Background: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes one of the most important modifiable risk factors for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are particularly vulnerable to CVD events. The addition of evolocumab to statins has shown marked reductions in LDL-C levels. The objective of this analysis is to demonstrate the clinical and economic value of LDL-C lowering with evolocumab from the Bulgarian public health care perspective. Methods: A disease-specific measure of health benefit was devised: Effectively treated patient-years (ETPYs) combine length of life with the likelihood of attaining best-practice recommendations on LDL-C lowering. “Effective treatment” was defined as a reduction in LDL-C levels of ≥50%. A Markov cohort state-transition model was adapted, considering a life-long treatment duration. Demographics, baseline characteristics and efficacy data were taken from the RUTHERFORD-2 trial. The model uses the relationship between LDL-C lowering and reduced CVD event rates observed in the meta-analyses conducted by the Cholesterol Treatment Trialists’ Collaboration. Outcomes and costs (from year 2015) were discounted at an annual rate of 5%. Sensitivity analyses were conducted to assess uncertainty surrounding the results. Results: The total incremental costs of evolocumab added to statins versus statins alone are BGN 120,329 while adding 9.30 ETPYs over lifetime. These results imply an incremental cost per ETPY of BGN 12,937 (US$ 7,215; € 6,604). The use of evolocumab is associated with a relative reduction in the CVD event rate by 38% (18% per 1 mmol/L). Conclusions: Adding evolocumab to statins may be considered cost-effective in light of an additional expense per patient-year gained in which individuals with HeFH receive effective treatment under the terms of international prevention guidelines. ETPYs are an intuitive and clinically meaningful measure of patient benefit that, in relation to costs, can support health care decision-making that considers quality of care.

Cardiovascular (CV) diseases are a major burden for all the healthcare systems around the world. Public health and medical advances continue to beneficially affect CV patients health. In the last decades, many new medicinal products for hearth problems were discovered and received a marketing authorization. Elevated low-density lipoprotein cholesterol (LDL-C) levels have been linked to major CV risk. The objective of this study was to review the medical needs in high-risk cardiovascular patients with familial hypercholesterolemia undergoing pharmacological treatment with statins and the degree of attained lipid control. A conclusion it can be stated, that there is a significant unmet medical need for a potentially effective treatment, which can supplement the statin therapy, enabling vulnerable populations to achieve sufficient control of LDL-C, and thus provide an alternative for patients with statin intolerance or where this group of medicinal products are not clinically appropriate.

Навлизането на новите дигитални технологии в различни бизнес отрасли води до множество промени в бизнес средата. Една от най-осезаемите такива промени е трансформацията във фармацевтичната индустрия, продиктувана от разширеното приложение на облачните системи, изкуствения интелект и машинното обучение.Използването на автономни устройства и системи е от жизненоважно значение при производството на лекарства с високо качество срещу по-малко разходи и време. Фазата на научноизследователска и развойна дейност за разработване на нов фармацевтичен продукт е много дълга и скъпа в сравнение с конвенционалните продукти, а използването на съвременни технологии в този процес може да донесе огромни ползи за фармацевтичната индустрия, като същевременно се предоставя възможност на регулаторните органи за проследяване на всички етапи от процеса с по-голяма прозрачност и по-малко усилия.