Assessment of US pathway for approving medical devices for rare conditions

BMJ (Online)

Volumn 348, Issue , 2014, Pages

  Hwang, Thomas J ^a,b   Carpenter, Daniel ^a   Kesselheim, Aaron S ^b  

^a HARVARD UNIVERSITY   (United States)

^b BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; DEVICE APPROVAL; DEVICE SAFETY; HEALTH CARE POLICY; HUMAN; INTERMETHOD COMPARISON; MEDICAL DEVICE; MEDICAL DEVICE REGULATION; PRIORITY JOURNAL; PUBLIC HEALTH; RISK ASSESSMENT; UNITED STATES;

CONSUMER PRODUCT SAFETY; DEVICE APPROVAL; GOVERNMENT REGULATION; GUIDELINES AS TOPIC; HUMANS; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84892737444     PISSN: None     EISSN: 17561833     Source Type: Journal    
DOI: 10.1136/bmj.g217     Document Type: Article

References (43)

1. US Food and Drug Administration. Humanitarian device exemption: overview. www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/ PremarketSubmissions/HumanitarianDeviceExemption/.
2. Kramer DB, Xu S, Kesselheim AS. Regulation of medical devices in the United States and European Union. N Engl J Med 2012;366:848-55.
3. Heneghan C. The saga of Poly Implant Prosthese breast implants. BMJ 2012;344:e306.
4. Cohen D. How a fake hip showed up failings in European device regulation. BMJ 2012;345:e7090.
5. Cohen D. Faulty hip implant shows up failings of EU regulation. BMJ 2012;345:e7163.
6. European Commission. Regulation of the European Parliament and of the Council on medical devices, and amending directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC) No 1223/2009. EC, 2012. http://ec.europa.eu/ health/medical-devices/files/revision-docs/proposal-2012-542-en.pdf.
7. Storz-Pfennig P, Schmedders M, Dettloff M. Trials are needed before new devices are used in routine practice in Europe. BMJ 2013;346:f1646.
8. Eikermann M, Gluud C, Perleth M, Wild C, Sauerland S, Gutierrez-Ibarluzea I, et al. Europe needs a central, transparent, and evidence based regulation process for devices. BMJ 2013;346:f2771.
9. 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.
10. European Parliament. Draft report on the proposal for a regulation of the European parliament and of the council on medical devices, and amending directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC) No 1223/2009. EP, 2013. http://bit.ly/1150IMJ.
11. Eucomed. No surprises in draft report on medical devices from Rapporteur Roth-Behrendt. Press release, 19 April 2013. www.eucomed.org/newsroom/108/57/No- surprises-in-draftreport-on-medical-devices-from-Rapporteur-Roth-Behrendt.
12. Cohen D. Devices and desires: industry fights toughening of medical device regulation in Europe. BMJ 2013;347:f6204.
13. Eucomed. €7.5 billion for unnecessary measures will be a blow to medical device innovation in Europe. Press release, 12 September 2013. www.eucomed.org/newsroom/115/57/17-5-billion-for-unnecessary-measures-will-be-a- blow-to-medical-device-innovationin-Europe.
14. Food and Drug Administration Safety and Innovation Act (FDASIA), Pub. L. 112-144. 2012.
15. Kaplan AV, Baim DS, Smith JJ, Feigal DA, Simons M, Jefferys D, Fogarty TJ, Kuntz RE, Leon MB. Medical device development: from prototype to regulatory approval. Circulation 2004;109:3068-72.
16. Food and Drug Administration. Summary of safety and probable benefit: King's College Hospital (KCH) fetal bladder drainage catheter (H970001). FDA, 1997.
17. Food and Drug Administration. Summary of safety and probable benefit: Ascension PIP (H010005). FDA, 2002.
18. Food and Drug Administration. Summary of safety and probable benefit: Medtronic Activa dystonia therapy (H020007). FDA, 2003.
19. Food and Drug Administration. Summary of safety and probable benefit: Cardio SEAL septal occlusion system (H990011). FDA, 2000.
20. Food and Drug Administration. Summary of safety and probable benefit: VOCARE bladder system (H980008). FDA, 1999.
21. US Food and Drug Administration. Listing of CDRH humanitarian device exemptions. www.fda.gov/medicaldevices/productsandmedicalprocedures/ deviceapprovalsandclearances/hdeapprovals/ucm161827.htm.
