Rare Diseases: Securing a better future for rare disease patients across Europe is a shared responsibility

Meeting unmet needs together is essential, explains Takeda Pharmaceuticals Chief Giles Platford.
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By Giles Platford

Giles Platford is President of the Europe & Canada region at Takeda

23 Feb 2021

The frameworks for addressing the needs of rare disease patients in Europe have undergone a remarkable transformation over the last 20 years, bringing significant improvements to the standards of care and innovation.

However, there remain multiple challenges throughout the patient journey, ranging from delays in diagnosis to inequitable access to treatment. In order to address the many lingering unmet needs, the entire rare disease ecosystem needs to be further strengthened through value-based, patient-centric partnerships at all stages of the patient journey.

As a values-based, R&D-driven biopharmaceutical company, Takeda is committed to building patient-centric value-based partnerships at all stages of the patient journey, with a shared purpose to deliver tangible and positive impact for the rare disease community. In recognition of our commitment to improving standards of diagnosis, care and treatment for rare disease patients, Takeda was honoured to be recognised this year with the prestigious EURORDIS 2021 Black Pearl Award for Patient Engagement.

“As a values-based, R&D-driven biopharmaceutical company, Takeda is committed to building patient-centric value-based partnerships at all stages of the patient journey, with a shared purpose to deliver tangible and positive impact for the rare disease community”

Through our collaborations and efforts to engage with patient groups, we strive to make improvements across the entire rare disease patient journey. One such collaboration early in the patient journey, ‘The Global Commission to end the diagnostic odyssey for children with a rare disease’, aims to bring forward actionable recommendations and pilot projects to shorten time to diagnosis.

Looking towards the end of the patient journey, RWE4Decisions is a multi-stakeholder initiative exploring how real-world evidence can support payer decisions and enable earlier access to highly innovative technologies and treatments. In rare diseases, real-world evidence is particularly important since it presents an additional route by which to demonstrate the value of treatments for small, heterogenous patient populations complementing the evidence generated through clinical trials.

Takeda also works closely with our trade associations, such as the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) to put forward constructive proposals for sustainable innovation and better access to rare disease treatments.

In light of the European Commission’s evaluation of the EU OMP Regulation, Takeda calls for a constructive and holistic approach that supports the sustainable development of innovative therapies addressing unmet medical needs and innovative access pathways so that rare disease patients benefit in a timely and equitable manner.

Given that 95 percent of rare diseases still have no EMA approved treatment, Takeda has actively supported the set-up of a multidisciplinary and cross-functional European Expert Group on Orphan Drug Incentives to bring forward common proposals to futureproof research & development of innovative rare disease and orphan medicines.

In the area of patient access, we work together with stakeholders to identify the root causes of differential access across Europe and find solutions based on evidence generation, joint clinical assessments and novel pricing models.

Partnerships have been critical to the positive evolution of the rare disease ecosystem across Europe over the past 20 years; and will be essential if we are to continue addressing unmet needs. The few initiatives I highlighted here are merely a snapshot of many ongoing multistakeholder efforts designed to improve standards of care and health outcomes for rare disease patients.

As we look ahead, the Rare 2030 study findings - which will be presented in the European Parliament on Rare Disease Day in February - will provide recommendations on the most critical areas needing government attention; and will hopefully guide rare disease policy in Europe for the next 10 years and beyond.

At Takeda, we look forward to continuing our active contribution to the debate, and to forging further value-based partnerships that bring forward actionable solutions to address the needs of rare disease patients.

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