The increasing reports and estimates of rheumatic diseases in CYP from African countries challenges the perception that these diseases are rare or non-existent in the WHO Africa region [6, 7, 27]. It necessitates an investment in healthcare for chronic inflammatory musculoskeletal disorders, and the provision of appropriate medicines to significantly improve quality of life and to reduce disability [28, 29].

As NEMLs inform the procurement and supply of medicines to meet priority healthcare needs in the public sector, the collation and comparison of NEMLs to the WHO EML for this study, highlights this aspect of care. It provides the data and impetus for stakeholders to advocate for updating their NEML to improve access and availability of the necessary medicines to treat CYP with rheumatic diseases, and simultaneously fuels discussions around financial risk protection for patients and their families in this setting.

Whilst work is ongoing to list medicines for systemic juvenile idiopathic arthritis i.e. tocilizumab and anakinra on the WHO EML, the latest versions of the model lists provide timely guidance for African countries to update their NEMLs to reflect standard care for rheumatic diseases in CYP. Only 4 countries have updated their NEMLs since 2021 and the majority of countries have a 50% or lower similarity with the 2021 WHO essential medicines model lists.

Conventional synthetic DMARDs (including the additional medicines not on the WHO EML i.e. mycophenolate mofetil, ciclosporin, and cyclophosphamide most commonly) appear to be more available and presumably due to their utility in adult rheumatic diseases; only 8 countries (South Africa, Ethiopia, Ghana, Nigeria, Kenya, Libya, Tunisia and Egypt,) listed biologic DMARDs, in keeping with more modern treatment approaches. Tumour necrosis factor inhibitors, as reflected in the WHO model lists, are noted for these 8 countries. The additional biologic DMARDs not listed on the WHO EML include tocilizumab in Nigeria, Libya and Kenya; rituximab in these 3 countries as well as in Ghana and South Africa; and anakinra only in Libya. Biologic DMARDs, while currently more costly than conventional synthetic DMARDs, have the potential to improve outcomes significantly [30, 31].

The provision of medicines is, however, dependent on adequate financing and robust pharmaceutical services [32], and must be adequately supported by health care policy. Notably, health care policy around priority diseases influences the development of NEMLs and STGs, and consequently the availability of medicines for patients managed by state health care services in particular. At the time of writing, there were no national healthcare policies in place for musculoskeletal healthcare in Africa, with only 6 countries in sub-Saharan Africa including the management of rheumatic diseases for CYP in STGs [33]. The expansion of rheumatology services across Africa [7, 27, 34, 35], supported by the clinician-led Paediatric society of the African League Against Rheumatism (PAFLAR) and the Global Paediatric Musculoskeletal Task Force, has provided evidence to address this challenge [5, 7, 15, 18, 20, 36]. However, to lever long term sustainable change, due consideration, coordination and funding at policymaker level is crucial. Furthermore adequate support for rheumatology services and the array of factors that influence access to appropriate care also need to be addressed; the Global Strategy for Musculoskeletal Health and the 2022 Australian ‘Inquiry into childhood rheumatic diseases’ describe strategies and adaptable exemplars for change at policymaker level, which may be implemented by the countries in Africa [10, 37].

However, the overall cost of treating rheumatic diseases in children, i.e. increased numbers of hospital visits, associated medical and non-medical costs including the high cost of biologic DMARDs, remains a major challenge [38,39,40]. Additionally, the main method of payment for about one third of healthcare services in the Africa region are financed non-sustainably and by ‘out-of-pocket’ expenditure [41]. These costs could be countered to some extent by improved clinical outcomes, the reduced costs of surgery, as well as the unmeasured longer term effects of chronic illness on mental health, and the impact on future productivity in society [42]. We therefore considered country specific factors including health care financing, which may affect the number of medicines on NEMLs and by proxy, their availability i.e. health expenditure per capita, socio-demographic index (a composite calculation of health expenditure per capita, average years of schooling and total fertility rate for females under 25 years), and the availability of rheumatology services, as predictors. As Gross Domestic Product has previously been shown not to predict the number of medicines on NEMLs [13, 14], this was not re-evaluated.

In our analysis, health expenditure per capita per se, did not predict the number of medicines on the NEMLs for rheumatic diseases in CYP. However, the availability of rheumatology services and socio-demographic index (SDI), were significant predictors of the number of medicines available, and the presence of tumour necrosis factor inhibitors on NEMLs.

