Event Recap – Alexion’s Rare Disease Policies (13 October 2022)
On 13 October 2022, Alexion held a high-level conference, as part of the “Rare Conversations” event series, on rare disease policies at the Bibliotheque Solvay in Brussels.
Alexion’s recent conference in Brussels, Rare Disease Policies 2022, was an insightful look into the possibilities for the future of the rare disease ecosystem. Highlighted throughout was the unyielding determination of rare disease stakeholders, patients, regulators, industry, researchers and others, to innovate in the rare disease ecosystem, as well as the need to work together to achieve this objective. This joint effort must occur to ensure a competitive and innovative atmosphere and strengthen Europe’s strategic autonomy on a global scale.
Yann Le Cam of EURORDIS stated aptly that ‘’We want Europe to be attractive. Not that (medical) science comes from here, goes to the US to be developed, then comes back to us as a product.’’
As part of the critical need to regulate effectively, the upcoming review of the Orphan Medicinal Products (OMP) Regulation and General Pharmaceutical Legislation (GPL) presents a timely opportunity to build a blueprint for future public/private partnerships. Collaborative work across sectors and stakeholders is a topic that holds immense promise and must be encouraged in the future. These public/private partnerships have the potential to drive investment for innovative pharmaceuticals, reinforcing Europe’s position as a leader in pharmaceutical innovation and ensuring that rare disease patients have access to the products that best reflect their specific needs.
As EUCOPE’s Vittoria Carraro put it during the conference: ‘’We cannot afford to go below our initial ambitions’’ when it comes to regulation.
The Orphan Drug Incentives (OD) Expert Group is acutely involved with the upcoming legislative revisions and their far-reaching implications. The upcoming revision of the OMP regulation is hugely important, as is the methodology used for Orphan Drugs under the new EU Health Technology Assessment (HTA) regulations. With EURORDIS and EUCOPE at the helm of the initiative, the group is currently focusing on developing concrete policy proposals for the revision of the OMP regulation, developing an operational framework for modulating incentives & policies, and developing proposals to contribute to a tailored methodology for OMPs as part of the EU HTA regulation.
Mentioned as a crucial component of any upcoming regulation, policy flexibility must be a part of any revision. This flexibility will allow for a more effective regulatory process. The integration of real-world evidence and the holistic assessment of a therapy is a giant leap forward that will ensure equitable market access across the EU of rare disease treatments for patients in critical need. Voiced as a topic of concern during the event was the lack of a truly synergistic pharmaceutical-regulatory system, a key area where targeted regulation could bring sustained success.
Assisting in this is a key area of interest for EUCOPE. Building a supportive rare disease regulatory ecosystem through the revision of GPL and OMP policies will allow for innovative products such as Substances of Human Origin (SoHOs), Advanced Therapy Medicinal Products (ATMPs), Advanced Diagnostics, and more, to reach the market efficiently.
EUCOPE will continue to play an integral role in further building the Rare Disease regulatory ecosystem. To ensure that Europe’s position as a leader in pharmaceutical innovation in the future, small and mid-sized companies will play a key role. The collaborative multi-stakeholder approach that EUCOPE takes was communicated during discussions as important for future progress at Alexion’s conference, reinforcing the need for EUCOPE to continue advocating for small and mid-size companies.
EUCOPE’s uncompromising amplification of the regulatory needs that small and mid-size pharmaceutical innovators have is, and will continue to be, an important piece of Europe’s rare disease ecosystem.