Drugs

Changes Sought in Molecularly Targeted Drug Studies

David McFarland

— 2 min read
Share

Three industry stakeholders say further information and clarification is needed in an FDA draft guidance on the implementation of pediatric studies of molecularly targeted oncology drugs under the FDA Reauthorization Act. The Biotechnology Innovation Organization (BIO) says that while the law contemplates only one molecularly targeted pediatric investigation, the draft guidance seems to raise the possibility of more than one clinical pediatric investigation and/or study. “We believe FDA should clarify in the final guidance a position consistent with the statute that only one molecularly targeted pediatric investigation will be required for any individual application and that only Pediatric Research Equity Act (PREA) or the new Section 504 study requirements can be imposed upon an individual application, not both,” the comment says.

BIO also says the issue of deferrals needs to be clarified to say that FDA may only defer the new investigation requirement with respect to an original application for a new active ingredient if it is first established that a molecular target is substantially relevant to the growth or progression of a pediatric cancer.

Pharmaceutical Research and Manufacturers of America says in its comment letter that the following areas would benefit from further discussion, confirmation, clarification, and/or revision in the final guidance: 

  •          clarify that either traditional PREA or the new investigation requirement will apply to an original application and not both;
  •          clarify FDA’s criteria for identifying a molecular target and determining whether it qualifies as substantially relevant to the growth or progression of a pediatric cancer;
  •         revise the draft to more clearly reflect that the agency must determine substantial relevance based on available information without requiring sponsors to conduct additional nonclinical studies;
  •          revise the draft to reflect that FDA may not defer its determination of substantial relevance;
  •          confirm in the final guidance that section 505(B) contemplates only one molecularly targeted pediatric clinical investigation;
  •          address how the agency’s proposed approach for issuing waivers and deferrals of the new investigation requirement aligns with the statutory standards;
  •         revise the draft guidance on written requests;
  •          clarify the discussion on early advice meetings;
  •          clarify the discussion of iPSPs for oncology drugs;
  •          advance international harmonization efforts for pediatric drug development plans; and
  •          issue modernized and comprehensive guidance on PREA and the Best Pharmaceuticals for Children Act.

Finally, Bayer says the focus on molecular mechanism of action is likely to increase competition for access to a limited patient population due to a small number of pediatric patients with the molecular target. It says the use of multi-stakeholder collaborations in clinical trials is an opportunity and the key to generating meaningful results in a limited patient population.

Read more