Regulatory Focus™ > News Articles > 2020 > 1 > FDA Offers Q&As on Submission of iPSPs for Cancer Drugs

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Posted 15 January 2020 | By Zachary Brennan 

FDA Offers Q&As on Submission of iPSPs for Cancer Drugs

2954 To help address a number of industry questions, the US Food and Drug Administration (FDA) on Wednesday published a new draft guidance on the submission of initial pediatric study plans (iPSPs) for oncology drugs, in light of amendments made by the FDA Reauthorization Act of 2017 (FDARA).

FDA explains how FDARA amended section 505B of the FD&C Act to require any new drug applications (NDAs) and biologics license applications (BLAs) submitted on or after 18 August 2020 to conduct pediatric investigations of certain molecularly targeted cancer drugs.

“FDARA thus created a mechanism to require evaluation of certain novel medicines that may have the potential to address an unmet medical need in the pediatric population,” FDA explains.

The 7-page draft, which comes in the form of a questions and answers (Q&As) document, discusses everything from when an iPSP is required, to when an iPSP can be abbreviated for sponsors seeking a waiver, to whether sponsors of combination drugs need to submit separate iPSPs for each of the drugs in the combination.

According to the draft, prior to 18 August, sponsors seeking a full waiver of pediatric studies, including because the new active ingredient is being developed for an indication on an FDA list of adult conditions that qualify for a waiver, “should complete only certain sections of the iPSP template included in Appendix 1 of the Draft iPSP Guidance” from 2016.

But on or after 18 August, the Q&A says sponsors of original or supplemental applications seeking a full waiver of pediatric studies for cancer drugs containing a new active ingredient, including for products directed at a molecular target included on FDA’s list of Non-Relevant Molecular Targets which Warrant Waiver, should also submit an abbreviated iPSP as described in the 2016 draft guidance. And sponsors “seeking a full waiver of pediatric studies for certain supplemental applications should continue to submit abbreviated iPSPs,” FDA adds.

If a product is granted an orphan designation, then it is only exempt from the Pediatric Research Equity Act (PREA) requirements under section 505B(k) of the FD&C Act if it is submitted prior to 18 August, the draft notes. “However, FDA recommends all sponsors developing such drugs submit an iPSP anyway because development timelines can be unpredictable and pediatric investigations will be required, unless waived, if the original application is submitted on or after August 18, 2020,” the agency adds.

The Q&A also addresses some of the more complicated situations, such as an application for a drug intended for the treatment of an adult cancer, with a new molecularly targeted ingredient that is determined to be substantially relevant to a pediatric cancer but has a proposed indication on the “rarely or never occur in pediatrics” list. FDA says that such a product will qualify for a waiver only if the application is submitted prior to 18 August.

“Nevertheless, even for products whose marketing applications are likely to be submitted before August 18, 2020, FDA recommends submission of an iPSP because, depending on the ultimate submission date of the marketing application, molecularly targeted pediatric cancer investigations may be required, and early discussion with the Agency prior to application submission may facilitate the development of studies to ensure that all relevant PREA requirements are met,” the draft adds.

Pediatric Study Plans for Oncology Drugs: Transitional Information Until Full Implementation of FDARA Section 504 Questions and Answers


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