Twenty years ago, I wrote an article for The Hematologist describing evidence-based medicine (EBM), an approach to health care decision-making that prioritizes evidence from research studies over personal experience or expert opinion. Initially met with skepticism, EBM principles are now widely accepted and deeply integrated in the daily practice of medicine, including among hematologists. Many forces are behind this shift, including regulatory standards for drug and device approval, medical education standards, and the concerted efforts of the American Society of Hematology (ASH).

As a result of this shift, landmark clinical trials have been conducted, and numerous new therapies have been introduced for hematology, significantly increasing therapeutic options and elevating the quality of care for many blood diseases.

With these advancements in clinical trials, hematology has also been transformed through the development and dissemination of clinical guidelines based on systematic reviews of published evidence. In place of textbooks and review articles, rigorously developed guidelines have helped standardize the treatment of hematologic conditions, reducing variability in patient care and ensuring that treatment decisions are based on the best available evidence. These guidelines have also eased the pathway to payment for expensive medications and allowed for studies of second- and third-line therapies against a background of well-founded and evidence-based care. Further, the guidelines have established future research needs by defining what is known and what is uncertain.

ASH has significantly advanced evidence-based hematologic care over the past two decades as a key player in the field. Its major initiatives, including the introduction in 2014 of a robust initiative to build new quality improvement programs and 2018’s establishment of the ASH Research Collaborative (ASH RC), have been instrumental in shaping the landscape of evidence-based hematology.

Since 2014, ASH quality programs have evolved to encompass many efforts to improve health care quality, i.e., ensure that care is safe, effective, timely, patient-centered, and equitable. For more information, see ASH’s clinical practice guidelines and quality care resources at hematology.org/education/clinicians/guidelines-and-quality-care.

Guideline Development:

Since 2018, ASH has published more than 20 formal guidelines on various hematologic diseases, including venous thromboembolism (VTE), immune thrombocytopenia, sickle cell disease (SCD), von Willebrand disease, and acute myeloid leukemia. Guidelines on another five diseases are currently in development. ASH guidelines are highly trusted partly because of the rigorous methods used to develop them, including systematic reviews of available evidence, explicit consideration of patient values, and decision frameworks for translating evidence into recommendations. ASH guideline panels include experts from multiple disciplines, as well as patient representatives. Conflicts of interest are managed through panel composition, disclosure, and recusal. With state-of-the-art methods, these ASH panels produce clinical recommendations that are clear, actionable, and explicit about supporting evidence.

Implementation Tools and Resources:

ASH also focuses on practical application, providing a suite of tools and educational resources — such as teaching slide sets, patient resources, and pocket guides — that facilitate the implementation of guidelines in clinical settings.

Quality Measurement and Reporting:

Many best practices can be measured for accountability and improvement. ASH has developed clinical quality measures for the diagnosis of VTE and the timely treatment of acute pain in patients with SCD based on guideline recommendations.

Collaboration and Advocacy:

Through partnerships with health care providers, regulatory bodies, and patient groups, ASH ensures that evidence-based practices are broadly adopted and integrated into health care policy and practice.

The ASH RC represents a significant expansion of ASH’s commitment to advancing hematology through evidence-based practice. This initiative aims to improve patient care by leveraging high-quality, real-world evidence that complements data from controlled trials. The ASH RC also explores how patient-reported outcomes can be used to inform better care. For more information, visit the ASH RC website at ashresearchcollaborative.org.

The ASH RC and similar projects undertaken by other peer organizations represent an innovative method for generating high-quality evidence outside the framework of traditional prospective clinical trials. While clinical trials remain the gold standard for high-quality evidence, their cost and complexity limit the number of such studies. Further, there are many questions, particularly regarding real-world practices, that cannot be addressed using traditional clinical trial methodologies.

In its initial phase, the ASH RC has focused on two disease states: multiple myeloma and SCD. The ASH RC has invested in two programs within these disease states to address the challenges of producing high-quality evidence. The first is a network-based approach of clinicians, care team members, and clinical investigators with a deep culture of cooperative research dedicated to advancing therapeutics and accelerating the generation of high-quality evidence that improves our understanding of hematologic conditions. The second is the Data Hub, an essential component of these networks. The Data Hub collects and aggregates national longitudinal real-world data to generate high-quality evidence and advance hematology research. The true value of these programs is that they are seamlessly integrated, creating a dynamic, clinician-and-patient-powered research network continuously driven by real-world data, creating limitless possibilities for prospective evidence generation and aggregated data analyses.

EBM in hematology is poised for further growth, enhanced by technological advancements such as artificial intelligence (AI) and big data analytics. These tools promise to refine our understanding of vast datasets, uncovering novel insights that could lead to more personalized and effective treatments. AI tools also promise to make systematic review and analysis of published data more efficient, speeding guideline development and revision.

However, technological advances are only an enabler of success. Advancing evidence will require the medical community to collaborate and focus on improving patient care. Further, such projects require funding, from multiple sources, which will require changing how funders prioritize evidence generation. Although funding that supports evidence generation needs to continue and ideally be enhanced, there also needs to be significant funding to support platforms that allow us to fully use the evidence we currently have available, whether that is in the form of synthesizing clinical trial results or gathering and analyzing real-world data through efforts like ASH RC.

It seems impossible that I wrote my earlier article in The Hematologist at the start of ASH’s evidence-based journey two decades ago. ASH’s progress over the last 20 years has been nothing short of remarkable. With the datasets and tools now available, I anticipate great things for evidence-based hematological care.

Dr. Crowther indicated no relevant conflicts of interest.