Conference “Medicines for rare diseases and children: learning from the past, looking to the future”

Background information

In the beginning of this century, the EU put in place two regulations: one for medicines for rare diseases (‘orphans’) in 2000 and one for medicines for children in 2006. The ultimate objective of the legislation was to increase therapeutic choices for children and patients suffering from rare diseases. How? By bringing more medicinal products for those therapeutic areas to the market.

Following the publication of the 2017 Commission Report on the Paediatric Regulation, the European Commission also announced a joint evaluation of both the paediatric and the orphan Regulations covering their effects and impacts so far.

To conduct this evaluation, the Commission will use the outcomes of various recent studies and of public and targeted consultations that were finalised earlier in 2019.

About the meeting

On 17 June 2019, the European Commission’s DG SANTE organised a conference on Medicines for rare diseases and children. About 150 experts were present from across the EU, representing national governments and health authorities, academia, patient and health professionals’ organisations and pharmaceutical industry.

The conference took place as part of the joint evaluation of both EU Regulations on Orphan Medicinal Products and Paediatric Medicines. It allowed stakeholders to make their voices heard and come forward with their views, suggestions and ideas with respect to the two Regulations.
The event was moderated by Professor Patrick Deboyser (European College of Parma) and opened by Commissioner Vytenis Andriukaitis and former MEP Françoise Grossetête.

More information:

• Programme for the day, including an outline for the topics of the break-out sessions.

Topics addressed

Olga Solomon (DG Health and Food Safety) and Michael Berntgen (EMA - European Medicines Agency) shared with the audience their perspectives on the two Regulations and to set the scene for some highly interactive and participatory sessions that determined the rest of the day.

In the morning, break-out sessions were organised during which five main topics were addressed: unmet medical need, incentives, medicines for children, ‘from R&D to patients’ and scientific developments.

Discussions focused among others on the following topics:

• The importance of a common understanding and of quantifying unmet medical need.
• The need to increase basic research and the collection and sharing of data in order to speed up the process from Research and Development to Patient.
• The need for incentives that support real innovation in the orphan therapeutic landscape
• Better guarantees that a reward is proportionate including a better coordination and identification of priorities.
• The need for a dedicated R&D strategy for paediatric only medicines development.
• The benefits of multi stakeholder engagement to overcome changing evidence standards, taking into account new scientific developments.

In the afternoon ‘Open Space’ participants had the possibility to come forward with their own themes, which they wanted to discuss with the audience.

Eight different themes were selected and formed the basis for dynamic discussions in small group sessions:

1. Making better use of shelved drugs;
2. How to avoid ‘evergreening’ of patents;
3. Further global harmonisation and coordination;
4. Innovation for Medicines;
5. The continuum of evidence generation;
6. Value-based pricing;
7. How to determine rarity;
8. More targeted treatments for children’s medicines.

In her closing address, DG Health and Food Safety Director General Anne Bucher underlined the importance of the conference, which has generated a wealth of ideas and suggestions. These will become an important input to the Regulations’ evaluation process including reflections on the future.

Outcomes

• Detailed Conference report
• Conference poster