Why Real-World Learnings Matter for People with Breast Cancer
When people understand basic concepts about their health, they’re better able to make more informed decisions. That’s why we are working to improve health literacy, clarify the language that we use around health topics, and return clinical trials data to participants.
A breast cancer diagnosis is life-changing; many people facing this diagnosis look to gather as much information about their prognosis and treatment options as possible. We know it’s not enough for us to simply develop breakthrough medicines. We must help patients understand cancer and cancer care to help them navigate the decisions they are facing.
The same holds true for the healthcare providers responsible for delivering care for cancer patients: The more information they have the better prepared they are to help patients address challenges and determine an appropriate treatment plan.
Later this month, Pfizer will present new findings from real-world studies regarding the treatment of patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer at the San Antonio Breast Cancer Symposium (SABCS), a scientific conference which highlights the latest research in breast cancer. Today we’re discussing the value of real-world evidence – together with clinical research – and the way it’s gathered.
We know that for both patients and healthcare providers, it’s helpful to understand the different ways that medicines can be studied.
Understanding randomized clinical trials
Randomized clinical trials (RCTs) are a type of controlled research study that can be used to evaluate if a treatment works. These study designs are considered the “gold standard” for testing new treatments.i RCTs are designed to show causality,ii and their results are generally used by regulatory authorities to determine if a medicine is safe and effective.ii
In an RCT for a potential new treatment, participants who meet specific eligibility criteria are randomly assigned to one of two or more different groups, where one group receives an established standard-of-care treatment and others receive the treatment being studied.i There are strict design criteria in RCTs to help researchers clearly evaluate findings by controlling other variables that can impact the outcome; for example, prior treatments or conditions that could independently impact the patients’ likelihood of responding or long-term survival.i
While RCTs are essential for understanding the risks and benefits of a treatment, there are some limitations.i The design criteria and other barriers to participation have led to certain communities being historically underrepresented in clinical trials for breast cancer, including older individuals,iii those with other health conditions,iv racial and ethnic minorities,v,vi and people living in underserved and rural areas.vii
Learning From Real-World Insights
While RCTs in breast cancer remain critical to evaluating if a medication can work and its risks, it is also important to gather real-world evidence (RWE).viii RWE is information about patients’ experiences with a treatment after it is approved, in everyday clinical practice. It can be developed from data collected during the course of routine care from medical records, payment claims, patient-reported information and prospectively collected by investigators once treatment has been decided.xiv,ix
The information from real-world studies can provide understanding of the effectiveness and safety of a medicine in a more diverse group of patients beyond RCTs. However, there needs to be appropriate context and acknowledgment of the limitations of real-world data and its interpretation. These limitations include that the patients are not randomly assigned to a treatment, therefore if a comparison is made between groups, statistical tools to adjust for differences in the patients’ demographic and clinical characteristics must be made to decrease the impact of bias. Other limitations include missing or inaccurate capture of data and the inability to adjust for a potential unknown variable that can influence treatment choice and outcomes.x Even though real-world studies are unable to determine causality, they offer an important complement to RCTs.xi
“Real-world evidence is a valuable tool to help us understand how a treatment may work in everyday medical practice,” said McRoy. “Although real-world evidence does not replace clinical trials, it can help fill data gaps in knowledge and inform personalized treatment plans.”
Pfizer’s Leadership in Generating Real-World Evidence
Pfizer is committed to the generation and use of RWE in breast cancer. Our real-world studies include data from over 20,000 people around the world,xii which enable Pfizer to expand upon the evidence from randomized clinical trials and gather important insights about how our breast cancer treatments work across a broader group of patients.
At the upcoming SABCS meeting, Pfizer will present new real-world data for its breast cancer treatments as well as updated findings from clinical research. The presentations include a U.S. study featuring the largest real-world dataset to date comparing the effectiveness of several CDK4/6 inhibitors, commonly used treatments for HR+, HER2− metastatic breast cancer. Additional presentations include data from around the world evaluating the effectiveness of Pfizer’s breast cancer treatments in routine medical practice among people often underrepresented in clinical research, including women aged 70 and older with significant comorbidities.
“Pfizer can learn something from every person who takes one of our breast cancer medicines,” said McRoy. “By combining real-world data with clinical research, we can help advance the care of people with breast cancer and ensure that healthcare professionals have the insights they need to make informed treatment decisions with their patients.”
i Zabor E, Kaizer A, Hobbs B. Randomized Clinical Trials. Chest. 2020;158(1S):S79-S87. doi:10.1016/j.chest.2020.03.013
ii U.S. Food and Drug Administration. Development & Approval Process | Drugs. https://www.fda.gov/drugs/development-approval-process-drugs
iii Dotan E, Lynch SM, Ryan JC, Mitchell EP. Disparities in care of older adults of color with cancer: A narrative review. Cancer Medicine. 2024;13(3):e6790. doi:10.1002/cam4.6790
iv National Institute on Minority Health and Health Disparities. Diversity and Inclusion in Clinical Trials. https://www.nimhd.nih.gov/resources/understanding-health-disparities/diversity-and-inclusion-in-clinical-trials.html
v American Cancer Society. Cancer Facts and Figures for African American/Black People 2022-2024.
https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/cancer-facts-and-figures-for-african-americans/2022-2024-cff-aa.pdf
vi Oyer RA, Hurley P, Boehmer L, et al. Increasing Racial and Ethnic Diversity in Cancer Clinical Trials: An American Society of Clinical Oncology and Association of Community Cancer Centers Joint Research Statement. J Clin Oncol. 2022;40(19)2163-2171. doi:10.1200/JCO.22.00754
vii Yabroff KR, Han X, Zhao J, Nogueira L, Jemal A. Rural Cancer Disparities in the United States: A Multilevel Framework to Improve Access to Care and Patient Outcomes. JCO Oncol Pract. 2020;16(7):409-413. doi:10.1200/OP.20.00352
viii Maruszczyk K, Aiyegbusi OL, Cardoso VR, et al. Implementation of patient-reported outcome measures in real-world evidence studies: Analysis of ClinicalTrials.gov records (1999-2021). Contemp Clin Trials. 2022;120:106882. doi:10.1016/j.cct.2022.106882
ix U.S. Food and Drug Administration. Real-World Evidence. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
x Katkade VB, Sanders KN, Zou KH. Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making. J Multidiscip Healthc. 2018;11:295-304. doi:10.2147/JMDH.S160029
xi Castelo-Branco L, et al. Ann Oncol. 2023;34:1097-1112.
xii Pfizer Data on File. November 2024.