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Twitter debate on access to medicines for ultra-rare diseases

On Monday 15 June, to raise awareness of the debate on access to medicines for ultra-rare diseases, Genetic Alliance UK co-hosted a digital twitter debate with Greg Mulholland MP and the House of Commons digital engagement team. Members of the public had the opportunity to feed their experiences into the debate happening the next day. On Tuesday 16 June, members of the public were permitted to use hand-held electronic devices in the Public Gallery at Westminster Hall, for the first time, during the debate “Access to drugs for rare diseases”.

Patients and their families told us about the impact that access to the right ultra-rare disease medicines have had on their lives. They also described the devastating effects of being denied medicines that they believe could help them.

Patients shared how their lives had been transformed when they had access to the medicines they needed. We heard stories from parents, of children now able to take part in the same activities as their peers as a result of their medication. For example, one parent told us about her daughter who could take part in her school sports day.

One patient told us about his fight to get the right medication, and his excitement in finally being able to get back to work:

“I’ve just got a job with a small company; they’ve given me time off for treatment. I’ve stabilised. It’s taken me 4 years, I can finally start putting my degree to use”.

Many stories, however, were about not having access to the right medicines, and showed the impact that this can have, not just on patients but on their families as well. Patients gave testimonials about the barriers that they had encountered on the way, such as clinicians not understanding the nature of the conditions, governance and bureaucracy, and the inequity between England, Scotland, Wales and Northern Ireland. Some highlighted that they struggle to access even the most basic therapies such as hydrotherapy – even when recommended by specialists.

“Many of us living with a Rare Disease could have better quality of life if top drugs and therapies were made available.”

Calls for a “robust and fair process for all those affected by ultra-rare diseases” showed that the issues faced by those who cannot access the medicines they need right now, are felt across the whole community. Many look to the future, knowing that developments in the treatment of their condition will come soon, and that access may be a problem if the system is not sorted. Others expressed concerns that one day they may no longer be able to access the medicines they need to effectively treat their condition.

The twitter debate’s hashtag, #RDdebate, reached 1.15 million people online – showing substantial engagement from patient organisations and patients themselves. The twitter debate was described as “historic” by Greg Mulholland MP, during the Westminster Hall debate on Tuesday. One really great outcome from the twitter debate is that so many MPs engaged in the debate on Tuesday. Over 15 MPs contributed to the debate and many of these used the testimonials that patients posted during our twitter debate, to inform their views and to direct questions to Life Sciences Minister, George Freeman MP.

You can view our Storify summary of the twitter debate here.

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