Posizione degli enti regolatori: FDA, EMA, AIFA

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Il carcinoma del colon-retto MSI-H.

Posizione degli enti regolatori (FDA, EMA, AIFA)

Roberto Bordonaro

Segretario Nazionale dell’Associazione Italiana di Oncologia Medica (AIOM) Struttura Complessa di Oncologia Medica

Clinical Trial Center ARNAS Garibaldi

Catania

Struttura Complessa di Oncologia Medica ARNAS Garibaldi – Catania

Centro di Riferimento Regionale per la diagnosi e la terapia dei tumori del colon-retto e dei tumori del

polmone

D.A. 06/06/2018 G.U.R.S. del 22/06/2018 parte I, n° 27

Percorso di gestione dei trattamenti antitumorali (UFA/ONCO/EMA)

(Certificazione ISO 9001-2015)

Clinical Trial Center (Certificazione ISO 9001 – 2015)

Phase I Controlled Clinical Trials Site

(Certificazione ISO 9001:2015)

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• Breakthrough Therapy Designation (BTD) was established by the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012;

• To expedite the development and review of drugs and biologics for serious diseases and conditions;

• In order to qualify for a breakthrough therapy designation, a candidate

therapy must be intended to treat a serious or life-threatening illness, and preliminary clinical evidence must indicate that the therapy may

demonstrate a substantial improvement over existing therapies on at least one clinically significant endpoint.

• Fast track, by contrast, may be granted based on either clinical or

nonclinical data, and these data need only demonstrate that the therapy has the potential to address an unmet medical need.

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Breakthrough therapy is distinct from FDA’s other expedited programs both in terms of the level of evidence required and the type of engagement that sponsors subsequently receive from the FDA during clinical development.

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Initial Interaction with Merck

• FDA discussed KN-16 with Merck in May 2015 on the basis of:

– ORR:

• 4/10 MSI-H CRC

• 5/7 MSI-H other tumors ( 0/18 MSS CRC) –

FDA discussed design of Keynote-164 for MSI-H colo-rectal cancers in which recommended enrollment of patients with other MSI-H GI cancers.

FDA recommended that Merck submit a BTDR.

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Il carcinoma del colon-retto MSI-H.