World Health Organisation Essential Medicines List: multiple sclerosis disease-modifying therapies application process

by Jennifer McDonell, MS Society of Canada

From Multiple Sclerosis Journal

Multiple sclerosis rates are increasing globally and while the number of treatments for the disease within the global market are also growing, access to them continues to present a concerning challenge in the treatment and care of MS. While access to safe and effective MS treatments predominantly impacts low-middle resource countries, it is also a barrier to disease management in high-resource countries.

As defined by the World Health Organization (WHO), ‘the model list of essential medicines contains the medications considered to be the most effective and safe to meet the most important needs of a health system’, subsequently it is often used by countries to establish their own lists of essential medications. The financial burden MS places on health and socioeconomic systems is vast and well documented, particularly in populations of MS patients who are untreated. Though there are more than a dozen medications for MS with regulatory approval globally, there are no medications on the EML for MS.

In 2018, the Multiple Sclerosis International Federation (MSIF) addressed this therapeutic gap by applying to the WHO to include three MS medications from different drug categories on the EML. Other MS medications have been submitted to previous versions of the EML, however none of the applications, including the recent MSIF submission have been successful.

MS is a complex disease, and its treatment can be equally as complicated. There is no MS treatment algorithm, no ‘one-size’ fits all. Robust clinical trial data demonstrating safety and efficacy, in addition to health and economic status, life stage, age, monitoring requirements, comorbidities, and other factors must be considered when selecting the best medication for a patient. How is ‘best’ defined though? Is it affordability? Safety during pregnancy? Avoiding agents that carry risks known to exacerbate certain pre-existing conditions? Pediatric patients, progressive patients, unresponsive patients, the list is extensive and unique to each person living with the disease. While all MS medications play an important role in the management of the disease, applying for all currently available medications on the international market was not a realistic goal. Instead, MSIF’s application aimed to establish a baseline of the minimum number of treatments that could treat the highest number of MS patients and following significant consideration, selected glatiramer acetate, fingolimod and ocrelizumab.

Though the application was unsuccessful, several other issues related to access to care and treatment emerged that will influence which medications are considered for future EML applications. For example, a greater understanding of resources available in low-to middle income countries is required to establish MS treatment guidelines that are representative of a country’s ability to provide optimal treatment and care. Off-label agents that may be considered out-of-date or lacking data in high-income countries may be the only treatments for people living with MS in low-resource settings due to lower cost and availability. The 2018 application to the WHO included MS medications with marketing authorization only, as phase three trial data for these agents was identified as priority, however it was suggested a more inclusive approach to MS medication selection be considered in subsequent EML applications.

The outcome of the application process was disappointing. However, the work done by MSIF and its partners has increased global awareness of the importance of access to MS treatments.  The MS movement has strengthened its role in advocating for access to treatment and care for people living with MS worldwide.