RARE Daily

Eurodis, Ahead of EC Release of Revisions to Orphan Regulations, Comments on Shortcoming of Leaked Draft

March 21, 2023

Rare Daily Staff

The European rare disease organization Eurodis, responding to a leaked draft of soon to be released legislative revisions to the Orphan Medicinal Products Revisions, offered its recommendations on steps Europe can take to accelerate the development of therapies for rare diseases.

Orphan Medicinal Products (OMP) regulation, similar to the Orphan Drug Act in the United States, was introduced in 2000 to incentivize drug companies to invest in the development of medicines to treat rare diseases.

While the regulations have had a positive effect on people living with a rare disease, the modernization of the regulations are “long overdue.” It noted that only 6 percent of known rare diseases have an approved treatment, and 22 percent of people with a rare disease cannot get the treatments they need because they are not available where they live.

“While the Commission’s proposed Revision has yet to be formally adopted, we perceived from a recently leaked draft text a number of shortcomings that we hope will be addressed—either by the Commission ahead of the official publication of the revision, or by policymakers across EU institutions as the Commission’s proposals make their way through the legislative process,” wrote Julien Poulain, Eurodis’ communications manager in a blog post.

The organization is calling for improvements in four key areas: addressing all unmet needs, strengthening patient engagement, sharpening the language to reduce room for interpretation, and increasing targeted incentives to encourage industry to develop medicines for conditions that currently have no therapeutic options and increasing the period of exclusivity for drugs that launch first in Europe.

“There is much that the Commission is planning to propose that we welcome, from maintaining the aforementioned prevalence threshold to keeping the rewards of market exclusivity lengthy and globally competitive,” wrote Poulain. “Yet it is vital that the proposed Revision to the OMP Regulation addresses the wide range of unmet medical needs affecting Europe’s rare disease population, and the European Commission must fulfil this obligation in the final stretch of preparing its proposals.”

Stay Connected

Sign up for updates straight to your inbox.