Aldevron Breakthrough Blog

Advancing through Disruption

Achieving advancement in ATMP regulation through disruptive innovation of European healthcare systems

Partnerships and transformation are a large part of our evolving biotech landscape. Not surprisingly, they were two common themes at this year’s Meeting on the Med in Barcelona.  As an example, attendees discussed how the lack of harmonization across EU member states has led to fragmented access to advanced therapy medicinal products (ATMPs), which varies from country to country. Chris Fox, president of Novartis Gene Therapies, used her company’s product, ZOLGENSMA®, as an example. It’s approved for use in 48 countries but is only accessible in 35. 

Shared responsibility, shared risk
With the increasing number of ATMPs being approved in Europe (6 in 2022 compared with 2-3 in 2020-2021), the consensus is that modernization of healthcare systems is essential to meet the needs of these new modalities. 

A solution-based payment approach is being touted as the way forward to shape the reimbursement model for new modalities. As the industry transitions away from traditional chronic treatment regimens toward effective one-time treatments, a bespoke, risk-shared, outcome-based approach is key to support access to these life-changing therapies. 

Partnerships and patients
In addition to modernization of existing reimbursement protocols, partnerships must be forged to improve access to ATMPs. These include those between drug manufacturers, regulators and reimbursors as well as academic research groups. And, if you apply a Quadruple Helix Model to this scenario, let’s not forget society at large, which puts patient communities at the heart of all decisions and actions. Speakers at the event agreed that listening to – and learning from – patients is imperative for the progression and strengthening of the European healthcare ecosystem.

Disruption to lead transformation
To meet the needs of new modalities and those of patients, innovative disruption of healthcare systems across Europe will be the next step. Synergizing with the awaited publication of the updated European Pharmaceutical Legislation, the European Medicines Agency (EMA) Network Strategy to 2025 will see the EMA and the Heads of Medicines Agencies (HMA) collaborate to streamline the decision-making progress thus driving quicker access to important medicines. The legislative updates are expected to provide the most significant overhaul of pharma legislation in the EU in decades. 

Utilizing Global Data
Healthcare industry professionals and regulatory bodies such as the EMA appreciate that using real world evidence (global data) opens new possibilities in the development and use of novel medicines. However, these medicines don’t reach patients across Europe with the same speed or in comparable quantities. 

To achieve unilateral access, more efficient partnerships between regulators, health technology assessment (HTA) bodies, other payers, and industry are needed. For example, the joint clinical assessment between the EMA and HTAs allow medicines to be evaluated in parallel with the European Health Data Space (EHDS), which already leverages big data and developments in analytics to complement clinical trial data. It is also a powerful tool to provide HTAs with post-approval data to assist their reimbursement decisions. An example of this is the rapid collection and analysis of data by EU member states during the COVID-19 pandemic.

Building partnerships is critical
From the discussions at Meeting on the Med, the general consensus is that transformative medicines require substantial change in the European healthcare ecosystem. This includes systemic change of healthcare systems, modernization and futureproofing of ATMP regulation, along with appropriate repayment models. Just as imperative, partnerships between academic, regulatory agencies, healthcare providers and industry need to be developed and strengthened. If effectively implemented, this will provide improved access and availability for patients to receive life-saving medicines.


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Bev Cameron, Ph.D.

Bev Cameron, Ph.D.

Bev Cameron, Ph.D., joined Aldevron as a Business Development Manager in February 2023, based in Europe. Following post-doc work in small molecule drug discovery she joined GE Healthcare as an AKTA chromatography and training specialist, then transitioned to account management. Prior to joining Aldevron, she was responsible for Clinical Partnerships in EMEA at Cytiva.

Topics: Cell Therapy, Gene Therapy, Immunotherapy, conferences, Thought Leadership