Clinical Research: Phase 1 - Phase 4

Applied Clinical Trials – Patient Engagement and Advocacy: Advancing the Cause of Clinical Drug Research

Duchenne muscular dystrophy (DMD) patients almost universally lose the ability to walk as the disease progresses. Patients generally understand that loss of ambulation is to be expected, and as was learned from a patient preference study, they tend to put greater priority on maintaining use of their hands. Yet for many years, DMD drug trials included a specific endpoint requiring that they enroll only patients who were still able to walk—denying eligibility to a potentially large number of participants.

That changed when a DMD-focused advocacy organization took the matter up with the Food and Drug Administration (FDA) and helped develop new trial guidelines that allow some non-ambulatory patients to participate in these trials.

Engaging patients in clinical trial design is essential to ensuring that researchers devise relevant criteria, establish realistic expectations, and pursue goals that are meaningful to the study participants who are central to the success of these costly and risky endeavors. The expanding number and influence of patient advocacy groups in recent years—the National Organization for Rare Disorders lists nearly 1,900 such organizations on its website—underscore the fact that patients are not passive subjects, but people who approach clinical trials with varying degrees of hope, fear, doubt, skepticism, anxiousness, and countless other emotions.

Some may find symptom relief or even a disease modifying therapy, while others won’t have the same outcomes. Some recognize that the study drug won’t benefit them personally, but enroll so they might help others. What unites patients and families who participate in clinical trials is their willingness to sacrifice their time, energy, and so much more for an exercise whose outcome is uncertain at best.

Read more at Applied Clinical Trials.