Regulatory Focus™ > News Articles > After 13 Years, FDA Finalizes Pediatric Trials Protection Rule

After 13 Years, FDA Finalizes Pediatric Trials Protection Rule

Posted 26 February 2013 | By Alexander Gaffney, RAC 

A new final regulation just released by the US Food and Drug Administration (FDA) amends an existing regulation to include minor additional safeguards for pediatric patients, including children, enrolled in clinical trials.

The long-anticipated rule has been in the works since 2001, when FDA released an interim rule meant to comply with the Children's Health Act of 2000, which mandates that all pediatric research in the US offer subject additional protections to ensure their safety.

These protections include requirements that institutional review boards review pediatric research and ensure that the research meets seven criteria:

  1. Risks to children are minimized.
  2. Risks to children are reasonable in relation to the perceived benefits.
  3. Subjects are selected equitably.
  4. Informed consent is obtained for all subjects and documented appropriately.
  5. Research data is monitored to ensure patient safety.
  6. Data is kept confidential.
  7. Safeguards are in place to protect the most vulnerable patients.

Other Changes

Importantly, the rule also calls on all companies to conduct a pediatric assessment for new products, including active ingredients, indications, dosing regimens and dosage forms, unless those requirements were explicitly waived or deferred by FDA.

Sponsors who complete the difficult pediatric trials are eligible for additional incentives, including six months of marketing exclusivity also known as pediatric exclusivity, which applies to all other forms of marketing exclusivity and not just the pediatric application. Those incentives were made permanent in 2012 under the FDA Safety and Innovation Act (FDASIA).

FDA said it has made four significant changes to the final rule relative to its interim rule:

  • The definition of guardian has been updated to exclude a phrase specifying that a guardian must have the ability to authorize medical care to account for the possibility that a guardian might not have this authority in some states.
  • An instance requiring parental permission has been removed because FDA found it was already required in another section.
  • A new paragraph requires institutional review boards to assess the level of risk to children participating in trials, which must determine if a trial represents "greater than minimal risk" to a patient, or just "minimal risk."
  • The rule now explicitly says that emergency research exceptions apply to children as well as adults.

The final rules come into effect 30 days after its publication in the Federal Register, scheduled for 26 February 2013. A similar rule was passed by FDA on 19 February 2013 pertaining to medical device submissions and pediatric data requirements.


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