CANCER THERAPY EVALUATION PROGRAM (CTEP), NCI

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CANCER THERAPY EVALUATION PROGRAM (CTEP), NCI
Dale Shoemaker, PhD Head, Regulatory Affairs,
CTEP, NCI
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ACCESS TO INVESTIGATIONAL AGENTS FOR PATIENTS
UNABLE TO PARTICIPATE IN CLINICAL TRIALS
Purpose To make available to individual cancer
patients and their physician investigational
agents that have notable clinical activity
against specific malignancies.
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MECHANISMS FOR EARLY ACCESS

• Special Exception Protocol
• Group C/Treatment IND Protocols
• Treatment Referral Center Protocols

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ISSUES TO CONSIDER IN A REQUEST FOR EARLY ACCESS

• Is the patient ineligible or unable to be treated
  on a research protocol?
• Have standard therapies been exhausted?
• Is there objective evidence that the
  investigational agent is active in treating the
  malignancy for which the request is made?
• Is the investigational agent likely to benefit
  the patient while posing an acceptable risk?

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SPECIAL EXCEPTION PROTOCOLS

• Functional equivalent of an emergency
  Investigational New Drug application (IND)
• A request may be made for any of the
  investigational agents sponsored by CTEP
• Generally not granted for agents in phase 1
  clinical development

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SPECIAL EXCEPTION PROTOCOLS (continued)

• Agents must have demonstrated efficacy in disease
  state for the request (generally agents in phase
  2 or phase 3 clinical development
• Approval depends on meeting the criteria outlined
  above and agent availability
• Requests reviewed and approved by physician staff
  in CTEP

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GROUP C PROTOCOLS

• Process initiated in the mid-1970s
• Request made to FDA for an investigational agent
  which has demonstrated reproducible efficacy in
  one or more specific types of tumor
• The agent is likely to alter the contemporary
  treatment of a tumor type and can safely be
  administered by properly trained health care
  providers without specialized supportive care
  facilities

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GROUP C PROTOCOLS (continued)

• FDA approval sought for those investigational
  agents for which an NDA or BLA will be submitted
  in the relatively near future
• CTEP has sponsored more than 20 Group C protocols
  with only one active Group C protocol at the
  present time

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TREATMENT REFERRAL CENTER PROTOCOLS

• TRC Protocols used for highly promising agents
  for diseases such as breast, prostate, lung, and
  ovarian cancer
• TRC Protocol is similar to a simple multicenter
  clinical trial (a large one-arm study with
  relatively open eligibility and simple
  objectives)
• TRC Protocols used to ensure equitable
  distribution of investigational agents with
  limited availability
• Safety and efficacy data are typically collected
• TRC Protocols initially offered to the
  NCI-designated clinical and comprehensive Cancer
  Centers

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CTEP TREATMENT REFERRAL CENTER

• Managed by the Pharmaceutical Management Branch,
  CTEP
• Provides information to community oncologists and
  other health care professionals about therapeutic
  options for cancer patients
• First priority is to refer patients to
  Cooperative Group or Cancer Center trials

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CTEP TREATMENT REFERRAL CENTER (continued)

• If patient unable to participate in a clinical
  trial, an early access mechanism might be
  considered
• Treatment Referral Center can be contacted by
  phone [phone]) or fax [phone])
• More information available on the CTEP home page
  (http//ctep.cancer.gov)

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SUBMISSION OF REQUEST

• Group C and TRC protocols have set criteria to
  determine patient eligibility while Special
  Exceptions are assessed on each request
• To properly evaluate each request, the following
  information is needed patient identifier
  (initials or identification number), age, sex,
  diagnosis, previous cancer therapy, current
  clinical status, intended dosage and schedule of
  intended agent, potential concomitant therapy,
  and pertinent laboratory data

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SUBMISSION OF REQUEST (continued)

• Each request is reviewed and assessed on the
  basis of established guidelines developed by
  staff oncologists for each investigational agent
• Response is provided as quickly as possible
  usually the same day of the request

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REQUIREMENTS FOR INVESTIGATORS

• Protocol standard protocol form to be completed
  and signed is provided
• FDA Form 1572
• IRB and Informed Consent
• Final Patient Report to be provided for summary
  of the treatment including efficacy and adverse
  events
• Agent Accountability records must be maintained
  by the investigator