Courtesy of the US National Institutes of Health

The number of children diagnosed with pediatric type 1 diabetes continues to grow across the globe. In response, throughout the last two decades new insulins and devices have been developed, tested, and approved for use in children. However, the current reality is that most children and adolescents do not reach the targets for metabolic control set by organizations such as the International Society for Pediatric and Adolescent Diabetes and the American Diabetes Association.

One complicating factor is that we cannot approach diabetes drug development among children the same way we do for adults. In the world of medicine, children are not simply ‘little adults.’ For example, pediatric patients may metabolize a drug differently than adults and they may experience side effects not relevant to adult patients, such as impact on growth or pubertal development. It is therefore very important that pediatric patients be included in development programs for drugs that will be prescribed to children, and there is legislation in many countries around the world addressing this issue. However, many challenges still exist in pediatric drug development.

Recently, a group of experts including Quintiles published a paper in the peer-reviewed journal Diabetes Care exploring different approaches to pediatric type 1 diabetes drug development and regulatory approval. While the paper focuses on the U.S. Food and Drug Administration, many aspects of the analysis are widely applicable in other countries too. 

Ten actionable suggestions were offered in an effort to improve pediatric type 1 diabetes drug development, including:

  • Involve patients to a great degree in the design, and execution, of clinical trials. As examples, the Center for Information & Study on Clinical Research Participation has helped incorporate patient advisory board panels into clinical trials to input on study protocols, communications, and conduct. Secondly, organizations such as PatientsLikeMe are creating new patient-driven research strategies by conducting studies using voluntarily submitted patient data: results are shared with healthcare and life sciences companies.
  • Ensure that differences in pediatric and adult study populations are reflected in the design, timing, and execution of trials. We already noted that children are not simply smaller versions of adults.  There are many differences including potential differences in the way drugs are processed in the body, the acceptability of wearing various medical devices, and ability to adhere to complex study protocols.
  • Develop a provisional or adaptive approval system. The traditional drug approval paradigm is binary: a drug goes from not being approved to being fully approved.  In contrast, an adaptive approval system is based on stepwise learning, and moving towards full approval in an iterative manner. Such a system could reduce the time and costs to market, potentially resulting in improved access to much-needed medicines.
  • Encourage different companies developing drugs to work together in a pre-competitive, collaborative manner. Establishing a clinical research infrastructure that allows sponsors to work together in a pre-competitive paradigm would help each company to bring new medicines to the patients that need them faster.
There is growing awareness and acceptance of the fact that multiple stakeholders — biopharmaceutical companies, regulatory agencies, physicians, patient advocacy groups, and patients themselves — can work much more closely to address unmet medical needs in this area.  This progress is very encouraging, offering considerable benefits to patients.