To address the scarcity of medical devices that are designed specifically for the pediatric population, FDA convened a public meeting and workshop August 13–14, 2018 to delve into the financial, regulatory, and design constraints associated with this challenging endeavor. Speakers at the workshop included FDA regulators, sponsors, clinicians, and members of the American Academy of Pediatrics. The prevailing message from the workshop was that while there are many barriers to the development of pediatric-specific devices, FDA and community in attendance is committed to developing innovative pathways and solutions to promote bringing pediatric devices to market.
1. Adult medical devices are often used off-label for pediatric patients
Both sponsors and FDA recognize that the use of adult devices for children is not ideal, but because of the many barriers to pediatric device development, off-label use is necessary and will continue until sufficient pediatric specific devices are available. Development of pediatric medical devices lags behind adult devices by five to ten years.
2. The development of pediatric medical devices is difficult for sponsors
Attendees to the workshop had meaningful discussion on how to expand the medical device market for pediatrics. However, presenters outlined the multitude of challenges faced by sponsors including the very small pediatric medical device market, evolving pediatric patient’s physiological development, and the great difficulty of running clinical trials in a pediatric population.
3. The pediatric medical device community is interested in incentives to develop pediatric medical devices
Dr. Lynne Yao, Director, Division of Pediatric and Maternal Health, Center for Drug Evaluation and Research, FDA, presented on current incentives and legislation in place for the development of pediatric-specific drugs which could be translated into an incentive system for development of pediatric-specific medical devices. Throughout the workshop, sponsor speakers emphasized the need for incentives to drive the development of pediatric-specific medical devices.
4. FDA provides data leveraging options to extrapolate adult data or use quality real-world data
The Pediatric Extrapolation for Devices (PEDs) team within CDRH works specifically to evaluate submissions for pediatric devices and highlighted that outside of randomized controlled trials for new devices, CDRH encourages extrapolation of known adult data to determine the safety and effectiveness of a device for pediatric use. FDA also highlighted their interest in evaluation of real-world data (e.g., clinical data from off-label use and data published in literature) to determine safety and effectiveness of new devices.
What about Human Factors?
Of particular concern is that the concept of human factors or device usability was never discussed at the workshop. It seems that the field of pediatric medical devices is still navigating ways to even develop new devices, so evaluation of device usability may not yet be a high priority. However, there are many opportunities to optimize designs and better serve pediatric patients with the application of human factors during the design process. It also is apparent that some sponsors are already focusing on usability for pediatric patients. For example, in August 2018, Vectura released study findings from a phase 2 trial evaluating the ability of young children and toddlers with asthma to use its nebulized inhaler. Vectura’s study enrolled 40 children, 1 to 4 years old, to examine their ability to use Vectura’s inhaler to deliver a fixed-dose formulation of the corticosteroid budesonide. According to the company, the study showed 90% of children age 2 and up were able to effectively use the mouthpiece which demonstrated tight control of breathing patterns with no loss of medication. Because of these findings, Vectura is also developing a facemask for children under 2. Usability studies are an important part to ensuring safety and effectiveness for devices which require primary use by children. Although it was not discussed at the workshop, it is important for sponsors to take the necessary steps to integrate human factors evaluations into the design process for new devices to help develop the safest products for end users (and not only rely on practices such as extrapolation of adult use data).
Important Human Factors Considerations for Pediatric Devices
1. The development of pediatric devices can benefit from human factors considerations
Because FDA’s workshop did not discuss usability, it seems sponsors may be missing the opportunity to integrate human factors into the design process in order to optimize use related safety and effectiveness. There is extensive scientific findings which highlight the developmental process of children and outline cognitive and motor stages of development that would be paramount to consider for this special and important user population.
2. It is vital that sponsors consider human factors for pediatric devices as they make plans for development of safe and effective devices
As demonstrated by the Vectura study, sponsors are aware that usability by children can be different than adult users. Sponsors should consider the necessity of conducting human factors evaluations with pediatric users early during the development process, such as when identifying a regulatory pathway and device development plan. It is also important to consider the specialized skills required to perform usability studies with children, as this user population presents different challenges to their adult counterparts.
3. Extrapolation of adult usability data may not sufficiently demonstrate use related safety and efficacy of devices for children
While the PEDS team within the CDRH highlighted their acceptance of extrapolated clinical adult data as a potential substitute to randomized controlled trial data, it must be highlighted that children are different from adults. Caution must be applied in assuming that adult usability data will be representative or applicable to children. Per Applying Human Factors and Usability Engineering to Medical Devices that was published by CDRH, FDA on February 3, 2016, “If intended users include a pediatric population, the testing should include a group of representative pediatric users; when a device is intended to be used by both pediatric and adult users, FDA views these as distinct populations.” In the same guidance, FDA highlights the following characteristics that impact the user’s ability to operate the medical device: physical size, strength, dexterity, coordination, sensory abilities, cognitive abilities, literacy, level of education and motivation to learn a new device. This is a non-exhaustive list of the numerous characteristics that likely will be different for pediatric and adult users, which will directly impact usability.
For sponsors committed to creating safe and effective medical devices for pediatric use, they should prioritize working with human factors professionals who have specific experience in medical devices and in navigating FDA regulatory pathways. Tools such as early expert/heuristic reviews in the design process can significantly improve device usability for children, and human factors formative and validation studies will help optimize the design and prepare evidence for sponsors and FDA to know pediatric devices are safe and effective for end users.
Agilis is a human factors partner for the global medical market. The firm helps new and established companies achieve successful human factors submissions while navigating the complex global regulatory landscape, resulting in safe and effective medical devices and combination products.