Last week I attended a symposium in Brussels on Improving Patient Access to Rare Disease Therapies. By involving all stakeholders and collaborating on a European level, we can ensure that all patients have access to live-saving approved treatments. This week’s blog discusses the important messages and discussions taken from the event.

The multi-stakeholder symposium was organised by Eurordis, a fantastic European patient-driven alliance, dedicated to improving the lives of rare disease patients and involving patients in everything they do! The symposium invited experts from a range of different fields to come together with the ultimate goal of improving patient access to rare disease therapies.

Yann Le Cann from Eurordis introduced the purpose of the symposium. Yann is all too aware of the challenges rare disease patients’ face, as his child has Cystic Fibrosis. He noted the huge improvements Europe has made in increasing the number of licensed rare disease therapies, with 30-50 new therapies expected every year by 2020. Despite progress, thousands of patients are unable to access these licensed therapies. This is costing patients’ lives.

Yann dedicated the symposium to Philip, a young patient with a rare disease, called Hunter Syndrome. Treatment for Hunter syndrome was approved in 2007, yet it took 7 years for Philip to be able to access it. He died last week due to inconsistent access to treatment. This personal story was a shocking reminder that his death could have been prevented had there been better processes in place. The need to discuss and collaborate on a European level is vital to improve the fragmented process that is taking lives.

‘We want Europe to be the best place in the world for rare diseases therapies’

Over the next two days, there were various speakers, panels and breakout sessions designed to highlight key challenges and encourage discussions and solutions between stakeholders. Cees Smit gave an important patient perspective on living with haemophilia and expressed how his life changed when the first licensed treatment was approved at age 16.

We also heard from Philippe de Becker, member of European parliament, who stressed that an EU approach can help improve patient access. Philippe noted the problems with the current fragmented system, as 28 member states all have different rules for how rare disease therapies are assessed. Huge economic differences in Europe mean high prices are manageable for some, yet unsustainable for others. By cooperating on a European level, we can ensure all patients across Europe have the same access.

‘Change can happen. Change will happen’

The second day focused on how we assign value and assess new therapies. We were invited to take part in a simulation exercise, where we were given four hypothetical new therapies and were given the task of deciding which two to fund. The exercise highlighted how challenging these decisions can be, particularly when faced with levels of uncertainty. It also showed how valuable the patient perspective is when faced with these decisions.

Charles L. Barker from CMI Concord Group stressed the importance of collaborations. The ultimate goal should be to win together rather than compete against one another. AKU advocate and chairman of trustees Nick Sireau shared his experience of using these collaborative techniques. After the failed nitisinone clinical trial in America, there was very little interest in AKU research, yet through determination and collaboration, we are currently running a European clinical trial, called DevelopAKUre.

‘The techniques you’ve discussed completely changed my life’

We ended the symposium with a panel discussion involving different stakeholders, where various important topics were discussed, including the nature of value, sustainability, innovation and improving quality of life.

The symposium was really valuable in bringing together stakeholders, highlighting challenges and most importantly, discussing ways to improve current services that are outdated and no longer fit for purpose. It was really refreshing to meet so many people passionate about working together to ensure all rare disease patients have access to appropriate treatment.