**Joint Workshop on Pediatric Oncology**

**July 18, 2002**

**Summary Questions**

Panel 1 summary questions

1.  Who should set priorities for pre-clinical testing and COG studies?

2.  How should choices made by the NCI pre-clinical consortium and COG
    be considered in FDA decisions regarding application of the
    pediatric rule and granting of pediatric exclusivity?

3.  What can the FDA and a sponsor do when a sponsor proposes
    exclusivity, but the NCI and COG do not conisder the drug a high
    priority for study?

4.  When and how should the FDA request pediatric formulation work as
    part of the NDA process?

Panel 2 summary questions

1.  When or under what circumstances would it be acceptable for new
    agents to enter clinical trials prior to or concurrent with the
    initiation of adult trials?.

2.  How can adult and pediatric development plans be coordinated to
    improve access for investigators to the most promising new cancer
    therapies during the drug development process?

3.  How could we employ, or would you accept some of the Bayesian or
    continual reassessment methods in clinical trial designs in the
    Phase I and Phase II setting? Could there be some relaxation of the
    traditional rules and look at historical controls and time to
    progression as endpoints that would achieve labeling for these
    studies?

4.  Is it within the FDA jurisdiction to be involved in the
    prioritization process? Could the FDA consider public policy and
    review of IND applications, or could it be required that IND
    submissions meet criteria for consortium study, and can that be a
    way to maximize the benefit of ODAC?

5.  Discussion of the ethical concerns regarding clinical trial
    requirements for samples that involve greater than minimal risk, or
    samples that were in some way sensitive in clinical trials?

Panel 3 Summary Questions

A.  Access to Investigational Drugs :

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1.  Would data from an expanded access program in children be more
    useful than data derived from expanded access programs involving
    adults (investigators more attuned to reporting requirements and
    capture of a greater proportion of the disease population) ?

2.  Could expanded access programs for childhood malignancies be
    detrimental to the process of developing new therapies since the
    numbers of patients available are limited and such a strategy could
    hamper accrual to protocols ?

3.  Should patients have to exhaust all research clinical trials before
    having access in an off-protocol setting ?

4.  How is and who decides "sufficient activity" and "acceptable risk"
    for expanded access programs and how can these criteria be made
    transparent to the public ?

5.  Should a consortium of representatives from NCI, FDA, COG, patient
    advocacy, and industry develop a "generic" protocol which can and
    should be used with each "compassionate use" request ?

6.  Should the FDA approve "compassionate use" requests only for
    investigational drugs that are being provided according to a
    consortium approved pediatric cancer specific protocol ?

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B.  Labeling :

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1.  What difference would it make if an agent has labeling, to industry,
    the public, physicians ?

2.  Should there be a statement in labeling regarding compliance with a
    written request ?

3.  Can criteria be modified for inclusion of information derived from
    studies of children with cancer in the label ?