Working group 4: Benefit/Risk assessment
Ensuring the safety, effectiveness and quality of human drugs is an increasingly complicated task that requires not only consideration of a multitude of complex factors but integration of multiple stakeholders’ perspective. Current thinking, including that of regulatory agencies, recognizes that the clinical trial review process could benefit from a more systematic and expansive approach to obtaining the patient and caregivers perspective on disease severity and current available options in a therapeutic area. Patients are uniquely positioned to inform regulators` understanding of clinical context, i.e., the unmet needs and meaningful impacts of a treatment for PWS, and contribute to the benefit / risk analysis.
The overall goal is to support the patients and caregivers` perspectives by using rigorous, generalizable and quantifiable risk-benefit assessment tools that will serve as scientific evidence. Understanding how disease severity vary across individuals with PWS, what matters to the patients and caregivers , and how PWS affects their quality of life will help regulators and researchers to better respond to unmet patient`s and caregivers` needs.
Specific aims include:
Aim 1: Measure the social, financial and psychological impact of PWS on caregivers.
Aim 2: Develop a video to illustrate what is PWS and how it impacts patients and caregivers
Aim 3: Assess patients and caregivers perspectives on benefits and risks regarding experimental treatments using rigorous scientific methods to document the priorities and preferences of patients and caregivers (J. Bridges, Ph.D., Johns Hopkins University).