Biologic DMARDs for children excluded from most national essential medicines lists
Biologic disease-modifying antirheumatic drugs used to treat juvenile rheumatic diseases are excluded entirely from national essential medicines lists in close to two-thirds of all countries in the world, according to data published in Pediatric Rheumatology.
“The World Health Organization (WHO) developed the Model list of essential medicines (WHO EML) ‘intended to meet the priority health care needs of a population’ in 1977, an influential template since adapted by countries worldwide,” Raphaël Kraus, MD, FRCPC, FAAP, of the University of Toronto, and colleagues wrote. “Subsequently, the WHO released a model list specifically delineating essential medicines for children (WHO EMLc). These essential medicines lists (EMLs) guide countries’ selection of drugs to fund, stock, prescribe and dispense.”
“The Lancet Commission, ‘Essential medicines for universal health coverage,’ affirms that countries ‘must implement a comprehensive set of policies to achieve affordable prices ...’ and equity in access,” they added. “A unique database of 138 national EMLs (71% of 195 countries) and associated country characteristics was recently compiled and demonstrates significant variation between countries in included medicines. We hypothesized that biologic DMARDs are underrepresented relative to conventional DMARDs in the model WHO and existing national EMLs.”
To analyze the presence of biologic DMARDs in essential medicines lists, as well as national characteristics that may contribute to their inclusion of absence, compared with conventional DMARDs, Kraus and colleagues made use of a previously compiled database, constructed in June 2017 and updated in January 2020. All essential medicines lists were included regardless of publication date and language. In all, the researchers included 138 national lists published between 2001 and 2017.
The researchers additionally aimed to include all systemic biologic and conventional DMARDs used in routine care for juvenile idiopathic arthritis, systemic lupus erythematosus, juvenile dermatomyositis scleroderma, systemic vasculitides and autoinflammatory disorders. Certain novel drugs, such as JAK inhibitors, were excluded because of their “limited clinical use,” the researchers wrote.
Country data collected included WHO region, population, life expectancy, infant mortality, gross domestic product (GDP) per capita, health care expenditure per capita, Gini index as a measure of income inequality and the corruption perception index. The researchers used regression modeling to examine how certain characteristics accounted for any differences between the lists.
According to the researchers, the 138 national lists ranged in size from 44 to 980 medicines, with a median of 308 and a mean of 366.9. All included countries listed at least one conventional DMARD. Eleven nations — or 7.97% — included all 10 conventional DMARDs, while 83.33% had five or more. GDP per capita was associated with the total number of conventional DMARDs included (1 = 1.02; 95% CI, 0.39-1.66).
Meanwhile, regarding biologic DMARDs, only three countries — or 2.2% — listed 10 or more, while 15 — or 10.9% — listed five or more, and just 47 — or 34.1% — listed at least one. As many as 65.9% of countries listed no biologic DMARDs.
European location (1 = 1.3; 95% CI, 0.08-2.52), life expectancy (1 = –0.7; 95% CI, –1.22 to –0.18), health expenditure per capita (1 = 1.83; 95% CI, 1.24-2.42) and conventional DMARDs listed (1 = 0.70; 95% CI, 0.33-1.07) were associated with the total number of biologic DMARDs included in a national list.
“Although biologic DMARDs are underrepresented in national EMLs and are more likely to be listed by high-income countries, this does not preclude their inclusion by less prosperous nations,” Kraus and colleagues wrote. “For example, only nine of the 42 countries (21.4%) listing rituximab (the most commonly listed biologic) are categorized as high-income; 33 of 42 (78.6%) are therefore low- or middle-income economies.”
“This indicates the potential for other countries to consider the listing of biologic DMARDs, which would ultimately lead to a lowering of their costs and a resultant increase in their cost-effectiveness, thus rendering them more attractive to governmental and other health payers,” they added. “Costs to health payers are likely to further decrease with the growth of the biosimilar market, driving price competition and improved patient access to biologic therapies. The inclusion of these medicines in a system of universal prescription drug coverage would ultimately abate the economic impact and improve the quality of life of children with systemic inflammatory disease.”