As we have discussed in previous posts, at the end of 2020, the European Commission set out its vision to build a European Health Union with its announcement of the new pharmaceutical strategy for Europe (the new Strategy). In 2021, the Commission has begun to implement the new Strategy, as discussed here. One area that was identified as in need of revision was the unmet medical needs in areas currently not within the scope of the legislation governing rare diseases and paediatric medicines. The Commission has been focused on this area for a number of years, and there have been a number of consultations and stakeholder engagements to explore possible changes to the legislative regime. We set out below a summary of the Commission’s proposals on orphan and paediatric medicinal products.
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Yesterday, the EMA launched a new secure online portal called IRIS for the submission of applications for orphan designation and the management of post-designation activities. The aim is for the portal to be used for all activities relating to orphan designation, including applying for orphan designation, requesting pre-submission meetings, responding to requests for supplementary information and transferring orphan designation to a new sponsor. The hope is that this will provide a “comprehensive procedural and scientific support system for orphan designations”, and IRIS is being treated as a “pilot for [a] future Agency-wide platform for procedure management”.…
Continue Reading Launch of the EMA’s Orphan Designation Portal
As reported in our previous post, at the end of last year, the European Commission published a draft amendment to Regulation No 847/2000 regarding the concept of “similar medicinal product” for the purposes of the Orphan Medicinal Products Regulation. Last week, the final Regulation was published: Regulation (EU) 2018/781. The new Regulation…
On 22 March 2018, the European General Court handed down its judgment in Case T-80/16 Shire Pharmaceuticals Ireland v EMA. Shire had sought annulment of the EMA’s decision refusing to validate its 2015 application for designation of its medicinal product Indursulfase-IT as an orphan medicinal product for the treatment of Hunter Syndrome. This decision is particularly important for innovator companies who invest in R&D for rare diseases, and will likely have an impact on incentivizing this type of research.
In 2001, a Shire product containing the active substance idursulfase was designated as an orphan medicinal product for the treatment of Hunter Syndrome. This was followed in 2007 by the grant of a marketing authorisation for the medicinal product Elaprase, containing the active substance idursulfase, and administered as a solution for intravenous infusion. In parallel, Shire started developing another medicinal product containing the same active substance. However this product, Idursulfase-IT, could be delivered intrathecally, whereas Elaprase did not cross the blood-brain barrier. Intrathecal administration of the new product allowed treatment of cognitive disorders associated with a severe form of Hunter Syndrome.
The European Commission has finally published the draft amendment to Regulation No 847/2000 regarding the concept of “similar medicinal product” for the purposes of the Orphan Medicinal Products Regulation. A key incentive of this Regulation is the ten-year market exclusivity that protects an orphan medicinal product, whereby national competent authorities shall not accept an application for authorisation, or grant an authorisation, or accept an application to extend an existing authorisation, “for the same therapeutic indication, in respect of a similar medicinal product” as the orphan product. Regulation 847/2000 defines “similar medicinal product” in this context, and sets out a number of examples of what kind of products will be similar.
Following a consultation last year, the Commission intends to make changes to the 17-year-old text to account for the rise of cell therapies and other advanced therapy medicinal products, the nature of which do not fit neatly into the current definitions.