Cost-effectiveness of novel systemic therapies for advanced breast cancer: Global representativeness and influence of funding.

Felippe Lazar Neto Instituto do Câncer do Estado de São Paulo, University of São Paulo, São Paulo, SP, Brazil info_outline Felippe Lazar Neto, Marina Melo, Cassio Murilo Trovo Hidalgo Filho, Maria Cecilia Mathias, Laura Testa, Alessandro Campolina

Authors Felippe Lazar Neto Instituto do Câncer do Estado de São Paulo, University of São Paulo, São Paulo, SP, Brazil info_outline Felippe Lazar Neto, Marina Melo, Cassio Murilo Trovo Hidalgo Filho, Maria Cecilia Mathias, Laura Testa, Alessandro Campolina Organizations Instituto do Câncer do Estado de São Paulo, University of São Paulo, São Paulo, SP, Brazil, Sao Paulo State Cancer Institute (ICESP), São Paulo, Brazil, Instituto do Câncer do Estado de São Paulo, University of Sao Paulo, São Paulo, Brazil, Oncoclínicas, São Paulo, Brazil, ICESP-FMUSP, São Paulo, Brazil, Instituto do Câncer de São Paulo, São Paulo, Brazil Abstract Disclosures Research Funding No funding received None. Background: Approval of expensive novel treatments increases costs of cancer care and undermines access, particularly in low and middle-income countries. As the most incident neoplasm, breast cancer (BC) therapies have an enormous budget impact if incorporated. Therefore, cost-effectiveness (CE) studies are essential to guide such decisions. Methods: A systematic literature search of Medline, Scopus, and Web of Science from January 1 st 2012 to July 8 th 2022 was conducted to identify CE studies of tumor-targeted systemic therapies for advanced BC. Articles without CE (or utility) ratio calculations were excluded. Information on the country and drug studied, authors’ conflicts of interests (COI) and funding, and authors' conclusions were manually reviewed by two independent investigators. Results: Of 1103 screened records, 74 studies comprising 212 CE comparisons were included. Most studied scenarios were from high-income countries (70%, 141/212), with only 10% from Latin America and none from Africa. The most studied drug classes were iCDK4/6 (23%), anti-HER2 directed therapy (21%), anti-PD1/PDL1 (12%) and endocrine therapy (11%). In 46% of the comparisons, a new drug combined with standard of care was compared to placebo and/or standard of care, and in 37% the compared treatment were alternative same class therapies. Nearly 46% (97/212) had pharmaceutical industry funding or author’s COI associated with industry. Overall, 56% of authors’ conclusions were unfavorable. Pharmaceutical industry related-studies were associated with high-income countries (75% vs. 64%, p = 0.006), same-class drug comparators (52% vs. 33%, p = 0.047), and favorable conclusions (60% vs. 15%, p < 0.001). A combination of drugs (addition to standard of care) was associated with unfavorable conclusions (56% vs 28%, p < 0.001). Conclusions: Industry-related studies target high-income countries' scenarios and are more likely to have favorable conclusions. As expected, the addition of drugs to standard treatments (combinations) is less likely to be cost-effective. Government funding in low and middle-income countries is needed to decrease the current literature gap and provide stakeholders with proper information to make incorporation decisions.