ESPE Yearbook of Paediatric Endocrinology

jchanoine@cw.bc.ca BMJ Global Health 2021;6:e007195. doi: 10.1136/bmjgh-2021-007195

Brief Summary: This review found that access to oral fludrocortisone and hydrocortisone remains suboptimal in the WHO Eastern Mediterranean Region. Improvement requires a collaboration between health professionals, families of patients, health authorities, pharmaceutical companies, and the WHO.

This review of access to hydrocortisone (HC) and fludrocortisone (FC), and to newborn screening for congenital adrenal hyperplasia (CAH), in 23 countries of the Eastern Mediterranean Region (EMRO) provides expected but previously undocumented evidence of unacceptable, potentially life-threatening care gaps for children with CAH. Access gaps in the region’s 13 low- and lower-middle-income countries (LMIC), such as consistent unavailability of both FC and oral HC, raise particular concern, including for similar or worse situations in LMICs across all other world regions. The detailed description and analysis of CAH care access gaps discerned by this author group provide first insights and consequently an opportunity to develop first steps towards gap closure. Of particular interest are, first, their documentation that in a region with high rates of consanguinity, and thus about twice higher incidence rates of CAH, universal newborn screening is unavailable except in 2 high-income countries (UAE and Kuwait). Second, availability of oral HC and FC correlates with presence of these medications in National Essential Medicines Lists (NEML; usually templated on the WHO Essential Medicines List that has listed FC and oral and injectable HC since 2009). Third, availability of FC and oral HC correlate with Gross National Income. Fourth, in many countries where access to FC and HC is limited or inconsistent, health care providers and families find creative but temporary and often unsustainable solutions to access medication, such as import by families and friends.

The authors conclude with constructive and realistic potential solutions to the medicine access gap, including the WHO prequalification process, group registration, pooled procurement, working with local pharma companies that will take responsibility for registration and distribution, developing national special access processes, and compounding using active pharmaceutical ingredient – emphasizing that all of the above require collaboration between several stakeholders including patient organizations, pediatric endocrinologists, national health authorities, manufacturers and the WHO.

Footnote: This commentary was kindly prepared by Julia E. von Oettingen, MD, PhD, MMSc, FRCP, Pediatric Endocrinologist, Assistant Professor, Montreal Children’s Hospital and McGill University, Montreal, Canada.