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AboutAboutIBRANCE experienceMechanism of actionDosing & MonitoringDosing & MonitoringDosing and administrationMonitoringRecommended dose modifications for IBRANCEWhat you need to know about IBRANCE tabletsClinical Trial DataClinical Trial DataIBR + AI Efficacy & SafetyIBR + AI Additional AnalysesIBR + Fulvestrant Efficacy & SafetyIBR + Fulvestrant Additional AnalysesReal-World Evidence​​​​​​​Real-World EvidenceIntroductionRandomized controlled trialReal-world study designReal-world study patient characteristicsReal-world effectiveness dataLabelLinkLinkLinkLinkLabelLinkLinkLinkLinkVideosPatient SupportPatient SupportMaterialsCoverage and accessPersonalized patient support
Prescribing Information for CapsulesPrescribing Information for Tablets Indications Patient Site
Real-World EvidenceIntroduction 

Real-world evidence can complement data from randomized controlled trials. It is important to understand both the randomized controlled trial results and the limitations of real-world evidence. 

Review results from PALOMA-2, the randomized controlled trial that evaluated IBRANCE + letrozole in postmenopausal women with estrogen receptor-positive (ER+)/HER2- mBC and no prior treatment in the metastatic setting.1

Understand the Study Limitations (expand here to view)
  • This study is a retrospective database study of electronic health records, which may have missing data (e.g., ECOG baseline scores [~30%]) or erroneous data entry 
  • Patients were not randomly assigned. Treatments were selected by prescribing physician
  • A causal relationship between treatments and outcomes cannot be determined
  • While statistical analyses* were used to balance baseline and clinical patient characteristics, unobserved variables cannot be fully addressed through these methods. The analysis may not adequately adjust for all variables (e.g., interval from initial breast cancer diagnosis to mBC diagnosis)
  • This analysis may not adjust for all sources of potential bias (e.g., immortal time bias)
  • May not fully account for intercurrent events (e.g., dose modifications, titration, ancillary and concomitant therapies)
  • Findings presented here may not be generalizable to patient populations not represented in the Flatiron Database2
  • Safety data were not collected as part of the study
Statistical analyses included sIPTW as the primary analysis and PSM as the sensitivity analysis.
Randomized Controlled Trials Are the Gold Standard and Can Be Supplemented by Real-World Data

Well-conducted RCTs are the gold standard for evaluation of efficacy and safety of a new drug. Due to narrow inclusion criteria for RCTs, real-world evidence may provide HCPs with helpful information about a drug in a large, heterogeneous population and among patients who would not have met clinical trial inclusion criteria.3,4 

RCTs: Gold Standard5
  • Designed to show causality6
  • Patients randomly assigned to treatment or comparator6,7
  • Data derived from prespecified, protocol-defined, uniformly assessed endpoints8
  • Designed to eliminate systematic biases when comparing drug treatments9
  • Highly monitored, controlled environment designed to control variability and maximize data quality7,8,10
  • Measure efficacy of an intervention (i.e., treatment performance under highly controlled conditions)8

  • Controlled patient inclusion and exclusion criteria often result in a narrower subset of patients than those encountered in routine clinical care11-14

