European Trade Group Warns Proposed Overhaul to Orphan Regulations Decrease Innovation and Hurt Patients

Rare Daily Staff

Proposed changes to the European Union’s Orphan Medicinal Product Regulation will reduce investment in rare disease drug development and stymie many rare disease patients from seeing a novel therapy for their conditions come to market, according to a new analysis prepared for an industry group.

The analysis, commissioned by the European Federation of Pharmaceutical Industry Associations and prepared by the rare disease strategic consultancy Dolon, found that the European Commission changes proposed in April 2023 as part of a wider overhaul to general pharmaceutical industry regulation could reduce R&D spending by $4.8 billion (€4.5 billion) and deprive about 1.5 million people with rare diseases of a novel treatment as an expected 45 products over a 15 year period would not come to market.

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

If broader changes to existing incentives for innovation are introduced, such as further reducing orphan market exclusivity and tighter criteria for securing orphan designation, Dolon warned that there could be 135 fewer orphan products between 2020 and 2035, and deeper impacts on patients, productivity, and research spending.

“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 European Federation of Pharmaceutical Industries and Associations. “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.”

Photo: Nathalie Moll, director-general of the European Federation of Pharmaceutical Industries and Associations