Identifying barriers to clinical trial participation with families affected by fragile X syndrome
Eley, Sarah Elizabeth Anne
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Fragile x syndrome (FXS) is thought to be the most common inherited form of intellectual and developmental disability and the most common known single gene cause of autism. The prevalence of FXS is approximately 1.4 per 10,000 males and 0.9 per 10,000 females. Learning difficulties in people with FXS can vary from mild to severe however it is most common to be within the mild to moderate range, with males often more affected than females. The current medical treatments for FXS are primarily symptomatic in nature; and include the use of selective serotonin re-uptake inhibitors for anxiety, stimulants for attention deficit hyperactivity disorder and antipsychotics for irritability or aggressive behaviour. However, our understanding of FXS has now progressed to a stage where treatments selected to target the underlying neurobiology are now being trialled. Experience conducting research studies at The Patrick Wild Centre into this condition has suggested that some families are reluctant to take part in clinical trials making recruitment challenging. Therefore, this study was designed to understand barriers to participation specifically amongst patients with the condition and their families. The primary aim of this study was to identify the barriers to research participation. The second aim of the study was to find out how barriers to clinical trial participation could be overcome in order to maximise recruitment for future studies. A mixed method design was used to collect both quantitative and qualitative data. Participants were identified from the UK Fragile X Society mailing list by a member of the Fragile X Society, The Patrick Wild Centre mailing list and through advertisements on the Patrick Wild Centre website, Facebook and Twitter feeds. A quantitative questionnaire was completed by 328 parents, carers, family members of individuals with FXS or by individuals with FXS. Following this, three focus groups took place in Chelmsford, Bristol and Edinburgh. This study demonstrated that there are many different factors which may negatively influence motivation to participate in a clinical trial in this group. However, the main barriers to participation were concerns about possible side effects, travel, requirement for blood tests and financial reasons. When these were explored further during focus groups it was evident that these barriers were not as clear as they may have appeared and had many complexities to them. The main way to overcome barriers that was repeatedly discussed in each focus group was the importance of accessible information in helping families to understand the study including any potential safety issues. This study highlights that while there is no one thing that researchers can do that will work for everyone, there are many things that researchers can do to improve participation such as making their research more individual, flexible and accommodating.