From pandemic to rare disease, medical innovation is the answer

DISCLAIMER: All opinions in this column reflect the views of the author(s), not of EURACTIV Media network.

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#WeWontRest, an initiative by the European Federation of Pharmaceutical Industries and Associations. [EFPIA]

This article is part of our special report Reviewing orphan drugs law: A tough equation.

Living through a global pandemic, we can take some comfort from the enormous collaborative research and development response that is taking place to find a way out from under the shadow of the COVID-19 crisis. Scientists around the world are working to find new diagnostics, treatments and vaccines to use in the fight against the coronavirus.

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

The polar opposite of a pandemic, a rare disease may only affect a handful of patients in a particular country, but to the patient, their family, carers and clinicians, the impact of their condition can be just as devastating. Often genetic, discovered in childhood and frequently severe, rare diseases are some of the most significant scientific challenges in medicine. Typically they affect only 1 in 2,000 people, but there are more than 6000 rare diseases meaning around 30 million Europeans are living with some form of rare condition.

Rare diseases certainly don’t attract the levels of media attention and interest that a public health crisis like COVID-19 does but for patients living with the 95% of rare diseases where no treatment options exist, the need for new diagnostics and medicines is every bit as real.

As the industry responsible for developing those new therapies, we are committed to achieving the crucial objective of finding new treatments for patients in areas of unmet medical need. It means supporting and strengthening the framework of incentives to drive further research into the next generation of treatments and cures for rare disease and paediatric treatments. Incentives drive investment, research and results. Results that mean new treatments and ultimately better outcomes for patients.

It is why EFPIA supports the existing European legislation on orphan medicines and paediatric medicines, while underlining the need to co-create vehicles to address issues around access to new treatments. Prior to the orphan legislation coming into force in 2000, there were just 8 treatments licensed for use to treat rare diseases, now there are 169, underlining the fundamental role that a predictable and stable incentives framework for research and development has. Any destabilisation of that framework threatens the investment in research and development in this area.

At the same time, faster, more equitable access to new rare disease treatments for patients across Europe is a shared goal and responsibility. Re-opening the Orphan Medicinal Product Regulation will not address the core challenges regarding unequal access and availability of orphan drugs within the European Union. Addressing this challenge requires a structured dialogue with relevant stakeholders, Member States and the European Commission sensitive to their respective competence areas, to find solutions to introduce these ground-breaking treatments.  That is why we reiterate the call to Member States and the Commission to set up a High-Level Forum composed of EU and national decision makers, patients, as well as the research and healthcare communities to find collaborative, multi-stakeholder solutions to these complex issues of access.

Considering the lack of innovation policy drivers in the Roadmap for the EU’s Pharmaceutical Strategy, it is all the more critical that we maintain a stable and predictable incentives framework that can continue to support the development of new treatments for rare disease patients in Europe. We have to work together to ensure access to new treatments and technologies today, medical innovation in rare diseases for tomorrow and sustainable healthcare systems in a globally competitive Europe. Destabilising investment in the discovery and development of new treatments for patients living with rare diseases by re-opening a legislation, proven to be effective in stimulating the development of new treatments, cannot be the right approach.

One crystal clear lesson from the COVID-19 pandemic has been that the answer lies in medical innovation. This is equally true for patients living with rare diseases.  Now is the time to re-build and re-invigorate rather than devalue Europe’s health research ecosystem, to make sure we address these  challenges and #WeWontRest until we make treatments for rare diseases less rare.

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