Postmarketing Trials and Pediatric Device Approvals

Hwang, Thomas J.; Kesselheim, Aaron S.; Bourgeois, Florence T.

doi:10.1542/peds.2013-3348

Cited by 46 publications

(41 citation statements)

References 19 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…7 As one part of this approach, the FDA can require additional clinical studies as a condition of PMA approval through its postapproval studies (PAS) program. 8 However, PAS may often be small, 9 delayed, 9,10 or not generalizable 5,11 ; in addition, only one-quarter of PAS required by the FDA between 2005 and 2011 were completed. 9 Beyond FDA-required postmarket studies, complementary sources of evidence may be generated through studies initiated by manufacturers or independent investigators, although manufacturers may conduct postmarket clinical studies primarily to comply with regulatory requirements.…”

Section: -5mentioning

confidence: 99%

Characteristics of Clinical Studies Conducted Over the Total Product Life Cycle of High-Risk Therapeutic Medical Devices Receiving FDA Premarket Approval in 2010 and 2011

Rathi

Krumholz

Masoudi

et al. 2015

JAMA

115

View full text Add to dashboard Cite

Administration (FDA) approves high-risk medical devices, those that support or sustain human life or present potential unreasonable risk to patients, via the Premarket Approval (PMA) pathway. The generation of clinical evidence to understand device safety and effectiveness is shifting from predominantly premarket to continual study throughout the total product life cycle.OBJECTIVE To characterize the clinical evidence generated for high-risk therapeutic devices over the total product life cycle. DESIGN AND SETTINGAll clinical studies of high-risk therapeutic devices receiving initial market approval via the PMA pathway in 2010 and 2011 identified through ClinicalTrials.gov and publicly available FDA documents as of October 2014. MAIN OUTCOMES AND MEASURESStudies were characterized by type (pivotal, studies that served as the basis of FDA approval; FDA-required postapproval studies [PAS]; or manufacturer/investigator-initiated); premarket or postmarket; status (completed, ongoing, or terminated/unknown); and design features, including enrollment, comparator, and longest duration of primary effectiveness end point follow-up. RESULTSIn 2010 and 2011, 28 high-risk therapeutic devices received initial marketing approval via the PMA pathway. We identified 286 clinical studies of these devices: 82 (28.7%) premarket and 204 (71.3%) postmarket, among which there were 52 (18.2%) nonpivotal premarket studies, 30 (10.5%) pivotal premarket studies, 33 (11.5%) FDA-required PAS, and 171 (59.8%) manufacturer/investigator-initiated postmarket studies. Six of 33 (18.2%) PAS and 20 of 171 (11.7%) manufacturer/investigator-initiated postmarket studies were reported as completed. No postmarket studies were identified for 5 (17.9%) devices; 3 or fewer were identified for 13 (46.4%) devices overall. Median enrollment was 65 patients (interquartile range [IQR], 25-111), 241 patients (IQR, 147-415), 222 patients (IQR, 119-640), and 250 patients (IQR, 60-800) for nonpivotal premarket, pivotal, FDA-required PAS, and manufacturer/investigator-initiated postmarket studies, respectively. Approximately half of all studies used no comparator (pivotal: 13/30 [43.3%]; completed postmarket: 16/26 [61.5%]; ongoing postmarket: 70/153 [45.8%]). Median duration of primary effectiveness end point follow-up was 3.0 months (IQR, 3.0-12.0), 9.0 months (IQR, 0.3-12.0), and 12.0 months (IQR, 7.0-24.0) for pivotal, completed postmarket, and ongoing postmarket studies, respectively.CONCLUSIONS AND RELEVANCE Among high-risk therapeutic devices approved via the FDA PMA pathway, total product life cycle evidence generation varied in both the number and quality of premarket and postmarket studies, with approximately 13% of initiated postmarket studies completed between 3 and 5 years after FDA approval.

show abstract

Section: -5mentioning

confidence: 99%

Characteristics of Clinical Studies Conducted Over the Total Product Life Cycle of High-Risk Therapeutic Medical Devices Receiving FDA Premarket Approval in 2010 and 2011

