Evaluating blinatumomab implementation in low- and middle-income countries: a study protocol

Abstract

Background The recent implementation of novel therapies has accelerated progress in pediatric cancer care. Despite the significantly poorer survival of patients in low- and middle-income countries (LMICs), administation complexities and other significant resource barriers have limited the translation of these novel therapies in these regions. This study aims to develop a model that can be used to support the implementation of novel therapies, such as blinatumomab (bispecific antibody therapy for B-cell acute lymphoblastic leukemia [B-ALL]) in LMIC centers, with the long-term goal of developing an implementation framework for similar future efforts. Methods In this study, mixed methods will be applied to understand the key contextual considerations that can be accounted for through a training program and prospectively designed implementation activities. The Consolidated Framework for Implementation Research will guide the activities related to implementation evaluation in parallel with a drug donation program. A multidisciplinary research team comprising high- and low-middle income healthcare professionals, industry, and implementation scientists has been assembled with the common goal of improving safe access to blinatumomab. To assess the factors affecting blinatumomab administration, semi-structured interviews with diverse collaborators and quantitative assessments of organizational characteristics will be conducted, together with quantitative and qualitative assessments of feasibility, acceptability, appropriateness, and cost of blinatumomab implementation. A quantitative assessment of stakeholder perceptions of different implementation strategies used as part of the multifaceted approach will also be performed. Finally, we will examine the key domains and processes used and construct the implementation roadmap for translation of novel therapies. Discussion This study will rigorously develop an implementation roadmap for translation of novel therapies in low-resource settings. The knowledge gained in the formative assessment will reveal the priority areas and key implementation strategies. Thereby, the resultant roadmap will facilitate future scale-out strategies for novel therapies in LMICs, thus increasing access, building capacity for management, and ultimately improving the care for children in LMICs.

Variations in global prices of chemotherapy for childhood cancer: a descriptive analysis

“…While the additional survival benefit of rituxmab in HICs has been modest, it is conceivable that the addition of targeted therapies, such as rituxumab for mature B-cell lymphoma or blinatumomab for B-ALL to lower intensity regimens in LMICs could result in a more significant survival benefit with lower treatment-related toxicity. 28 Using a comparative regimen approach, combined with increased transparency and graphically depicting price variations, advocates can use the data from this study to support market-shaping initiatives and ultimately increase access to novel therapies in low- and middle-income countries.…”

“…Novel therapies introduced in HICs are rarely available in low- and middle-income countries for a variety of regulatory and practical reasons. 28 However, affordability is also a key barrier. For example, the addition of rituximab to a chemotherapy backbone for the treatment of pediatric mature B-cell lymphoma has become standard of care in HICs but remains out of reach in many low- and middle-income countries due to the near order in magnitude increase in price.…”

Transforming Childhood AML Care in India

“…In the context of childhood ALL, blinatumomab, a bispecific T-cell engager, has been made available in selected LMICs through industry partnership as a part of the Blincyto Humanitarian Access Program (BHAP) program. 6 In addition, indigenous CD19 CAR T-cell for childhood ALL is also currently being manufactured in India. While novel agents are accessible for childhood ALL, the situation is different for childhood AML.…”

“…supportive therapy is required for early life up to adolescence. 6 Patients should be administered conjugated and unconjugated S. pneumoniae and Haemophilus influenzae conjugated Neisseria meningitides vaccines for immunization 7. We are still performing immunoglobulin replacement every 3 weeks for our 4-year-old patient.…”

Infusion of 14 days Blinatumomab in Combination with Chemotherapy for 46-day MRD+ Pediatric B-ALL Patients in Intermediate/High-Risk Group Results in MRD Conversion and BiTE Induced Immune Response to Malignant Cells

“…Considering the above evidences and the high cost of blinatumomab therapy 13 , we propose to add 1 course of blinatumomab to the original treatment for MRD + patients at the day 46 of initial induction therapy, and adjust the duration of treatment to 14 days to explore the effectiveness and safety of shortening the duration of continuous infusion in MRD + children with newly diagnosed B-ALL.…”