24-25 - TREATMENTS: Single dose innovation

Innovation requires change. In a world geared towards chronic therapy and disease management, it’s important to demonstrate the benefit and lifelong potential of innovative, single-dose treatments. It is the boost decision-makers need to help them make informed decisions on reimbursement and change the current healthcare paradigm.

Take gene therapy, for example: the first one for spinal muscular atrophy (SMA), Zolgensma, was approved in the US in May 2019 and in Europe in May 2020; a huge milestone for patients and industry alike. It represented a completely new approach to SMA disease treatment – addressing the root cause of the disease and delivering the therapy as a one-time dose.

Previously, the standard of care for treating SMA was a medicine delivered as an injection to the spine which required four initial loading doses and then a maintenance dose every four months, for life. With such innovation, the new therapy has necessitated change in how healthcare systems currently value, reimburse and provide access to such medications. Healthcare systems are adapting – or have already adapted – to accommodate such revolutionary treatments, ensuring access to eligible patients in many countries.

We need to be willing to share risk. As with any new, revolutionary treatment that comes to market, there is some level of uncertainty.

With many rare diseases there is limited data available at the time of approval, mainly due to the size of the population that is available for clinical studies. This is also true for one-time therapies, particularly in relation to long-term data. It is not feasible, however, to wait for long-term data before making the therapy available to patients in need.

Many innovative treatments are designed to significantly improve patients’ lives. Thus, we need to challenge the status quo of healthcare systems and traditional access pathways. Only then can we share the potential benefits of medicine innovation among patients, industry and wider society. One way to do this is for stakeholders to be willing to find risk mitigating tools such as annuity payments and outcomes-based agreements.

It is vital to provide one-time treatments for genetic diseases and equally important to realise why. Take SMA for example – this rare, devastating genetic disease leads to progressive muscle weakness, paralysis and when left untreated in its most severe forms, permanent ventilation, or death in 90% of cases by age two.

Just over a decade ago, there were no treatments for SMA available, but now there are three approved treatments in Europe. Patients need to be treated as soon as possible. We now know that the earlier a baby is diagnosed and treated, the better the outcome. Given the nature of the disease, a child with SMA will always require some form of follow-up care management, but the vision behind one-time gene therapies is to help limit chronic treatment burden.

We need to adapt to understanding and valuing the lifetime of a treatment vs an annual cost or a defined period of time especially as it relates to one-time therapies. Currently, caring for and treating patients with SMA cost healthcare systems between €2.5m to €4m per patient in the first ten years of their life alone – much more than a single dose of gene therapy for SMA.

We appreciate that a large upfront cost is daunting and requires a refocus of healthcare system resources and priorities, but cumulative savings over time will be realised, allowing for reallocation of savings and resources into other areas in need.

There are several factors that impact access agreements for innovative therapies, not least, proven clinical efficacy and favourable benefit-risk profile, the benefit to healthcare systems and the value to society.

The most important attribute that all our successful access and reimbursement agreements share is the ability to be flexible and work with existing frameworks and processes as well as forge new pathways. Novartis Gene Therapies is constantly in talks with government bodies and local stakeholders across Europe to ensure access to Zolgensma, offering flexible options such as retroactive rebates, deferred payments and instalment options, and outcomes-based rebates.

It’s these tailored solutions that have enabled children with SMA and their families across Europe to experience the transformational benefit of gene therapy.

So far, we’ve seen great success with our customised reimbursement solutions across Europe, including in England, Scotland, Italy, France, Germany, Spain, Russia and Portugal, as well as countries in Africa and the Middle East, such as Egypt and Israel.

We’ve worked collaboratively with markets to establish access pathways tailored specifically to their healthcare and patient access systems ensuring reimbursement is sustainable, whether that be via national reimbursement funds, national health services, third-party payers or innovative medicines initiatives.

Meanwhile our access work continues to build momentum. Every single day, our teams across Europe and the Middle East and Africa (EMEA) region are collaborating with officials in their markets to ensure access to all eligible patients in need.

Of course, achieving reimbursement is a huge step in providing patients with access, but that’s not the only milestone. As with any specialist technology, healthcare professionals need to be trained properly in its use, delivery and distribution. Novartis Gene Therapies has been and continues to work with healthcare systems across the region to support specialised treatment centres, equipping them with the skills needed specifically for treating patients with gene therapy.

It’s essential to keep collaborating in order to raise the profile and currency of gene therapy – working with other groups to bring our treatment to eligible patients in their countries and regions is vital.

Successful negotiation is a two-way street, and we appreciate the leap that decision makers are adopting when introducing a completely new treatment method to their systems. Our strong belief in the value of this innovative treatment is rewarded by the fact that many patients gain access to it and are able to have their lives transformed.

We will only be successful if we continue to work together. We understand that changing the way we look at transformative therapies is not something that can happen overnight, but we are confident that working together in a collaborative spirit will pave the way for even more countries to embrace one-time therapies.

We truly believe that all patients deserve the option for potentially transformative outcomes and that these options will be recognised by the increasing number of modern, culture-shifting healthcare systems.