Disrupting cell and gene therapy commercialization with innovative payment models

Elisabetta Zanon is the Director of European Public Affairs and Advocacy at the Alliance for Regenerative Medicine, an international advocacy organization championing the benefits of engineered cell therapies and genetic medicines for patients, healthcare systems, and society. The Alliance aims to shape the policy and regulatory environment that supports the development and commercialization of cell and gene therapies representing its 400+ member organizations including small biotechs, large pharma companies, academic centers and patient organizations.

Here, Elisabetta discusses the regulatory landscape for cell and gene therapies in Europe, the challenges associated with their commercialization and how innovative new payment models could help improve their uptake.

What are the public and regulatory perceptions of regenerative therapies in Europe?

We have now seen a number of regulatory approvals for cell and gene therapies in Europe by the European Medicines Agency. But, these approvals are just the tip of the iceberg. There are many ongoing clinical trials and further candidates in the development pipeline that could reach patients going forward. The potential offered by these therapies is absolutely phenomenal; in the future we could potentially see upwards of ten approvals per year.

Now what is the perception? The perception, I would say, is that these therapies are a disruptive innovation. Our healthcare systems need to adapt to accommodate regenerative medicines, which represent a paradigm shift compared to traditional medicinal products. Currently, healthcare systems are not ready for this; science is moving much faster than our healthcare systems are updating.

For healthcare systems to maximize the impact that cell and gene therapies could have on our society, we need changes from regulatory, health technology assessment and pricing perspectives. We need to help inform pricing and reimbursement bodies on the value of these therapies, because if you compare these transformational therapies with the existing, conventional therapies, they are often far more expensive at the moment of administration, but they offer a durable effect over time and as such they are cost effective in the medium and long term. So the potential is there, but that there is a lot of work required for us to realize it.

How can healthcare systems adapt to accommodate regenerative medicines?

We need to start with the health technology assessment, which is the way healthcare systems assess the value of a therapy after it has received regulatory approval, ultimately to help them decide if they will pay for it. Most health technology assessments rely on information from randomized controlled trials that essentially test the innovative therapy against a competitor or the current standard of care, or a placebo group, for example, in a randomized controlled trial.

Very often, randomized controlled trials are not possible for cell and gene therapies for several reasons. Often the therapy is for a rare disease and the number of patients is so small that you cannot have a control group. Alternatively, it can be unethical to have a placebo group as the treatment requires invasive administration, so you would have to conduct an invasive procedure without giving a patient the option of receiving the transformational therapy.

As a result, regenerative medicines are often assessed in single arm trials. Health technology assessment bodies then claim that we don’t have enough evidence to properly estimate the clinical value of this therapy against the standard of care or against the control group and so healthcare systems may refuse to pay for it. There is this level of uncertainty where the evidence, which is considered sufficient by the regulator to give authorization, is later considered to be insufficient by technology assessment bodies or pricing and reimbursement bodies. This is a big challenge.

What do you see as the solution to this challenge?

We need more alignment between the regulatory and health technology assessment bodies in terms of the evidence needed for both of them to come to a decision.

How else can we adapt to facilitate the adoption of these therapies?

We need to implement more innovative payment models to alleviate the impact of the high cost of these therapies. Instead of paying the full cost of a therapy at the moment of administration, we could split the payment of that therapy over a number of years. You could also link the payment to the outcome: if the outcome is good, then you continue paying as long as the therapeutic effects of the therapy persist; if the outcome is not the one you expected, then you can stop the payment.

These innovative payment models are very important for our sector to ensure a significant take up of these therapies going forward, because the cost of these therapies can be significant at the point of administration. If you have a chronic patient receiving a treatment over the course of their life, the cost of the treatment is split over a long period of time. Transformational therapies like cell and gene therapies are most often only administered once and then they have a very durable effect over a long period of time. Therefore, they can seem more expensive at first, but they lack the hidden cost of continual repeat treatment. For healthcare systems, this can be difficult from a spending perspective.

However, while I think that introducing these innovative payment models can help make costs more manageable, it will, of course, requires healthcare systems to begin more structured follow-up with patients to capture outcomes. As a result we often notice resistance from healthcare systems to adopt these innovative payment models due to the additional work and cost associated with that follow up.

Are the companies making these therapies enthusiastic about these payment models?

Mostly, yes. I cannot, of course, speak for individual companies, but we have seen a number of therapies going to market and being commercialized using these innovative approaches and these payment models have eased discussions and negotiations, ultimately leading to an increased uptake of these innovative therapies.

This is vitally important as in recent years we have seen seven products that received marketing authorization in Europe be withdrawn from the market due to the difficulty in commercializing them. So around one-third of the products approved by the European Medicines Agency have been withdrawn from the market mainly for commercial reasons. We need creative solutions to ensure that the products authorized by the European Medicines Agency are commercially viable in Europe.

If there was one thing that you could ask for to improve outcomes for the regulatory approval and commercialization of these therapies, what would it be?

This is really challenging because it’s the whole ecosystem that matters. We are talking of really disruptive innovation here. These therapies are very, very different compared to traditional medicine but we still rely on the old system for the authorization, assessment and commercialization of these products. We need this system to develop to support these advances, all the way through the development, testing, authorization, clinical assessment, health technology assessment, cost and ultimately, the commercialization of these products.

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The opinions expressed in this interview are those of the interviewee and do not necessarily reflect the views of RegMedNet or Taylor & Francis Group.