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.

Commission evaluation of orphan and paediatric legislation

On 11 August 2020, the Commission published its evaluation of the legislation governing medicines for rare diseases and children. The evaluation aimed to assess the strengths and weaknesses of Orphan Regulation 141/2000 and Paediatric Regulation 1901/2006, and took into account recent studies on paediatric and orphan medicines, as well as extensive consultation with stakeholders.

The evaluation showed that the regulations have stimulated research and development of medicines to treat rare diseases and of medicines for children. However, the regulations were stated to have not adequately supported development in areas where the need for medicines is greatest. A number of shortcomings were noted, including:

  • 95% of rare diseases still have no treatment option;
  • Failure to sufficiently address the highest unmet needs for children (e.g. mental and behavioural disorders and neonatology);
  • Lack of consistency in accessibility and availability to all EU patients across the Member States;
  • Inadequate measures to adopt scientific and technological developments.

There was particular focus in the report on the incentives under the regulations, and whether these encouraged the development of medicines. It was noted that advances in science, such as personalised medicine and the use of biomarkers, add complexity to the regulatory framework, and could lead to a proliferation of protection periods. In addition, the report questioned whether the protection periods were justified in all cases or truly stimulated development. In relation to paediatric medicines, it noted that there is no dedicated instrument to direct development, and development was largely driven by adults’ needs. Further, it was noted that methods available to shorten protection periods, such as to reduce the 10 year orphan exclusivity to 6 years if the product is sufficiently profitable, were under-used. The procedures were also noted to be inefficient and burdensome.

The aim was for the report to guide future changes to the legislative framework, and to improve and accelerate patients’ access to safe, effective and affordable medicines. The revision of the two pieces of legislation is also one of the actions of the new Strategy.

Commission consultations

Following this report, the Commission has published consultations seeking views on suggested amendments to the legislation.

In November 2020, the Commission published an Inception Impact Assessment, which proposed various options and invited citizens and stakeholders to provide feedback. The options in relation to paediatric medicines were:

  1. 6 month SPC extension will remain the main reward for completion of the PIP;
  2. 6 month SPC extension only for medicines addressing unmet needs for children;
  3. 6 month SPC extension for completion of the PIP AND novel reward for medicines addressing unmet needs;
  4. Novel reward for medicines addressing unmet needs (no 6 month SPC extension).

The options in relation to orphan medicines were:

  1. Market exclusivity will remain the main reward (but its duration will be variable);
  2. Market exclusivity will remain the main reward (but its duration will be variable) AND changes would be made to the criteria for designation to better identify rare diseases;
  3. Market exclusivity will remain the main reward (but its duration will be variable) AND novel reward for medicines addressing unmet needs;
  4. Novel reward for medicines addressing unmet needs (no market exclusivity).

There were many and wide ranging responses to the consultation, with differing views on the effectiveness of the current regime. EFPIA, for example, noted that any revision of the rewards and incentives should be designed to maintain and improve Europe’s strong basis for innovation, and that reducing rewards or limiting them to less prevalent diseases would risk leaving out significant numbers of patients. EFPIA also noted that there were many factors outside of the legislation, such as pricing and reimbursement and the operation of the national healthcare system, that may impact the access and adoption of these medicines.

Having considered the responses to the Impact Assessment, in May 2021 the Commission launched a public consultation to revise the legislation on medicines for children and rare diseases. Interested parties are invited to share their views via a questionnaire until 30 July 2021. The questionnaire focuses on barriers to development of treatment for rare disease and for children, and asks for views on a number of approaches that could be used to better address the needs of patients. For example, responders are asked whether additional incentives should be offered where the medicine is developed with the potential to address unmet needs of patients, and for views on the best way to reward innovation where other treatments are available. Comments are also requested on whether access to medicines in all Member States should be a condition on obtaining any reward.

The current intention is for the Commission to publish its proposal for new legislation in the first quarter of 2022.