Ensuring Safe and Timely Access to Advanced Cell and Gene Therapies

Dermot Mcgrath Reports

The development of advanced therapy medicinal products (ATMPs) for treating eye diseases has become a rapidly expanding field in recent years. Based on genes, tissues, or cells, ATMPs have the potential to revolutionize the treatment of a wide range of ocular conditions such as age-related macular degeneration, retinitis pigmentosa, Leber’s congenital amaurosis, Stargardt’s disease, optic nerve pathology, and limbal stem cell deficiency, among others.

With the number of approved ATMPs expected to increase, the legislative, regulatory, and access frameworks need to be sufficiently flexible and robust to ensure that patients across Europe have equitable access to these potentially life-changing treatments.

With this in mind, the European Alliance for Transformative Therapies (TRANSFORM) recently launched an MEP Charter in the European Parliament. The Charter presents seven main policy recommendations to promote safe and timely access to ATMPs while ensuring Europe remains attractive for investment and promoting healthcare systems’ sustainability.

The Charter launch included several roundtable discussions with TRANSFORM Alliance and MEP Interest Group members. Speaking at the panel discussion on new approaches to foster safe and timely patient access to ATMPs in Europe, Professor Mor Dickman of Maastricht University, Netherlands, and representative of the European Alliance for Vision Research and Ophthalmology (EU EYE), said it was a particularly exciting time to work as an ophthalmologist.

“We have some of the best scientists in the world working in Europe, and we have seen some very exciting developments in recent years in the field of ophthalmology,” he said. “We now have the first cell-based therapy with marketing approval to treat corneal blindness and the first gene therapy to treat inherited retinal disease with marketing approval. This is exciting for us as clinicians, but first and foremost, for patients who can experience life-changing therapies.”

Fragmented landscape

Prof Dickman said it was important for Europe to draw on the experience of such breakthrough treatments to improve the process of taking ATMPs from the lab to the bedside.

“This is a very fragmented landscape, and there is a clear need for harmonization, even though health is the prerogative of the European member states. What happens, for example, if an ATMP is approved, patients start benefiting from it, and then the company decides to abandon it?” he observed. “It has happened in the past and will undoubtedly occur again. One possible solution might be to apply the principle of hospital exemption (HE), which allows for ATMP use without marketing authorization under certain strictly controlled circumstances. The COVID-19 pandemic has really made clear how important it is that Europe becomes self-sufficient.”

Another key issue relates to data collection both within and across the continent’s borders, which Prof Dickman said is very complex due to the number of partners involved.

“What are the standard data sets, what are the outcome measures, and how do we collect them considering the issues of interoperability? How do we find funding for registries? And if one does receive funding, how do we ensure the sustainability of these registries once the funding is over? There is a need to impose data collection in a way that collects long-term data and captures patient-reported outcome measures (PROMs) in an independent platform in collaboration with the industry,” he said.

Lessons from COVID

The recent COVID-19 pandemic, Prof Dickman said, has also demonstrated the benefit of international collaboration in working towards a common goal.

“COVID showed how regulatory authorities throughout the world could come together and approve a vaccine in a historically short time. So, there should be room for ATMPs as well,” he said. “Someone earlier mentioned the FDA as our competitor, whereas I think they are our partner. We need to work together and harmonize our processes with the United States to make sure there is one pathway to the patient.”

Registries can play an important role in helping patients gain access to groundbreaking new treatments, Prof Dickman said, citing the Dutch RD5000 registry for patients with inherited retinal diseases (IRDs).

“Anticipating that more and more advanced therapies will become available soon, we’ve set up a registry where all these patients with IRDs in the Netherlands are registered,” he said. “It essentially means we are building an infrastructure in how we can reach these patients as and when a new treatment becomes available. I think if we had multinational registries or European registries with similar information, we could reach our target populations much more quickly.”

Another advantage is how they foster collaboration and enable benchmarking.

“One of the major benefits is they create networks where people learn from each other, share their experiences, and improve,” he added. “For surgeons, it allows benchmarking so you can compare your performance to others and see that what you are doing adjusted to the patients that you are treating is delivering the same results.”

European Alliance for Vision Research and Ophthalmology

Help us to build a healthier Europe!

Go to https://www.eueye.org or send us an email at info@eye.org to learn about our work with the European Commission and the European Medicines Agency.

Mor M Dickman MD, PhD is professor of ophthalmology at the University Eye Clinic, Maastricht UMC, Netherlands. m.dickman@maastrichtuniversity.nl

Prof Dickman’s work with the European Parliament is part of the greater efforts of EU EYE to increase awareness at the EU level about the research and policy needs in ophthalmology. This work is continuously supported by Dr Ioanna Psalti, who develops and finely tunes the communication and visibility strategy for EU EYE.

Tags: cell therapy, gene therapy