orphan

Subscribe to orphan via RSS

Today, 26 April 2023, the European Commission (the Commission) published its long-awaited proposed amendments to the EU regulatory framework for medicinal products (the Proposals). We set out the key takeaways from these Proposals in our Advisory.

This is the culmination of a number of years’ work by the Commission, starting with the new pharmaceutical strategy for Europe (the Strategy) announced on 25 November 2020. We set out a summary of and reasons behind the Strategy in a previous Advisory, noting that the Strategy sought to ensure a high level of public health by increasing the availability, accessibility, and affordability of medicinal products throughout the EU and harmonize the internal market.

To meet these goals, the Commission has proposed substantial changes to the EU regulatory system, via a new Directive that will replace Directive 2001/83 (medicinal products for human use), and a new Regulation that will replace Regulation (EC) No 726/2004 (authorization and supervision of medicinal products), Regulation (EC) No 141/2000 (orphan medicinal products), and Regulation (EC) No 1901/2006 (pediatric medicines). The Proposals include changes to the regulatory protections available for medicinal products and orphan medicinal products and a new procedure relating to shortages of medicinal products. Continue Reading Proposed Amendments to the EU Regulatory Framework for Medicinal Products

In September 2021, we posted about the European Commission’s implementation of its new pharmaceutical strategy (which was also discussed in more detail in our posts on the strategy, proposed amendments to orphan and paediatric legislation and the industry response). Readers will be aware that the focus of the Strategy is on the availability, accessibility and affordability of medicinal products, based on the view that current incentive models do not provide an adequate solution for unmet medical needs or appropriately incentivise investment in innovation. As part of its work on the revision of the EU pharmaceutical legislation, the European Commission launched a public consultation to seek views on the current framework and on some of the proposals for changes in order to support the European Commission’s impact assessment for the revision of the legislation.

Following this consultation, the European Federation of Pharmaceutical Industries and Associations (EFPIA) published an article entitled “Back Innovation, Boost Access” with its response to the EU pharmaceutical strategy consultations and some of the concerns raised by the Commission. This expresses EFPIA’s position that innovation is only meaningful if patients have access to it, but highlights that access is not always in the control of the pharmaceutical companies. It also describes the current status of access to medicines in the EU Member States, some of the reasons for the delays to access and EFPIA’s proposals to improve patient access to innovative medicines.Continue Reading EFPIA’s Response to EU Pharmaceutical Strategy Consultations

We discussed in previous posts the Commission’s announcement at the end of 2020 of its new pharmaceutical strategy for the EU. One topic 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.  We have previously discussed recent consultations and stakeholder engagements by the Commission to explore possible changes to the legislative regime in these areas, including the possibility of reducing the ten-year market exclusivity period for orphan medicines and changing the criteria for determining the rarity of a disease.

Following the responses to these consultations, in May 2021, the Commission launched its latest public Consultation on the proposed revisions to the legislation.  The Commission’s statement accompanying the launch asserts that its evaluation “revealed shortcomings in the current system concerning in particular the development of medicines in areas of high unmet need for patients and their accessibility to all EU patients across the Member States.” Industry bodies representing the innovative pharmaceutical industry have now published their responses to the Consultation, as summarised below.
Continue Reading Industry’s response to the Commission’s proposed amendments to the EU orphan and paediatric legislation