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July 31, 2012 Dr. Dianne Murphy Director, Office of Pediatric Therapeutics

Pediatric Product Development : A Permanent Seat at the Research Table! Presented To: NIH CTSA Consortium: Child Health Oversight Committee. July 31, 2012 Dr. Dianne Murphy Director, Office of Pediatric Therapeutics FDA, Office of the Commissioner.

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July 31, 2012 Dr. Dianne Murphy Director, Office of Pediatric Therapeutics

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  1. Pediatric Product Development: A Permanent Seat at the Research Table!Presented To: NIH CTSA Consortium:Child Health Oversight Committee July 31, 2012 Dr. Dianne Murphy Director, Office of Pediatric Therapeutics FDA, Office of the Commissioner

  2. Major Pediatric Elements of the recent FDA legislation: (FDA Safety & Innovation Act = FDASIA) • Makes Pediatric product development a PERMANENT part of FDA’s law! • Requires early consideration of the pediatric use possibilities for all products being developed. • Thus, transforms the approach to product development at FDA in that pediatrics is now a PART of the process, not “additional”

  3. PediatricLegislation: Details Permanent Reauthorization of Pediatric Legislation Both the exclusivity (BPCA: Best Pharmaceuticals for Children) and the requirement (Pediatric Research Equity Act- PREA) programs now permanent Earlier submission requirements for Pediatric Study Plans Sponsors required to submit plans earlier in the drug development process End of Phase 2 instead of at time of marketing application Opportunity for Agency to work with Sponsor to ensure rational product development Permits EARLY discussion of fundamental approaches that are unique to pediatrics that need to be discussed: extrapolation, endpoint validation, formulations May allow for sponsor to begin nonclinical studies and have adequate time to determine what data is necessary to support dosing, safety, and efficacy in the pediatric population 3

  4. Pediatric Legislation: Details More Transparency and Tracking • Retroactive retrieval of Medical, statistical and pharmacology reviews of pediatric studies under prior peds legislation submitted between 1/2002 and 9/2007 and posting on the Web. • Posting annually the basis of extensions of deferred studies and requires FDA to track Increased enforcement mechanism • Authority to issue and publically post non-compliance letters for overdue studies.

  5. Pediatric Study Plan Timelines/Mandates • Pediatric Study Plans due by 60 days following End of Phase 2 (EOP2) meeting • FDA must meet with applicant, or provide a written response, no more than 90 days after receipt of plan. • Once applicant has documented agreement on the plan, FDA has 30 days to confirm. • Pediatric Review Committee (PeRC) will review the plans and make recommendations. • Mandate for better tracking of demographics of pediatric trials • Mandate for more transparency of studies and activities 5

  6. Pediatric Legislation: Additional Components • The Pediatric Advisory Committee is made permanent • The Pediatric Oncology Advisory subcommittee of the Oncology Advisory Committee is to exist as long as the Oncology AC does. • The Office of Pediatric Therapeutics will hire expertise in pediatric epidemiology and neonatology. Minimum of 5 year requirement for neonatology. • The Peds Review Committee (PeRC) will have neonatology expertise

  7. Pediatric Legislation: Additional Components • Rare Pediatric Diseases: - FDA shall hold at least 1 public meeting to discuss ways to encourage development of new therapies for rare diseases - FDA shall issue a report that includes a strategic plan for accelerating development for pediatric rare diseases. This includes many pediatric cancers. • Pediatric Device Consortia: - FDA to issue proposed rule re Consortia - Tracking of Pediatric uses of devices

  8. Pediatric Legislation: Reports • Within 4 years and then every 5 yrs. Report to Congress on implementation, including assessments and data on timeliness & effectiveness of pediatric study planning, efforts to increase studies for the neonatal population, and data on drugs and biological products for pediatric cancers. To be posted on FDA’s web site and made publically available.

  9. Pediatric Legislation: Reports • At least 6 months prior to the mentioned Report to Congress, input will be obtained from patient groups, consumer groups, industry, academia, and other interested parties. • By 18 months of enactment FDA will hold a meeting to discuss ways to encourage development of therapies for rare diseases and provide a report as noted earlier.

  10. Process Development • Guidance on pediatric legislation implementation ( 1year) • Standard Operating Procedures must be developed for the PeRC review of pediatric study plans and posted on the Web. (by 1 year) • Promulgation of Regulations on Pediatric Study Plans (1 year)

  11. So Why Do You Need To Know This? • How does this affect us? -as researchers - as advocates for children -as providers of health care • What can we do? - familiarity with requirements for studies being submitted to FDA - be knowledgeable re “extrapolation” - endpoint validation activities

  12. FDA’s OPT Homepage:www.fda.gov/pediatrics

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