The 2019 Global Burden of Disease study reported the median SDI for countries in the WHO Africa region as 0.41 (IQR 0.34–0.52) and ranged from 0.081 (Somalia) to 0.724 (Seychelles), with 56% of countries in the low SDI range. This measure is indicative of poorer health outcomes in the region, which may be further affected by a complex array of challenges [1]. While the 8 countries which have listed biologic DMARDs on their NEMLs have disparate values for health expenditure per capita and SDI, the unifying factor appears to be the availability of established rheumatology services (adult and/or paediatric). As advocacy and demand are important in driving down the cost of these medicines, perhaps through pressure on manufacturers and funders, the role of rheumatologists to modernise care, has been demonstrated to be of particular importance in this setting.

However, a high similarity with the WHO EML does not necessarily translate to the direct availability of medicines for patients. Given the wide variation in GDP, health expenditure per capita, SDI and the appropriate prioritisation of communicable diseases in the WHO Africa region, improving care for rheumatic diseases in CYP remains complex. Many African countries have overburdened, heterogeneous healthcare systems which may include private health care and varying support from non-governmental organisations, where access to medicines may differ to that of patients dependent on state sponsored healthcare. Additionally, pharmaceutical services in institutions across Africa have required support from several globally funded programs to address operational gaps [43,44,45,46].

We therefore looked further to successful strategies that tackle high direct costs and the equitable distribution of medicines, that may be extrapolated to close ‘treatment gaps’ in this context.

In line with WHO Universal Health Coverage agendas (Sustainable Development Goal 3.8) and frameworks for the care of the chronically ill, the successful implementation of a universal access program for JIA in Chile as an example, resulted in timely diagnosis, higher rates of clinical remission and lower rates of complications [38]. This program was financed by a 1% increase in state value added tax; a strategy that may be difficult to implement in many regions of Africa, particularly after the devastating financial effects of the COVID-19 pandemic on many economies, as well as that of recent natural disasters, ongoing conflict and corruption [47, 48]. Nonetheless, the program highlights the positive effects of an enforced government mandated policy.

There are also several aspects of the successful multi-sectoral model for HIV/AIDS (WHO UNAID 3 by 5 initiative and others such as End TB and malaria) [49] which could be emulated in a globally driven strategy for rheumatic diseases in CYP. Tanzania and other East African countries implemented a ‘Non-Communicable Diseases Prevention and Control Programme’ in recent years, which reinforces multi-sectoral involvement and strong government commitment in effecting change [50, 51]. The system provides screening, prevention, diagnosis and treatment of hypertension and diabetes for people living with HIV, integrated with standard HIV healthcare services. Plans for extension to include those without HIV, is currently in progress. Supported by the WHO, and external funding (the United Nations program on HIV/AIDS (UNAIDS), the United States President’s Emergency Fund for Aids Relief (PEPFAR) and the Global Fund), these programs provide cost-effective care and medicines using existing infrastructure, with better outcomes as demonstrated in the INTE-Africa study [50,51,52].

In another positive step towards early recognition, diagnosis and treatment of rheumatic diseases in CYP, the Western Cape region of South Africa has included an extensive section on musculoskeletal disorders and inflammatory arthritis in the ‘Practical Approach and Care Kit for children’, a primary care manual complementing the WHO Integrated Management of Childhood Illnesses guideline and STGs [53].

While only 6 countries in sub-Saharan Africa have paediatric rheumatic diseases noted in STGs, this may be improved over time with lessons learned from these programs, and with adequate training and support, these models of primary care which includes the provision of appropriate medicines, could be successfully implemented for CYP with rheumatic diseases.

By prioritising rheumatic diseases and considering novel financing involving the Global Fund, managed entry agreements for new medicines, and the use of the Medicines Patent Pool as exemplified in these programs, medicines could be procured at a much lower cost and as part of a comprehensive package of care, affording patients and their families financial risk protection.

Pharmaceutical agreements may also encourage the expedited development of approved biosimilars (at a lower cost), paediatric medicine formulations and formulations which promote patient empowerment e.g. self-administered subcutaneous injections or oral medications, instead of intravenous administration. Oversight of patients requiring these medicines, including surveillance and treatment for TB and other infections, may pro-actively be achieved by adapting the monitoring systems already in place, as has been explored for other non-communicable diseases, and integrating telemedicine programs for paediatric rheumatology where in-person consultations are not feasible [54, 55]. These strategies may also be explored as potential avenues for the de-centralisation of care for CYP with rheumatic diseases once diagnosed, and the appropriate level of training has been achieved.

Maintaining up to date NEMLs would ensure the provision of a range of medicine for standard care. Countries which do not distinguish between the medicines listed for adults and children, or have medicines listed for rheumatic diseases but were updated before 2021, have the opportunity to advocate for ‘Medicines for musculoskeletal disorders’; and for the sub-section ‘Juvenile joint diseases’ to be aligned with the latest 2023 WHO model lists. For countries with an NEMLc, harmonising the medicines listed with the NEML and with the WHO model lists, is essential to ensure appropriate access to medicines for continuity of care throughout the life course.