As corporate commitment and cost to develop a treatment for any Rare Disease increases, the risk for smaller biotech companies and even large pharma increases, too. So when I read this article outlining results from a Phase 1b Study of QR-010, I kept thinking about the next critical decisions and trial development ahead for them.
More specifically, I wanted to remind all companies brave enough to support the development of a rare disease treatment of the importance of involving their commercial team or an early asset or commercial development agency in developing and defining clinical trial endpoints correctly. Does the QR-010 team know the importance of including or not including a patient QOL tool like the CFQ-R in Phase 2 or 3? Is a change in sweat chloride still the most meaningful proof of concept to key stakeholders? Is there a clinical trial endpoint that can separate their product from others and motivate physicians to prescribe the treatment, or bolster the argument for access among payers, or be the differentiating reason patients and caregivers decide to choose the drug? Throughout my twenty years in pharmaceutical marketing research, clients continue to tell me about their need to ensure that the asset in development will have a unique characteristic that can be marketed. Yet, these same clients struggle to involve their commercial teams early enough in the study design process, when the clinical trial endpoints can be defined and refined in order to support claims that will establish the unique product advantages in the marketplace.
A number of exploratory efficacy endpoints were assessed in the multiple dose groups: respiratory symptoms (as measured by a validated patient-reported outcome tool, the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS), lung function (as measured by mean absolute change in percent predicted forced expiratory volume in 1 second, or ppFEV1), sweat chloride test and weight change