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Center for Health + Technology [CHeT]

URMC / Center for Health + Technology / Our Expertise / CHeT Outcomes

 

CHeT Outcomes

CHeT Outcomes

The CHeT Outcomes team specializes in developing and validating highly sensitive, disease-specific outcome measures, reported by patients (PRO: patient-reported outcomes) and caregivers (CR: caregiver-reported outcomes, also known as ObsRO: observer-reported outcomes). These measures are designed for use in therapeutic trials and FDA drug-labeling claims. Our group has developed and individually validated more than 200 disease-specific instruments and over 1000 subscales that quantify symptomatic disease burden during clinical trials. These instruments are capable of reliably measuring how a patient feels and functions, can reduce sample size requirements, and are designed to detect meaningful changes in health prior to traditional and generic outcome measures.

Our disease-specific instruments have demonstrated superior responsiveness in detecting clinically relevant changes in patient conditions compared to traditional measures. View our 5 Pivotal Studies to learn more about these findings.

Our group will collaborate with you to develop and fully validate a disease-specific outcome measure for any disease or provide consultation regarding outcome measure selection, use, optimization, and analysis. If you would like more information about how you can obtain a license to use our health indexes in your study, please contact HealthIndexes@chet.rochester.edu

 

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Our Instruments

Our instruments measure the multifaceted, patient-perceived disease burden in individual diseases. Our team of epidemiologists, biostatisticians, qualitative researchers, patient advocates, linguists, computer programmers, outcomes researchers, and physicians has developed patient-reported and caregiver-reported outcome measures for adult and pediatric populations, including instruments for the following diseases:

