Pre-Approval Access

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About Celgene’s Pre-Approval Access Program

At Celgene, we believe that wherever possible, use of an investigational therapy for a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of new medicines and, consequently, to wider availability for patients. We encourage patients to speak with their physicians about eligibility to enroll in any of Celgene’s clinical trials. Learn more about?Celgene clinical trial programs.

We are committed to developing safe and effective therapies for patients and strive to put patients first. As part of that commitment, when patients with a serious or life threatening unmet medical need are unable to access our investigational therapies through a clinical trial and have exhausted all other treatments, Celgene may be able to provide access through its Pre-Approval Access Program either as a Single Patient Request (e.g. Compassionate Use) or Expanded Access Program.

To be considered for pre-approval access to a Celgene investigational therapy, the request must be submitted by a qualified and licensed physician with expertise and facilities appropriate for the administration of the investigational therapy, monitoring, managing and reporting side effects.

Evaluation Criteria

In determining whether access is appropriate outside of a clinical trial, Celgene will evaluate each request based on the following general criteria:

  • The investigational therapy must be part of a Celgene clinical development program. Once a therapy is approved by the country’s Health Authority to treat a particular disease, it will no longer be available for pre-approval access to treat that disease in that country.? In certain countries (including the United States), approval of the therapy by the Health Authority to treat any disease shall preclude access to the therapy through Celgene’s pre-approval access program;
  • Providing access to the Celgene investigational therapy must not interfere with the completion of a clinical trial supportive of a Health Authority approval or compromise the potential development of the therapy;
  • There are no beneficial alternative treatments available (including participation in ongoing relevant clinical trials);
  • The efficacy and safety data available at the time are sufficient to determine whether the potential clinical benefit to patient would likely outweigh the possible risks, including the outcome of the disease;
  • The intent for use is limited to providing the patient with a clinical benefit in the treatment of the underlying disease, not a symptom or comorbidity of the disease, unless such symptom or comorbidity is itself serious, debilitating or life-threatening;
  • A clinical study is not available, or the patient is not eligible (i.e. does not qualify);
  • Patients with underlying medical conditions that may pose safety risks that have not been sufficiently characterized/studied would not be eligible to participate;
  • An appropriate dose and/or treatment regimen for the investigational therapy has been determined for the disease for which treatment is sought;
  • There is manufacturing capacity and Celgene has adequate supply of the investigational therapy;
  • Access is compliant with local rules and laws;
  • Health Authority and / or Institutional Review Board / Ethic Committee approval is obtained (when required); and
  • Additional criteria regarding the therapy, its development program, the patient, or other circumstances may impact eligibility.

Additional Criteria for Single Patient Requests

  • The disease is serious, debilitating or life threatening; and
  • The patient has exhausted all available therapies typically used to treat the disease and is no longer responsive to, or able to tolerate, these treatments.

Additional Criteria for Expanded Access Programs:

  • The program is limited to groups of patients who generally have the same stage, extent and type of disease or medical condition that was evaluated in the key study/studies that will be submitted to the Health Authority in support of an application for approval of the medicine;
  • Celgene must consider the risk-benefit to be robust and persuasive and reasonably likely to be supported by the Health Authority; and
  • Based on discussions with the Health Authority, Celgene must have reason to believe the Health Authority is likely to approve the therapy for use in this population of patients.

Process for Requesting Access

The request for access to Celgene investigational therapy must come from the patient’s treating physician.? The qualified physician may request information on Celgene’s Pre-Approval Access Programs (e.g. Expanded Access Programs including any request for single patient “Named Patient Program” access and emergency access requests) by contacting Celgene’s Medical Information Department at 1-888-771-0141 or 1-908-673-9800 or by completing?the?online request form. ?Celgene strives to respond to expanded access requests as quickly as possible, usually acknowledging receipt of a request within 24 hours.? Celgene cannot make a guarantee that the investigational therapy will be available to a particular patient. Any Pre-Approval Access to investigational therapies must always comply with the applicable country-specific laws and regulations, including importation requirements, approvals from applicable regulatory bodies, and by an appropriate Institutional Review Board or Ethics Committee.? Celgene may require agreements and/or certifications executed by the treating physician and other information to be provided prior to final approval of any request.

If you are a patient with questions concerning access to our investigational therapies, either through a clinical trial or through expanded access, please consult with your treating physician.