This year we commemorate Rare Disease Day on the 29th February, the rarest of days. This globally coordinated movement aims to raise awareness about rare diseases and the impact they have on both the people living with them and their families. Rare Disease Day looks at the 36 million people in the EU who live with a rare disease1 to make sure, they are not overlooked.
At this time, it is crucial to shine a spotlight on the pressing issues surrounding rare diseases in the European Union to understand the full picture of the challenges and barriers faced by this community. The current review of the EU Pharma Package, including that of the Orphan Drug Regulation, stands as a pivotal moment for policymakers, healthcare advocates and industry leaders to join forces in addressing the unique challenges faced by those affected by a rare disease.
As previously outlined in The Parliament, Takeda’s view towards the review of the Orphan Drug Regulation remains the same. We must continue to focus on the following:
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Elevating rare diseases as public health priority.
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Building partnerships across the medicine lifecycle and patient journey to further strengthen the rare disease eco-system.
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De-linking access requirements from the modulation of incentives for innovation together with adopting a broad definition of orphan designation and unmet medical need.
The EU Pharma Package review is the right moment for policymakers to strengthen the rare disease eco-system through recalibrating the regulatory landscape, to ensure that it fosters innovation and accelerates the development of innovative therapies for rare diseases. By providing a supportive environment for research and development, we can unlock new possibilities and deliver hope to those grappling with these often-devastating conditions.
“By providing a supportive environment for research and development, we can unlock new possibilities and deliver hope to those grappling with these often-devastating conditions.”
When it comes to incentives related to innovation, these must be robust and targeted, reflecting the distinct challenges associated with rare diseases. The development of orphan drugs, designed to treat diseases affecting a small patient population, requires significant investments and a tailored regulatory framework. Only through a predictable incentives framework can we encourage pharmaceutical companies to invest in research and development for rare diseases, ultimately bringing transformative therapies to those in need.
The Orphan Drug Regulation has proven to work, and I still consider it a great European success story, let us strengthen the framework that was set more than 20 years ago.
A pivotal moment to place rare diseases at the forefront of the political agenda
I would like to take this opportunity to shift my focus away from the ongoing discussions around the Pharma Package and look at what lies beyond. The upcoming elections for the European Parliament and an incoming European Commission present an invaluable opportunity to place rare diseases at the forefront of the political agenda. I call upon the new European Commission and all Members of the European Parliament to recognise the urgency of addressing rare diseases as a public health priority and to commit to championing policies that support research, access to treatment, and an improved healthcare infrastructure for people living with a rare disease.
Beyond rhetoric, what is needed is a concrete commitment from the European Commission to formulate and implement a comprehensive European Action Plan on Rare Diseases. Such a plan should encompass a multifaceted approach, incorporating research and development incentives, streamlined regulatory pathways, and enhanced collaboration between industry, academia, and healthcare providers. It is only through a coordinated effort that we can hope to make meaningful progress in the rare disease space.
For us at Takeda, partnerships are pivotal in driving innovation and ensuring that innovative therapies reach patients quickly. Collaboration between pharmaceutical companies, academic institutions, healthcare providers, and patient advocacy groups is essential in navigating the complex landscape of rare diseases. By fostering an ecosystem of cooperation and knowledge-sharing, we can accelerate the development and delivery of life-changing treatments to those who need them most.
“When it comes to incentives related to innovation, these must be robust and targeted, reflecting the distinct challenges associated with rare diseases.”
As we envision a future where no patient is left behind, it is important to acknowledge the interconnections of stakeholders and the importance of a united front in the fight against rare diseases. Takeda is committed to playing its part by actively seeking collaborations and partnerships that bring together diverse expertise and resources. Together, we can amplify our impact and drive positive change for the rare disease community in the European Union.
In conclusion, Rare Disease Day serves as a moving reminder of the challenges faced by individuals living with rare diseases. As we commemorate this day, let us not only raise awareness but also take concrete actions to address the unique needs of those living with a rare disease.
Looking ahead to the elections for the European Parliament and the incoming European Commission, a European Action Plan on Rare Diseases is not just a wish but a necessity, and it is only through partnerships and collaborative efforts that we can truly make a difference.
Read more about Rare Disease Day on our Rare Disease Hub or in our Rare Disease supplement.