Elevating innovation through partnerships
Today, European institutions are leading the biggest legislative revision of the EU’s pharmaceutical framework we have ever seen[1]. A revision that is needed after more than 20 years. Reflecting on this legislative review, I believe all stakeholders involved and affected by this revision should ask the following question; how can we work together with all stakeholders across the entire ecosystem to provide solutions that make a difference for all?
This review is an important milestone in how we foster innovation and how we focus on patient care within the European Union. This ‘once-in-a-generation’ reform of the EU Pharma Package represents a real chance to boost innovation in Europe, and this will not be without challenges. The months ahead will be critical and will set the groundwork for how the EU will be looking at bringing innovation to patients who need it in the future. I am convinced that we need to strengthen our innovative ecosystem to do so.
At Takeda, we support the Commission’s objectives for the revision of the EU pharmaceutical legislation: the ambition for timely access and availability of approved medicines across member countries, more efficient regulatory processes, and an attractive, innovation-friendly environment. We welcome proposals to streamline regulatory processes, enhance digitalization and an increased use of real-world evidence. We also welcome the recognition in the proposal of the achievements of the Orphan and Paediatric Regulations as European success stories by maintaining the threshold for orphan designation at a prevalence of 5/10,000[2].
This review is an important milestone in how we foster innovation and how we focus on patient care within the European Union.
Takeda believes that the existing framework for incentives and rewards linked to regulatory data protection and orphan market exclusivity is effective and predictable. This framework has been instrumental in driving innovation in rare diseases and addressing unmet needs in patient care. Takeda supports maintaining and strengthening this framework, further incentivizing the development of medicines that target all unmet needs and emphasizes the necessity of legislative predictability.
One of the key debates on the EU Pharma Package revolves around how to define “unmet medical need”. We would argue that a precise, legally binding definition is not an effective solution for directing investments into specific areas of health care. A narrow definition of unmet medical need assumes that patient needs are met as soon as a treatment is authorized. However, this does not accurately reflect the dynamic nature of patients’ needs. Medical needs can change over time, and therefore, any definition should be continuously revised to remain relevant, patient-focused and, most importantly, be supported by all relevant stakeholders, especially the patient community.
Looking ahead, the upcoming review of the EU Pharma Package must be part of a broader effort to support patient care.
Another point of contention is the linkage between access requirements and incentives for innovation. This approach will not improve patient access to medicines effectively. Requiring companies to launch approved medicines in all markets within two or three years of EU approval does not consider the complex barriers to patient access. Takeda suggests exploring alternative proposals developed as multistakeholder solutions, such as innovative value-based contracts and equity-based tiered pricing[3]. These approaches could offer more flexibility in ensuring patient access while maintaining incentives for innovation.
We want to emphasize the industry’s commitment to file for pricing and reimbursement across the Union within two years of marketing approval, demonstrating its dedication to facilitating access for patients.
Collaboration as a solution
So how can we overcome these issues? For Takeda, the answer lies in partnerships. Partnerships are the cornerstone in building an innovation-friendly ecosystem. Collaboration among various stakeholders, including patient groups, industry and academics, is crucial throughout the whole drug development lifecycle. Initiatives such as the Rare Diseases Moonshot[4], European Expert Group on Orphan Drug Incentives[5], Together4RareDiseases[6], TRANSFORM[7], and RWE4Decisions[8] exemplify the power of collective action to build a more robust innovation ecosystem in Europe.
Collaboration among various stakeholders, including patient groups, industry and academics, is crucial throughout the whole drug development lifecycle.
Looking ahead, the upcoming review of the EU Pharma Package must be part of a broader effort to support patient care. Rather than attempting to solve all issues through a single legislative initiative, it should be viewed as one step in a more holistic approach. Building upon the successes of the current framework and fostering collaboration through multistakeholder partnerships will be essential to shaping the future of patient care in Europe. By fostering partnerships and taking a holistic approach, Europe can continue to take a global leadership role in innovation and provide options for those in need.
References:
Horgan, D., Koeva-Balabanova, J., Capoluongo, E., Jagielska, B., Cattaneo, I., Kozaric, M., & Malats, N. (2022). Making Sure That Orphan Incentives Tip the Right Way in Europe. Healthcare (Vol. 10, No. 9, p. 1600). MDPI.
Aartsma-Rus, A., Dooms, M., & Le Cam, Y. (2021). Orphan medicine incentives: how to address the unmet needs of rare disease patients by optimizing the European orphan medicinal product landscape guiding principles and policy proposals by the European expert group for orphan drug incentives (OD Expert Group). Frontiers in Pharmacology, 12, 3666.
Takeda. (2023): Annual integrated Report 2023.
Moonshot RD (2022). Rare disease moonshot initiative. https://www.rarediseasemoonshot.eu. Accessed 4 October 2023.
Together4RD (2023). What is Together4RD. . Accessed 4 October 2023.
European Confederation of Pharmaceutical Entrepreneurs EUCOPE (2021): TRANSFORM: Multi-stakeholder approach to gene and cell therapies . Accessed 4 October 2023.
RWE4Decisions (2023). Real World Evidence for Decisions. Co-Creating Real World Evidence for Decision-Making. Accessed 4 October 2023.
About Takeda
Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. www.takeda.com
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Created October 2023
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[1] Horgan, D.; Koeva-Balabanova, J.; Capoluongo, E.; Jagielska, B.; Cattaneo, I.; Kozaric, M.; Tumiene, B.; El Ahl, J.-P.; Lal, J.A.; Kalra, D.; et al. Making Sure That Orphan Incentives Tip the Right Way in Europe. Healthcare 2022, 10, 1600. healthcare10091600
[2] Aartsma-Rus, A., Dooms, M., & Le Cam, Y. (2021). Orphan medicine incentives: how to address the unmet needs of rare disease patients by optimizing the European orphan medicinal product landscape guiding principles and policy proposals by the European expert group for orphan drug incentives (OD Expert Group). Frontiers in Pharmacology, 12, 3666.
[3] Takeda. (2023): Annual integrated Report 2023.
