Bronchiectasis patient feedback influences change in future trial design

A workshop on the design of clinical trials for bronchiectasis treatments was organised by the US Food and Drug Administration (FDA) in June to review their current advice to researchers.

The FDA is responsible for approving the licensing of drugs in the USA. Their decisions can also be important for those working in Europe, because pharmaceutical companies may stop developing their drugs if they are not approved in the USA.

This workshop follows an FDA licensing panel for a potential new bronchiectasis treatment earlier this year – which 12 members of the ELF bronchiectasis patient advisory group (PAG) took part in. Although the drug was not approved, the FDA had committed to reviewing the guidance they provide drugs companies in trials for treatments.  

At this workshop, views from our bronchiectasis PAG were presented by Professor James Chalmers (Chair of EMBARC). PAG members were asked three questions to establish their views on clinical trials. The US COPD Foundation also collated responses to the same questions – and there were presentations from patients and carers on the day.

Our patient question results

What would you consider the most important endpoint to measure in clinical trials of antibiotics?

The top two answers were:

• 40% said quality of life
• 28% said frequency of exacerbations

Would you be willing to do a trial and potentially get placebo (a fake medicine) for 2 years or would you have concerns about this?

• 44% said they would not be willing participate
• 34% would prefer a shorter duration
• 22% were unwilling to participate in a trial with a placebo

How much do you worry about the risk of antibiotics causing resistance in bacteria compared to the potential benefit of antibiotics for treating bronchiectasis?

• 55% worry
• 45% do not worry

Following the workshop, the FDA is now likely to: 

• Recommend quality of life or the frequency of exacerbations as the preferred endpoints for trials.
• Recommend research into different ways of measuring symptoms in trials – e.g. smart phone diaries.
• Review its guidance around the length of trials, as patients and clinicians said that 2-year studies are not fair to patients and could result in bias towards less severely ill individuals.
• Encourage companies who have previously conducted trials to share their data with researchers.
• Encourage more studies on questionnaires used for bronchiectasis to establish which are useful to measure treatment effects.

For more information about bronchiectasis visit the bronchiectasis patient priorities website.