Today, EFPIA has filed its response to the European Commission public consultation on the revision of EU rules on medicines for children and rare diseases. To support our submission and to inform the evaluation of the EU Paediatric and Orphan Medicinal Products Regulations, EFPIA has published a report by Dolon and illustrative case studies on five different therapies developed in collaboration with the Office of Health Economics.

Children and people living with a rare disease have greatly benefited from the progress achieved through the European Paediatric and Orphan Regulations. The Paediatric Regulation contributed to significantly expanding research and development, increasing the number of medicines delivered to children and enhancing the paediatric-specific expertise. The EU Orphan Regulation, since its advent in 2000, has incentivised the pharmaceutical sector to deliver treatments for up to 6.3 million patients in the EU.

The regulatory and economic incentives introduced by both Regulations have been a great success, progressing care in many overlooked conditions and supporting the creation of numerous SMEs in Europe. These incentives have worked and should remain. Pharmaceutical innovation does not occur in a vacuum, as the European Commission considers changes to legislation and the incentives framework, it will be important to maintain and strengthen Europe’s delicate innovation ecosystem. In that context, aiming to reduce the period of market exclusivity, narrow development incentives, promote faster generic competition and cap the aggregate number of patients that can benefit from a designated orphan product, is likely to drive research investment away from meeting the needs of patients and weaken a sector at the very heart of EU competitiveness and service to citizens. Instead, we need to maintain broad incentives to facilitate multiple avenues of research, consider additional, targeted incentives for certain treatments and identify and resolve the multi-factorial barriers to access. Economic incentives are not the only solution to tackle the challenges in underserved areas for children and people living with rare diseases. The report and case studies that we publish today take an evidence-based, holistic approach to identifying and addressing the scientific, regulatory and economic challenges involved in meeting the needs of children and people living with rare conditions across Europe and beyond. The case studies emphasise the complexity of the research ecosystem and show that development of new therapies is driven by scientific or technological developments in unrelated areas.

Speaking on the publication of the report and case studies, EFPIA Director General, Nathalie Moll said: “Science, economics, and policy coalesce to inform investment decisions. It follows that these three dimensions must be addressed in concert for innovation to flourish in extremely rare and paediatric-onset diseases. There is a risk that the Commission’s current proposals may lead us to the worst-case scenario; limiting innovation without addressing access. Over recent months we have seen the power of science and innovation in addressing health challenges, as demonstrated by the hard-won progress in fighting COVID-19. To bring that power to bear on the needs of children and people living with rare disease means all stakeholders having a ‘moon-shot’ mindset and working in partnership; to foster an innovation oriented eco-system for rare and paediatric disease addressing the scientific, regulatory and economic barriers to innovation and access. Our hope is that the report and case studies we publish today present a tangible contribution to the creation of such an eco-system.”

EFPIA’s response to the consultation will be made public on the Commission’s website and is also available here.