Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal. For many CMT patients, their condition is manageable and not worth the risk of potential harmful effects of drugs or other therapeutic options. However, many are terribly suffering and would benefit from participating in clinical studies that could have therapeutic benefits. In addition to safety and efficacy, the measurement of patient-reported outcomes is critical when clinical trials are developed for inherited neuropathies. It is well documented that for CMT (and many of the neuromuscular diseases), there are poor clinical outcomes measurement instruments and methods for validating the potential use of treatments for these diseases. HNF has developed two clinical studies that can increase the chances of successful future trials and ensure drug therapies will come to market for CMT.

Assuming the hurdles are overcome in the development of safe treatments, it’s still imperative to the success of therapy development to have valid primary and secondary outcomes measures.  Although there are some valid novel instruments such as, NIH’s PROMIS® and Neuro-QOL, in comparison to more established tools, such as the Child Health Questionnaire, Short Form- 12/36, and EQ-5D we still lack strong methods that measure QoL as well as outcomes measures for the disease.

Later this year, HNF will launch a health-related QoL clinical study in collaboration with three neuromuscular centers that will enhance clinical endpoints used for determining benefits or negative effects of potential treatments. Our goal is to develop a clear method for determining the clinical utility of potential treatments for CMT and related neuromuscular diseases. The study will be led by Dr. Renée J. Goldberg Arnold, PharmD, President and CEO, Arnold Consultancy & Technology LLC, NYC, where she develops and oversees outcomes research and affiliated software for industry and the federal government. Her special interest in evidence-based health derives from her research that deals with use of technology to collect and/or model real-world data for use in rational healthcare decision-making by healthcare practitioners and policy makers. Dr. Arnold’s academic titles include Adjunct Associate Professor, Master of Public Health (MPH) program, Department of Preventive Medicine at the Mount Sinai School of Medicine, where she has developed the pharmacoeconomics coursework and is a preceptor for MD/MPH students completing their MPH practicums. She is also Full Adjunct Professor, Div. of Social Sciences, at Long Island University College of Pharmacy and Health Sciences, including as a preceptor for students completing rotations in health outcomes and pharmacoeconomics. Dr. Arnold is a founding member of the International Society for Pharmacoeconomics and outcomes Research (ISPOR) and is an author/coauthor of numerous articles and book chapters in the areas of pharmacology, pharmacoeconomics and cost containment strategies. As Dr. Renee Arnold has adjunct faculty positions at several institutions (Mount Sinai, LIU) and has led multiple US-government, as well as commercially-funded studies, she is well qualified to provide a mutually supportive interaction between scientists conducting clinical research.

If you are interested in supporting our clinical study, please contact Allison Moore at [email protected].