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Rare Disease Research In Europe – Will Academia Pick Up The Gauntlet?

Updated: Jan 13, 2021

Over the past few decades, the European Commission has sought to activate the pharmaceutical industry to research rare diseases. In the 80s and 90s, pharmaceutical companies and policy makers alike began to recognize that low patient numbers make clinical trials relatively laborious. Additionally, recouping investment costs once the drug is approved and marketed is challenging when there are few patients. Taken together, this provides little incentive for research and development. The EU Regulation on Orphan Medicinal Products (2000) sought to address these problems and incentivise research to benefit rare diseases.

The European Commission recently took the 20th anniversary of the Orphan Regulation as an opportunity to re-evaluate its impact on medicinal products for use in the paediatric population. Their evaluation leans on three independent studies, reports by the European Commission and the European Medicines Agency, as well as numerous public and stakeholder consultations. These Regulations, the Commission concluded, have fostered the development and availability of medicines. Most efforts have, however, been focused on specific therapeutic areas. Therefore, no advances have been made across the board. The high prices of developed drugs combined with the prolonged market exclusivity have caused issues of access to increase substantially.

The evaluation has directly informed a new European Commission Pharmaceutical Strategy for Europe. In this strategy, the Commission proposes shortening administrative procedures and investing more money on rare disease research, in addition to promoting increased and faster sharing of research data. This final proposition will likely not sit well with the pharmaceutical industry. It does, however, seem to open the door for academia to play a larger role than thus far evidenced.

The European Medicines Agency is right on the money here. They launched an initiative last month, announced in Nature, providing free scientific advice to academia and non-profit organisations working on orphan drugs. The goal, the Agency says, is to enhance the chance of success through aligning research with regulatory requirements early on in the drug development process. Time, and perhaps another evaluation, will tell whether these and dovetailing initiatives to stimulate academic research on orphan medicinal products will deliver. In the Netherlands, at least, universities are primary players in several Social Pharmaceutical Innovation initiatives, so early signs are hopeful.


Written by: Tineke Kleinhout-Vliek,




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