This article appeared in “Le Chaînon – la revue des associations de patients et de proches”, N°52 in September 2020.

Research carried out by Professor Sandy Tubeuf and Dr Setti Rais (Louvain Institute of Data Analysis and Modeling in Economics and Statistics) on economic issues in the context of rare diseases shows that, while orphan drug regulation has indeed led to an increase in the number of clinical trials and scientific publications concerning rare diseases, this increase has had at least two perverse effects :

• Investment in research and development mainly benefits rare diseases that occur in adulthood, and rare diseases that affect early childhood receive the least investment, despite the fact that one in two rare diseases affects infants ;
• Even though there are more orphan drugs on the market, the very high prices charged by pharmaceutical companies are an obstacle to effective access to these drugs.

To alleviate these difficulties, these authors recommend, firstly, that research be genuinely promoted by establishing and pooling databases that will enable real progress to be made in our knowledge of rare diseases.

Secondly, the authors insist on the need to increase coordination and thus the bargaining power of political decision-makers in relation to manufacturers, in order to balance the sharing of drug development risks and better negotiate drug prices.

Thirdly and lastly, to mitigate the profit motive of industrialists, the researchers advocate fairer pricing based on greater transparency of research and development costs and consideration of the common good of society.

Professor Sandy Tubeuf, Health Economics, UCL and Romain ALDERWEIRELDT, Foundation 101 Genomes

For more information :

https://www.regardseconomiques.be/index.php/index.php?option=com_reco&view=article&cid=198.