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Home » Rare diseases » Rare disease clinical trials continue, thanks to new technology

Dr. Carlos Camozzi

Chair of the Rare and Orphan Advisory Board, Simbec-Orion

Despite the ongoing COVID-19 pandemic, clinical trials continue, including those that feature patients with rare diseases which can often be the hardest to establish.

When it comes to clinical trials, an incredible amount of work has to be done behind the scenes before any patient analysis can take place. It is particularly challenging however, when conducting trials for rare disease treatments. Dr Carlos Camozzi, Chair of the Rare and Orphan Advisory Board, Simbec-Orion, explains:

“Clinical trials are certainly a challenge when it comes to rare diseases, because of the small number of patients, the rapid, progressive deterioration puts patients’ lives at risk and demands a sustainable commitment from all stakeholders.”

Setting up clinical trials

There are several steps that need to be taken before the start of a clinical trial in rare diseases. The first is to create reliable and validated biological, clinical and imaging markers that are needed to support the final application to regulators. Global recruitment of patients can also take a long time.

“Patient groups are there not just to support people with rare diseases, but also to provide a reality of what the patient experiences. Obviously when patients are enrolled in a trial, we listen to their needs and tailor their treatment but before a trial starts, a patient group can help not only spread the word about the trial, but also direct the main concerns and queries from the community.”

The impact of COVID-19 on trials

Clinical trials for rare diseases come with other challenges, such as little treatment data and often no possibility to compare the safety and efficacy of the new treatment. Neither can any of the patients involved be given a placebo, so it is incredibly important to listen to their concerns. With the onset of COVID-19, trials have had to face drastic changes.

Dr Camozzi explains, “Many trials had to stop, as hospital settings were diverted for other uses and new protocols had to be created. That’s why it is so vital to create a link between clinical research organisations and the patients who can advocate for them. Health authorities who have been very considerate of those with rare diseases and their stakeholders have also been working hard to speed up the process.”

Despite the pandemic, the application of digital technology has helped develop trials when medical settings have not been available. From healthcare at home to mobile devices, home delivery of the medication and apps, both clinical trials and the search for innovative treatments are once again able to continue.

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