August 5, 2020
Measuring Clinical Outcomes
In most studies, it’s easy to know when an investigational drug or device is effective. You can count tumor cells in a sample, families can report the number of symptom free days, or you can count how many times a patient used a rescue medication. In rare neuromuscular disorders, it can be difficult to know how best to demonstrate efficacy. The symptoms and functional impacts can be entirely different for people of different ages, genotypes, and stages of disease progression. An outcome measure can perfectly capture a drug effect at one stage but completely miss the effect in another.
TRiNDS Study Measurements provides specialized neuromuscular expertise to help researchers choose and implement the most appropriate study outcomes. Our experts help researchers select the appropriate outcome measures for the planned study population during protocol design. We create training plans to guide how central trainers and site staff are trained to administer the outcome measures. During the study, we provide central data review to monitor whether site staff are administering and scoring the measures correctly over time. Our team provides refresher trainings and continuing education to study teams to keep their skills current throughout the study. We match our services to the unique needs of every trial sponsor and their sites to maximize data quality without duplication of efforts.
TRiNDS is a contract research organization (CRO) dedicated to conducting rare and neuromuscular disease research. We are currently accepting new clients developing products for rare disease orphan disease, and neuromuscular patient populations. Contact TRiNDS today to request a quote.
