, costs, and Incremental Budget Impact (IBI). Results -The total number of treatments was underestimated by 10% (6,243) and the total expenditure was overestimated by 463% (US$ 4.7 billion). In five years, the total difference between the estimated values and real IBI reached US$ 1.1 billion. A current expenditure of US$ 1.0 was observed for every US$ 5.60 of estimated expenditure. Conclusion -The higher estimates from the BIA might cause decision makers to be more cautious in the introduction of the new drug to reduce the opportunity costs for other interventions.

Impacto orçamentário da incorporação do rituximabe para o tratamento de leucemia linfocítica crônica no SUS

“…Faleiros et al (Faleiros et al, 2022) compararam os valores de uma AIO da incorporação do adalimumabe para o tratamento de artrite reumatoide com as evidências de mundo real, e os resultados do estudo demonstraram que os custos estimados para cinco anos foram 463,3% maiores do que os custos de mundo real. Esse estudo também considerou que dois fatores foram os principais causadores da divergência: a estimativa incorreta da população e a variação de preços do medicamento ao longo dos anos (Faleiros et al, 2022). A AIO demonstra apenas a estimativa dos gastos para o detentor do orçamento em função da adoção de uma alternativa terapêutica, não sendo de sua abrangência verificar os benefícios para a saúde e para a economia.…”

Assessing the Accuracy of Sales Forecasts Submitted by Pharmaceutical Companies Applying for Reimbursement in Austria

“…Also, this applies not only to pharmaceutical sales forecasts submitted by market authorisation holders to payers. In a budget impact analysis from Brazil, the estimated costs for adalimumab for the treatment of rheumatoid arthritis were overestimated by 463% (US $ 4.7 billion) due to uncertainties in the number of patients treated and to variations in prices (Faleiros et al, 2021). In a study from Thailand, the theoretically estimated constant budget impact per patient and year for the treatment of lung cancer with pemetrexed was initially overestimated by 34% (US $ 11,881 vs. 8,834) (Sooksriwong and Chanjaruporn, 2011).…”

“…This requires standardization of methodology, e.g., based on the ISPOR report (Sooksriwong and Chanjaruporn, 2011). We are aware of the fact that this per se cannot solve the problem (Faleiros et al, 2021). However, valid international, cross-system comparisons may be useful to provide answers to the following non-exhaustive list of questions: are prevalence data consistently inaccurate?…”

Challenges with PCSK9 Inhibitors Use in Asia Given Real-World Data Implications

“…Careful cost-effective analysis helps more effectively balance intervention costs with savings through proactive interventions and increased quality of life benefits. In the case of PCSK9 inhibitors, some researchers suggested that overestimates of budget impact by healthcare system decision makers contributed to current reimbursement structures 8,9) . And prelaunch predictions of the budget impact of newly developed drugs were reported to be considerably overestimated versus the actual sales; furthermore, PCSK9 inhibitors had a higher than average prediction "error" 8) .…”