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Designing Rare Disease Patient-Friendly Clinical Trials

Last week, we discussed the unusual clinical trial recruitment challenges faced by pharma and biotech companies when trying to develop new orphan therapies. This week, we will propose potential solutions to approach trial participant recruitment as well as retention.


Because there are dramatically fewer patients in any rare disease population, recruitment strategies must take this into consideration when planning this stage of development. The geographic net must be cast larger, across multiple regions. Rare disease advocacy organizations can be instrumental by developing patient registries specific to individuals interested in clinical trial participation.


A stumbling block for many patients when they consider being part of a clinical trial is their location. Many are not close to a trial center, and the distance they would need to travel for follow-up is prohibitive. A practical and very effective solution to this obstacle would be at home monitoring. Certain hospitals and health systems employ at home nursing services who could visit the patient’s home for blood draws and other follow-up. There are also partner labs with visiting nurses or phlebotomists who can also perform these services.


By making clinical trial participation less of a burden for eligible patients, the pharma industry can increase the pace of developing necessary, life-saving therapies for communities who have been without FDA-approved medications for too long.

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