| Autor: |
RR Sales, DM Oliveira, BC Silva, JVP Neto, EM Fagundes, RLG Cunha, MT Laloni, CG Ferreira, P Nazareth-Junior |
| Jazyk: |
angličtina |
| Rok vydání: |
2024 |
| Předmět: |
|
| Zdroj: |
Hematology, Transfusion and Cell Therapy, Vol 46, Iss , Pp S1265- (2024) |
| Druh dokumentu: |
article |
| ISSN: |
2531-1379 |
| DOI: |
10.1016/j.htct.2024.09.2214 |
| Popis: |
Introduction: The increasing cost of novel technologies in the oncohematology field, with increasingly flexible regulatory criteria for approval by regulatory agencies, has raised concerns among private Health Technology Assessment (HTA) bodies. The ASCO framework and ESMO-MCBS-H are validated tools designed to assess clinical benefit in oncology. Objectives: This study aims to evaluate the utility and limitations of these tools in integrating new technologies into the institutional protocol of an Oncology Network in Brazil. Material and methods: We applied the ASCO and ESMO tools validated to hematologic scenario to evaluate the clinical benefit of drugs approved by the Brazilian regulatory agency (ANVISA) for hematological malignancies in the last two-years. In addition, we assessed the quality of evidence according to the GRADE framework and the risk of bias in the ROB-2 tool. Results: Our analysis included 12 new drug approvals and five advanced therapy indications for CAR-T cells for a total of 17 assessments. Of these, five drugs were not covered by supplementary health insurance. Eight (47%) trials were phase-III, and nine (53%) were phase-II or I/II. Nine trials (53%) were randomized, and only one (6%) was blinded. Eight (47%) reported progression-free survival or event-free survival as the primary endpoint. Noteworthy, one of the trials does not have efficacy measures as a primary endpoint. The mean of ASCO score was 41.04±8.94, with only one study (6%) meeting the cut-off score of 45 for relevant clinical benefit. For the ESMO-MCBS-H, only two trials (11,7%) achieved grade 4, indicating a substantial magnitude of clinical benefit. Curiously, the only trial that passed the GRADE and ROB2 tools did not pass the ASCO or ESMO assessments. Discussion: The use of classical evidence assessments together with validated scores to assess clinical benefit in oncohematology has not led to the approval of any of the most recent approvals in hematological malignancies in Brazil. While the ESMO-MCBS-H provided assessment forms specifically designed for hematological malignancies, it is unclear how the ASCO framework is appropriate for assessing the value of treatments for blood cancers. GRADE and ROB2 are the main references for assessing the certainty of evidence and can be used by health care providers. They are recommended for use in systematic reviews and decision-making by HTA agencies such as ANVISA and NICE. However, they have rigorous criteria for evaluating trials that are sometimes unattainable in some clinical scenarios or treatment peculiarities, such as those that prevent blinded interventions. Such conditions are often found in hematological malignancies. Real-world evidence (RWE) is a supplemental source of data, especially relevant in populations that are under-represented in clinical trials, such as the Brazilian population. However, there is still room for improvement in terms of standardisation and tools for RWE quality assessment. Conclusion: Our findings highlight the current challenges of incorporating new technologies in the private Oncology Network in Brazil and the urge need for new tools to ensure confidence in the healthcare decisions in line with contemporary trends in oncohaematology. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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