Medical & Surgical Devices for Children: MDUFA IV Public Meeting. July 13, Tamar Magarik Haro Associate Director Dept. of Federal Affairs

Transcription

1 Medical & Surgical Devices for Children: MDUFA IV Public Meeting July 13, 2015 Tamar Magarik Haro Associate Director Dept. of Federal Affairs

2 American Academy of Pediatrics The American Academy of Pediatrics (AAP) is a professional organization of 64,000 primary care pediatricians, pediatric medical subspecialists, and pediatric surgical specialists Dedicated to the health, safety, and well-being of infants, children, adolescents, and young adults since its founding in 1930 AAP maintains numerous organized sections with device expertise: Allergy and Immunology Anesthesiology and Pain Medicine Cardiology and Cardiac Surgery Clinical Pharmacology and Therapeutics Gastroenterology Genetics and Birth Defects Hematology/Oncology Hepatology and Nutrition Infectious Diseases Nephrology Neurological Surgery Neurology Ophthalmology Orthopaedics Otolaryngology Pediatric Dentistry and Oral Health Pediatric Pulmonology Perinatal Pediatrics Plastic Surgery Radiology Rheumatology Urology Surgery

3 Children and Device Regulation Children are a small and vulnerable population Since 1977, the AAP has advocated strongly that medicines used in children should be tested in children The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have been hugely successful innovations for children resulting in more than 580 drug labeling changes Just as in drugs, children need medical and surgical devices made with their unique needs in mind and studied in pediatric populations Children have unique medical and surgical device needs related to size, growth, and physiological differences Differences between drug and device markets call for unique policy solutions for children

4 The Pediatric Devices Initiative Beginning in June 2004, a series of stakeholder meetings yielded recommendations for improving the availability of pediatric medical and surgical devices. The meetings were hosted by: American Academy of Pediatrics Elizabeth Glaser Pediatric AIDS Foundation National Organization for Rare Disorders (NORD) National Association of Children's Hospitals Advanced Medical Technology Association (AdvaMed) Representatives from government, industry, medicine and patient groups participated

5 Pediatric Medical Device Safety and Improvement Act of 2007 and 2012 Reauthorization The law in 2007 and 2012: Increased FDA authority for tracking of pediatric device approvals Draft guidance implementing the tracking provision Incentivized device development by removing the humanitarian device exemption (HDE) profit cap for pediatric devices Reauthorized in 2012 with a narrow expansion for adult-only HDEs Established non-profit consortia to stimulate device development Reauthorized in 2012 law at $5.25 million per year Enhanced postmarket surveillance for pediatric devices Enhanced the federal response for pediatric device needs

6 Recent Milestones in Pediatric Devices Publication of draft guidance Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices Authority for pediatric extrapolation of efficacy for PMAs and HDEs given in 2007 has great potential for pediatric labeling if used appropriately Creation of the Chief Pediatric Medical Officer position in CDRH Success of the Pediatric Device Consortia Program Funded at $3 million per year and the nine FDA-funded consortia assisted in advancing the development of 440 proposed pediatric medical devices FDA Public Workshop on Developing Medical Devices for Pediatric Patients with Rare Diseases (Jan. 2014) Draft guidance on pediatric uses of medical devices

7 Looking Forward: MDUFA IV Process Progress has been made but we re still just getting started the law was an important first step Meaningful and robust integration of pediatrics in the regulatory process for devices More proactive approach to pediatric submissions Consideration of targets and a strategic plan for development of devices for unmet needs in children

8 Pediatric Opportunities in FDA Device Reform Maintain the integrity of the FDA review process, striking the right balance between safety and effectiveness (or probable benefit) with access to new devices Build a more established pediatric infrastructure within CDRH Begin the conversation about pediatrics sooner in the product life cycle Better use of existing tools e.g. innovation 2.0 pathway and the PDC program for unmet pediatric medical device needs identified by FDA Certain authorities of the Pediatric Medical Device Safety and Improvement Act will need to be reauthorized by Sept. 30, 2017

9 Next Steps Opportunities for improvements to the development of safe medical devices for children through Congressional action on FDA user fee and other programs between now and 2017 Consideration of new methods, opportunities, incentives for pediatric device development

10 Contact Information Tamar Magarik Haro Associate Director, Department of Federal Affairs American Academy of Pediatrics