Training Workshop on Pharmaceutical Development with focus on Paediatric Formulations

1
Regulatory requirements for the development of
medicinal products for pediatric use
Training Workshop on Pharmaceutical Development
with focus on Paediatric Formulations Protea
Hotel Victoria Junction, Waterfront Cape Town,
South Africa Date 16 to 20 April 2007
2
Regulatory requirements for the development of
medicinal products for pediatric use

• Presenter
• Dr Tom Sam
• Industrial Pharmacy Section
• International Pharmaceutical Federation (FIP)

• The significant contributions to this
  presentation
• of the following persons is gratefully
  acknowledged
• - Nicole Herijgers - NV Organon
• - Lisette Vromans - NV Organon

3
Outline

• Regulatory environment
• Timing of pediatric studies during development
• Type of development studies needed
• Addressing pediatric information in the labelling
• Need for special drug formulations

4
Introduction

• Use of unlicensed and off-label medicines in
  children is wide-spread
• gt 60 of all medicines used in children have
  never been tested and are not authorised in the
  peadiatric population (i)
• These products have only been tested in adults,
  not necessarily in the same indication or same
  disease.
• Lack of information and appropriate
  pharmaceutical formulations may expose children
  to unwanted side effects or underdosing without
  the expected efficacy.
• i Le Cam, Y. Medicines for children better,
  more and faster. Regulatory Rapporteur. May 2005
  8.

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Requirements (1)

• Canada - Registration of pediatric drug
  follows normal procedure.
• Australia - Market authorisation may be
  denied / delayed, if clinical pediatric data is
  not included but is deemed appropriate.
• India - If new drug is intended to treat both
  adults and pediatric patients, the pediatric
  population should be included in the clinical
  trials from an early point onwards. If pediatric
  data is not included, this needs to be justified
  in detail.
• China - Registration of pediatric drug
  follows normal procedure. Clinical trials on
  children are discouraged unless the drug-use is
  limited to only the pediatric population.

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Requirements (2)

• Japan - No mandatory pediatric clinical trials.
  There are however, ongoing projects organized by
  the MHLW (Ministry of Health, Labour and
  Welfare), which aim to facilitate an accelerated
  review of off-label pediatric drugs.
• South Korea - Non-established legislation for
  pediatric drugs.
• Switzerland - Pediatric studies are not mandatory
  for drug registration, unless the drug has
  potential pediatric use.

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US legislation on pediatric medicines

• Pediatric Rule issued by FDA in December 1998
  (i).Required the conduct of pediatric studies
  and development of pediatric formulationsThe
  Pediatric Rule suspended in October 2002.
• Pediatric Research Equity Act (PREA) law in 2003
  (ii,iii).
• Pediatric Exclusivity Provision - complementary
  to the PREA,which provides an incentive for
  companies who perform clinical trials in the
  pediatric population (iv).
• i Pediatric Rule was codified at 21 CFR 314.55
  and 601.27 with additional amendments to 21 CFR
  201, 312, 314 and 601.
• ii Pediatric Research Equity Act of 2003
  (http//www.fda.gov.cder.pediatric/index.htm
  accessed on 27 March 2006)
• iii Draft Guidance for Industry