• Clinical Trials in Paediatric IBD

    Children with inflammatory bowel disease (IBD) have more extensive and aggressive disease than adults, making it crucial that new treatments become available sooner. Unfortunately, it takes an average of seven years between approval being given for a new drug in adult practice and it becoming widely available to children. There are many reasons for this long delay, including the time taken to receive approval from the drug regulators for use in children.

    Although children are clearly different to adults in many aspects, for the purposes of treatment of IBD there are many similarities, and it is generally thought that the way the drug works is similar in both groups. In fact, children have had a better reported response to infliximab than those in adult trials. Due to the similar response to treatment, is has been suggested by the authors of this paper that there are alternative ways to enable faster availability of treatments for children.

    The method for obtaining drug approval involves several stages; the final stage before the drug is licensed is testing of a new drug against a placebo (dummy drug). The investigators then examine the effect of the new drug in order to determine whether it improves symptoms sufficiently and safely enough to be used widely. The authors of this paper believe that it is not ethical to deprive children of potential treatment, which has already been proven to be effective in adults, and may improve their disease; as such, a placebo should not be given. Instead, the same drug should be used but at a different dose or different timing (i.e. given every eight weeks versus every 12 weeks). However, it remains crucial that trials are performed in children to ensure the appropriate dosage, safety and usage of the drug. These trials would require fewer patients and, as a result, could be done more promptly. If accepted by the drug regulators, licences could then be approved sooner.

    This approach to avoid the use of a placebo has recently been suggested, but has not yet been approved by the drug regulators. Many key features need to be clarified and established to ensure that new treatments are both safe and effective for children. If these issues can be addressed, a more streamline path can be provided to ensure that children with IBD have access to new treatment options in a timely way and that no child will be offered a knowingly inferior treatment.

    FC

    http://journals.lww.com/jpgn/Abstract/2016/01000/Use_of_Placebo_in_Pediatric_Inflammatory_Bowel.35.aspx