Horizon Scanning for Paroxysmal Nocturnal Hemoglobinuria in Brazil: Insights on Past Trends and Future Prospects
Author(s)
Ludmila P. Gargano, BPharm, MSc1, Lucas L. Tôrres, BPharm, MSc2, Diego Kashiura, BPharm2, Felipe G. Thies, BSc, PhD2.
1Postgraduate Program in Medicines and Pharmaceutical Services, Federal University of Minas Gerais, Belo Horizonte, Brazil, 2Novartis, São Paulo, Brazil.
1Postgraduate Program in Medicines and Pharmaceutical Services, Federal University of Minas Gerais, Belo Horizonte, Brazil, 2Novartis, São Paulo, Brazil.
Presentation Documents
OBJECTIVES: Conduct a horizon scan for Paroxysmal Nocturnal Hemoglobinuria (PNH), evaluate the timeline from US regulatory approval to Brazilian approval, examine the timeline from regulatory approval to reimbursement decisions in Brazil, and investigate the reimbursement status in the UK for drugs under evaluation in Brazil.
METHODS: We identified PNH technologies through an advanced search in the Cortellis database for Phase 3 studies. Regulatory approval dates for PNH therapies were verified on the FDA and ANVISA websites. Reimbursement decisions were obtained from the Conitec website in Brazil, and the reimbursement status in the UK for drugs not yet approved in Brazil was verified on the NICE website.
RESULTS: We identified 10 treatments for PNH, six of which had FDA approval: eculizumab (2007, intravenous C5 inhibitor), ravulizumab (2018, intravenous C5 inhibitor), pegcetacoplan (2021, subcutaneous C5 inhibitor), iptacopan (2023, oral factor B inhibitor), danicopan (2024, oral factor D inhibitor used with eculizumab or ravulizumab), and crovalimab (2024, intravenous/subcutaneous C5 inhibitor). At the time of the study, only eculizumab, ravulizumab, and pegcetacoplan had regulatory approval in Brazil, while iptacopan, danicopan, and crovalimab were still under assessment. The time from FDA to ANVISA approval was 3,650 days for eculizumab, 255 days for ravulizumab, and 802 days for pegcetacoplan. The time from ANVISA approval to Conitec submission was 394 days for eculizumab, 1,374 days for ravulizumab, and 266 days for pegcetacoplan. The average time from submission to Conitec's final recommendation was 262 days, with positive recommendation for eculizumab and ravulizumab. The three drugs under ANVISA assessment in Brazil received NICE recommendations in 2024: iptacopan (September), danicopan (October), and crovalimab (November).
CONCLUSIONS: Recent cooperation between ANVISA and the FDA promises to accelerate drug approvals in Brazil. Three new therapies, including an oral monotherapy currently under ANVISA evaluation, could significantly improve pharmaceutical services by enhancing distribution and addressing geographical disparities.
METHODS: We identified PNH technologies through an advanced search in the Cortellis database for Phase 3 studies. Regulatory approval dates for PNH therapies were verified on the FDA and ANVISA websites. Reimbursement decisions were obtained from the Conitec website in Brazil, and the reimbursement status in the UK for drugs not yet approved in Brazil was verified on the NICE website.
RESULTS: We identified 10 treatments for PNH, six of which had FDA approval: eculizumab (2007, intravenous C5 inhibitor), ravulizumab (2018, intravenous C5 inhibitor), pegcetacoplan (2021, subcutaneous C5 inhibitor), iptacopan (2023, oral factor B inhibitor), danicopan (2024, oral factor D inhibitor used with eculizumab or ravulizumab), and crovalimab (2024, intravenous/subcutaneous C5 inhibitor). At the time of the study, only eculizumab, ravulizumab, and pegcetacoplan had regulatory approval in Brazil, while iptacopan, danicopan, and crovalimab were still under assessment. The time from FDA to ANVISA approval was 3,650 days for eculizumab, 255 days for ravulizumab, and 802 days for pegcetacoplan. The time from ANVISA approval to Conitec submission was 394 days for eculizumab, 1,374 days for ravulizumab, and 266 days for pegcetacoplan. The average time from submission to Conitec's final recommendation was 262 days, with positive recommendation for eculizumab and ravulizumab. The three drugs under ANVISA assessment in Brazil received NICE recommendations in 2024: iptacopan (September), danicopan (October), and crovalimab (November).
CONCLUSIONS: Recent cooperation between ANVISA and the FDA promises to accelerate drug approvals in Brazil. Three new therapies, including an oral monotherapy currently under ANVISA evaluation, could significantly improve pharmaceutical services by enhancing distribution and addressing geographical disparities.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA36
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases