On Wednesday 25 November, the European Commission published its long-awaited pharmaceutical strategy, in which it commits to examining ways to improve accessibility, sustainability and security of supply in Europe (see EUROPE 12608/24). In an inception impact assessment, published alongside the strategy, the Commission puts forward a number of specific options regarding orphan and paediatric medicines.
The impact assessment is open until 6 January 2021 for comment under the ‘better regulation’ procedure.
A commitment made even more necessary by the crisis
At a press conference, Health Commissioner Stella Kyriakides observed that presenting a strategy of this kind was included in the mission letter she received from President Ursula von der Leyen at the beginning of her term in office. Since then, we have lived through the Covid-19 pandemic: the document attempts to respond to all of the pre-existing challenges and the challenges brought to the fore by the crisis.
The Communication, which is over 20 pages long, consists of a list of actions, mainly involving initiating studies or discussions between Member States and/or with industry.
It has four objectives: - to ensure accessibility to affordable medicines and fulfilling unmet medical needs; - to support a competitive, innovative and sustainable European pharmaceutical industry; - to enhance security of supply; - to project a strong EU voice globally.
Fairly vague promises
With regard to strategic autonomy, which was one of the main lessons from Covid-19, the Commission prefers structured dialogue before considering legislative measures.
On the difficult issue of shortages of medicines, the Commission is waiting for the results of a study launched this year before considering regulatory changes. These could include “stronger obligations on industry to ensure the supply of medicines, earlier notification of shortages and withdrawals, enhanced transparency of stocks across the supply chain, and a stronger coordinating role for the European Medicines Agency in monitoring and managing shortages”.
With regard to generic and biosimilar medicines, the Commission undertakes to examine targeted policies that support greater competition. This may include further clarifying the provisions for the conduct of trials on patented products to support generic and biosimilar marketing authorisation applications (the so-called ‘Bolar’ provision).
Impact assessment on orphan and paediatric medicines
The Commission is most active in the area of modernising the regulations for paediatric and orphan medicines (1901/2006 and 141/2000 respectively), and has published an inception impact study on the issue. In her speech, Commissioner Kyriakides regretted the fact that 7,000 citizens in the EU are living with a rare disease and 95% of them have no treatment options.
In its impact assessment, the Commission reflects on how to improve incentives for the pharmaceutical industry.
With regard to paediatric medicines, it proposes making extensions of the Supplementary Protection Certificate (SPC) conditional on timely completion of a Paediatric Investigation Plan (PIP) and on the product’s placement on the market in most/all Member States, with the possibility of other rewards. Other options include replacing the SPC with another reward or keeping it solely for paediatric medicines.
With regard to rare medicines, the Commission is considering changing the current threshold for identifying rare diseases that need specific support for developing medicines, i.e. to “fewer than 5 patients in 10,000”, and changing the market exclusivity incentive.
The inception impact assessment will be followed by a new, more detailed impact assessment in the first quarter of 2022.
No change in pricing and reimbursement
Unsurprisingly, the pharmaceutical strategy does not really address the issue of pricing and reimbursement. A European official acknowledged the problems posed by the lack of transparency, but noted that the issue of pricing falls under domestic jurisdiction. “This strategy will not propose any change in competences. However, we are planning to foster a dialogue on prices and reimbursement. We’ll develop cooperation in a group of competent authorities. We hope with this to foster an environment for greater transparency. Also, an initiative to foster transparency on research costs (and make a link with the cost of medicines)”, our source notes.
The Commission's strategy can be found at: https://bit.ly/2V05vBV and the inception impact assessment at: https://bit.ly/39cfjks (Original version in French by Sophie Petitjean)