Real-World Evidence: Why Are We Still Waiting?

INTRODUCTION

Temporomandibular disorders (TMD) represent a profoundly challenging and often misunderstood set of conditions that deeply impact the lives of millions. As patients and advocates, we are deeply grateful to the editors of the International Journal of Translational Medical Research and Public Health for this opportunity to raise an urgent issue: the need for better evidence.

Patients understand that medical advancements carry uncertainties. What troubles us is not the unknown–it is the delays in discovering critical answers. Why aren’t the data we contribute as part of our care being used more effectively to shed light on treatment safety and efficacy?

I write this as a patient and advocate who has faced the consequences of inadequate evidence firsthand. Decades ago, I co-founded The Temporomandibular Joint Association (TMJA) to prevent others from enduring what I experienced: harm caused by treatments that lacked scientific evidence. The TMJA exists to demand better science for TMD and related conditions and to advocate for treatments that help rather than harm.

THE PROMISE OF REAL-WORLD EVIDENCE (RWE)

The 21^st Century Cures Act was a mixed blessing. By accelerating the approval of treatments and medical devices, it promised faster access to life-saving innovations while addressing inefficiencies in identifying adverse effects post-approval. Yet for temporomandibular joint (TMJ) patients–and many others–that promise remains unfulfilled. The infrastructure needed to generate meaningful RWE has not been fully realized.

Instead, we find ourselves grappling with the risks of an accelerated approval system. While the act shortened the time to market for treatments, it also reduced the number of patients required in clinical trials, leaving us with insufficient data to assess the long-term risks of these therapies. For TMJ treatments, this has led to devastating consequences – patients harmed by poorly tested devices and procedures.

BRIDGING THE GAP

The concept of RWE is not hypothetical. Coordinated Registry Networks (CRNs) already demonstrate their potential in specific medical areas. These networks bring together caregivers, researchers, and patients to collect and analyze high-quality data that not only tracks treatment safety and efficacy but also improves the quality of care. CRNs should be the gold standard for creating the evidence we need–but they remain underfunded and underutilized.

Over the past decade, billions of dollars have been invested in initiatives such as Sentinel and PCORnet, with mixed results. These approaches often lack the detailed data necessary for meaningful insights, like the unique device identifier. By contrast, CRNs provide a robust framework for generating actionable evidence, yet they have not received the level of investment required to expand their reach and impact.

A CALL TO ACTION

As we take stock of the progress made, it is clear that a strategic evaluation of RWE initiatives is overdue. Patients deserve evidence-based care informed by the most promising methodologies, such as CRNs. This requires bold leadership and a commitment to putting patients first – not only in rhetoric but also in action.

We commend the editors of this journal for dedicating this special issue to RWE. Their focus on bridging the gap between research and clinical practice aligns with what patients have been calling for science that translates into safer, more effective care.

IN MEMORIAM

This piece was drafted by Terrie Cowley and finalized after her passing by her close colleagues at The TMJA. Terrie was the co-founder and longtime president of The TMJA whose tireless advocacy was fueled by her personal experiences as a TMJ implant patient. Her efforts helped bring about transformative changes, including the establishment of the TMJ FDA Medical Device Epidemiology Network and the landmark 2020 report on TMD by the National Academies of Sciences, Engineering, and Medicine.

Terrie’s greatest legacy, however, lies in the countless lives she touched. She listened. She fought. She gave voice to those in pain. As we continue her work, we honor her life of purpose and passion — a beacon of hope and determination for TMJ patients everywhere.

CONCLUSION AND IMPLICATIONS FOR TRANSLATION

Temporomandibular Disorders demand evidence-driven action. Patients are ready to contribute, yet the infrastructure to transform real-world experience into reliable evidence remains underdeveloped. Coordinated Registry Networks offer a clear path to safer, more effective care, but will only occur with sustained investment and a commitment to patient centeredness. It is time to turn data into insight, and insight into treatments that truly help—putting patients, not uncertainty, at the center of care.