Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA)
Support for European legislation on orphan medicines, coupled with better collaboration among stakeholders, will improve access to treatment for patients with rare diseases.
In normal times, rare diseases don’t always receive a high level of attention from the medical community. But, during the COVID-19 pandemic, it can seem that rare disease is even further down the agenda. “An incredible amount of attention has understandably been dedicated to COVID-19,” says Nathalie Moll, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA). “This has had an impact on those living with other health conditions, including rare diseases. Throughout the crisis, pharma companies have worked tirelessly to ensure the continuity of clinical trials in extremely challenging circumstances.”
30 million Europeans living with some form of rare condition – delivering new diagnostics and treatments for people living with rare conditions is more pressing than ever.
But delays in access to orphan medicines are not a just a result of the COVID-19 crisis. “There has been a disparity of access for a number of years,” says Moll. “It is not a COVID-19-related issue.” Still, in the age of the coronavirus – and with around 30 million Europeans living with some form of rare condition – delivering new diagnostics and treatments for people living with rare conditions is more pressing than ever.
A two-pronged approach to improve treatment access
“For 95% of rare diseases, no treatment exists,” says Moll. “So there are two areas of critical importance. Firstly, we need to discover, develop and deliver more innovations and treatment options for people with this unmet medical need.” One way of doing this is to support the existing European legislations on orphan medicines and paediatric medicines. Before the orphan legislation came into force in 2000, just eight treatments were licensed for use to treat rare diseases. Now there are 169. “Having stable regulation in place inspires and drives investment in rare disease research,” notes Moll.
Secondly, where a new treatment option becomes available, all patients must be assured of access to it. “No one part of the healthcare equation can fix this problem,” says Moll. “We need to get all stakeholders around the table to discuss it, which is why we’ve called for a High-Level Forum – including EU and national decision-makers, patients, healthcare providers and new treatment innovators – to understand the causes of delay and outline potential solutions. If we do both of these things, we can make a real difference to people with rare diseases.”