22. US Food and Drug Administration. Summary of safety and probable benefit: EXCOR Pediatric Ventricular Assist Device (H100004). FDA, 2011.
23. Dhruva SS, Bero LA, Redberg RF. Strength of study evidence examined by the FDA in premarket approval of cardiovascular devices. JAMA 2009;302:2679-85.
24. Chen CE, Dhruva SS, Redberg RF. Inclusion of comparative effectiveness data in high-risk cardiovascular device studies at the time of premarket approval. JAMA 2012;308:1740-2.
25. Pricewaterhouse Coopers. Medical technology innovation scorecard: the race for global leadership. 2011. http://pwchealth.com/cgi-local/hregister.cgi? link=reg/innovationscorecard. pdf.
26. Center for Thoracic Insufficiency Syndrome, Children's Hospital of Philadelphia. Frequently asked questions about the vertical expandable prosthetic titanium rib (VEPTR). www.chop.edu/service/thoracic-insufficiency- syndrome-center/veptr-faqs.html.
27. Gadepalli SK, Hirschl RB, Tsai WC, Caird MS, Vanderhave KL, Strouse PJ, et al. Vertical expandable prosthetic titanium rib device insertion: does it improve pulmonary function? J Pediatr Surg 2011;46:77-80.
28. Groenefeld B, Hell AK. Ossifications after VEPTR (vertical expandable prosthetic titanium rib) treatment in children with thoracic insufficiency syndrome and scoliosis. Spine (Phila Pa 1976) 2013;38:E819-23.
29. Emans JB, Caubet JF, Ordonez CL, Lee EY, Ciarlo M. The treatment of spine and chest wall deformities with fused ribs by expansion thoracostomy and insertion of vertical expandable prosthetic titanium rib: growth of thoracic spine and improvement of lung volumes. Spine (Phila Pa 1976) 2005;30(suppl):S58-68. (Pubitemid 41292204)
30. Campbell RM Jr. VEPTR: past experience and the future of VEPTR principles. Eur Spine J 2013;22(suppl 2):S106-17.
31. Bernad DM. Humanitarian use device and humanitarian device exemption regulatory programs: pros and cons. Expert Rev Med Devices 2009;6:137-45.
32. Fins JJ, Mayberg HS, Nuttin B, Kubu CS, Galert T, Sturm V, et al. Misuse of the FDA's humanitarian device exemption in deep brain stimulation for obsessive-compulsive disorder. Health Aff (Millwood) 2011;30:302-11.
33. Eydelman MB, Chen EA. The FDA's humanitarian device exemption program. Health Aff (Millwood) 2011;30:1210-2.
34. Chimowitz MI, Lynn MJ, Derdeyn CP, Turan TN, Fiorella D, Lane BF, et al. Stenting versus aggressive medical therapy for intracranial arterial stenosis. N Engl J Med 2011;365:993-1003.
35. US Food and Drug Administration. Stryker Wingspan stent system: safety communication-narrowed indications for use. FDA, 2012. www.fda.gov/Safety/ MedWatch/SafetyInformation/SafetyAlertsforHumanMedicalProducts/ucm314836.htm.
36. US Food and Drug Administration. Information for physicians and patients on the withdrawal of two humanitarian device exemptions (HDEs) for patent foramen ovale (PFO) occluders. FDA, 2006. www.fda.gov/MedicalDevices/ DeviceRegulationandGuidance/HowtoMarketYourDevice/PremarketSubmissions/ HumanitarianDeviceExemption/ucm135747.htm.
37. Kramer DB, Tan YT, Sato C, Kesselheim AS. Postmarket surveillance of medical devices: a comparison of strategies in the US, EU, Japan, and China. PLoS Med 2013;10:e1001519.
38. Garber AM. Modernizing device regulation. N Engl J Med 2010;362:1161-3.
39. US Food and Drug Administration. Strengthening our national system for postmarket device surveillance. FDA, 2012. www.fda.gov/AboutFDA/CentersOffices/ OfficeofMedicalProductsandTobacco/CDRH/CDRHReports/ucm301912.htm.
40. Normand SL, Hatfield L, Drozda J, Resnic FS. Postmarket surveillance for medical devices: America's new strategy. BMJ 2012;345:e6848.
41. Thompson M, Heneghan C, Billingsley M, Cohen D. Medical device recalls and transparency in the UK. BMJ 2011;342:d2973.
42. Kramer DB, Xu S, Kesselheim AS. How does medical device regulation perform in the United States and the European union? A systematic review. PLoS Med 2012;9:e1001276.
43. Basu S, Hassenplug JC. Patient access to medical devices-a comparison of US and European review processes. N Engl J Med 2012;367:485-8.