ASH 2023: Advancing Potential Breakthroughs for Patients with Hematologic Disorders

This weekend, Pfizer will be joining the hematology/oncology community at the 65^th Annual Meeting of the American Society of Hematology (ASH), the premier professional society of clinicians and scientists dedicated to conquering blood diseases.

Blood diseases can disrupt a broad range of essential functioning – from providing the body with oxygen and nutrients, to fighting the growth of cancer, to preventing excessive bleeding. Pfizer is advancing novel approaches to blood diseases with next-generation treatments that could improve outcomes for patients while lessening the burden of disease on everyday life, and this year’s meeting marks significant progress toward that goal.

We are proud to present data from 10 Pfizer medicines in our growing hematology portfolio, including pivotal findings for people living with hemophilia A and B without inhibitors, initial Phase 2/3 results for a next-generation treatment in sickle cell disease (SCD), and the latest results from our multiple myeloma (MM) program, featuring extended follow-up data from our pivotal MagnetisMM-3 study.

These data will help advance the understanding of future medical breakthroughs and established medicines, as well as tackle the challenges patients living with these disorders face. Three key themes have emerged with this year’s data.

Rethinking established approaches to uncover the next wave of treatments

For decades, the most common treatment for hemophilia A and B has been factor replacement therapy infused intravenously, often multiple times per week. There have been notable medical advancements over this time – including several that Pfizer has been involved in through our more than 30-year history in the rare hematology space. But the burden that comes with managing the disease while maintaining bleed control remains a persistent challenge.

A U.S. survey of people living with hemophilia A and B receiving today’s standard of care found that only one-third of patients in the study had high treatment compliance – defined as taking 75% or more of their prescribed infusions. It also found that the time-consuming nature of the treatment was the most significant challenge.^i,ii Further, nearly three out of five people that took the less-than-prescribed number of infusions reported that time commitment was the primary reason for missing treatment.

This year, Pfizer will be presenting data on newer treatment mechanisms in hemophilia, which are being studied with the goal of establishing safety and efficacy of these investigational therapies while potentially reducing the burden of frequent infusions. We will also present research from a next-generation SCD treatment that will build on today’s standard of care.

Addressing health disparities for patients with blood disorders

Multiple myeloma and SCD are conditions that face stark racial disparities. Members of the Black community are twice as likely to be diagnosed with MM and twice as likely to die from it. Yet, they are often underrepresented in clinical trials, which leaves limited evidence-based data for doctors when making treatment decisions. People living with SCD – a condition most common in various minority populations – have also long been overlooked when it comes to medical research.

As part of Pfizer’s commitment to health equity, we have made concerted efforts to address these disparities. This includes diversifying our MagnetisMM clinical trial program, with support from our Diversity in Clinical Trials Center of Excellence, which better enables us to explore how specific racial and ethnic groups may respond to treatment with all the rigor of randomized controlled trials.

At ASH, we will present a new analysis of Black patients with relapsed or refractory MM (RRMM) from our MagnestisMM studies that explores the safety and efficacy of our MM treatment in these patients. This evidence may help healthcare professionals make more informed treatment decisions with their Black patients.

Pfizer’s work to reduce health inequity extends to our SCD advocacy and research as well. SCD is a lifelong, debilitating inherited blood disorder characterized by hemolytic anemia, which drives vascular inflammation, acute pain crises and progressive end organ damage, and occurs particularly among those of African, Middle Eastern, and South Asian descent.

Historically, there has been a high unmet need for therapies that address SCD and its acute and chronic complications. ASH will feature 14 Pfizer-led presentations specific to SCD, including data illustrating insights from social media listening about the experiences of patients and their caregivers.

Harnessing digital capabilities to gain medical insights

Pfizer is committed to addressing the needs of the underserved SCD community. At this year’s meeting we will present findings from a listening study which gathered insights across publicly accessibly social media in the UK, using artificial intelligence-powered theme detection through natural language processing to quantify prevalence and sentiment. We also partnered with data analysts and a patient living with SCD to develop search terms and conduct a human-led analysis to contextualize and validate the findings.

This novel method is a valuable tool that could advance our understanding of the real-life impacts of pain crises, access to care, and racial bias and stigmatization by analyzing candid online commentary. Researchers believe this method could potentially become useful for better understanding the experiences of people with other types of diseases in the future as well.

We look forward to sharing these collective findings at ASH this year as we continue our work to address the broad spectrum of patient needs across hemophilia, SCD and MM.

ⁱ Thornburg CD, Duncan NA. Treatment adherence in hemophilia. Patient Prefer Adherence. 2017;11:1677-1686  
https://doi.org/10.2147/PPA.S139851.  
ⁱⁱ Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001;7(4):392-6.