RWE: Complements RCT Findings15

Example

  • Assess association; unable to determine causality4,6
  • Patients are not randomized; although statistical analyses may be used to balance patient characteristics, bias related to treatment selection and unobserved variables may not be fully addressed4,8
  • Based on endpoints as defined for a real-world setting, which may rely on a treating physician's clinical assessment and not standard RCT criteria4
  • Potential variations in follow-up and adherence in patients seen in routine clinical practice4
  • RWD sources may have missing data and erroneous data entry4,16
  • Measure the effectiveness of an intervention (i.e., treatment performance under real-world conditions)4
  • May include patients excluded or underrepresented in RCTs; in terms of age, race, socioeconomic status, geography, clinical setting, and disease severity or due to patient history and patient willingness to seek treatment12
Observational retrospective analyses are not intended for direct comparison with clinical trials and may introduce bias. NEXT: Randomized controlled trial summary See the data LoadingReferences:Finn RS, Martin M, Rugo HS, et al. Palbociclib and letrozole in advanced breast cancer. N Engl J Med. 2016;375(20):1925-1936. doi:10.1056/NEJMoa1607303Rugo HS, Brufsky A, Liu X, et al. Real-world study of overall survival with palbociclib plus aromatase inhibitor in HR+/HER2- metastatic breast cancer. NPJ Breast Cancer. 2022;8(1):114. doi:10.1038/s41523-022-00479-xGyawali B, Parsad S, Feinberg BA, Nabhan C. Real-world evidence and randomized studies in the precision oncology era: the right balance. JCO Precis Oncol. 2017;1:1-5. doi:10.1200/PO.17.00132DeMichele A, Cristofanilli M, Brufsky A, et al. Comparative effectiveness of first-line palbociclib plus letrozole versus letrozole alone for HR+/HER2- metastatic breast cancer in US real-world clinical practice. Breast Cancer Res. 2021;23(1):37. Published online March 24, 2021. doi:10.1186/s13058-021-01409-8Subbiah V. The next generation of evidence-based medicine. Nat Med. 2023;29(1):49-58. Khozin S, Blumenthal GM, Pazdur R. Real-world data for clinical evidence generation in oncology. J Natl Cancer Inst. 2017;109(11):1-5. doi:10.1093/jnci/djx187Booth CM, Tannock IF. Randomised controlled trials and population-based observational research: partners in the evolution of medical evidence. Br J Cancer. 2014;4;110(3):551-555.Singal AG, Higgins PDR, Waljee AK. A primer on effectiveness and efficacy trials. Clin Transl Gastroenterol. 2014;5(1):e45. doi:10.1038/ctg.2013.13US FDA. Designing Sound Clinical Trials That Incorporate Real-World Data. Accessed January 23, 2024. https://www.fda.gov/drugs/regulatory-science-action/designing-sound-clinical-trials-incorporate-real-world-dataUS FDA. Framework for FDA's Real-World Evidence Program. Accessed January 23, 2024. https://fda.gov/media/120060/download?attachmentMaissenhaelter BE, Woolmore AL, Schlag PM. Real-world evidence research based on big data: Motivation-challenges-success factors. Onkologe (Berl). 2018;24(Suppl 2):91-98.Beaulieu-Jones BK, Finlayson SG, Yuan W, et al. Examining the use of real-world evidence in the regulatory process. Clin Pharmacol Ther. 2020;107(4):843-852.Blonde L, Khunti K, Harris SB, et al. Interpretation and impact of real-world clinical data for the practicing clinician. Adv Ther. 2018;35(11):1763-1774.Webster J, Smith BD. The case for real-world evidence in the future of clinical research on chronic myeloid leukemia. Clin Ther. 2019;41(2):336-349. Castelo-Branco L, Pellat A, Martins-Branco D, et al. ESMO guidance for reporting oncology real-world evidence (GROW). Ann Oncol. 2023;34(12):1097-1112.Carroll OU, Morris TP, Keogh RH. How are missing data in covariates handled in observational time-to-event studies in oncology? A systematic review. BMC Med Res Methodol. 2020;20(1):134. To view a copy of the license for Rugo HS, Brufsky A, Liu X, et al (NPJ Breast Cancer. 2022;8(1):114), please visit https://creativecommons.org/licenses/by/4.0/
Real-World Evidence

Access the publication.
The FDA-approved Prescribing Information for IBRANCE includes data from the PALOMA clinical trials. However, some data reported in this publication may be different from, or are not included in, the IBRANCE Prescribing Information. Please review results from PALOMA-2 (RCT) provided on this page. The study met its primary endpoint of progression-free survival but did not meet the secondary endpoint of overall survival.

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Indications IBRANCE® (palbociclib) 125 mg capsules and tablets are indicated for the treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer (mBC) in combination with:
 
  • an aromatase inhibitor as initial endocrine-based therapy, or
  • fulvestrant in patients with disease progression following endocrine therapy
Important Safety Information Neutropenia was the most frequently reported adverse reaction in PALOMA-2 (80%) and PALOMA-3 (83%). In PALOMA-2, Grade 3 (56%) or 4 (10%) decreased neutrophil counts were reported in patients receiving IBRANCE plus letrozole. In PALOMA-3, Grade 3 (55%) or Grade 4 (11%) decreased neutrophil counts were reported in patients receiving IBRANCE plus fulvestrant. Febrile neutropenia has been reported in 1.8% of patients exposed to IBRANCE across PALOMA-2 and PALOMA-3. One death due to neutropenic sepsis was observed in PALOMA-3. Inform patients to promptly report any fever.

Monitor complete blood count prior to starting IBRANCE, at the beginning of each cycle, on Day 15 of first 2 cycles and as clinically indicated. Dose interruption, dose reduction, or delay in starting treatment cycles is recommended for patients who develop Grade 3 or 4 neutropenia.
 

