Rare Diseases: Why medical innovation needs to be one step ahead

Accelerating innovation key to ensuring that no one is left behind in fight against rare diseases, says Nathalie Moll EFPIA Director General.
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By Nathalie Moll

Nathalie Moll is Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA)

24 Feb 2021

As we come to celebrate Rare Disease Day 2021, we share two aspirations with the wider health and research community: to ensure faster, more equitable access to treatment for people living with rare disease in Europe and to meet the needs of people living with the 95 percent of rare conditions where currently no treatment options exist.

Discovering and developing new treatments is the first step to addressing unmet medical needs and creating access to medicines for people living with a rare disease. It requires developing a shared understanding of how and where medical innovation is generated. Then creating a policy framework to support that journey.

“Rare Disease Day brings people together to develop a shared understanding of the barriers and delays to access to medicines for rare disease”

Typically, it follows the science and stems from a dynamic, vibrant and connected research ecosystem. For example, breakthroughs like gene therapy and cell editing have paved the way for the science to be applied to a range of disease areas and rare genetic conditions. If we look at mRNA technologies that have come to prominence as the basis for a number of the leading COVID-19 vaccines, the science was developed in the 1990s and, as its potential has begun to be understood, it is now being explored for treatments of different types of cancer, TB and a range of vaccines.

This is how innovation works. You foster, incentivise and support a broad research ecosystem so that science can explore a range of avenues and when the breakthroughs come, new treatments follow. This knowledge is critical as the EU considers options around the framework of incentives for orphan medicine research. Any changes to the incentives’ framework must be the subject of a detailed impact assessment. Decades of experience in generating medical innovation have taught us that narrowing incentives, to focus only on ultra-rare conditions for example, will stem rather than accelerate the flow of new treatments for rare conditions.

The second step to faster, more equitable access for patients in Europe begins with regulatory approval. Industry, regulators and health systems need to work together to make the most of 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. There is an opportunity with the European Pharmaceutical Strategy to evolve Europe’s world-class regulatory processes as an important step towards faster access for patients.

Once a treatment is licenced for use, industry, healthcare systems and governments share the responsibility of ensuring that patients get access to that new treatment. We believe it is time for a different type of conversation to happen, one that brings patients, industry, healthcare systems and governments around the table to collectively find new ways to sustainably finance innovative treatments such as cell and gene therapies that replace a lifetime of care often with a one-off treatment.

If we really want to address the access issue, we need to build and agree on a shared, evidence-based understanding of the root causes of shortages, barriers and delays in access to medicines before going on to co-create pragmatic solutions to those hurdles that work for patients. Rare Disease Day brings people together to develop a shared understanding of the barriers and delays to access to medicines for rare disease, as well as to collectively understand where rare disease innovation comes from, which will be critical for getting the next steps right and to ensuring that no one is left behind. 

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