“When my son was diagnosed with cancer, he was treated with old, off-label drugs.  The same happened two years later when he relapsed two years later, despite a poor prognosis. Not a single experimental drug was on trial to give us hope. We have to change this and revise the Paediatric Medicines Regulation to ensure new cancer drugs with a relevant mechanism of action reach young patients faster and safely and get approved for paediatric use”

Delphine Heenen, Committee Member of Childhood Cancer International – Europe shared her powerful testimony during a policy dialogue organised by the Association of European Cancer Leagues (ECL), the European Fair Pricing Network (EFPN), the European Society for Paediatric Oncology (SIOP Europe) and the European Public Health Alliance organised to discuss the improvements needed to the 2000 Orphan Medicinal Products Regulation and the 2006 Paediatric Regulation to ensure the best treatment for children and those suffering from rare diseases as “one-size-fits-all” incentives do not lead to a sustainable and equitable pharmaceutical system. 

The recommendations from the same organisations, published in conjunction with the policy dialogue call for development and equal access to affordable medicines for children and patients with rare diseases, which is patient-centred, based on transparency and accessibility, driven by patient and public health needs. In addition, ECL, EFPN, SIOP Europe and EPHA make the following recommendations:

  • Setting research priorities that target unmet medical needs based on transparent and objective criteria.
  • Strive for future-proofed incentives and investments which are targeted to support  to support early-stage medicine development (e.g. for children) and ensure medicines with proven added value and medicines in areas where there currently are no treatments
  • Set conditionalities to guarantee the correct implementation of the current Regulations and tackle the wide inequalities in availability, accessibility, and affordability in the EU
  • Increase competition to reduce prices
  • Support research and scientific developments geared towards unmet patient needs

The European Commission has just opened a consultation to review the legislation on paediatric and rare diseases treatments which closes on 31 July 2021. 

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