Improving the quality of life of people living with a rare disease
28.9.2022
Question for written answer E-003235/2022
to the Commission
Rule 138
Stelios Kympouropoulos (PPE), Maria da Graça Carvalho (PPE), István Ujhelyi (S&D)
People living with a rare disease suffer from many chronic and debilitating manifestations of their condition and have a lower quality of life than the general population. The disease mechanisms for many rare and ultra-rare diseases are still unknown, which makes diagnosing them challenging and researching and developing treatments unpredictable. One approved orphan medicinal product does not equate to the alleviation of all unmet needs.
In the context of the revisions of the EU’s general pharmaceutical legislation, the Orphan Medicinal Products Regulation[1] and the Paediatric Regulation[2], and to ensure that the unmet medical needs of the up to 36 million people living with a rare disease are not overlooked:
- 1.How will the Commission further encourage key innovations in Europe, while acknowledging that there are varying levels of unmet medical needs across different rare diseases?
- 2.Would it consider including, for example, ultra-rare haematological disorders in the socio-economic disease burden assessment project of the European Partnership for Rare Diseases under Horizon Europe? Will it also consider looking at the mental burden of rare diseases?
- [1] Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products, OJ L 18, 22.1.2000, p. 1.
- [2] Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004, OJ L 378, 27.12.2006, p. 1.