Objective This article reviews the assessment pathways that have been implemented
worldwide to facilitate access to drugs for patients with rare diseases. Methods The …

Objectives: The aim of the study was to compare the access of patients with rare diseases
(RDs) to biotechnological drugs in several Central and Eastern European countries …

The interest to rare diseases has increased in the recent decades. Legislation seeks to
facilitate patients' access to innovative and effective treatment and to define incentives for …

Background Policy makers face a lot of challenges in the process of drug reimbursement
decision-making, especially in the context of entering the market of more and more …

Provision of the latest innovative and advanced therapies for rare diseases (RDs) patients,
following the international therapeutic recommendations, is crucial and necessary for both …

There are problems people don't want to talk about, and one of them is incontinence in
adults. This is a fairly common and delicate problem. It negatively affects human life. This …

The interest to rare diseases has increased in the recent decades. Legislation seeks to
facilitate patients' access to innovative and effective treatment and to define incentives for …

Introduction Spinal muscular atrophy (SMA) is a very serious debilitating rare condition
mainly affecting newborns and infants. Areas covered The aim of current chapter is to …

Untitled Page 1 Page 2 2 АНОТАЦІЯ Даценко ІСНауково-методичні підходи до
удосконалення фармацевтичного забезпечення хворих на легеневу гіпертензію …

0 АННОТАЦИЯ Проанализированы современные подходы к организации оказания
фармацевтической помощи больным орфанным заболеваниям в системе …