Cell and gene therapies (CGTs), also classified as Advanced Therapy Medicinal Products (ATMPs), have been hailed as revolutionary, but the path towards realising their potential has also seen pitfalls and disappointments along the way since the first attempts some decades ago.
Today, CGTs offer new horizons for people living with some rare, lifelong diseases. However, despite their promise, these therapies entail a range of scientific, logistic, regulatory and economic challenges. There is a clear need for inclusive dialogue to define a way forward in ensuring safe and affordable access to these novel treatments.
The current landscape: hopes and hurdles
In several disease areas, CGTs already exist and have transformed patients’ lives. Speaking at a debate convened by the European Health Forum Gastein and CSL Behring on 15th of June, Avril Daly (EURORDIS) stressed that we often think about CGTs as futuristic products, while they are already being administered “in the now”.
Thus, a main objective should be providing access for patients and their families and “turning hope into reality”. Bertalan Meskó (Medical Futurist Institute) reinforced this notion by urging to “make patients the point of care and involve them at the highest level of decision-making”.
However, where there are expectations and hope, there are also concerns and uncertainties.
Speaking in a personal capacity, Ilona G. Reischl (Austrian Medicines and Medical Devices Agency), commented that she sees a trivialisation of CGTs in Europe: we are discussing complex medicinal products and CGTs should be treated as such, she emphasised, underlining that there is a need for appropriate regulation and for clinical trials in order to provide product specific information.
It is also crucial to collect real-world data to help answer important questions about the durability of treatment responses to CGTs and to inform novel payment approaches for potentially curative, one-off therapies.
Further, health systems must adapt to accommodate this paradigm shift in care as they have done previously with ground-breaking, disruptive medical innovations.
Pooling expertise, setting up clinical frameworks and providing the means and infrastructures to enable the specialist care needed to deliver CGTs is now top of the agenda.
Equally important is ensuring appropriate infrastructure and processes to access diagnosis and genetic testing, as a first step for patients in receiving these transformative therapies.
However, time is of the essence for patients with rare diseases lacking treatment options or with poor quality of life. Opportunities to provide therapies that save, change or enhance these patients’ lives cannot be missed.
What may be seen as a small improvement can in fact have a huge impact of patients’ quality of lives, and the way we measure improvements need to change to reflect properly patients’ experiences and expertise, noted Avril Daly.
If indeed CGTs will transform tomorrow’s healthcare paradigm, we are not far from tomorrow, explained Dan Hart (Royal London Hospital Haemophilia Centre). The scientists have done the work, and it is now up to us to overcome some of the ethical implications linked with ensuring equal access across Europe.
Several EU policy initiatives offer possibilities to define a European policy and regulatory environment that addresses current challenges and is future-proof with respect to embracing medical innovation and ensuring patient access while maintaining European leadership in this field.
These include the upcoming EU Pharmaceutical Strategy, the European Health Data Space (EHDS), and the evaluation/revision of existing legislation including the EU Cross-border Healthcare Directive and the EU Blood, Tissues and Cells Directive. Andrzej Ryś (European Commission Directorate General for Health and Food Safety), stressed the need for continuity and for legislation that is adaptable and reduces administrative burden.
Alignment between the relevant EU legislation and between European and national frameworks will be paramount, said Ilona G. Reischl, including rules on hospital exemption (HE) which allow for the use of an ATMP without a marketing authorisation under certain circumstances.
“The willingness of all to realise ATMPs is there, and the stars are aligned”, said Ryś, while MEP Cristian Silviu Buşoi outlined that by 2025 the European Medicines Agency shall approve 10-20 CGTs annually.
Paige Bischoff (Alliance for Regenerative Medicine) reflected on the fact that the EU Pharmaceutical Strategy refers to ATMPs as a major milestone of innovation. What is key now is greater harmonisation across Europe to support the Strategy, especially in legislation regulating genetically modified organisms (GMOs).
The EHFG debate revealed a consensus that allowing creativity and flexibility at all levels can help provide an environment where patients can best benefit from CGT. For example, legislative frameworks need to be adaptable to innovation, and COVID-19 served as an example of how to adapt quickly to change.
From a pricing and reimbursement perspective, new payment models are needed to reflect the novel features of CGTs. One size will not fit all, as every CGT is different. Diane Kleinermans (Belgian National Institute for Health and Disability Insurance) highlighted that thinking out of the box will be important to answer to the needs inherent to new technologies and therapies.
Further, international cooperation is the way forward, and the BeNeLuxA initiative is one example of this, she explained, as a platform for collaboration between five countries to monitor the whole life spectrum of medicines, organise joint Health Technology Assessments and conduct horizon scanning.
EU-level support for the continuous monitoring of quality and harmonised data, including via the EHDS, will be a building block to guarantee patient access to safe and effective CGTs across Europe and to address burning questions such as how safe is safe enough, or when health systems should pay for treatment given uncertainties in the durability of patient responses.
Reducing fragmentation, standardising data across borders and the increased use of real-world evidence will be key to supporting the acquisition of longer-term data, Bischoff asserted.
Cooperation between medical experts, policymakers, regulators, hospitals and academic developers, patients, health technology assessors, payers and industry will be decisive in transforming healthcare. Advancing CGTs requires collaboration as well as patient-centricity, working with and for patients.
The field of CGTs is deeply complex, as outlined by Vivienne Parry, science writer and broadcaster, and facilitator of the debate – but the fact is that CGTs are real and being administered now.
They disrupt traditional ways of providing healthcare, of developing, assessing and paying for treatments, and challenge us all to rethink existing policies and practices – or, as Bertalan Meskó pointed out at the beginning of the debate, to shift mindsets to incorporate change, as technological adoption needs to be accompanied by cultural adoption.
This article reflects on a more extensive discussion that took place during the webinar “Transforming the future of healthcare – do cell and gene therapies hold the key?”, organised by the European Health Forum Gastein and CSL Behring on 15th June 2021. Ilona Reischl and Diane Kleinermans spoke in a personal capacity during this meeting. To watch the recording of this webinar, please visit https://www.youtube.com/watch?v=QL6OYmoJIzg