Poor control of cardiovascular disease accounts for a substantial proportion of the disease burden in developing countries, but often essential anticoagulant medicines for preventing strokes and embolisms are not widely available. In 2019, direct oral anticoagulants were added to the World Health Organization's WHO Model list of essential medicines. The aims of this paper are to summarize the benefits of direct oral anticoagulants for patients with cardiovascular disease and to discuss ways of increasing their usage internationally. Although the cost of direct oral anticoagulants has provoked debate, the affordability of introducing these drugs into clinical practice could be increased by: price negotiation; pooled procurement; competitive tendering; the use of patent pools; and expanded use of generics. In 2017, only 14 of 137 countries that had adopted national essential medicines lists included a direct oral anticoagulant on their lists. This number could increase rapidly if problems with availability and affordability can be tackled. Once the types of patient likely to benefit from direct oral anticoagulants have been clearly defined in clinical practice guidelines, coverage can be more accurately determined and associated costs can be better managed. Government action is required to ensure that direct oral anticoagulants are covered by national budgets because the absence of reimbursement remains an impediment to achieving universal coverage. Tackling cardiovascular disease with the aid of direct oral anticoagulants is an essential component of efforts to achieve the World Health Organization's target of reducing premature deaths due to noncommunicable disease by 25% by 2025.
Poor control of cardiovascular disease accounts for a substantial proportion of the disease burden in developing countries, but often essential anticoagulant medicines for preventing strokes and embolisms are not widely available. In 2019, direct oral anticoagulants were added to the World Health Organization's WHO Model list of essential medicines. The aims of this paper are to summarize the benefits of direct oral anticoagulants for patients with cardiovascular disease and to discuss ways of increasing their usage internationally. Although the cost of direct oral anticoagulants has provoked debate, the affordability of introducing these drugs into clinical practice could be increased by: price negotiation; pooled procurement; competitive tendering; the use of patent pools; and expanded use of generics. In 2017, only 14 of 137 countries that had adopted national essential medicines lists included a direct oral anticoagulant on their lists. This number could increase rapidly if problems with availability and affordability can be tackled. Once the types of patient likely to benefit from direct oral anticoagulants have been clearly defined in clinical practice guidelines, coverage can be more accurately determined and associated costs can be better managed. Government action is required to ensure that direct oral anticoagulants are covered by national budgets because the absence of reimbursement remains an impediment to achieving universal coverage. Tackling cardiovascular disease with the aid of direct oral anticoagulants is an essential component of efforts to achieve the World Health Organization's target of reducing premature deaths due to noncommunicable disease by 25% by 2025.
Questo Dossier è dedicato alla presentazione del background scientifico e dei risultati del Progetto AFO (Appropriatezza farmaci oncologici) sviluppato dall'Agenzia sanitaria e sociale della Regione Emilia-Romagna nell'ambito del Programma Ricerca e innovazione (PRI E-R). Dal punto di vista del background scientifico l'elemento di originalità del progetto è stato la sperimentazione dell'uso del metodo GRADE (Grading of Recommendations Assessment, Development and Evaluation) per la produzione e graduazione di raccomandazioni cliniche relative all'effetto degli interventi sanitari. Il metodo GRADE è nato dall'attività avviata nel 2000 di un gruppo di lavoro internazionale che ha ritenuto necessario mettere a punto un approccio unificato alla produzione di raccomandazioni cliniche in presenza di una molteplicità di sistemi di grading tra loro non sempre coerenti e la cui variabilità mette in serio pericolo la fruibilità dello strumento linee guida/raccomandazioni. Dal punto di vista dei contenuti e delle modalità operative, il Progetto AFO - che ha anche ottenuto un finanziamento da parte del Ministero del Lavoro, della salute e delle politiche sociali nell'ambito del Programma Ricerca finalizzata (Fondo ex art. 12 DLgs 502/1992) - è nato con l'obiettivo di sperimentare la possibilità di coinvolgere gruppi multidisciplinari di clinici nella produzione di raccomandazioni sull'uso appropriato di farmaci oncologici, sia nuovi sia già registrati ma in evoluzione per quanto riguarda le indicazioni cliniche. Nella prima parte del Dossier, dopo una breve introduzione relativa alla filosofia del programma PRI E-R e agli scopi del progetto AFO, viene presentato il metodo GRADE, le sue assunzioni e le tappe operative di applicazione. Nella seconda parte vengono presentati i risultati del lavoro dei tre panel multisciplinari che hanno prodotto complessivamente 32 raccomandazioni per la terapia adiuvante e la fase avanzata del trattamento dei tumori della mammella, del colon retto e del polmone. Conclude la seconda parte del Dossier una discussione circa le criticità di applicazione del metodo e le potenzialità e i limiti del suo utilizzo per la produzione di strumenti utili al miglioramento dell'appropriatezza d'uso dei farmaci. Nell'Appendice viene infine riportato, a scopo esemplificativo, il testo di due raccomandazioni relative all'uso del trastuzumab nella terapia adiuvante del tumore della mammella e delle fluoropirimidine nella terapia adiuvante del tumore del colon. ; This report is devoted to the presentation of the scientific background and results of the AFO (an acronym standing for Appropriateness of Oncologic Drugs) project developed by the Health Care and Social Agency of Emilia-Romagna region in the framework of the Research and Innovation program (PRI E-R). From the methodological standpoint the novelty of the project stems from the utilisation of the GRADE method (Grading of Recommendation, Assessment, Development and Evaluation) for the production and grading of clinical recommendations on the effects of health care interventions. The GRADE method originated from the work of an international working group that, starting 2001, was convened to go beyond the variations in the existing methods for producing and grading clinical recommendations. These variations were mostly due to conceptual inconsistencies that, if not properly recognised and addressed, would jeopardise the credibility of the entire guidelines movement. From the subject matter standpoint the AFO project - which was also partially supported by a research grant from the Italian Ministry of Welfare, Health and Social Affairs - was conceived to test the feasibility of a working methodology where multidisciplinary panels of clinicians would be convened to produce clinical recommendations on newly registered drugs as well as those already in widespread use but whose indications change over time. In the first part of the report, after a brief introduction on the working hypotheses and objectives of the PRI ER program and the AFO project, the GRADE method is presented and discussed. In the second part the results of the activities of the three panels that produced a total of 32 recommendations for the adjuvant and advanced treatment of breast, colorectal and lung cancer are reported. The final session of the report addresses some open issues relative to the application of the GRADE method to the production of clinical recommendations construed with the explicit aim of improving quality and appropriateness of care in oncology. In the Appendix, two examples of the recommendations produced though this project are presented: they refer to Trastuzumab in the treatment of early breast cancer and Fluoropirimide in the adjuvant therapy of colon cancer.