Severe, life-threatening, or fatal interstitial lung disease (ILD) and/or pneumonitis can occur in patients treated with CDK4/6 inhibitors, including IBRANCE when taken in combination with endocrine therapy. Across clinical trials (PALOMA-1, PALOMA-2, PALOMA-3), 1.0% of IBRANCE-treated patients had ILD/pneumonitis of any grade, 0.1% had Grade 3 or 4, and no fatal cases were reported. Additional cases of ILD/pneumonitis have been observed in the post-marketing setting, with fatalities reported.
 

Monitor patients for pulmonary symptoms indicative of ILD/pneumonitis (e.g., hypoxia, cough, dyspnea). In patients who have new or worsening respiratory symptoms and are suspected to have developed pneumonitis, interrupt IBRANCE immediately and evaluate the patient. Permanently discontinue IBRANCE in patients with severe ILD or pneumonitis.
 

Based on the mechanism of action, IBRANCE can cause fetal harm. Advise females of reproductive potential to use effective contraception during IBRANCE treatment and for at least 3 weeks after the last dose. IBRANCE may impair fertility in males and has the potential to cause genotoxicity. Advise male patients to consider sperm preservation before taking IBRANCE. Advise male patients with female partners of reproductive potential to use effective contraception during IBRANCE treatment and for 3 months after the last dose. Advise females to inform their healthcare provider of a known or suspected pregnancy. Advise women not to breastfeed during IBRANCE treatment and for 3 weeks after the last dose because of the potential for serious adverse reactions in nursing infants. 
 

The most common adverse reactions (≥10%) of any grade reported in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (80% vs 6%), infections (60% vs 42%), leukopenia (39% vs 2%), fatigue (37% vs 28%), nausea (35% vs 26%), alopecia (33% vs 16%), stomatitis (30% vs 14%), diarrhea (26% vs 19%), anemia (24% vs 9%), rash (18% vs 12%), asthenia (17% vs 12%), thrombocytopenia (16% vs 1%), vomiting (16% vs 17%), decreased appetite (15% vs 9%), dry skin (12% vs 6%), pyrexia (12% vs 9%), and dysgeusia (10% vs 5%).
 

The most frequently reported Grade ≥3 adverse reactions (≥5%) in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (66% vs 2%), leukopenia (25% vs 0%), infections (7% vs 3%), and anemia (5% vs 2%).
 

Lab abnormalities of any grade occurring in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were decreased WBC (97% vs 25%), decreased neutrophils (95% vs 20%), anemia (78% vs 42%), decreased platelets (63% vs 14%), increased aspartate aminotransferase (52% vs 34%), and increased alanine aminotransferase (43% vs 30%). 

 

The most common adverse reactions (≥10%) of any grade reported in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (83% vs 4%), leukopenia (53% vs 5%), infections (47% vs 31%), fatigue (41% vs 29%), nausea (34% vs 28%), anemia (30% vs 13%), stomatitis (28% vs 13%), diarrhea (24% vs 19%), thrombocytopenia (23% vs 0%), vomiting (19% vs 15%), alopecia (18% vs 6%), rash (17% vs 6%), decreased appetite (16% vs 8%), and pyrexia (13% vs 5%).
 

The most frequently reported Grade ≥3 adverse reactions (≥5%) in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (66% vs 1%) and leukopenia (31% vs 2%). 
 

Lab abnormalities of any grade occurring in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were decreased WBC (99% vs 26%), decreased neutrophils (96% vs 14%), anemia (78% vs 40%), decreased platelets (62% vs 10%), increased aspartate aminotransferase (43% vs 48%), and increased alanine aminotransferase (36% vs 34%). 
 

Avoid concurrent use of strong CYP3A inhibitors. If patients must be administered a strong CYP3A inhibitor, reduce the IBRANCE dose to 75 mg. If the strong inhibitor is discontinued, increase the IBRANCE dose (after 3-5 half-lives of the inhibitor) to the dose used prior to the initiation of the strong CYP3A inhibitor. Grapefruit or grapefruit juice may increase plasma concentrations of IBRANCE and should be avoided. Avoid concomitant use of strong CYP3A inducers. The dose of sensitive CYP3A substrates with a narrow therapeutic index may need to be reduced as IBRANCE may increase their exposure.
 

For patients with severe hepatic impairment (Child-Pugh class C), the recommended dose of IBRANCE is 75 mg. The pharmacokinetics of IBRANCE have not been studied in patients requiring hemodialysis

Please see full Prescribing Information for IBRANCE capsules and tablets.

IndicationsIBRANCE® (palbociclib) 125 mg capsules and tablets are indicated for the treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer (mBC) in combination with:
  • an aromatase inhibitor as initial endocrine-based therapy, or
  • fulvestrant in patients with disease progression following endocrine therapy