The CHeT Outcomes focuses on developing and validating disease-specific patient-reported outcome measures (PROMs) for use in clinical trials and FDA drug-labeling claims. Dr. Heatwole’s group has developed 62 instruments, including short forms and translated versions in 10 languages.
Over the last 10 years, our PROMs have been extensively used in therapeutic trials worldwide, including those sponsored by academia, industry, and government and they have been recognized by the NIH common data elements initiate as a highly recommended way to measure a patient’s multifactorial burden of disease.
As therapies are developed, outcome measures that can reliably and sensitively detect meaningful changes in disease burden over time are critical. The FDA has identified disease-specific PROMs as a valid, responsive, and potentially ideal mechanism to measure therapeutic change during clinical trials. Our lab develops these instruments from the ground up to fully satisfy all FDA criteria.
Our instruments measure the multifaceted, patient-perceived disease burden in individual diseases. Our team of epidemiologists, biostatisticians, qualitative researchers, patient advocates, linguists, computer programmers, and neurologists has developed disease-specific PROMs including instruments for the following diseases:
- Congenital myotonic dystrophy
- Facioscapulohumeral muscular dystrophy
- Huntington’s disease
- Myotonic dystrophy type-1
- Myotonic dystrophy type-2
- Spinal muscular atrophy
- Charcot-Marie-Tooth disease