Finding solutions for access to new treatments in rare disease

This week has seen extensive coverage of issues around access to new treatments for rare disease in Belgium. As with any case involving such severe disease, particularly involving children, our first thoughts are with Pia and her family and all those affected by Spinal Muscular Atrophy or any other rare disease. The case of Pia underlines the collective responsibility of regulators, industry and health systems to find solutions together to bring new therapies to patients who can benefit across Europe.
Zolgensma® is one of a new type of treatments.  A one-time gene therapy that, as well as other cell and gene therapies, have the potential to transform the outlook for many patients living with rare diseases.  These advanced, complex therapies may replace a life-time of treatment, give patients and their families hope of a different future but create challenges in managing their introduction in to health systems.
Traditional medicine reimbursement models cover from diagnosis through treatment until a patient is cured or their condition better managed.  In the case of many chronic conditions this can be over the lifetime of the patient.  Companies are reimbursed each time their medicine is used, spreading the costs to the healthcare system across the patient pathway. One-time cell and gene therapies change that paradigm. Not only in the complexity of development, diagnosis and use, also by delivering a one-time treatment with a life-time of patient benefit where the costs are borne by the health system upfront.  Clearly this is extremely challenging for health systems already under pressure from increased demand for their services. When viewed as a one-time cost these new treatments may seem expensive and should be valued in the context of replacing the costs of a life-time of treatment, transforming the life of the patient and the ground-breaking science involved.
From the companies that develop these new treatments, to the regulators that licence them, from the clinicians and nurses that care for the patients to most importantly, the patients and families themselves, everyone across healthcare wants to see patients like Pia get access to life-changing treatments like Zolgensma®.  So what needs to happen?
First of all, to make these new treatments available to patients like Pia as early as possible underlines the importance of the speed of regulatory decision making. Although Zolgensma® is approved for use in the US, it has not been approved for use in Europe at this time.  Normally, before new treatments can be made available to patients in Europe they need to be licenced by the EMA and then go through national pricing and reimbursement procedures before being reimbursed by health systems. In the case of Zolgensma®, it is unlikely to be approved by EMA before Q4 2019 and then will go through national decision-making processes – hence Pia’s family’s desperate drive to fundraise and self-finance a treatment currently only approved in the US.  In Europe, our industry, regulators and health systems need to work together to make new treatments available to patients faster.  From the regulatory perspective, that means maintaining Europe’s world class regulatory system, making the most of adaptive pathways, real world evidence and advances in data analytics to evolve our systems and processes to keep pace with the rapidly advancing science and changing treatment paradigms.  From the health systems perspective it may translate into a need to consider supporting authorization and payment of early access treatment, such as we’ve seen happen in France and Portugal.
Once a treatment is licenced for use in Europe, it’s about ensuring that patients get access to that new treatment. It is here where our industry, healthcare systems and governments need to find new ways to organize care and provide coverage for therapies that provide a lifetime of benefits in just a few treatments. We need to work together to find new models to finance these treatments that ensure access for patients and sustainability for health systems. We believe it is time for a  different type of conversation and EFPIA supports the EU Health Summit’s call for a multi-stakeholder High-Level Forum on Access and Healthcare Transformation [1] to bring all actors together to discuss how to ensure access to new treatments and technologies today, medical innovation for tomorrow and sustainable healthcare systems in a globally competitive Europe.
Finally, for every case where a new treatment has been developed there are many more rare diseases where currently no treatment option exists.  The EU Regulation N.141/2000 on Orphans Medicinal Products (OMPs) has been instrumental in fostering and supporting research into new treatments for people living with rare diseases. Since its adoption in 2000, more than 160 OMPs had been approved by the European Medicines Agency (EMA) by 2018, where only 8 existed before the Regulation came in to force. Today, 95% of rare diseases are still without a treatment option, so much more needs to be done for the 30 million Europeans living with a rare disease. We need to support the legislation, work collaboratively on its implementation and create an environment in Europe that realises the potential of rapidly advancing science like gene and cell therapies for the benefit of those patients.
By working together to maintain Europe’s world-class regulatory system, address issues of access and availability of orphan treatments and by building on the progress initiated by the EU OMP Regulation, our hope is that we can deliver a brighter future for the one in seventeen Europeans living with rare diseases.

[1] A Shared Vision For Health, Paving the Way in 2019, Recommendations in Full. EU Health Coalition 2019

Nathalie Moll

Nathalie Moll joined the European Federation of Pharmaceutical Industries and Associations (EFPIA) as Director...
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