When we talk about progress in myeloma, we often focus on the things that are easiest to measure: response rates, progression-free survival, overall survival, and a variety of laboratory results. These metrics are critically important and have helped drive remarkable advances in treatment over the past two decades. 

But as someone who has spent more than 20 years working alongside patients and families affected by myeloma, I can tell you that patients measure success differently. 

Patients care deeply about living longer. But they also care about how they live. 

How Treatments Feel and How They Affect Living 

Many myeloma treatments come with side effects that can have a profound impact on quality of life. Fatigue, peripheral neuropathy, diarrhea, recurrent infections, cognitive changes, and other treatment-related effects can affect a person’s ability to work, travel, participate in family activities, or simply enjoy everyday life. 

Yes, the myeloma may be controlled, but sometimes that control comes at a cost. 

For some people, it means being reluctant to leave the house because they are worried about finding a bathroom in time. For others, it means being unable to walk comfortably because of neuropathy or hesitating to hug a grandchild for fear of catching an infection. These realities do not always appear in clinical trial results, yet they are often the outcomes that matter most to patients and their families. 

This is why Patient-Reported Outcome Measures (PROMs) are so important. 

What Is PROMs? 

PROMs surveys allow patients to directly report how they feel, how they function, and how treatment affects their daily lives. This kind of data helps us understand the human impact behind the clinical data and ensures that decisions are informed not only by what treatments do to the disease, but also by what they do to the person. 

Why Are PROMs Important? 

At Myeloma Canada, we have long believed that patients must be involved in defining the outcomes that matter most. This belief is reflected in our own research on PROMs, which explored the importance of capturing the patient perspective throughout the treatment journey. The findings reinforced what many patients have been telling us for years: quality of life matters. Patients want treatments that not only extend life but also allow them to live that life as fully as possible. 

I encourage you to read Gabriele Colasurdo (Director of Science and Research)’s article, where he describes a recent PROMs review that Myeloma Canada participated in. 

The inclusion of meaningful patient-reported outcomes must begin early, during the design of clinical trials. If we are not measuring what matters to patients, we risk developing evidence that fails to capture the full impact of treatment. 

This is one of the reasons I serve on the Community Advisory Board of Myeloma Patient Europe (MPE). Through this work, I have the opportunity to provide direct input to pharmaceutical companies on the patient perspective as they develop their clinical trial programs. 

People sometimes ask why I participate through a European organization rather than solely through Canadian initiatives. The answer is simple. Decisions about the design of global clinical trial programs are rarely made in Canada. They are typically made by pharmaceutical company headquarters in Europe or the United States. If we want the patient voice to influence what outcomes are measured, which side effects are prioritized, and what questions are asked, we need to be present where those decisions are being made. 

Patient-reported outcomes are equally important in the growing field of Real-World Evidence (RWE) 

Around the world, healthcare decision-makers are increasingly using RWE to complement clinical trial data and support reimbursement, funding, and policy decisions. Real-world evidence helps us understand how treatments perform outside the controlled environment of a clinical trial and in the everyday lives of patients. PROMs are an essential part of that evidence because they reflect the actual perspective of the patient. This kind of data is patient-reported. 

Increasingly, health technology assessment bodies, payers, and healthcare systems are recognizing that patient-reported outcomes are not simply “nice-to-have” data points. They are critical evidence. 

The Real-World Impact of Real-World Evidence 

This week, I had the opportunity to participate in an educational session organized by the RWE4Decisions initiative, an international learning network that brings together health technology assessment bodies, payers, researchers, industry, and patient representatives to explore how real-world evidence can better support healthcare decision-making. The discussions focused on real examples where RWE has influenced decisions and on the ongoing work needed to improve the quality, consistency, and usefulness of these data. 

What struck me most was the growing recognition that stakeholders across the healthcare ecosystem are working toward a common goal: making better decisions informed by patients. There is still work to do, particularly in ensuring patients remain involved in defining what data are collected, how they are interpreted, and how they are ultimately used. But there is also a growing understanding that patient experience is not secondary evidence. 

I often reflect on a conversation I had years ago while working in the pharmaceutical industry. I asked a senior international oncology clinical trial leader how he incorporated the patient perspective into the trials he was designing. 

His answer was immediate: 

“That’s easy. I ask the doctor what the patient wants.” 

At the time, that response reflected a mindset that was still common across healthcare. 

Today, thankfully, we know better. 

We have learned that physicians are experts in treating disease. Researchers are experts in generating evidence. Regulators are experts in evaluating data. But patients are the experts in living with myeloma.The only way to understand what matters to patients is to ask patients directly. 

While there is still much work to do, we are making progress. More clinical trials are incorporating patient-reported outcomes. More healthcare decision-makers are considering patient experience in their evaluations. More healthcare systems are recognizing the value of real-world evidence. Most importantly, more patients are being invited to participate in shaping the decisions that affect their lives. 

At Myeloma Canada, we will continue to advocate at both the Canadian and international levels to ensure that the patient voice is not simply heard, but acted upon. Because when we measure what matters to patients, we make better decisions, develop better treatments, and ultimately move closer to our vision of a life uninterrupted by myeloma. 

Warmly,

Martine