04 October 2019

Only a small proportion of trials of sickle cell disease (SCD) are multi-national, according to a new analysis.

The US is responsible for 53% of trials, but just 17% are being conducted across national borders, according to analysts at GlobalData.

The analysis was released to mark the end of Sickle Cell Disease Awareness Month in the US.

Analysts identified trials conducted between 2014 and 2019 and found that 24% were phase I trials and 52% phase II trials. Some 14% were at phase III.

60% of trials were conducted independently of commercial sponsorship, the analysts found. About 20% of trials were conducted in Europe, including 3.2% in the UK compared with 3.9% in France.

The most researched drug is hydroxyurea, the researchers found, but a substantial number of trials have involved Voxelotor, a drug which stabilises the structure of HbS and so prevents sickling.

GlobalData analyst Mohamed Abukar said: “Due to the trial phase distribution showing a greater proportion of early phase trials, it is not surprising that 82.9% of SCD trials are single-country trials, with only 17.1% being multinational.”

He added: “At present, bone marrow transplant is the only cure for SCD. It is typically reserved for younger patients due to the increasing risk of morbidity and mortality in patients with advancing age.”

Source: GlobalData


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