Essential medicines concept and health technology assessment approaches to prioritising medicines: selection versus incorporation

Brhlikova et al. Journal of Pharmaceutical Policy and Practice https://doi.org/10.1186/s40545-023-00595-4 (2023) 16:88 Journal of Pharmaceutical Policy and Practice Open Access COMMENTARY Essential medicines concept and health technology assessment approaches to prioritising medicines: selection versus incorporation Petra Brhlikova1* , Thilagawathi Abi Deivanayagam2,3 , Zaheer‑Ud‑Din Babar4 , Claudia Garcia Serpa Osorio‑de‑Castro5 , Rosângela Caetano6   and Allyson M. Pollock1    Growing expenditure on medicines is impacting the sustainability of health systems [1]. The global pharmaceutical market is estimated to grow at a rate of 3–6% annually through 2027, surpassing US$1.9 trillion by 2023. An average of 65 new drugs are expected to be launched per year, primarily oncology, immunological, anti-diabetic, and obesity drugs, resulting from a continuous stream of innovative products [2]. Medicines are also the biggest driver of out-of-pocket payments (OOPs) and catastrophic health expenditure globally, with spending on medicines creating a greater financial burden for households than spending on inpatient or outpatient services [3]. On the one hand, overdiagnosis, inappropriate prescribing and medicine use may lead to over-treatment, inappropriate treatment, and health hazards [4]. On the *Correspondence: Petra Brhlikova 1 Population Health Sciences Institute, Newcastle University, Baddiley‑Clark Bldg, Newcastle Upon Tyne NE2 4AX, UK 2 Lancaster Medical School, Faculty of Health and Medicine, Lancaster University, Lancaster, UK 3 Institute for Global Health, University College London, London, UK 4 Department of Pharmacy, University of Huddersfield, Huddersfield, UK 5 Departamento de Política de Medicamentos E Assistência Farmacêutica, Escola Nacional de Saúde Pública Sergio Arouca, Fundação Oswaldo Cruz, Rio de Janeiro, Brazil 6 Departamento de Política, Planejamento E Administração Em Saúde, Instituto de Medicina Social, Universidade Do Estado Do Rio de Janeiro, Rio de Janeiro, Brazil other hand, lack of access to affordable medicines is a significant barrier to accessing health care [5]. In this commentary, we look at two paradigmatic approaches to prioritising medicines for use in health systems—the Essential Medicines concept, upon which the process of selecting essential medicines is based, and Health Technology Assessment, which is the process for comparing individual medicines using an aggregate of analytical tools, mainly involving cost-effectiveness. The paper intends to highlight gaps in research and lack of evidence for these approaches mainly on their effectiveness in containing costs, ensuring access and appropriate medicine use. Medicines selection based on the WHO essential medicines concept For the last four decades, the WHO has made strenuous efforts to enable universal access to affordable and essential medicines. In 1975, in response to growing concerns over the increasing number of medicines in the market and the need to ensure that government procurement and prescribers focus on key medicines to meet public health needs, the WHO took the discussion to the World Health Assembly [6]. It resulted in the development of the essential medicines (EM) concept, and its main strategy, the Essential Medicine List (EML), also known as the Model list. The EML is a limited list of medicines covering all therapeutic classes and is the most widely used tool for prioritising medicines. © The Author(s) 2023. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativeco mmons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. Brhlikova et al. Journal of Pharmaceutical Policy and Practice (2023) 16:88 The EM concept is a programme which covers all aspects of drug management, including procurement, storage, distribution, prescribing and use. It recognises the need to rationalise and prioritise the selection of effective medicines for proven health needs. The EM concept is rooted in evidence-based and priority needbased selection. Essential medicines are selected on the basis of population need, efficacy, safety, effectiveness, quality, and affordability and are deleted/ deselected if better alternatives become available or if shown to be ineffective or harmful [7, 8]. The WHO also requires evidence of market registration, i.e. producers have a licence for manufacturing the drug for use. Pricing of medicines and affordability to the health system and public purse underpins essential medicines policy [5]. The alignment between EML which guides medicine procurement, and thus availability, and treatment guidelines which support prescribing is crucial. The concept has been voluntarily adopted and implemented by 156 out of 193 WHO member states [7], which develop and implement their own lists, inspired by the WHO EML. National EMLs may be further adapted for use at state, regional and district levels and within the different levels of the health system [7]. Tools for implementation of the list are Standard Treatment Guidelines (STGs), which may be developed prior to or after inclusion of medicines on to the list [9] and drug formularies, which include indications and information for the prescriber [10]. The budgetary impact on health systems in countries which adopt the EM concept is unknown. In many countries there is under-registration of essential medicines and many low-income countries do not procure all medicines on their EMLs. They may also use additional levels of prioritisation (Vital Essential Necessary, VEN classification) when budgets are tight. This economic classification is for example used at different levels of the health system in Jamaica, Trinidad and Tobago, and Uganda [11–13]. While in 1977, the EML selection criteria considered only off-patent, generic medicines, and emphasised the need to select effective and safe low-priced medicines [14], novel on-patent and high-cost medicines have been incorporated into the WHO EML in recent years. This further strain on the budgets of low- and middle-income countries and inc (...truncated)