Postmarketing trials and pediatric device approvals

Pediatrics

Volumn 133, Issue 5, 2014, Pages

  Hwang, Thomas J ^a,b   Kesselheim, Aaron S ^b   Bourgeois, Florence T ^c,d  

^a HARVARD UNIVERSITY   (United States)

^b BRIGHAM AND WOMEN S HOSPITAL   (United States)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Children; Clinical trial; Device safety; Medical device; Pediatric; Postmarketing

Indexed keywords

ADULT; ARTICLE; CARDIOVASCULAR EQUIPMENT; CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); DEVICE APPROVAL; DEVICE SAFETY; DIGESTIVE THERAPEUTIC DEVICE; EVIDENCE BASED MEDICINE; FOOD AND DRUG ADMINISTRATION; GYNECOLOGICAL AND OBSTETRIC EQUIPMENT; HUMAN; INTERMETHOD COMPARISON; MARKETING; PATIENT SAFETY; PRIORITY JOURNAL; TREATMENT INDICATION; UROLOGICAL EQUIPMENT; ADOLESCENT; CHILD; COHORT ANALYSIS; COMPASSIONATE USE; FEMALE; LEGISLATION AND JURISPRUDENCE; MALE; MIDDLE AGED; POSTMARKETING SURVEILLANCE; RANDOMIZED CONTROLLED TRIAL (TOPIC); UNITED STATES; YOUNG ADULT;

ADOLESCENT; ADULT; CHILD; COHORT STUDIES; COMPASSIONATE USE TRIALS; DEVICE APPROVAL; EQUIPMENT SAFETY; FEMALE; HUMANS; MALE; MIDDLE AGED; PRODUCT SURVEILLANCE, POSTMARKETING; RANDOMIZED CONTROLLED TRIALS AS TOPIC; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION; YOUNG ADULT;

EID: 84899845894     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2013-3348     Document Type: Article

References (32)

