Last week, Oliver attended the project board meeting of ASTERIX. Oliver represents the DevelopAKUre clinical trials on their patient think tank, and in today’s blog explains how the involvement helps the AKU Society.
ASTERIX is a European Commission funded project, similar to
DevelopAKUre. The name is short for: ‘Advances in Small Trials dEsign for Regulatory Innovation and eXcellence’. The project has two main aims:1) Redesign clinical trial to allow for more input from patients.2) Redesign clinical trials to meet the challenge of doing research in a small group of patients (such as in rare diseases).Typically, clinical trials are designed with little consultation of patients, and usually recruit thousands of patients. For a trial like DevelopAKUre, there are very few AKU patients in Europe, and so our target number was small. By involving the AKU Society as a partner, the study has direct input from patients. Since DevelopAKUre is therefore quite unique in clinical trials, we were asked to contribute some of our experience to ASTERIX.
A rare disease clinical trial like DevelopAKUre involves 140 patients. When compared to most clinical trials which would involve thousands of patients, this means that most rare disease clinical trials are underpowered (i.e. they have too few patients which makes the results more open to coincidence. If 10 people out of 100 experience a side effect, it looks like the likelihood of that side effect is 10%. However if 10 people out of 10,000 experience a side effect, it looks like a chance of 0.1%). More patients mean that you can trust the results more – statisticians call this the power of the trial.
Since clinical trials in rare diseases all tend to have this problem of being underpowered, and most unfortunately are designed without patient input; ASTERIX decided to focus in this area. At the end of their project, ASTERIX will create guidelines with recommendations on how to design future trials. To ensure those recommendations are useful, they created a patient think tank of 10 patient advocates who could bring their own experiences into the discussion on how best to change clinical trial design. As a member of the think tank, I get to share our experience from DevelopAKUre.
Last Spring, I took part in a structured interview with ASTERIX, answering detailed questions about the role of the AKU Society in DevelopAKUre, how we work with patients, what changes we achieved in DevelopAKUre and what changes we would like to see in future clinical trials. My interview was written up, along with the 9 other patient advocates and together formed the basis of a few longer discussions at the project board meeting.
These discussions will help the ASTERIX team to create guidelines for future designs of clinical trials. There is still another 2 years in the project, and so still time to collect in more information and experience of unique clinical trials. As patient advocates, the think tank made it clear that we would expect patient involvement to be a key priority, and so hope that in future, more clinical trials will consult with patients to ensure their views and experiences are taken into consideration.
If any AKU patients enrolled into SONIA 2 or SOFIA want more information about the ASTERIX project, or want to include their views on clinical trials in my feedback, please do contact me: [email protected].