Our patients have spoken: “We want earlier diagnoses to stop disease progression and better biomarkers for earlier diagnosis”
On March 8th, 2017, the Arthritis Foundation, US Food and Drug Administration (FDA), osteoarthritis (OA) patients, drug developers, health care providers and academic researchers came together to discuss the serious burden of OA disease, particularly the most significant OA-related symptoms, the impact of those symptoms, currently available treatments and ideal treatments.
The Voice of the Patient Report is a summary report resulting from this Arthritis Foundation externally-led OA Patient-Focused Drug Development meeting, a parallel effort to the FDA’s Patient-Focused Drug Development Initiative.
The purpose of this meeting was for the FDA to gain an up-to-date understanding of the treatment outcomes most meaningful to patients in the current landscape of OA therapy. This meeting provided key drug development stakeholders the opportunity to hear directly from patients, caregivers, and other patient representatives about their experiences with OA and its treatments. Results of this meeting suggested that current patient preferences are not merely for symptom modifying treatments; most importantly, a paramount patient priority is for disease modifying treatments that prevent disease early in its course and halt OA disease progression and onset of disability.
The patient input generated through this meeting strengthens the drug development community’s understanding of the burden of OA on patients and the burden of disease management with the treatments currently used to treat OA and its symptoms. Input was provided to directly support the FDA’s benefit-risk assessments for medical products under review. This input is also of value to the drug development process more broadly. For example, it may be useful to drug developers to consider the things that matter most to patients when defining clinical endpoints – dealing with the symptoms that affect a patient’s quality of life like fatigue, functional limitations, impaired mobility and pain while assessing the joint and structural changes.
Most notably, this report emphasizes the need for drug developers to prioritize the focus to disease prevention and stopping progression instead of merely treating symptoms which have no effect on improving the joint environment. Patients emphasized the dire need to explore options which limit disease progression or prevent it altogether. The results inform the design of clinical trials for OA to facilitate selection of outcomes now most meaningful to patients.