Rathi

Krumholz

Masoudi

et al. 2015

JAMA

115

View full text Add to dashboard Cite

show abstract

“…In certain premarket studies, safety and effectiveness of medical devices used in adolescents have been extrapolated from adult data. Hwang et al 3 found that most of the recently approved pediatric devices have been approved on the basis of adult studies. There are unique issues that should be considered in device use for adolescents.…”

Section: Medical Device Study Considerations For Adolescentsmentioning

confidence: 99%

Medical Devices and Adolescents

Samuels‐Reid

Cope

2016

JAMA Pediatr

View full text Add to dashboard Cite

Use of medical devices in adolescents presents many challenges. The Center for Devices and Radiological Health of the US Food and Drug Administration (FDA) defines the pediatric population as birth through age 21 years, with the adolescent age group defined as ages 12 through 21 years. 1 Following the Food and Drug Administration Amendments Act of 2007, the FDA implemented a requirement that medical device manufacturers provide readily available information in certain premarket applications on pediatric patients who have the disease or condition that the device is intended to treat, diagnose, or cure, even if the device is intended for adult use. 2 It is imperative to ensure appropriate focus on adolescents. The adolescent population is not monolithic. It has unique subgroups spanning different age groups with various stages of development, and it includes gender-specific variations-all important factors for manufacturers, investigators, and clinicians to consider. In many cases adolescents are treated as young adults, and the fact that they are still part of the pediatric population is often lost. There are special issues that should be considered in any treatment plan for adolescents that involves medical devices.

show abstract

“…In other words, the projected profitability from pediatric device development is outstripped by the resources required to bring a device to market. As such, most devices used in pediatrics have not been rigorously studied in pediatric patients (10) and many unmet needs exist in this population. Programs and initiatives designed to help innovators address urgent unmet clinical needs such as those for pediatric patients are discussed below.…”

Section: The Unmet Clinical Needmentioning

confidence: 99%

Proceedings from Heart Rhythm Society’s emerging technologies forum, Boston, MA, May 12, 2015

et al. 2016

View full text Add to dashboard Cite

Physicians are in an excellent position to significantly contribute to medical device innovation, but the process of bringing an idea to the bedside is complex. To begin to address these perceived barriers, the Heart Rhythm Society (HRS) convened a forum of stakeholders in medical device innovation in conjunction with the 2015 HRS Scientific Sessions. The forum facilitated open discussion about medical device innovation, including obstacles to physician involvement and possible solutions. This report is based on the themes that emerged. First, physician innovators must take an organized approach to identifying unmet clinical needs and potential solutions. Second, extensive funds, usually secured through solicitation for investment, are often required to achieve meaningful progress developing an idea into a device. Third, planning for regulatory requirements of the U.S. Food & Drug Administration (FDA) and Centers for Medicare and Medicaid Services (CMS) is essential. In addition to these issues, intellectual property and overall trends in health care, including international markets, are critically relevant considerations for the physician innovator. Importantly, there are a number of ways in which professional societies can assist physician innovators to navigate the complex medical device innovation landscape, bring clinically meaningful devices to market more quickly, and ultimately improve patient care. These efforts include facilitating interaction between potential collaborators through scientific meetings and other gatherings; collecting, evaluating, and disseminating state-of-the-art scientific information; and representing the interests of members in interactions with regulators and policy makers.

show abstract

Postmarketing Trials and Pediatric Device Approvals

Cited by 46 publications

References 19 publications

Characteristics of Clinical Studies Conducted Over the Total Product Life Cycle of High-Risk Therapeutic Medical Devices Receiving FDA Premarket Approval in 2010 and 2011

Characteristics of Clinical Studies Conducted Over the Total Product Life Cycle of High-Risk Therapeutic Medical Devices Receiving FDA Premarket Approval in 2010 and 2011

Medical Devices and Adolescents

Proceedings from Heart Rhythm Society’s emerging technologies forum, Boston, MA, May 12, 2015

Contact Info

Product

Resources

About