WHO endorses landmark public health decisions on Essential Medicines for Multiple Sclerosis

Today the World Health Organization (WHO) published the new editions of the Model Lists of Essential Medicines (EML) and Essential Medicines for Children (EMLc) which include important new medicines for the treatment of multiple sclerosis, cancer, infectious diseases, and cardiovascular conditions, among others.  The updated Model Lists aim to facilitate greater access to innovative medicines that show clear clinical benefits. These treatments could have a very large public health impact globally without jeopardizing the health budgets of low- and middle-income countries.

“For over 40 years, countries all over world have relied on the WHO Essential Medicines List as a definitive, evidence-based guide the most important medicines for delivering the biggest health impact,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General. “Rising prices and supply chain disruptions mean that all countries now face increasing problems in ensuring consistent and equitable access to many quality-assured essential medicines. WHO is committed to supporting all countries to overcome these obstacles to increase access with equity.”

For the 2023 update, 85 applications, encompassing over one hundred medicines and formulations, were considered by the WHO Expert Committee on Selection and Use of Essential Medicines.  The recommended changes bring the total number of medicines on the EML and EMLc to 502 and 361, respectively.

Full details of the Expert Committee’s recommendations, describing the additions, changes and removal of medicines, and decisions not to recommend medicines are available in the Executive Summary here.

Medicines for multiple sclerosis (MS)

Multiple sclerosis is a chronic, debilitating disease of the nervous system affecting approximately 2.8 million people worldwide. Until now, no medicines for its treatment have been included on the EML. In 2023 three medicines that can delay or slow its progression – cladribine, glatiramer acetate and rituximab – are added to the EML, filling an important gap  given the large global burden of MS. Listing of these medicines as treatment options for MS with different routes of administration, different prices (with the availability of generics and biosimilars) and different recommended uses, is aimed at facilitating improved access to treatment for people living with MS around the world. The decision to support off-label use of rituximab is supported by strong evidence of its efficacy and safety for this indication. This recommendation, which is in line with previous recommendations by the Expert Committee, could lead to major health benefits worldwide.

“The List is an important tool for achieving universal health coverage, providing guidance to governments, health facilities and procurers on which medicines are the best value in terms of benefits for individuals and communities. The EML includes medicines only on the basis of solid evidence for safety and efficacy. Approved indications within national jurisdictions or the availability of on-label alternatives is not a decision criterion,” said Secretariat of the WHO EML, Dr Benedikt Huttner.  “Given the evidence base and the increased affordability of rituximab, including the availability of prequalified biosimilars, it has been prioritized over on-label alternatives as an essential medicine to treat relapsing-remitting and progressive MS”.

Cardiovascular medicines

Fixed-dose combinations of multiple medicines (commonly called ‘polypills’) for the prevention of diseases of the heart and blood vessels, notably cholesterol-lowering agents with one or more blood pressure lowering agents with and without acetylsalicylic acid (aspirin) have been added to the EML for the first time.  Based on recommendations by previous EML Expert Committees, scientists from multiple countries conducted milestone trials confirming the benefit of these combinations for both primary and secondary prevention of heart disease.

Infectious diseases

New medicines listed for infectious diseases include:

• ceftolozane + tazobactam, a ‘reserve’ group antibiotic effective against multi-drug resistant bacteria, including difficult-to-treat infections caused by carbapenem resistant Pseudomonas aeruginosa;
• pretomanid for treatment of multidrug-resistant or rifampicin-resistant tuberculosis;
• ravidasvir (to be used in combination with sofosbuvir) for the treatment of chronic hepatitis C virus infection in adults;
• monoclonal antibodies for Ebola virus disease.

Cancer medicines

Two new cancer treatments have been added: pegylated liposomal doxorubicin for Kaposi sarcoma and pegfilgrastim to stimulate production of white blood cells and reduce the toxic effect of some cancer medicines on the bone marrow. The indications for several cancer medicines for children already included on the EMLc were extended to include new types of childhood cancers (anaplastic large cell lymphoma, Langerhans cell histiocytosis and Burkitt lymphoma). Additionally, those not recommended for inclusion encompass several patented, highly priced cancer medicines because of concerns about affordability and feasibility in low-resource settings. Some of these had been evaluated and rejected during past Committee meetings – PD-1 / PD-L1 immune-checkpoint inhibitors and osimertinib for lung cancers, and CDK4/6 inhibitors for breast cancer.

Other updates

Diabetes – current listings for human insulin on the EML and EMLc are extended to include cartridge and pre-filled pen delivery systems due to their potential advantages for patients over vials and syringes in terms of ease of use, greater accuracy of dosing and improved adherence.

Mental health conditions – a comprehensive review of medicines for mental health and behavioral disorders has led to updates to the EML and EMLc to ensure strong alignment between the Model Lists and recommendations in WHO guidelines, including the addition of two new medicines – acamprosate and naltrexone – for treatment of alcohol use disorder.

Essential medicines for children – updates were made to listed formulations of over 70 medicines the EMLc to ensure appropriate dosage forms and strengths for use in children aged up to 12 years are included. Ready-to-use therapeutic food is added to the EMLc for the treatment of severe acute malnutrition in infants and children up to 5 years old.

Applications not recommended: A total of 32 applications were not recommended including: glucagon-like peptide-1 receptor agonists for weight loss in obesity, risdiplam for treatment of spinal muscular atrophy, donepezil for treatment of dementia due to Alzheimer disease, CAR-T cell therapies for lymphoma and fast-acting oral transmucosal fentanyl for breakthrough cancer pain. Details for these decisions can also be found in the Executive Summary.

Note to Editors

The meeting of the 24th WHO Expert Committee on the Selection and Use of Essential Medicines was held at WHO Headquarters in Geneva from 24 to 28 April 2023. The Expert Committee considered 85 applications for medicines to be added to the 22nd WHO Model List of Essential Medicines (EML) and the 8th WHO Model List of Essential Medicines for Children (EMLc). WHO technical departments were involved and consulted with regard to applications relating to their disease areas.

The updated Essential Medicines Lists include 24 new medicines for adults and 12 new medicines for children and specify new uses for 16 already-listed medicines. New formulations of 19 medicines on the EML and 48 medicines on the EMLc have been added. The changes recommended by the Expert Committee bring the number of medicines deemed essential to address key public health needs to 502 on the EML and 361 on the EMLc. While these numbers may seem high, they are only a small proportion of the total number of medicines available on the market.

Launched in 1977 largely to promote better access to medicines in developing countries, the WHO Model Lists have become a global policy tool for decisions related to the selection and universal coverage of medicines within all health systems.  The Model Lists are updated every two years by an Expert Committee, made up of recognized specialists from academia, research and the medical and pharmaceutical professions, to address new health challenges, prioritize highly effective therapeutics and improve affordable access.

Around the world, more than 150 countries use the WHO Model Lists to guide decisions about which medicines represent the best value for money, based on evidence and health impact.