CONSENSUS ON BARRIERS AND UNMET NEEDS IN HEMOPHILIA CARE IN BRAZIL: A MODIFIED DELPHI STUDY
Author(s)
Margareth C. Ozelo, MD, PhD1, Franciele Iachecen, MSc2, Bruna Webler, MS2, JULIA LIMA, MSc2, Ana Dantas, MD3, Mariana Alves, PhD3, Ana Karolina Nunes, PhD3, Rodrigo Alexandre, PharmD, MSc3, Daniela V. Pachito, MD, PhD3, Ieda S. Pinto, MD4;
1University of Campinas, Hemocentro UNICAMP, Campinas, Brazil, 2IQVIA Solutions, Sao Paulo, Brazil, 3Pfizer Brazil, Sao Paulo, Brazil, 4Federal University of Para, Belem, Brazil
1University of Campinas, Hemocentro UNICAMP, Campinas, Brazil, 2IQVIA Solutions, Sao Paulo, Brazil, 3Pfizer Brazil, Sao Paulo, Brazil, 4Federal University of Para, Belem, Brazil
OBJECTIVES: Despite therapeutic advances, hemophilia care in Brazil still relies on the prophylactic treatment with coagulation factors for most patients and continues to face significant challenges related to treatment burden, access barriers, suboptimal adherence, and residual disease. This study sought consensus among Brazilian hematologists regarding unmet needs, barriers to care, and desired features of new technologies for hemophilia A and B.
METHODS: A modified Delphi panel was conducted with 12 hematologists from specialized public health centers across all five geographic regions of Brazil. Experts reviewed 39 statements across five domains: unmet needs, residual disease and adherence, extrinsic barriers, intrinsic barriers, and valued attributes of novel therapies. Consensus was defined as ≥80% agreement, with three rounds of evaluation.
RESULTS: Of the 39 statements, 23 reached consensus after three rounds. Persistent unmet needs were identified for all clinical profiles, including patients with and without inhibitors. There was agreement on consolidating clinical protocols into a single national document (83.3%). Key extrinsic barriers included long travel distances (83.3%), social vulnerability (83.3%), limited access to rehabilitation (91.7%) and acute care (83.3%), insufficient specialized centers (90%), failures in referral systems (90%), and lack of specialized emergency support (100%), scarcity of trained professionals (91.7%), poor care coordination (83.3%), and delayed adoption of new therapies (91.7%). Intrinsic barriers included venous access difficulties in children (100%), lack of social support, infusion-related discomfort, mental health issues (80-91.7%), challenges in transitioning to self-infusion (91.7%), and functional limitations in older adults (83.3%). Desired features of new technologies included subcutaneous administration (100%), reduced dosing frequency (100%), and no need for laboratory monitoring (83.3%).
CONCLUSIONS: High consensus among specialists highlights priority barriers and unmet needs in hemophilia care in Brazil, as well as attributes of innovative therapies to guide health policy.
METHODS: A modified Delphi panel was conducted with 12 hematologists from specialized public health centers across all five geographic regions of Brazil. Experts reviewed 39 statements across five domains: unmet needs, residual disease and adherence, extrinsic barriers, intrinsic barriers, and valued attributes of novel therapies. Consensus was defined as ≥80% agreement, with three rounds of evaluation.
RESULTS: Of the 39 statements, 23 reached consensus after three rounds. Persistent unmet needs were identified for all clinical profiles, including patients with and without inhibitors. There was agreement on consolidating clinical protocols into a single national document (83.3%). Key extrinsic barriers included long travel distances (83.3%), social vulnerability (83.3%), limited access to rehabilitation (91.7%) and acute care (83.3%), insufficient specialized centers (90%), failures in referral systems (90%), and lack of specialized emergency support (100%), scarcity of trained professionals (91.7%), poor care coordination (83.3%), and delayed adoption of new therapies (91.7%). Intrinsic barriers included venous access difficulties in children (100%), lack of social support, infusion-related discomfort, mental health issues (80-91.7%), challenges in transitioning to self-infusion (91.7%), and functional limitations in older adults (83.3%). Desired features of new technologies included subcutaneous administration (100%), reduced dosing frequency (100%), and no need for laboratory monitoring (83.3%).
CONCLUSIONS: High consensus among specialists highlights priority barriers and unmet needs in hemophilia care in Brazil, as well as attributes of innovative therapies to guide health policy.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD92
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)