From Rare to Neglected Diseases: A Sustainable and Inclusive Healthcare Perspective for Reframing the Orphan Drugs Issue

Abstract

This work is about how healthcare issues can be reframed from a sustainable and inclusive development perspective. Focusing on the case of orphan drugs and rare diseases, first, a country-based review of the main regulatory approaches to orphan drugs is conducted; then, the main contributions of the literature are reviewed to identify dominant views and the way the problem is more commonly framed. The main findings reveal that the dominant regulatory approaches and theoretical interpretations of the problem are mainly based on economic considerations. However, this does not seem to have led to very satisfactory results. Reflecting upon what the sustainability perspective can highlight with reference to healthcare, substantial connections between the orphan drugs issue and that of neglected diseases are highlighted. These connections suggest reframing the orphan drugs issue as a social equality and inclusiveness problem, hence the need to adopt a sustainable and inclusive development perspective. As a key sustainable development goal (SGD) to be shared by all nations, healthcare should always be approached by putting the principles of sustainable and inclusive development at the core of policy makers’ regulatory choices. Accordingly, we think that the orphan drugs issue, like that of neglected diseases, could be better faced by adopting a social equality and inclusiveness perspective.

Are rare diseases overlooked by medical education? Awareness of rare diseases among physicians in Poland: an explanatory study

“…For this reason, in 2009 the Council of the European Union recommended that RDs should be prioritized and that all member states should adopt a national strategy for RDs [ 10 ]. Nevertheless, even though the right to healthcare of all Polish citizens is protected constitutionally and everyone is entitled to equal access to public healthcare, RDs are often overlooked or even neglected by drug makers, doctors, developers, government officials and policy makers, public health programs and news media [ 11 , 12 ]. For example, although most European countries have either implemented or created national plans for RDs, Poland still has not adopted such a strategy [ 13 , 14 ].…”

Modern views on medical provision of patients with rare diseases of the circulatory system in Ukraine and the world

“…Першою країною, в якій було прийнято у 1983 р. пакет законів щодо РЗ, зокрема рідкісних захворювань системи кровообігу, стали США [10]. В Україні наказом Міністерства охорони здоров'я України (МОЗ України) від 27 жовтня 2014 р.…”

Orphan Drug Policy

“…They disproportionately impact children: 50% of new cases are in children and are responsible for 35% of deaths before the age of 1 year, 10% between the age of 1 and 5 years, and 12% between 5 and 15 years [1]. However, the most neglected diseases by pharma industries are tropical diseases that are caused by parasites and spread by insects or contaminated water or soil, for example [2]. Development of orphan drug policies is least concern of pharmaceutical companies.…”