Rare and ultra-rare diseases impact up to 36 million people in the European Union, creating significant challenges in healthcare systems across the region. Research into treatments for these conditions also presents unique challenges. The current European legislative landscape accelerated research and innovation, but there is still no approved treatment for 95 per cent of rare diseases. The upcoming revision of the General Pharma and Orphan Medicinal Products legislation provides policymakers in Europe with the chance to strengthen the regulatory framework and create an innovation-orientated ecosystem capable of delivering treatments for the millions of people affected by rare conditions. This was the focus of a recent debate held by The Parliament in partnership with Alexion, AstraZeneca Rare Diseases in the European Parliament.
The overall objectives of the European Commission were welcomed by the different participants, but the unintended consequences of the revision raised concerns. Central to the debate was the proposed Commission revision to reduce the timeline for market exclusivity for orphan medicines, such as those developed to address rare diseases. MEP Billy Kelleher (Renew Europe/Fianna Fáil), host of the event, applauded the EU’s capabilities for its prompt action during the pandemic, noting that the same pragmatism is what the EU needs when dealing with rare diseases.
Discussing access to medicines, Kelleher compared the cases of Germany and Ireland to illustrate a clear example of disparity inside the EU due to differences in reimbursements — a reality that inevitably affects patients with rare diseases. “If you’re in Germany, you’re very lucky. You’ll access medicines in a very timely manner. If you’re in Ireland, you’re not so lucky,” he said, noting that the EU average is 518 days.
The Irish MEP called for a collaborative approach to address disparities between countries and the deficiencies of services in the smaller nations. Issues with pricing and reimbursement remain a challenge across the EU with different economies and healthcare budgets at a Member State level, he said, leading to fewer medicines available in poorer countries.
Addressing unmet medical needs
Explaining the Commission’s mindset towards incentives for the development of orphan drugs, Kaja Kantorska, policy officer for pharmaceuticals at the Commission, said the EU executive is trying to create a system where innovative products addressing unmet needs reach patients: “We tried to make some changes to the orphan system to adapt it with innovative products and to direct investments to areas where there are no treatment options to reward as well [as to] those developers which are game-changers.”
MEP Pernille Weiss (DK/EPP), the European Parliament’s rapporteur for the revision of the EU pharmaceutical legislation, opposed the Commission’s view on the proposed timelines and said the period of market exclusivity should be longer. “One of the very key factors [of the framework] is the system of incentives and the regulatory data protection – this is the beef on my side,” argued Weiss. The Danish MEP moved the baseline from six years to nine years and added an extra six months so that innovators can have an extra year of intellectual protection if the medical product meets unmet needs. Weiss made clear that a strong pharmaceutical industry in Europe should be a priority for the competitiveness of the Union and to secure access to innovative medicines for patients.
Incentives key to drug development
Dr Annette Bakker, president of the Children’s Tumour Foundation, welcomed the Commission’s efforts to strive for a balanced approach and applauded MEP Weiss’s amendments on the current proposal. Overall, patient organisations have reacted similarly to the Commission’s proposal, saying it will likely undermine access to medicines since the industry would have less incentives to invest in research, thus resulting in fewer orphan drugs. Dr Orla Galvin, Executive Director of the European Federation of Neurological Associations, asked for more support to back neurological research: “At the moment we are reliant on the pharmaceutical industry to invest and support brain neurological patients and their families.” Speaking on behalf of the European Confederation of Pharmaceutical Entrepreneurs, Secretary General Alexander Natz concurred: “If we’re putting too many ‘ifs’ around incentives and IP rights for pharmaceutical companies, that is a bit of an issue.”
The Commission’s Kantorska said the proposal tries to find a balance but emphasised that it also attempts to help developers ensure that medicines reach patients in need: “We have seen that 95 per cent of rare diseases have no treatment and we didn’t have in the regulation any tool to help boost the innovative R&D for the medicines. By adding this additional year of market exclusivity – this would help in early stages to develop, discuss protocols and quick assessment.”
Dr Anita Hill, a veteran healthcare professional working with rare diseases for over two decades in the UK’s NHS, joined Alexion determined to help more patients survive from rare diseases and provide them with an increased quality of life. Hill agreed that the orphan market exclusivity must be extended, as well as calling for increased investment into innovative drug development. “The amount of investment that goes to research astounded me when I joined the industry,” she said. “I think that’s because of what we don’t see on the outside as clinicians: the number of failures that it takes to get to effective therapies in the end.” Most importantly, through her personal experience as a medical doctor on the front line, she pointed out the importance of defining unmet medical needs, taking into consideration the actual needs of patients who want to live and not merely survive. The definition of unmet medical need is likely to become a core topic of discussion for the revision, for both industry and patient organisations.
Taking steps towards an effective and collaborative policy
When looking at the definition of unmet needs, Kantorska explained that the EU executive created a notion of exceptional therapeutical advancement, which is not easy to explain in a regulatory document. “The next steps are for the European Medicines Agency to meet with all the stakeholders and discuss this concept and make these criteria understandable, agreeable by everyone and predictable for the developers,” she said. MEP Weiss commended the Commission’s good intentions but remarked that the investors need to be part of this discussion. “By having a more technocratic speculative approach of what is highly unmet, how will that be understood and used by investors?”
Weiss concluded by highlighting the proposal on joint procurement as part of the Commission’s revision and noted that it would be problematic if it’s regarded as a silver bullet for everything. Looking forward, Weiss said the input received in the event provided several insights on how to focus on the development of a joint procurement to move decisions forward at a member state level.
In partnership with
This article was produced in partnership with Alexion.