"Access to Medicines in the Context of Rare Diseases" by Prof. Sandy Tubeuf and Romain Alderweireldt

2020 / Saturday, January 2nd, 2021

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

The research carried out by Professor Sandy Tubeuf and Dr Setti Rais (Louvain Institute of Data Analysis and Modeling in Economics and Statistics) on the economic stakes in the context of rare diseases shows that, although the regulation of orphan drugs has 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:

  • Investments in research and development mainly benefit rare diseases that emerge in adulthood, and rare diseases that affect young children receive the lowest investments, even though one in two rare diseases affect young children;
  • Even though there are more orphan drugs on the market, the very high prices charged by pharmaceutical companies are a barrier to effective access to these drugs.

To overcome these difficulties, these authors recommend, first, to effectively promote research by establishing and sharing databases that will allow real progress in knowledge on rare diseases.

Second, the authors emphasize the need to increase coordination and thus the negotiating power of political decision-makers in relation to industrialists in order to balance the sharing of the risks of drug development and to better negotiate their prices.

Finally, thirdly, in order to mitigate the profit objective of industrialists, the researchers advocate the application of fairer prices that would result from greater transparency of research and development costs and consideration of the common good of society.

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

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