Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team and over 1600 stakeholders from the rare disease community to look at what’s next on the path to protect rare disease patients.
Soraya Bekkali, Senior Vice President for International Commercial Operations at Alexion kicked off the conference. In her opening remarks she issued a reminder that despite significant progress in Europe, there is a long way to go: “Talk will only get us so far. We find ourselves at a tipping point where our efforts over the last decade will either deliver continued progress building on our achievements or be undermined by well-intended policies which could impact innovation” Bekkali told the summit. Setting the scene for the event to come, she issued a powerful reminder that “policy choices made today will have significant long-term impact”.
The European Commission’s Arjon Van Hengel, Deputy Head of Unit for Health Innovations & Ecosystems, provided attendees with greater details about the rare disease and orphan drugs elements of the Commission’s new proposed revision of its Pharmaceutical Legislation. The revision of the legislation focuses on six key objectives: access, availability, affordability, competitive regulatory framework, environmental sustainability, and combatting antimicrobial resistance. Underpinning these six objectives is the establishment of a single market of medicines in the EU.
Across several keynote discussions and panel, Van Hengel reiterated the Commission’s commitment to patients across Europe. “Not everyone is happy with everything across the proposed legislation. There are elements that one stakeholder likes and another stakeholder does not like, but in the end what we are going for is improving the situation for the patients, so that is the background of the revision of this legislation” he explained.
Challenges posed for the industry by the legislation along with fears of unintended consequences were highlighted across the summit. One consistent concern throughout the course of the discussions was around the entrenched nature of the European Commission’s definition of an unmet medical need. According to Yann Le Cam, CEO of EURORDIS, the definition “doesn’t reflect patients and science”. “Until you’re cured, the work is not done” argued Le Cam. With this legislation set to be binding for another extended period, calls for flexibility in definitions were made at the event. Toon Digneffe, Head of Public Affairs & Public Policy for Europe & Canada at Takeda reiterated the positive moves made in the European Parliament where MEPs have led calls for the definition of an unmet medical need to be wider and patient-centric to provide the needed degree of flexibility.
Concerns were also raised about how the legislation could impact SMEs and smaller innovative companies who so often begin the research into treatments for rare diseases. Alexion’s Bekkali reaffirmed the importance of these smaller companies: “We must be mindful not to disincentivise investment into early research. That’s where innovation comes from, especially in the biotech startup ecosystem.” If this is not reflected in legislation, “we will lose a lot of small companies bringing innovation to patients with legislation” according to Sophie Schmitz, Managing Partner at Partners4Access.
A recognition of success and optimism for the future were plenty as attendees looked back at the progress made over the last 20 years. “I have no doubt that there is a success story behind the regulation so far, but there is still lots to be done. The original intention to provide access for patients must be maintained” explained Cecile De Coster, Executive Director for Global Regulatory Affairs & Development Strategy at Alexion. Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products at the Medicines Evaluation Board touched on her experience as a regulator, highlighting that “without the EU’s regulation, we wouldn’t have achieved what we see today”. Gemma Mayman, Global Rare Disease Commercial Lead at Pfizer, added how since the EU passed its previous legislation on rare diseases we have seen the approval of over 200 orphan drugs in the EU.
The fact that 2023’s summit focused around patients rather than pricing was a “step in the right direction” according to one attendee. The role of patients in the rare disease community has been a success story, and one which participants were keen to see continue. Formalised networks such as EURORDIS were the focus of several discussions, and their ability to mobilise patients and allow them to speak directly to policymakers about their experiences have been critical to the recognition of rare diseases according to Alexion’s Josep-Maria Espinalt, Vice President and Head of Alexion Shared Markets. EURORDIS’ Le Cam expressed his desire to see further collaboration between the European institutions and patients. “We need to continue to listen to patients to address their needs. We must consolidate patient focus on all levels, not just on management boards”.
Better awareness of rare diseases in the wider world was something celebrated amongst attendees and speakers. “In 2023, I think there are no people who have never heard about rare diseases and this is very important” stated Stoyanova-Beninska. This awareness has led to greater focus for early diagnosis of rare diseases. Further steps need to be taken to harness the potential to improve patients’ lives, as was illustrated by recent advances in newborn screening. In the UK, Lucy Mckay, CEO of Medics4RareDiseases, explained how screening is widely available with 98% of babies screened in recent years. This early diagnosis was crucial for patients, as Kelly du Plessis, CEO of Rare Disease South Africa, provided the shocking statistic that “every 2 weeks a child loses an IQ point and this is not reversible”.
The coming together of stakeholders for the improvement of lives is important in any sector. For Alexion’s Bekkali, the World Orphan Drug Congress “has established itself as a platform where we come together as a community to share best practice and also come together with a common purpose to take on the next steps of our journey”.
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