Newsmaker Interview: Who is Looking Out for Ophthalmology?

It is hard enough to keep up with the daily demands of a busy ophthalmology practice, much less with the sometimes bewildering EU regulatory landscape. Nonetheless, policy decisions made in the EU parliament affect everyone doing research or clinical work. EuroTimes Editor-in-Chief Sean Henahan spoke with Mor Dickman MD, PhD, who provided insight on current initiatives affecting ophthalmology.

Busy clinicians barely have the time to read the newspaper, much less lobby the EU. What do they need to know?

There is a lot of frustration among our clinical colleagues because there is little understanding of how the European mechanism works and where we can influence matters that are important to us, even in the short and medium term. You achieve that only by engaging with the regulators and policymakers to explain what is important, what should be done, what should not be done, what would be cumbersome and unnecessary, and what is really needed.

In terms of a regulatory landscape in Europe, who is looking out for ophthalmology?

An organisation called the European Alliance for Vision Research and Ophthalmology (EU EYE) engages with regulators and policymakers to advance the interests of the ophthalmology community and ensure we are represented in the wide context of EU health policies. This alliance of major ophthalmology societies is a non-profit, pan-European advocacy organisation. It is important to mention the ESCRS played a key role in establishing this. Peter Barry was the first president of EU EYE, and Paul Rosen is continuing this work.

The gamechanger was the recognition of EU EYE as an eligible organisation for the EMA, the European Medicines Agency. I want to congratulate Ioanna Psalti on her work in achieving this. I represent the EU EYE at Health Care Professional Workgroup (HCPW) at the EMA. This is an interesting forum where we gather medical specialists from all the specialties in Europe and provide the EMA and its scientific committees with recommendations related to medicines and medical devices.

You are also involved with the European Alliance for Transformative Therapies (TRANSFORM). Tell us about that.

TRANSFORM is a multi-stakeholder alliance that connects members of the EU parliament with policymakers, medical experts, patient groups, medical associations, scientists, and industry. All these stakeholders are involved in this very exciting field of advanced therapy medicinal products (ATMPs). These therapies have tremendous potential to influence how we may treat eye diseases, but also the implications for the sustain-ability of our healthcare systems. It is about all stakeholders coming together for a common goal.

For ophthalmology, this relates to cell therapies. The field of ophthalmology has been quite pioneering in this area. Holoclar, the first stem cell therapy approved by EMA in 2015, was for treating limbal stem cell deficiency. One of the first gene therapies approved by EMA was Luxturna to treat retinal degeneration related to a mutation in the RP65 gene.

Since 2007, all the regulatory work related to ATMPs is coordinated by one body, the EMA. Within the EMA, the committee for advanced therapies is responsible for proving scientific opinions on product quality, safety, and efficacy. In one of the most interesting developments in recent years, the EMA is inviting clinical researchers—especially academics and non-profit organisations—to engage with the regulatory bodies at a very early stage to gain an understanding to help them overcome obstacles they may encounter. Initiatives such as the PRIME project focus on driving these products quickly from the lab to the bedside.

What ophthalmology ATMPs are in the pipeline now?

Several ATMPs are in various stages of clinical development, including cell therapies for age-related macular degeneration, inherited retinal disorders, and corneal diseases. A second group are gene therapies using adenovirus vectors, 

developed mostly for inherited retinal diseases, including choroideremia and Leber congenital amaurosis. A third and very exciting category is RNA-based therapies for inherited retinal diseases such as Stargardt disease and cornea diseases such as Fuchs’ dystrophy.

What about the costs of these new therapies?

Cost is a very important question. Normally, you price a new treatment against an alternative, but these conditions have no alternative, which means it will be expensive. Research and development costs are very high, and the regulatory pathway is also quite expensive. We are experimenting to see if we can improve this situation by using the hospital exemption rule more often—a European legislative initiative that guarantees patient access to novel therapies based on the use of ATMPs in the member states.

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