CSL Behring
The CSL Behring story spans decades of innovation, collaboration, patient focus, and an ongoing drive for excellence.
ID: 322536115782-29
Lobbying Activity
Response to Pharmaceutical Strategy - Timely patient access to affordable medicines
7 Jul 2020
CSL Behring appreciates the opportunity to comment on the EU Commission’s roadmap for its Pharmaceutical Strategy (the Strategy). We commend the Commission’s commitment to ensuring that patients in Europe have access to medicines they need and the efforts to create the environment for Europe to be a world leader in medical innovation.
CSL Behring is a leading researcher and manufacturer of life-saving biotherapies used in the treatment of rare and life-threatening diseases. CSL Behring believes that it is critical for the Strategy to account for the particularities of different types of biopharmaceutical products, such as plasma-derived medicinal products (PDMPs), gene therapies and other advanced therapy medicinal products (ATMPs). Overall, the strategy should have a clear focus on how regulatory and access frameworks can be improved to enable broad and rapid patient access to these therapies in Europe.
As PDMPs are used for the treatment of rare and life-threatening diseases and also represent potential critical therapies in pandemic situations, the availability of plasma in Europe should be one of the main strategies for avoiding supply disruptions. In recent years, the global demand for PDMPs, and consequently the need for plasma donations has grown and is expected to grow further. The uniqueness of plasma as a source material, complexity of the manufacturing process and long lead times require a special focus to ensure adequate supply is maintained.
The Commission has a key role to play in realizing the full potential that gene therapies and other ATMPs can deliver to patients and national payers in Europe. The Strategy should consider the uniqueness of rare diseases and foster the adoption of novel financing mechanisms that enable manufacturers and Member States to enter into outcome-based agreements over time. Regulators and Health Technology Assessments (HTA) bodies should align on patient experience data generation throughout the product lifecycle. With the implementation of the Clinical Trial Regulation, it would be beneficial to re-assess the applicability of GMO requirements to ensure optimisation of processes for ATMPs containing GMOs currently under development. Broader evidence generation strategies such as the use of real-world evidence likewise is critical to support the introduction of ATMPs. These initiatives call on the Strategy to set a vision for stronger cooperation among stakeholders, including industry, patient representatives, regulatory authorities, HTA bodies, and payers.
CSL Behring supports inter-disciplinary cooperation to facilitate the development of new products, particularly in areas of unmet medical need and rare diseases. A transparent, dynamic and competitive regulatory framework which allows flexible and accelerated regulatory assessments to further strengthen innovation in Europe is needed. The regulatory pathways introduced during the COVID-19 crisis could be leveraged and applied to the areas of unmet medical needs. Moreover, the review of orphan and paediatric regulations calls for an increased linking of paediatric requirements and orphan designations to accelerate orphan paediatric medicines development.
The recent adoption of the ICH Q12 Guideline is also a unique opportunity to increase flexibility and streamline regulatory lifecycle management. CSL Behring is urging a revision of the EU variation framework, with the aim of fully implementing innovative tools laid down in ICH Q12, specifically the Established Conditions and Product Lifecycle Management concepts.
For the goal of ensuring patient access to safe and affordable medicines, it is important to consider all potential root causes for supply disruptions, including healthcare system priorities, complex shortage reporting systems and reimbursement policies in Europe. A coordinated action in Europe with a platform allowing quicker flow of information between regulatory authorities, HTA bodies and the industry is needed.
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