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FDA Pediatric Oncology Subcommittee Meeting

FDA Pediatric Oncology Subcommittee Meeting. “Company Perspective”. Key Questions to Address (Company Perspective). Question #1-When in the development of a new oncolytic should pediatric studies be undertaken?

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FDA Pediatric Oncology Subcommittee Meeting

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  1. FDA Pediatric Oncology Subcommittee Meeting “Company Perspective”

  2. Key Questions to Address(Company Perspective) • Question #1-When in the development of a new oncolytic should pediatric studies be undertaken? • Question #2-What factors influence the decision whether or not pediatric studies are undertaken? • Question #3-Should pediatric studies be performed only by cooperative groups?

  3. Key Questions to Address • Question #1-When in the development of a new oncolytic should pediatric studies be undertaken? • Question #2-What factors influence the decision whether or not pediatric studies are undertaken? • Question #3-Should pediatric studies be performed only by cooperative groups?

  4. Drug Date of Initial FDA Approval Date of 1st Adult Study Date of 1st Pediatric Study Difference in Years Between Studies Gemcitabine 1996 1991 Ongoing 10+ Paclitaxel 1992 1986 1993 7 Cladribine 1993 1984 1991 7 Irinotecan 1996 1993 (US) 1999 (US) 6 Temozolomide 1999 1992 1998 6 Docetaxel 1996 1992 1997 5 Tretinoin 1995 1988 1992 4 Topotecan 1996 1992 1993 1 Imatinib 2001 2001 Ongoing 1+ Historically When Are Pediatric Studies Performed?

  5. ASPHO Survey1(Interim Results) 1Survey of 91 ASPHO Members-October 2002

  6. Key Questions to Address • Question #1-When in the development of a new oncolytic should pediatric studies be undertaken? • Question #2-What factors influence the decision whether or not pediatric studies are undertaken? • Question #3-Should pediatric studies be performed only by cooperative groups?

  7. ASPHO SURVEY1-INFLUENTIAL FACTORS2 • Pediatric preclinical data • Drug with new mechanism or target • Positive data from adult phase I or II • Availability of drug for pediatric studies • Other factors 1Survey of 91 ASPHO Members-October 2002 2Rate from 1-7; One being least influential

  8. “Most Influential-#7” #Responders choosing “7”

  9. Key Questions to Address • Question #1-When in the development of a new oncolytic should pediatric studies be undertaken? • Question #2-What factors influence the decision whether or not pediatric studies are undertaken? • Question #3-Should pharmaceutical companies conduct pediatric studies outside of cooperative groups?

  10. ASPHO Survey1 1Survey of 91 ASPHO Members-October 2002

  11. Comments- “No” • Patient numbers too small-direct competition with COG studies • Definitely “no”; “That would be a terrible mistake” • Concerned about “conflict of interest” • Cooperative group(s) are cornerstone of our success against pediatric cancer • More convincing if studies are done within Cooperative group setting • Cooperative group mechanism in concert with industry and the NCI, when necessary, should meet all industry and FDA requirements

  12. Comments-“Yes” • The Cooperative groups have a problem with congestion; Speed! • Cooperative group is a monolith • Any route necessary if it means getting phase I studies in children • Resources and infrastructure • Opportunity for smaller institution

  13. “Company Prospective” Each new agent needs to be considered separately (there is no standard approach) Early Pediatric Studies Late Pediatric Studies CMC Formulation-oral Stability (Drug supply) Toxicology “Unaccepted” toxicities Unusual target organs Medical/Scientific Similar disease process Similar target expression (Novel mechanism) Regulatory Pediatric Rule Business Development FDAMA/BPCA Unmet Need Data Management Cooperative group

  14. Summary- “Company Perspective” • Strong shift towards not if, but when, should pediatric studies be undertaken • Most pediatric oncologist believe studies should be done early versus late • Company involvement is OK-But! • Perception that conducting studies outside cooperative groups could speed up the process? • Companies are showing increased interest in developing new agents in children • FDAMA and Pediatric Rule • Many factors influence decision to conduct studies in children • Industry views are similar to those of pediatric oncologist, but there are obvious differences

  15. Questions-Discussion?

  16. “Other Influential Factors” • A company that is not overtly hostile towards pediatric studies (7) • Favorable side-effect or toxicity profile (6) • Unmet need (5) • Ease of study (5) • Foreign experience (4)

  17. Recent/Ongoing Pediatric Phase I Studies ABT-751 (ST) Arsenic Trioxide (L) BMS 247550 (ST) Clofarex (L) Carbo/Irinotecan (ST) DX-8951f (ST) Fenretinide (ST) Doxorubicin Liposome (ST) Flavopiridol (ST) Gadolinium Texaphyrin Cisplat/VP16/Bleo/Amifostine (GC) IT-Busulfan (BT) Iodine I131 MIBG (NB) Melphalan/Buthionine Sulfoximine Irinotecan Oxaliplatin (ST) R115777 PS-341 SCH 66336 (BT) Squalamine/Carbo (ST) SU5416 (BT) Temozolomide/O6BG Thiotepa/Carbo/Topotecan (ST) Irinotecan/VCR (ST) XR 9576 (ST) STI571 (ST) Holmium Ho 166 IL-2 Expanded CD8 Cells (NB) Rituximab -Glucan MoAb 3F8 (NB) hu14.18/IL-2 (NB) MoAb Ch14.18/IL2 (NB)

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