EFPIA response to Inception Impact Assessment on Orphan and Paediatric Regulations

EFPIA members are committed to ensuring that unmet needs are addressed and that available treatments reach all European patients. Children and rare disease (RD) patients have greatly benefited from the progress achieved through the Paediatric and Orphan Regulations. The Paediatric Regulation is meeting best-case expectations from its impact assessment (IA) conducted in 2004. The Orphan Regulation has incentivised the pharmaceutical sector to deliver treatments for up to 6.3M patients in the EU. Any revision of the rewards and incentives should be designed to keep and improve Europe’s strong basis for innovation and should avoid measures aimed to address affordability issues which are best dealt with elsewhere.

We agree that new incentives, in addition to dedicated research funding and accelerated regulatory pathways, can stimulate R&D in areas of unmet need. For many diseases, scientific knowledge and diagnostic capabilities must be built, and clinical endpoints developed. Any process to identify unmet needs must build on a multi-stakeholder discussion and include inter alia patients and their caregivers. Said system must also spill over to and be recognised by downstream decision-makers, ie HTA bodies and payers.

Reducing rewards for fulfilling the PIP obligations or reducing incentives to focus only on ultra-RD where no treatment exists or on first in class innovation fail to understand the science behind biopharmaceutical research. Such measures will not redirect R&D into areas of needs but rather risk undermining Europe’s attractiveness as a region for innovation. Limiting the RD-designation criteria to less prevalent diseases risks leaving out significant numbers of pathologies and patients; reducing prevalence thresholds or using a cumulative prevalence criterion fails to recognise the importance of follow-on innovation to achieve meaningful patient outcome.

Developing medicines for children and RD patients only provides a very marginal economic case, which is why these areas are not attractive to generic and biosimilar developers. The concept of overcompensation is not established: the staff working document (SWD) showed that only 14% of OMPs have yearly turnover of >€100 million. Making rewards and incentives conditional on launch obligations fails to understand the economics behind biopharmaceutical research and the complexities of launching medicines across the EU.

We call on the Commission to work with stakeholders and urgently identify the root causes of access differences to find targeted solutions. Root causes are multifactorial and cannot be reduced to company decisions. Any access solutions will require a close collaboration of national healthcare systems (eg HTA and P&R bodies) on issues such as pan-European evidence generation, meaningful joint clinical assessments, and novel pricing and payment models. They require solidarity between Member States (MS), a recognition that wealthier EU MS should not benefit from the lower prices that ought to be available, in the interest of patient access, to poorer MS.

We welcome the Commission’s intention to keep pace with scientific and technological developments and to ensure that procedures remain efficient. Regulatory authorities should be entrusted to ensure that the system works and keeps up with global regulatory and scientific developments. Key scientific and procedural developments can be consolidated in guidance, eg on issues such as biomarker-defined conditions, or class waivers for paediatric development.

We call on the Commission to consider all options through a thorough IA, including those identified as ‘baseline’ and to acknowledge that their SWD is based on data up to 2017, which neither reflects the actions agreed in the 2018 EC-EMA paediatric action plan, nor the Commission notice 2016/C 424/03 on the Orphan Regulation or scientific and regulatory progress. EFPIA stands ready to work with the Commission to ensure future development of medicines.

The full response can be accessed here.