Pfizer recently announced that it has initiated a multi-center, open-label expanded access program (EAP) in the United States for the investigational CDK 4/6 inhibitor, palbociclib.  Through the program, palbociclib is being made available for use in combination with letrozole for post-menopausal women with hormone receptor-positive, HER2-negative advanced breast cancer for whom letrozole is considered appropriate therapy. 

Healthcare professionals and patients can learn more about the palbociclib EAP by visiting www.clinicaltrials.gov (trial number: NCT02142868).  In addition, for HCPs, below are answers to some frequently asked questions about the palbociclib EAP. 

If you are a patient interested in participating in the palbociclib EAP, you should speak with your physician to understand if palbociclib is an appropriate treatment option for you.  All requests for access to palbociclib through the palbociclib EAP must come through a treating physician. 

Palbociclib is an investigational therapy and is not approved for any indication in any markets.  Pfizer is currently conducting clinical trials to evaluate the safety and efficacy of palbociclib in women with advanced breast cancer. 

FAQs

1. What is an EAP (Expanded Access Program)?
   
   Under its expanded access programs, the U.S. Food and Drug Administration (FDA) works with companies to allow access to investigational therapies outside of a clinical trial to patients with serious or life-threatening illnesses for whom there are no comparable or satisfactory alternate therapies.  For more information, visit www.fda.gov.

2. What is the difference between an EAP and Compassionate Use?
   
   FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case, compassionate use basis for an individual patient, as well as expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.

   This type of FDA-approved access to investigational drugs is sometimes called “expanded access” and other times “compassionate use.”  For palbociclib, access provided on a case-by-case basis to an individual patient for whom there is no appropriate clinical trial option is referred to as compassionate use.  Access provided through this program for post-menopausal women with hormone receptor-positive, HER2-negative advanced breast cancer who are eligible for letrozole therapy is referred to as Expanded Access.

3. What is the difference between the EAP and the ongoing Phase 3 trials for palbociclib?
   
   Pfizer has established a comprehensive development program to evaluate the safety and efficacy of palbociclib in hormone-receptor positive (HR+), HER2- breast cancer across stages and treatment settings.  This includes PALOMA-2 (also known as Study 1008) and PALOMA-3 (also known as Study 1023). 

   PALOMA-2 is a randomized (2:1), multi-center, double blind Phase 3 study that evaluates palbociclib in combination with letrozole versus letrozole plus placebo as first-line treatment for post-menopausal patients with ER+, HER2- advanced breast cancer.  PALOMA-3 is a Pfizer-sponsored, randomized (2:1), multi-center, double blind Phase 3 study that evaluates palbociclib in combination with fulvestrant versus fulvestrant plus placebo in women with hormone receptor-positive (HR+), HER2- metastatic breast cancer whose disease has progressed after prior endocrine therapy.  The PALOMA-2 and PALOMA-3 trials are ongoing but no longer recruiting new patients.

   With recruitment of new patients to our Phase 3 PALOMA-2 and PALOMA-3 trials now complete, Pfizer has initiated the Palbociclib EAP for post-menopausal women with hormone receptor-positive, HER2-negative advanced breast cancer for whom letrozole is considered appropriate therapy.  This program will provide a mechanism by which eligible women who may potentially benefit from treatment with palbociclib can gain access to this investigational therapy at this time. Pfizer has submitted an NDA to the FDA for palbociclib, combined with letrozole, for the treatment of postmenopausal women with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer who have not received previous systemic treatment for their advanced disease.

   A number of international, investigator-led Phase 3 trials of palbociclib in breast cancer are also ongoing.  For more information about ongoing clinical trials of palbociclib, please visit www.clinicaltrials.gov.

4. I am a physician and believe my patient may be a candidate for the palbociclib EAP.  How can I find out more information about participating?
   
   Physicians seeking more information about the palbociclib EAP can call 1-800-420-6755 or e-mail [email protected] for further details.  These channels are specifically for U.S.-based doctors who may have eligible patients.

5. What about patients who are not eligible to participate in this expanded access program?  How can they receive palbociclib?
   
   Patients should always discuss their treatment options with their physician.
   
   In addition to the palbociclib EAP, palbociclib is currently being evaluated in a comprehensive clinical development program that includes Pfizer-sponsored and investigator-initiated research in breast cancer and other cancers.  Patients or physicians seeking more information about available palbociclib clinical trial options can visit www.clinicaltrials.gov or contact Pfizer Medical Information at 1-800-438-1985 in the U.S.

6. Do patients participating in the palbociclib EAP have to pay for the drug?
   
   Palbociclib is an investigational therapy.  Patients in the U.S. who receive palbociclib through the expanded access program for advanced breast cancer will not pay for palbociclib while they are participating in the program. 
   
   Women enrolled to the study will be treated with palbociclib in combination with letrozole as per local standard of care.  Payment for letrozole therapy should be discussed with the patient’s insurance carrier.

7. What about patients outside the U.S.?  How can they receive palbociclib?
   
   Patients should always discuss their treatment options with their physician.
   
   Palbociclib is currently being evaluated in a comprehensive, international clinical development program that includes Pfizer-sponsored and investigator-initiated research in breast cancer and other cancers.  Patients or physicians seeking more information about local palbociclib clinical trial options can visit www.clinicaltrials.gov.

   If participation in a clinical trial is not an option, physicians are able to request palbociclib for single-patient compassionate use by e-mailing [email protected].  All requests for access to palbociclib through the compassionate use program must come through a treating physician.