EU Orphan Regulation evaluation; meeting unmet need and facilitating access

EFPIA notes the publication of the Study to support the evaluation of the EU Orphan Regulation (Technopolis, July 2020) and the accompanying Staff Working Document (SWD). The study “finds that the EU Orphan Regulation has contributed to important strides in the field of rare diseases and development of orphan medicines. Since the Regulation was introduced more products have come on the market. There is also a promising pipeline of products under development, that may bring real value to patients for whom currently no treatment options exist.”[1]
EFPIA strongly believes that the evidence in both the study and SWD supports keeping the existing legislation on orphan medicines in place, while underlining the need to co-create vehicles to drive research to areas of unmet medical need and address issues around access to new treatments.
According to the SWD, the Orphan Regulation has contributed to faster availability in the EU[2] (average 9 months earlier) and “has also stimulated wider availability of Orphan medicines (...) to 6.3 million patients in the EU”[3]. However, while access issues persist, the Technopolis report notes that “the observed problem [availability of, and access to orphan medicines] can only be addressed by a EU Regulation to a very limited extent, as a substantial part of the observed unevenness stems from national policies and decision-making processes”. The SWD then specifies that the “availability, accessibility and affordability of (orphan and paediatric) medicines for patients across the EU are influenced to a great extent by factors that go beyond the Regulations and/or EU competence”[4]. These include external reference pricing, the size of the population as well as the organization of the health system and national administrative procedures, national pricing and reimbursement decisions and prescription decisions by medical professionals.
As also seen in the SWD, the Paediatric Regulation has played a key role in transforming that landscape and stimulating the development of medicines for children. The number of paediatric development programmes is increasing, there is a growing paediatric research infrastructure and overall more clinical trials to support regulatory submissions. EFPIA is currently working with other stakeholders on a number of pragmatic, non-legislative measures that can be taken to improve the efficiency and speed of paediatric development, including establishing an inventory of disease-based unmet paediatric needs, improving the efficiency of paediatric investigation plans and global collaboration.
EFPIA Director General, Nathalie Moll said. “The Technopolis report and the SWD emphasize the significant impact of the OMP Regulation, while recognising that more needs to be done on access to new treatments and incentivising research in areas of unmet medical need.”
The root causes of delays and unavailability of medicines are multi-factorial; including late start of market access assessment, duplicative evidence requirements, and national pricing and reimbursement policies[5].
Nathalie Moll continued. “Rather than re-open legislation proven to generate new treatments for children and for people living with rare disease, we reiterate the call on the Commission to set up the appropriate multi-stakeholder High Level Forum composed of EU and national decision makers, the research and healthcare communities, to concretely and constructively address these issues and ensure that no one living with a rare disease is left behind and that more and more treatments are developed for our children. This would complement well the non-legislative initiatives already ongoing by different stakeholders (EMA, patient groups and industry) following the 10-year evaluation of the Paediatric[6] Regulation. We put forward our suggested actions and call on the full implementation of the EMA’s Action Plan on Paediatrics[7] and hope that a High-level Forum is established to also address challenges around orphan medicines.”
Particularly on medicines for rare diseases, Ms. Moll went on to say. “As the industry responsible for developing therapies for patients with rare diseases, we share the acute desire to drive research to areas of unmet medical need. As the report states, “Overall, the EU Orphan Regulation has contributed to addressing some of the unmet needs for patients with rare conditions, but the unmet need remains considerable, in terms of availability of treatments as well as of access.[8]” Addressing the needs of patients living with the 95% of rare diseases where no treatment option exists, means supporting and strengthening the framework of incentives to drive further research into the next generation of treatments and cures for rare diseases and paediatric treatments. The report proves that incentives drive investment, research and results. That means new treatments for patients.”
As the report indicates. “it is important to bear in mind that any modifications to the regulatory framework, particularly to the instrument of market exclusivity, solely for the purpose of better bringing in line costs and rewards could have the undesirable ‘side-effect’ of also slowing down much needed innovation. It is nonetheless worth considering whether and where other forms of incentives could be as effective, or more so in stimulating research and product development for rare diseases.”[9]
In light of the evidence and conclusions of the study and the SWD, EFPIA considers that when looking at future policy options to update the legal framework, the Commission should focus on non-legislative options considering that a re-opening of the Orphan Medicinal Product Regulation is not likely to address the core challenges regarding unequal access and availability of orphan medicines within the European Union. A targeted response to the access and availability challenges does not lie in changing the legislation and requires a structured dialogue with relevant stakeholders, Member States and the European Commission in light of their respective competence areas to find solutions to introduce these ground-breaking treatments. The same is valid for the Paediatrics Regulation, which 10-year evaluation in 2017 concluded that the challenges could be addressed in parallel via non-legislative measures.
Nathalie Moll concluded. “Considering the lack of innovation policy drivers for our industry in the new EU Industrial Strategy and the concerning content of the Roadmap to the EU pharmaceutical strategy, it is all the more critical that we maintain a stable and predictable incentives framework that can continue to support the development of new treatments for rare disease patients in Europe[10]. We have to work together to ensure access to new treatments and technologies today, medical innovation for tomorrow and sustainable healthcare systems in a globally competitive Europe. Destabilising investment in the discovery and development of new treatments for patients living with rare diseases by re-opening a legislation, proven to be effective in stimulating the development of new treatments, cannot be the right approach.”

[1] p.18 Study to support the evaluation of the EU Orphan Regulation, Technopolis, (July 2020)
[2] p.44, Staff Working Document Evaluation, European Commission, (August 2020)
[3] p.46, Staff Working Document Evaluation, European Commission, (August 2020)
[4] p.17, Staff Working Document Evaluation, European Commission, (August 2020)
[8] p10, Study to support the evaluation of the EU Orphan Regulation, Technopolis, (July 2020)
[9] p309, Study to support the evaluation of the EU Orphan Regulation, Technopolis, (July 2020)