Coordination between the Member States to guarantee a high level of health protection with regard to Sanfilippo syndrome

16.1.2019

Question for written answer E-000211-19
to the Commission
Rule 130
Antonio López-Istúriz White (PPE)

Sanfilippo syndrome is a rare disease that presents in children between two and six years of age and causes intellectual deterioration and developmental delays. Children suffering from the disease usually do not survive past adolescence. In many Member States, gene therapies are being developed to treat this fatal disease and other rare and ultra‐rare diseases. However, lengthy bilateral negotiation processes between pharmaceutical laboratories throughout the Member States are denying patients access to treatment, resulting in discrimination against children in the EU who are suffering from these diseases and who, in many cases, do not receive treatment in time.

Article 168 TFEU states that the Commission may encourage cooperation between the Member States. The EU urgently needs to take steps to make the Member States work together and speed up negotiation processes so that they can provide access to these treatments throughout the EU. We are talking about saving children’s lives and helping families affected by serious disorders like Sanfilippo syndrome.

1. What is the Commission doing to ensure that children with rare diseases receive treatment in time?

2. Will it take steps to encourage the Member States to work together to improve access to advanced treatments for rare diseases?