SIOPE and CCI-E joint position paper:
The European Society for Paediatric Oncology (SIOPE) and Childhood Cancer International – Europe (CCI-E) are representing respectively health professionals and parents, patients and survivors in paediatric oncology across Europe. Childhood cancer is the leading cause of death by disease in children over the age of one, with 35,000 new diagnoses annually. Over 60% of Europe’s 500,000 childhood cancer survivors face long-term side effects in adulthood. Survival rates vary by up to 20% between Eastern and Western Europe due to disparities in access to standard-of-care diagnosis, treatment and research. Today, neither the EU Paediatric nor the EU Orphan Regulation have significantly improved young cancer patients' survival chances, with only 16 anticancer medicines authorised for a specific paediatric cancer indication in the last ten years, compared to over 150 for adult cancers. We are calling for a Revision of EU Pharmaceutical Legislation to accelerate innovation and address the unmet medical needs of young cancer patients.
SIOPE and CCI-E have identified 8 key priority topics, which are essential for the Childhood Cancer Community. We have detailed in our joint position paper which topics should be upheld, and which ones should be amended to improve the outcomes of young people with cancer across Europe. Priority Topics:
1. Definition of Unmet Medical Need (UMN) and High Unmet Medical Need (high UMN)
Directive Article 83 and 70, Regulation Article 162
We welcome the introduction of important concepts of UMN and HUMN in the EU Commission’s proposal for the Revision of the EU Pharmaceutical Legislation. We think the UMN and HUMN concept should allow a medicinal product to be considered as addressing those needs if it reduces acute or long-term toxicity.
2. Molecular Target (MOA)
Regulation Article 75
We welcome the introduction of paediatric medicine development based on the molecular target. Indeed, under the current framework, there is no obligation for a medicine developer to submit a Paediatric Investigation Plan (PIP) if the medicine originally developed for an adult cancer does not exist in children, even when the medicine has a relevant mechanism of action for a given type of paediatric cancer from a biological/molecular perspective.
3. Early Start of the Peadiatric Investigation Plan (PIP)
Regulation Article 76
We would like to safeguard incentives and obligations around Paediatric Investigation Plans to achieve medicine development for Children with Cancer in Europe.
4. Academic Repurposing
Regulation Article 48
We highly stress the support of the possibility for a non-for-profit entity’s (academical) studies outcome to be put on the product information of medicines via a procedure led by EMA instead of a solely industry powered one. This measure supports academic-led research, which is essential in rare disease areas for medicine development and avoiding off-label medicine prescription, as well as an increase in access to lifesaving medicines for patients all over Europe.
5. Improved Access to Novel Medicines (Equal Access)
Directive Article 59, 81 and 82
In order to close the gap of 20% difference in survival rates between East and West Europe medicine must be made available in all member states instead of just financially favourable ones for pharmaceutical companies due to country size and economic power.
6. Improved Access to Essential Medicines (Critical Medicine List)
Regulation Chapter X
We expect as patient and healthcare organisations to be involved in the consultation process of the establishment of the Critical Medicine List, to avoid shortages of crucial medicines in paediatric cancer.
7. First-in-Child Innovation
Regulation Article 71
The current proposal does not include specific incentives for first-in-child development and first-in-child marketing authorisation of medicines. Therefore, we strongly recommend including a first-in-child marketing authorisation incentive, as this would be expected to increase commercial interest in the development of medicines specific to paediatric cancers (and paediatric rare diseases).
8. Hospital Exemption
Directive Article 2
It is essential that it stays possible for hospitals to create treatment options for patients for whom no or less favourable medicine options are available to cure their life-threatening diseases. Academia should be allowed to continue to drive innovation in paediatric oncology and address challenges of ATMP production through a cross-border network of academic manufacturing centres that enables decentralised and standardised production of ATMPs under harmonised Good Manufacturing Practices (GMP).