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Frequently Asked Questions

Dr. Chad Heatwole has ~20 years of experience in developing regulatory-level outcome measures for use in industry trials. As a clinical neurologist with NIH fellowship training in experimental therapeutics, Dr. Heatwole completed a master’s degree in clinical investigation focused on clinical outcomes development. Dr. Heatwole’s team comprises a vast network of subject matter experts, including patients, qualitative research specialists, biostatisticians, patient advocates, outcomes researchers, linguists, computer programmers, and physicians. Dr. Heatwole and CHeT have developed unparalleled expertise in the methodology for the development and validation of patient and caregiver-reported outcome measures. All of our instruments have been developed using rigorous regulatory guidelines for patient-reported (PROs) and caregiver-reported outcome measures (ObsROs) and have been designed to focus on what is most important to patients, quantify disease burden, be highly responsive to therapeutic intervention, and help support FDA drug labeling claims.
All of our patient-reported outcome (PRO) instruments are intended for the patient to complete the instrument independently and do not need to be administered by a trained coordinator or health provider. In this way, our instruments can be administered in-person or remotely. Many of our Health Indexes also have a caregiver reported (CR) outcome variation. These observer reported outcome measures (ObsRO) are intended for a caregiver or someone close to the patient to complete on the patient’s behalf.
While generic PROs are available for measuring disease progression in clinical trials, disease-specific outcome measures have higher precision, simpler application, better responsiveness, greater relevance, fewer ceiling effects, heightened sensitivity to detect clinically significant therapeutic changes over time when compared to generic instruments, and an ability to reduce clinical trial sample size/cost-efficiency. During prior therapeutic trials, our instruments have been more sensitive in detecting a clinically-relevant response to a therapy compared to other instruments including the SF-36, Neuro-QoL, and PROMIS-57 tools.
The average time to complete each of our instruments varies by disease and ranges from 5 minutes to 15 minutes. Each instrument has a short-form version, an abbreviated valid representation the total instrument, which can be completed in less than one minute. Unlike many other PROs, our Health Indexes are custom-designed to measure what is most important to patients with individual diseases. In general, the full version of each instrument is recommended for regulatory studies; however, our short forms provide versatility in instances where a shorter completion time is required or desired. Despite the completion time of our instruments, during head-to-head studies, patients have reported that they prefer our instruments (given their relevant content and similar application) to shorter generic and semi-generic instruments including the SF-36 and Individualized Neuromuscular Quality of Life Questionnaire (INQoL), and SymptoMScreen.
Our instruments have been used in over 100 studies. These have included studies sponsored by industry, academic groups, and foundations. The type of studies that utilized each of our instruments can be found on each instrument’s page at HealthIndexes.com.
In order to maximize the internal consistency of the subscales while maintaining theoretical justifiability, our instrument methodology does not permit changing the wording or order of questions. If you require a modification or fit-for-purpose version, let us know and we’ll discuss how we can help.
Fees vary based on the type of study, length, number of patients included, and other factors. We would be happy to discuss the licensing processes and fees with you based on your particular needs. Please contact our team at HealthIndexes@chet.rochester.edu.
Yes! Custom translations are available for all instruments and can be created for new languages as needed. To see existing language and cultural translations, click the specific instrument on HealthIndexes.com.
All instruments can be formatted for digital or paper use. We can work with your team for custom digital purposes, such as a direct to patient app or web version. Depending on the version, instruments range from 10 questions on the short forms up to about 80 questions for the full instrument. Each question has the same 6 point Likert scale responses which reduces participant burden.
Although each instrument’s questions are unique and specific to the population studied, the general structure is similar across all of the Health Indexes. If you are interested in obtaining a viewing license to review a complete instrument, please reach out to HealthIndexes@chet.rochester.edu.
Scoring is performed using a proprietary algorithm to enhance the meaningfulness and responsiveness of the instrument. The algorithm allows for the prioritization of the most important issues within a subscale as well as a total score to properly prioritize changes in the most important symptomatic areas. The algorithm also accommodates for missing data and allows for quantification of all individual areas of disease burden. All subscales are scored on a scale of 0 to 100 with 0 representing no disease burden and 100 representing the maximum level of disease burden. Subscale scores are also weighted to generate a total score (0-100) representing overall disease burden. Changes in individual subscale scores (representing a granular area of symptomatic burden) or changes in total disease burden (as measured by the total score) can be utilized and predetermined in a therapeutic study depending on the expected effects of a therapeutic agent.
All of our instruments are developed using extensive patient (and/or caregiver) input and validated using published FDA guidance. All instruments are developed using qualitative interviews, large cross-sectional studies, beta testing, statistical and psychometric analysis, reliability testing, and known groups testing. Each instrument undergoes extensive psychometric analysis and question selection optimization to maximize the responsiveness, reliability, and relevance of each of the questions and subscales.
Our work has been supported by the NIH, numerous foundations, the DOD, the CDC, New York State, and multiple pharmaceutical groups.
Yes, we have extensive expertise in working with industry and foundations to build disease-specific regulatory grade outcome measures to augment future clinical trials.
Our instrument subscales are designed to address all of the symptomatic areas that are most important for a particular disease. Individual diseases have a unique profile of symptomatic burden. Our multifactorial disease-specific outcome measures comprehensively measure and quantify this unique symptomatic profile for patients with select diseases. This provides an advantage over generic instruments which either do not measure the symptoms that are most important to a disease population or ask about symptoms that are not relevant to patients.
Yes! During the development of our Health Indexes, we validate each of the subscales as well as the total instrument. Internal consistency, reliability, standardized scoring, responsiveness, and known groups validity are optimized for each subscale.
Our Health Indexes total scores and subscale scores have been shown to strongly correlate with functional measures traditionally used in clinical trials, such as clinician assessment (e.g., strength and function test), laboratory evaluations (e.g., upper and lower extremity lean body mass, electromyographic myotonia), and patient-reported outcome measures (e.g., SF-36 physical-role score, INQoL score). In addition, all of our instruments are designed to be able to differentiate between populations with higher or lower disease burden.
Yes. Our instruments for myotonic dystrophy, spinal muscular atrophy, facioscapulohumeral muscular dystrophy (FSHD), Charcot-Marie-Tooth disease (CMT), and Huntington’s disease have all been used to show a beneficial response to a therapy during a clinical trial.
Yes. Our instrument for Huntington’s disease (HD-HI) was used in, and demonstrated a therapeutic effect of Valbenazine in a study of patients with Huntington’s disease.
On a case-by-case basis, Dr. Heatwole and his team are available to provide consultation regarding their outcome measures as well as outcome measure selection, outcome measure validation, outcome measure optimization, clinical trial design, regulatory pathways, and outcome measure analysis during therapeutic trial.
We would be happy to set up a time to talk. Please contact our team at HealthIndexes@chet.rochester.edu.