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Should patients with rare neurodegenerative diseases have the “right to try” experimental treatments? 

Posted: 26.07.23

Aisling Cantwell, a MSc Drug Development Science student at King’s College London, would like to invite you to complete the following questionnaire, which aims to increase understanding of the views of rare disease caregivers on the principle of the “right to try”. 

In the US, the Right to Try Act, enables non-trial access of unlicensed investigational drugs to patients with life-threatening conditions or diseases that have exhausted all alternative therapeutic options and cannot take part in clinical trials.  

In many rare neurodegenerative diseases, like Niemann-Pick, patients may have limited treatment options and life expectancies, and may not be able to participate in clinical trials. This raises the question as to whether these patients should have a right to try.  Aisling is investigating whether patient advocates and caregivers are in favour of implementing the ‘right to try’ in the UK.

This questionnaire consists of 30 questions and is open to UK based adult caregivers of patients affected by rare neurodegenerative diseases, like Niemann-Pick. The questionnaire will take approximately 5-10 minutes to complete, and participation is entirely voluntary. All data will be fully confidential and anonymised.

To participate, you can access the questionnaire here: