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In Small Doses

Meeting the need for medical devices for a paediatric population is a far from straightforward proposition. Following a string of legislative changes in the US, the Paediatric Trials Network is well-placed to use strategic partnerships to promote the development of medical devices for children.

In an attempt to save a life, a paediatrician places an adult coronary artery stent in a newborn infant’s collapsed airway.The ‘one size fits all’ tracheostomy tube is too long and the doctor is forced to trim it off and file the end smooth.The baby needs a ventricular assisting device to live, but it is unavailable – what should be done then? This is a typical example of the challenges paediatricians and surgeons face every day as they strive to meet the needs of the children entrusted to their care.

Unmet Needs and Major Barriers

Although difficult to substantiate, opinions prevail that there are many unmet needs for medical devices utilised in the care of the paediatric population. In April 2004, a public law was passed in the US to initiate a process whereby these perceived needs could be systematically addressed (1).This law required the Secretary of Health and Human Services (HHS) to submit a report on the barriers to the availability of devices intended for the treatment or diagnosis of diseases and conditions that affect children. Sponsored by the US Senate Health, Education, Labor and Pensions Committee (HELP), and hosted by the American Academy of Pediatrics, the Elizabeth Glaser Paediatric AIDS Foundation, the National Organization for Rare Disorders, and the National Association of Children’s Hospitals, a series of meetings of stakeholders to discuss the development and availability of paediatric devices began in the same year on 28 June. Representatives from the clinical community, medical device manufacturers and trade organisations, federal agencies, biomedical technical societies, academia and paediatric patient advocacy organisations participated in the meeting.Needs were identified as fitting into one of three categories:

  • No device is available to treat a paediatric indication
  • Off-label use of an adult device is being used to satisfy the need
  • A paediatric device is available but it does not meet the specific needs of the particular population

The stakeholders concluded that there was no question that the paediatric population – ranging from neonates to adolescents – needed devices and that the focal point was how those needs could be met (2). Paediatric device development presented challenges beyond devices for adults. These included: the variation in size; continued growth and development; the diversity of the device industry; the relatively short life cycle of devices as opposed to pharmaceuticals; R&D costs for devices would be limited to only paediatric applications; legal liability; intellectual property concerns; and reimbursement issues. Additional requirements for clinical trials leading to regulatory clearance were also of concern.Major barriers to paediatric device development were noted to include:

  • A relatively smaller market for paediatric devices
  • The costs involved in conducting paediatric studies, especially for low volume disorders
  • The relatively short life span of a device generation and the ability of competitors to design around patents
  • The lack of information about paediatric needs
  • The lack of extensive collaborative networks of paediatric investigators, academic institutions, government and industry

Legislative Changes

A legislative response was created by Senate HELP committee staff, and concluded with the passage of the Title III Paediatric Medical Device Safety and Improvement Act of 2007. In particular, this legislation included a modification to the Humanitarian Device Exemption, allowing the use of up to 4,000 devices per year, and permitting companies to make profits on these devices for children. It also encouraged paediatric medical device development at NIH through the examination of its current status of federally funded paediatric medical device research, and identifying of any gaps in such research, revisions in its agenda for improving paediatric medical device development, FDA clearance or approval of paediatric medical devices, and evaluation of its short- and long-term safety and effectiveness of paediatric medical devices.

The Office of Orphan Products Development was tasked to develop the Paediatric Device Consortia Grant Program in order to support non-profit consortia in promoting paediatric device development (3).The goal for the consortia was that it would facilitate the development, production and distribution of paediatric medical devices by:

  • Encouraging innovation and connecting qualified individuals with paediatric device ideas with potential manufacturers
  • Mentoring and managing paediatric device projects through the development process, including product identification, prototype design, device development and marketing
  • Connecting innovators and physicians to existing federal and non-federal resources
  • Assessing the scientific and medical merit of proposed paediatric device projects
  • Providing assistance and advice as needed on business development, personnel training, prototype development and postmarketing needs

The Office of Orphan Products Development solicited grant applications from non-profit consortia and the recipients were the Michigan Paediatric Device Consortium, the Paediatric Cardiovascular Device Consortium (Boston, MA & Atlanta, GA), the University of California, San Francisco Paediatric Device Consortium, and the MISTRAL Device Consortium (Palo Alto, CA). Subsequently, the Michigan Paediatric Device Consortium partnered with the Paediatric Medical Device Institute (Roanoke,VA), and the MISTRAL Device Consortium entered into a one-year partnership with the Institute for Paediatric Innovation (Boston, MA). Applicants were judged on the organisational capacity of their proposed consortiums to exert a sustained, powerful influence on the field of paediatric device development, as well as an assessment of potential device projects. Those who received funding scored best in their unique abilities to serve as a national platform to advance the development of paediatric medical devices, while supporting device projects whose outcomes could have a significant impact on the practice of paediatric medicine.

These consortia have supported 96 projects falling into most of the developmental categories depicted in the FDA Center for Devices and Radiological Health’s Total Product Life Cycle as shown in Figure 1. Seventy-five of the 96 projects are currently classified as ‘active’ projects with a key metric for success of movement along the life cycle, such as from prototype to preclinical. Few have reached the market, but consortia personnel are working closely with the FDA to facilitate and accelerate device development through access to resources.

Paediatric Trials Network

In April 2010, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) published a solicitation of proposals for a contract to support clinical activities for a newly evolving Paediatric Trials Network (PTN) (5). This $95 million effort was established to provide the requisite personnel, facilities and services to establish, conduct and support paediatric clinical trials and related services including: expert consultation; paediatric clinical pharmacology; product formulation; drug distribution; and device development.  The contract was awarded to the Duke Clinical Research Institute in September 2010, but involves an international network and infrastructure. PTN is studying product formulation, drug dose, efficacy, safety and device development.

Data collected from the trials will help inform paediatric drug labelling and provide regulators, paediatricians and researchers with current information on how children respond to medication.The primary goal is to generate the data necessary to produce evidence-based clinical practice guidelines.The PTN has a strategic partnership with the NICHD to effectively study therapeutics of critical need, priority drugs, and therapeutic and diagnostic devices in children.The Duke Clinical Research Institute (DCRI)-PTN comprises six core components (see Figure 2) with broad oversight by the Network Steering Committee.The six PTN team cores are pharmacometrics, clinical pharmacology, clinical operations, devices, safety and ethics, and mentorship.The backbone of each core comprises investigators and operations staff with a long and productive history of working together through NICHD-sponsored grants, networks and contracts.

The PTN will conduct 16 trials over the next five years with research spanning a variety of therapeutic areas, including cardiovascular diseases, cancer, infectious diseases, gastroenterology, respiratory diseases, neonatology and medical devices.Trials involving several medications and one medical device are currently underway. A New Year’s baby started the year off for the Paediatric Trials Network, with the first subject enrolled in the metronidazole trial on 1 January 2011, a mere 93 days after funding was approved.

References

  1. The Medical Devices Technical Corrections Act of 2004 (MDTCA), Public Law 108-214, April 1, 2004
  2. Report to Congress: Barriers to the Availability of Medical Devices Intended for the Treatment or Diagnosis of Diseases and Conditions that Affect Children, US Department of Health and Human Services, Food and Drug Administration, October 2004
  3. Paediatric Device Consortia Grant Program (P50), Request For Applications (RFA) Number: RFA-FD-09-007
  4. Feigal D, CDRH Vision, total product life cycle (unpublished), available at www.fda.gov/ohrms/dockets/ac/01/slides/3799s1_11_Feigal/sld003.htm, accessed 7 August 2011
  5. Paediatric Trials Network: NIH-NICHDCRMC- 2010-02, Department of Health and Human Services, National Institutes of Health, National Institute of Child Health and Human Development

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Andre Muelenaer holds academic appointments at Virginia Tech Carilion School of Medicine, Virginia Tech, and the University of Virginia. He is Section Chief, Paediatric Pulmonology/Allergy, at the Carilion Clinic Children’s Hospital. His BS and MS are from Virginia Tech, and MD is from Eastern Virginia Medical School. He did his paediatric residency at William Beaumont Army Medical Center and his fellowship in paediatric pulmonology was at the University of North Carolina. Andre is cofounder of the non-profit Paediatric Medical Device Institute. He maintains a telemedical practice in North Carolina, and serves as part-time medical director for several biotechnology companies in Virginia. Email: aamuelenaer@carilionclinic.org

Alfred L Wicks received his PhD from Michigan Tech University. Since joining the faculty of the Mechanical Engineering Department at Virginia Tech in 1986, Alfred has developed numerous techniques for applying scanning laser technology to dynamics measurements. Alfred is the co-founder of the Pediatric Medical Device Institute (PMDI), a non-profit organisation focusing on the development of delivering paediatric devices to the bedside. He has also served as Technical Chairman of the International Modal Analysis Conference for the last 20 years. Email: awicks@vt.edu

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