Eurordis favors EU vote advancing rare disease research, treatments

Parliament supports reforms to pharmaceutical legislation for member states

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by Mary Chapman |

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Eurordis has welcomed as a “significant stride forward” the latest European Parliament plenary vote for compromise amendments to bridge the gap between accelerated scientific progress and patient care.

The recent vote was in favor of proposed reforms to the European Union’s general pharmaceutical legislation, which includes proposed regulatory updates on pediatric as well as orphan medicines. Such medicines are used for the diagnosis, prevention, or treatment of a chronically debilitating rare condition — one affects no more than 5 in every 10,000 EU residents — such as aromatic l-amino acid decarboxylase (AADC) deficiency.

Eurordis, which stands for the European Organization for Rare Diseases, is a nonprofit alliance of more than 1,000 rare disease patient organizations from 74 countries. It aims to improve the lives of the 30 million individuals living with a rare disorder in Europe.

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Reforms to advance ‘treatment development and access,’ Eurordis says

Specifically, members of the European Parliament at large — meaning they represent the entire EU population — voted in favor of amendments passed in March by the Parliament’s Committee on the Environment, Public Health, and Food Safety. At the time, Eurordis lauded the vote as a “meaningful step” forward.

These amendments seek to improve the development and availability of treatments for rare diseases within the EU. As proposed by the European Commission, the amendments maintain the nine years of market exclusivity currently given to orphan medicines. However, market exclusivity increases up to 11 years for treatments for diseases that currently need them the most.

Under the PRIME scheme in Europe, which works to advance the development of medicines for unmet medical needs, orphan medicines would be covered. There would also be greater incentives for academic research and mandatory patient participation in European Medicines Agency (EMA) consultations, particularly when defining guidelines to identify unmet medical needs.

Moreover, the amendments advocate for the establishment of a comprehensive policy framework for rare disorders, changes to regulatory data protection, and more efficient processes for advanced treatments.

“Today’s plenary vote by the European Parliament on pharmaceutical reforms marks a significant stride forward for the 30 million Europeans living with rare diseases and their families. Against the backdrop of 94% of rare diseases still lacking a dedicated treatment, we welcome the genuine political will that has been demonstrated to improve treatment development and access,” Valentina Bottarelli, Eurordis’ public affairs director, said in the organization’s statement.

Eurordis specifically supports parliamentary approval of “crucial” proposals, including the introduction of progressive market exclusivity for rare disease treatments and the refinement of criteria covering “significant benefit” and “high unmet medical needs,” Bottarelli said.

“These changes, along with enhancing the PRIME scheme and ensuring patient participation in EMA consultations, represent not just progress but the result of our community’s advocacy to close the gap between fast-paced scientific discoveries and patient care,” she noted.

In its reaction to the parliamentary vote, Eurordis reiterated its long-standing call for a European Action Plan for Rare Diseases, citing a “strong and broad political consensus” on what it calls an urgent need. The organization also called for incorporating an orphan drug development plan into the legislation to advance the development of more and better treatments.

“As we progress to the next stages of the proposed reform, Member States should acknowledge and build upon the Parliament’s recommendations,” Bottarelli said.

AADC deficiency, a genetic disorder, affects the nervous system, hindering nerve cell communication. The disease causes developmental delays and problems with movement and behavior, and patients generally require lifelong care. Globally, fewer than 350 cases have been reported.

The disease’s estimated prevalence in the EU is 1 in every 116,000 residents.