Proposed changes to EU regulation risks progress on orphan drugs

The European Medicines Agency building

Plans to overhaul the EU regulation on rare disease therapies risk undermining two decades of progress, a new report claims.

The assessment, commissioned by the European Federation of Pharmaceutical Industries and Associations (EFPIA), was conducted by Dolon, a strategic consultancy specialising in rare diseases.

The European Orphan Medicinal Product Regulation was introduced in 2000 to incentivise the development of new medicines for people living with rare disease. As a result, the number of EU-approved medicines for rare diseases was in single digits two decades ago. To date, over 205 new treatments for orphan diseases have been approved.

The new study analysed the potential impact on innovation of the changes proposed by the European Commission in April 2023 as part of a wider overhaul of the general pharmaceutical package.

It found that, as currently written, the proposals would have a negative impact on the development of 45 products in Europe corresponding to a 12% decrease in innovation. This could deprive around 1.5 million European rare disease patients of a novel treatment option and would mean a further decrease by €4.5 billion in R&D spending in Europe.

“Europe’s policy and legislation on orphan medicines have been a success. They are a poster child for how the right combination of incentives and support can and do stimulate the development of medicines for people with rare diseases,” said Nathalie Moll, Director-General of the EFPIA. “This robust report on the impact of the Commission’s currently proposed regulatory changes should give us all pause for thought. We have come too far together to put progress for patients at risk.”

In contrast, the US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases.

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