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Home » Rare diseases » From lab to clinic: what charities are doing to power rare disease progress

Dr Catriona Crombie

Head of Rare Diseases, LifeArc 

Charities play a vital role in attracting investment towards rare diseases and supporting research that might not otherwise make it to the clinic.

The rare disease community has long been underserved, with many people struggling to receive the diagnosis and treatment they desperately need. Finding collaboration, funding and the right support are all hurdles for researchers to overcome in bringing innovations from the lab to the clinic. This is where charities and not-for-profits have a vital role to play.    

How charities encourage investment in rare disease 

While exciting progress has been made in rare disease research, investing in rare conditions — especially ultra-rare conditions — can carry more risk for commercial companies than common conditions. However, charities have been stepping in and partnering with industry to help progress these innovations towards patients.  

They can fund early-stage work to help reduce the risk and provide resources and advice to get medicines or new ways to diagnose a rare disease through trickier parts of their journeys. This can make it easier and more appealing for investment from industry partners.  

Access to new tests and treatments
through clinical research is crucial for
individuals living with rare diseases.

Moving research from the lab into medical breakthroughs 

Access to new tests and treatments through clinical research is crucial for individuals living with rare diseases. Many times, though, research gets stuck between the lab and the next phase of development due to a lack of investment, resources or knowledge. 

This ‘translation’ of science from the lab into results that benefit patients is the key to unlocking innovations. Some medical charities are targeting their efforts in this space, despite its tricky and (at times) risky nature.  

They help provide funding, identify the commercial potential of research and assist with the things a commercial company might not: early-stage development; advice on licensing; collaboration; intellectual property protection; and more.  

Our ambition is to transform the way diseases are identified and treated.

Advocating for the bigger picture in rare disease 

Addressing the challenges faced by the rare disease community requires not just research but policy change, coordinated investment, collaboration and education. Charities and the UK Government are working to build a nationwide ecosystem of researchers and partners to do just that. 

They can also use their neutrality to bring different parties (with different interests and needs) together for a common aim. This can help influence business models and solutions that might not have been found otherwise. 

There is no such thing as a disease too rare for investment. By driving innovations from the lab to the clinic, charities are helping to ensure that no rare disease goes unnoticed or untreated. 

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