Cell and gene therapies (CGTs), also classified as Advanced Therapy Medicinal Products (ATMPs), are hailed as revolutionary, but the road to realizing their potential has also brought pitfalls and disappointments along the way since the first attempts a few decades ago.
Today, CGTs offer new horizons to people living with some rare, lifelong diseases. However, despite their promise, these therapies pose a number of scientific, logistical, regulatory, and economic challenges. In order to ensure safe and affordable access to these novel treatments, an inclusive dialogue is clearly needed.
The current landscape: hopes and hurdles
CGTs already exist in several disease areas and have changed patients’ lives. In a debate convened by the European Health Forum Gastein and CSL Behring on June 15, Avril Daly (EURORDIS) emphasized that we often regard CGTs as futuristic products while they are already being administered “in the now”.
A primary goal should therefore be to provide access to patients and their families and “turn hope into reality”. Bertalan Meskó (Medical Futurist Institute) reinforced this notion by urging “putting patients at the center of care and involving them in decision-making at the highest level”.
But where there are expectations and hopes, there are also worries and uncertainties.
Ilona G. Reischl (Austrian Drugs and Medical Devices Authority) said personally that she sees a trivialization of CGTs in Europe: We are discussing complex drugs and CGTs should be treated as such, she stressed, stressing that there is appropriate regulation and clinical trials required to provide product-specific information.
It is also crucial to collect real world data to answer key questions about the persistence of treatment responses to CGTs and to inform new approaches to payment for potentially curative single-use therapies.
In addition, health systems must adapt to this paradigm shift in care, as they have previously done with groundbreaking, disruptive medical innovations.
The pooling of expertise, the establishment of clinical frameworks and the provision of the means and infrastructure to enable the specialist care required for the provision of CGTs is now high on the agenda.
Equally important is ensuring the appropriate infrastructure and process for accessing diagnosis and genetic testing as a first step for patients receiving these transformative therapies.
However, time is of the essence for patients with rare diseases who lack treatment options or who have poor quality of life. The opportunity to offer therapies that will save, change or improve the lives of these patients is not to be missed.
What can be viewed as a small improvement can indeed make a big impact on patients ‘quality of life, and the way we measure improvement needs to change to adequately reflect patients’ experiences and expertise, Avril noted Daly.
If CGTs do change tomorrow’s health paradigm, we are not far from tomorrow, said Dan Hart (Royal London Hospital Haemophilia Center). The scientists have done the work and it is now up to us to overcome some of the ethical implications of ensuring equal access across Europe.
Several EU policy initiatives offer opportunities to define a European political and regulatory environment that meets current challenges and is future-proof in terms of absorbing medical innovations and ensuring patient access, while maintaining European leadership in this area.
These include the upcoming EU Pharmaceutical Strategy, the European Health Data Space (EHDS) and the assessment / revision of existing legislation, including the EU Cross-Border Healthcare Directive and the EU Blood, Tissues and Cells Directive. Andrzej Ryś (Directorate General for Health and Food Safety of the European Commission) stressed the need for continuity and adaptable legislation that reduces the administrative burden.
Alignment between relevant EU legislation as well as between European and national frameworks will be of the utmost importance, said Ilona G. Reischl, including rules on hospital exemption (HE), which allow the use of an ATMP in certain circumstances without a marketing authorization.
“Everyone is ready to implement ATMPs, and the stars are aligned,” said Ryś, while MEP Cristian Silviu Buşoi outlined that the European Medicines Agency should approve 10-20 CGTs annually by 2025.
Paige Bischoff (Alliance for Regenerative Medicine) reflected on the fact that ATMPs are identified as an important innovation milestone in the EU pharmaceutical strategy. The key now is greater harmonization across Europe to support the strategy, particularly on legislation regulating genetically modified organisms (GMOs).
The EHFG debate found a consensus that creativity and flexibility at all levels can help create an environment in which patients can best benefit from CGT. For example, legal frameworks must be adaptable to innovations, and COVID-19 served as an example of how one can adapt quickly to change.
From a pricing and reimbursement perspective, new payment models are needed to reflect the novel characteristics of CGTs. One size doesn’t fit all as every CGT is different. Diane Kleinermans (Belgian National Institute for Health and Disability Insurance) emphasized the importance of thinking outside the box in order to meet the demands of new technologies and therapies.
In addition, international cooperation is the right way to go, and the BeNeLuxA initiative is an example of this, she explained, as a platform for cooperation between five countries in order to monitor the entire life spectrum of drugs, to organize joint health technology assessments and Perform scans.
Support at EU level for the continuous monitoring of quality and harmonized data, including through the EHDS, will be a building block to ensure patient access to safe and effective CGTs across Europe and to answer burning questions about how safe it is safe enough or when? Health systems should pay for treatment because of uncertainties about the durability of patient responses.
Reducing fragmentation, standardizing data across borders, and making greater use of real-world evidence will be keys to supporting longer-term data collection, Bischoff said.
Collaboration between medical experts, policymakers, regulators, hospitals and academic developers, patients, health technology evaluators, payers and industry will be critical in transforming healthcare. The further development of CGTs requires collaboration and patient-centered work with and for patients.
The field of CGTs is deeply complex, as outlined by Vivienne Parry, science writer and broadcaster and presenter of the debate – but the fact is that CGTs are real and are now being administered.
They disrupt traditional pathways of health care, the development, evaluation and payment of treatments and challenge us all to rethink existing policies and practices – or, as Bertalan Meskó pointed out at the beginning of the debate, to change mindsets to include changes such as technological ones Adoption must be accompanied by cultural adoption.
This article reflects a more detailed discussion that took place in the context of the webinar “Transforming the future of healthcare – do cell and gene therapys hold the key?” By the European Health Forum Gastein and CSL Behring on June 15, 2021. Ilona Reischl and Diane Kleinermans spoke personally during this meeting. To view the recording of this webinar, please visit https://www.youtube.com/watch?v=QL6OYmoJIzg
source https://dailyhealthynews.ca/do-cell-and-gene-therapies-hold-the-key-euractiv-com/
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