1. US Food and Drug Administration. FY 2014 Congressional budget request: justification of estimates for appropriations committees. Available at: www.fda.gov/downloads/ AboutFDA/ReportsManualsForms/Reports/ BudgetReports/UCM349751.pdf. Accessed August 6, 2013
2. Beekman RH III, Duncan BW, Hagler DJ, et al; Workgroup on Pediatric Cardiac Devices, Section on Cardiology and Cardiac Surgery, American Academy of Pediatrics. Pathways to approval of pediatric cardiac devices in the United States: challenges and solutions. Pediatrics. 2009;124(1). Available at: www.pediatrics.org/cgi/content/full/124/ 1/e155
3. Cecchin F, Atallah J, Walsh EP, Triedman JK, Alexander ME, Berul CI. Lead extraction in pediatric and congenital heart disease patients. Circ Arrhythm Electrophysiol. 2010;3(5):437-444
4. Atallah J, Erickson CC, Cecchin F, et al; Pediatric and Congenital Electrophysiology Society (PACES). Multi-institutional study of implantable defibrillator lead performance in children and young adults: results of the Pediatric Lead Extractability and Survival Evaluation (PLEASE) study. Circulation. 2013;127(24):2393-2402
5. McElhinney DB, Bergersen L, Marshall AC. In situ fracture of stents implanted for relief of pulmonary arterial stenosis in patients with congenitally malformed hearts. Cardiol Young. 2008;18(4):405-414
6. Breinholt JP, Nugent AW, Law MA, Justino H, Mullins CE, Ing FF. Stent fractures in congenital heart disease. Catheter Cardiovasc Interv. 2008;72(7):977-982
7. Sutherell JS, Hirsch R, Beekman RH III. Pediatric interventional cardiology in the United States is dependent on the off-label use of medical devices. Congenit Heart Dis. 2010;5(1):2-7
8. Kramer DB, Xu S, Kesselheim AS. Regulation of medical devices in the United States and European Union. N Engl J Med. 2012;366(9): 848-855
9. Kaplan AV, Baim DS, Smith JJ, et al. Medical device development: from prototype to regulatory approval. Circulation. 2004;109 (25):3068-3072
10. Medical Device User Fee and Modernization Act of 2002, Pub L No. 107-250
11. Pediatric Medical Device Safety and Improvement Act of 2007, Pub L No. 110-85
12. US Food and Drug Administration. Pediatric report to Congress, FY 2008-2011. Available at: www.fda.gov/MedicalDevices/ ProductsandMedicalProcedures/ ucm135104.htm. Accessed July 18, 2013
13. US Food and Drug Administration. Premarket approval. Available at: www.accessdata.fda. gov/scripts/cdrh/cfdocs/cfpma/pma.cfm. Accessed July 18, 2013
14. US Food and Drug Administration. Listing of CDRH humanitarian device exemptions. Available at: www.fda.gov/medicaldevices/ productsandmedicalprocedures/deviceapprovalsandclearances/ hdeapprovals/ucm 161827.htm. Accessed July 18, 2013
15. Federal Food, Drug, and Cosmetic Act, x515(a)
16. US Food and Drug Administration. Postapproval studies. Available at: www.accessdata. fda.gov/scripts/cdrh/cfdocs/cfpma/pma-pas. cfm. Accessed July 18, 2013
17. Sapirstein JS. FDA Pediatric Advisory Committee. Medtronic Melody Transcatheter Pulmonary Valve (TPV) presentation. Available at: www.fda.gov/downloads/Advisory- Committees/CommitteesMeetingMaterials/ PediatricAdvisoryCommittee/UCM319365.pdf
18. Ulrich LC, Joseph FD, Lewis DY, Koenig RL. FDA's pediatric device consortia: national program fosters pediatric medical device development. Pediatrics. 2013;131(5):981-985
19. US Food and Drug Administration. Draft guidance: providing information about pediatric uses of medical devices under section 515A of the Federal Food, Drug, and Cosmetic Act. Available at: www.fda.gov/downloads/ MedicalDevices/DeviceRegulationandGuidance/ GuidanceDocuments/UCM339465.pdf. Accessed July 2, 2013
20. US Food and Drug Administration. "Proposed rule: pediatric uses of devices; requirement for submission of information on pediatric subpopulations that suffer from a disease or condition that a device is intended to treat, diagnose, or cure (finalized in January 10, 2014)". Fed Regist. 2013; 78(33):11612-11617
21. Sibbald B, Roland M. Understanding controlled trials: why are randomised controlled trials important? BMJ. 1998;316(7126): 201
22. Garber AM. Modernizing device regulation. N Engl J Med. 2010;362(13):1161-1163
23. Dhruva SS, Bero LA, Redberg RF. Strength of study evidence examined by the FDA in premarket approval of cardiovascular devices. JAMA. 2009;302(24):2679-2685
24. Dolcimascolo F. Pediatric cardiac devices - an FDA pediatrician's perspective of the challenges and potential solutions. J Cardiovasc Transl Res. 2009;2(2):147-149
25. Hwang TJ, Carpenter D, Kesselheim AS. Assessment of US pathway for approving medical devices for rare conditions. BMJ. 2014;348:g217
26. Martinez-Castaldi C, Silverstein M, Bauchner H. Child versus adult research: the gap in high-quality study design. Pediatrics. 2008; 122(1):52-57
27. Caldwell PH, Murphy SB, Butow PN, Craig JC. Clinical trials in children. Lancet. 2004; 364(9436):803-811
28. Steinbrook R. Testing medications in children. N Engl J Med. 2002;347(18):1462-1470
29. Campbell B, Stainthorpe AC, Longson CM. How can we get high quality routine data to monitor the safety of devices and procedures? BMJ. 2013;346:f2782
30. US Food and Drug Administration. Strengthening our national system for postmarket device surveillance. 2012. Available at: www. fda.gov/AboutFDA/ CentersOffices/OfficeofMedicalProductsandTobacco/ CDRH/CDRHReports/ ucm301912.htm. Accessed July 2, 2013
31. Li JS, Eisenstein EL, Grabowski HG, et al. Economic return of clinical trials performed under the pediatric exclusivity program. JAMA. 2007;297(5):480-488
32. Federal Register (codified as 21 CFR xx814.104, 814.122) Development