SMASH: An initiative for equitable access to precision medicine for rare or severe lipid disorders

Abstract

Background. Despite significant improvements in our knowledge of the biological basis of rare or severe lipid disorders and the refinement of their clinical management, equity challenges and barriers to access are gradually emerging, particularly in low-middle-income countries or remote regions. SMASH (System and Molecular Approaches of Severe Hyperlipidemia) is a global initiative with the goal of making precision medicine innovations available without discrimination for patients affected by rare or severe lipid disorders. Objectives. SMASH main objective is to facilitate access to accurate diagnosis and optimal treatment for patients affected by rare or severe lipid disorders regardless of where they live, their gender, ethnicity, or socioeconomic status. Overview. SMASH is an international initiative comprising five interrelated components: SMASH-Access, -Natural History, -Trials, -e-Share, and -Biorepository. SMASH has selected as templates four severe lipid disorders that have in common the accelerated development of precise diagnosis and the emergence of innovative treatments that represent equity challenges: HoFH (homozygous familial hypercholesterolemia), persistent chylomicronemia, LCAT (lecithin-cholesterol acyl transferase) deficiency, and severely elevated Lp(a). Access issues are broad and not limited to clinical or socio-economic factors. Several environmental variables are also contributory. Summary. SMASH is developed to conceive, support, or catalyze initiatives that might improve our understanding of rare or severe dyslipidemias and facilitate access to innovation for those affected. It will not duplicate ongoing initiatives but will support them. A system approach and a structured collaborative effort is mandatory to provide fair access to emerging treatments to patients in both developed countries and emerging economies.