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Food and Drug Administration Amendments Act of 2007 Reauthorization of Pediatric Initiatives Lisa L. Mathis, M.D. Pediatric and Maternal Health Staff Office of New Drugs April 2008 Acronyms BPCA – Best Pharmaceuticals for Children Act FDAAA – Food and Drug Administration Amendments Act

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food and drug administration amendments act of 2007 reauthorization of pediatric initiatives

Food and Drug Administration Amendments Act of 2007Reauthorization of Pediatric Initiatives

Lisa L. Mathis, M.D.

Pediatric and Maternal Health Staff

Office of New Drugs

April 2008

acronyms
Acronyms
  • BPCA – Best Pharmaceuticals for Children Act
  • FDAAA – Food and Drug Administration Amendments Act
  • PAC – Pediatric Advisory Committee
  • PREA – Pediatric Research Equity Act
  • WR – Written Request

FDAAA: ODAC Pediatric Subcommittee April 08

objectives
Objectives
  • Brief Overview of Pediatric History at FDA
  • Major changes to Title V: Best Pharmaceuticals for Children Act of 2007 (BPCA)
  • Major changes to Title IV: Pediatric Research Equity Act of 2007 (PREA)

FDAAA: ODAC Pediatric Subcommittee April 08

slide4

FDA and Pediatrics

  • 1979 Labeling Requirement
  • 1994 Pediatric Labeling Rule
  • 1997 FDA Modernization Act (FDAMA)
  • 1998 Pediatric Rule
  • 2002 Best Pharmaceuticals for Children Act (BPCA)
  • 2002 Pediatric Rule Enjoined
  • 2003 Pediatric Research Equity Act (PREA)
  • 2007 FDA Amendments Act of 2007
    • Pediatric Medical Device Safety and Improvement Act
    • Pediatric Research Equity Act (PREA)
    • Best Pharmaceuticals for Children Act (BPCA)

FDAAA: ODAC Pediatric Subcommittee April 08

slide5

Childhood Cancer Mortality 1975-2003

FDAAA: ODAC Pediatric Subcommittee April 08

bpca 2007
BPCA 2007

FDAAA: ODAC Pediatric Subcommittee April 08

slide7

Improvements to BPCA

BPCA 2007

  • Pediatric Subcommittee of the Oncologic Drugs Advisory Committee
    • Extended Committee to 2012
    • May make recommendations directly to the Internal Review Committee on the implementation of BPCA
  • Expands role of NIH to “pediatric therapeutics” not just drugs

BPCA 2003

  • Clarified role of the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee
  • Previouspriority list was for off patent drugs only

FDAAA: ODAC Pediatric Subcommittee April 08

internal review committee pediatric review committee perc
Internal Review CommitteePediatric Review Committee (PeRC)
  • Established to provide oversight to required and requested pediatric activities within CBER/CDER
  • Expertise mandated
  • Pediatric Oncology is represented

FDAAA: ODAC Pediatric Subcommittee April 08

national institutes of health
National Institutes of Health
  • Expanded role includes establishing a “List of Priority Issues in Pediatric Therapeutics”
    • Previously NIH was charged with a list of off patent drugs not eligible for market exclusivity
    • Expert meeting in June-July 2008
  • Law requires consideration of available information on drugs and biologics

FDAAA: ODAC Pediatric Subcommittee April 08

slide10

Improvements to BPCA (cont)

BPCA 2007

  • Drugs
  • Studies voluntary
  • Studies on entire active moiety
  • May include on & off label indications
  • WR may be issued for orphan indications
  • Preclinical studies may be requested
  • Priority review for all applications

BPCA 2003

  • Drugs
  • Studies voluntary
  • Studies on entire active moiety
  • Had to choose between on or off label indication
  • WR may be issued for orphan indications
  • Only includes studies in pediatrics
  • Priority Review supplements only

FDAAA: ODAC Pediatric Subcommittee April 08

preclinical studies
Preclinical Studies
  • Law states that pediatric studies and preclinical studies may be included in a Written Request
    • BPCA 2002 only allowed for the request of studies in the pediatric population
    • Could not previously require a preclinical study as a term of the WR
  • Law does not allow for preclinical studies only

FDAAA: ODAC Pediatric Subcommittee April 08

slide12

Improvements to BPCA (cont)

BPCA 2007

  • All adverse events must be submitted with application
  • 6 months to review studies and determine if terms of WR met
  • Must have 9 months of exclusivity at time of determination
  • Written requests become public
  • Studies must be added to label
  • Full reviews posted
  • Adverse events review for peds

BPCA 2003

  • No requirement to submit adverse events
  • 3 months to review studies and determine if terms of WR met
  • Could submit on day of patent expiration
  • Written request confidential
  • Labeling not required
  • Summaries of reviews posted
  • Adverse events reviewed for peds

Sunset for PREA and BPCA Oct 1, 2012

FDAAA: ODAC Pediatric Subcommittee April 08

prea 2007
PREA 2007

FDAAA: ODAC Pediatric Subcommittee April 08

slide14

Improvements to PREAbasics remain the same

PREA 2007

  • Drugs and biologics
  • Studies mandatory
  • Studies for orphan indications exempt
  • Required studies only on drug/ indication under review

PREA 2003

  • Drugs and biologics
  • Studies mandatory
  • Studies for orphan indications exempt
  • Required studies only on drug/ indication under review

FDAAA: ODAC Pediatric Subcommittee April 08

required studies only on drug indication under review application of prea
Required Studies Only on Drug/ Indication Under Review:Application of PREA
  • Pediatric studies required and a pediatric assessment must be submitted for NDA/BLA or supplements with
    • New active ingredient
    • New indication
    • New dosage form
    • New dosing regimen or
    • New route of administration
  • Applies only to the indication included in the submission
    • Limits scope of required studies

FDAAA: ODAC Pediatric Subcommittee April 08

application of prea
Application of PREA
  • Indications that are likely to be studied under PREA
    • Leukemia/Lymphoma
    • Supportive care
      • Pegfilgrastim

FDAAA: ODAC Pediatric Subcommittee April 08

slide17

Improvements to PREA (con’t)

PREA 2007

  • Age appropriate formulation required
  • Study results must be included in labeling
  • Full reviews posted
  • Pediatric adverse events reviewed and presented to PAC

PREA 2003

  • Age appropriate formulation required

Sunset for PREA and BPCA Oct 1, 2012

FDAAA: ODAC Pediatric Subcommittee April 08

conclusions
Conclusions
  • Reauthorization provides continued incentive and requirement for pediatric studies and pediatric oncology still plays a major role in the new legislation
  • Despite progress in pediatric oncology drug development specifically, improvements in survival rates have slowed
  • Scientists in every sector must collaborate using tools available to them to provide new opportunities for treatment
  • We can use legislation to improve this collaboration

FDAAA: ODAC Pediatric Subcommittee April 08

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