On November 1, 2024, Knowledge Ecology International (KEI) submitted a proposal for the addition of risdiplam to the World Health Organization’s (WHO) Model List of Essential Medicine (EML). You can access the full proposal here: risdiplam2WHOEML1Nov2024
The EML, updated every two years, identifies medicines that the WHO considers most effective and safe for addressing major public health needs worldwide. Its inclusion criteria consider a treatment’s clinical efficacy, safety, cost-effectiveness, and potential to address unmet needs, amongst other considerations. Serving as a blueprint that countries can use to develop their own essential medicines lists, the EML sends a signal about a medicine’s value in improving public health. Inclusion on this list can also raise awareness of a medicine’s benefits, highlighting it for priority use and potentially influencing accessibility and affordability worldwide.
Following KEI’s submission, the EML Secretariat reviews it and publishes it for public comment on the WHO website in late January 2025, allowing stakeholders to provide feedback. This open-comment period allows stakeholders, such as patients and advocacy organizations, to contribute insights and perspectives that may influence the Expert Committee’s assessment. The Expert Committee, consisting of a diverse panel of experts, will meet in Spring 2024 to review and make a recommendation on risdiplam’s inclusion. A final decision is anticipated by Summer 2025.
This is KEI’s second submission for risdiplam’s inclusion on the EML, incorporating several new elements and an important clinical data update. This application highlights recent evidence, notably from the October 2024 RAINBOWFISH study, which underscores the critical value of presymptomatic treatment in reducing SMA-related disability and mortality. KEI’s application emphasizes the potential of risdiplam to significantly improve the quality of life for individuals with spinal muscular atrophy (SMA), especially when treatment is initiated early.
In addition to our strong recommendation for the inclusion of risdiplam on the core EML, KEI additionally recommends that the World Health Organization (WHO) consider a new category for essential yet expensive medicines alongside the recommendation for the government to take measures to obtain an affordable version of the medicine.