‘Moonshot’ mindset can make the impossible possible

Over the last 20 years we have witnessed some incredible advances in orphan and paediatric medicine. Now is the time to come together and push the boundaries of what is possible.
People living with rare and paediatric diseases face some of the most severe, debilitating and challenging health conditions. From diagnosis to treatment, patients, their carers and families live in hope of advances in medicine.
Science, economics, and policy coalesce to shape the outlook for people living with rare and paediatric diseases and their families. Some of the hurdles are well-known: the small number of patients with rare conditions makes it difficult to conduct vital research, while trials on children come with added complexity. And even when a new treatment is developed the potential patient population where it can be used is by definition, small.
In the late 90ies, the pipeline of orphan and paediatric medicines in Europe was worryingly empty which caused European policymakers to seriously step up and develop an enabling environment, in consultation with key stakeholders: the EU Orphan Medicinal Product Regulation.
Thanks to a range of incentives for research and development, the implementation of the Regulation has led to over 190 new treatments for around 90 rare diseases authorised. The adoption of the EU Paediatric Regulation in 2006 has stimulated paediatric research resulting in 296 new paediatric medicines approved between 2007-2019[1] and a 50% increase in the proportion of paediatric clinical trials between 2007 and 2016.
Building on success
20 years since the implementation of these regulations, we can  safely say that regulatory and economic incentives have successfully sparked increased scientific activity and supported the creation of SMEs working on conditions which had previously been overlooked. Looking at the current pipeline, 40% of therapies in development are orphan drugs[2]. The Orphan Regulation alone has incentivised our industry to deliver treatments for up to 6.3 million patients in the EU.
In July this year, EFPIA published a report by Dolon which explores how to build on these successes, along with a series of case studies looking into the key factors that drive the development of orphan and paediatric medicines. The report clearly shows  that while real progress has been made, there is scope to deliver more for people living with peadiatric and rare conditions.
The lack of therapeutic options in some areas is a consequence of existing scientific, regulatory and economic barriers to development. These are further compounded by uncertainties relating to pricing and reimbursement. It is clear to all that there is no single solution to stimulating medicines’ development in extremely rare and paediatric-onset diseases.
We also know that future medical innovation will be influenced both by Member State life science policy and the implementation of the EU Pharmaceutical and Industrial Strategies. That’s why it is critical that we develop European policies that support, rather than hinder Member States’ ambitions to boost their life sciences sectors. Innovations that can transform the lives of patients come from advances in science more broadly. The more scientific avenues we incentivise the greater the chance of finding new treatments in areas of unmet medical need.

Nathalie Moll

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

What should we do next? How do we foster and support a research eco-system that can deliver the next generation of treatments and cures? We need to share a ‘moonshot’ mindset so as to drive scientific breakthroughs, reduce barriers and develop well-calibrated incentives.
To shoot for the moon means thinking big. It means applying what we have learned since the inception of the OMP Regulation in 2000, developing a shared understanding of how innovation in rare and paediatric disease happens and then ensuring our policy framework helps deliver more for patients. And of course, we know ambitions of this scale are never realised alone. Collaboration will be key.
If we can support, incentivise and harness the incredible science, create the right policy framework and work together then we have a real shot at making the impossible possible.