Development of Pediatric Illustrations for Concept Elicitation Interviews

In 2018, researchers at Endpoint Outcomes embarked on an exercise to develop illustrations to assist pediatric research participants in communicating their disease experience to our interviewers.  A list of concepts commonly found in pediatric diseases was developed based on a review of generic pediatric health-related quality of life measures.  Draft illustrations were initially developed by a professional illustrator to reflect the core concepts.  The draft illustrations were then reviewed by staff at Endpoint Outcomes to ensure they reflect the relevant concepts and illustrations were subsequently altered if necessary. The illustrations have been developed to be gender and ethnicity/race neutral and to focus on disease deficits.  Currently, the illustrations are being pilot tested in two pediatric studies in rare diseases. A formal evaluation of the content validity of the measures is planned for later in the year. For additional information on this work please contact Shayna Egan:

The Patient’s Perception of Burden of Disease in Pyruvate Kinase Deficiency

A manuscript co-authored by Shayna Egan, Aisling Ryan, Ted Wells and Brooke Witherspoon was recently accepted by the European Journal of Haematology. The article titled, The Burden of Disease in Pyruvate Kinase Deficiency: Patients’ Perception of the Impact on Health-Related Quality of Life, is currently available in Epub ahead of print.

Boston Company Retreat

The Long Beach and Boston offices came together for a week long retreat to discuss current developments and methodological issues in our field: patient experience data, regulatory issues, classical and modern psychometrics, interpretation of meaningful change on PROs, interviewing difficult research participants, placebo response in clinical trials, wearable devices, readability testing, the use of cartoon illustrations in pediatric studies and reporting of cognitive interview results.  The retreat concluded with a cruise around the Boston harbor and tours of several local craft breweries.


The Patient’s Perspective on Neuropathic Pain

A recent publication co-authored by Aisling Ryan, Ted Wells and Chris Evans discusses the development of a conceptual model of symptoms and impacts in neuropathic pain in the US and Japan: Hwang S, van Nooten F, Wells T, Ryan A, Crawford B, Evans C, English M.  Neuropathic pain: a patient-entered approach to measuring outcomes.  Health Expectations 2018;1-13.

Novel migraine-specific PRO being presented at the American Headache Society

Mary Lynn Cala, Nicole Lyn, Christina Graham and Chris Evans are co-presenters of a poster at the 60th Annual Scientific Meeting for the American Headache Society in San Fransisco from June 28 to July 1, 2018. The poster presentation is about a novel migraine-specific PRO measure to assess functioning based on activity impairment related to migraine in episodic and chronic migraine patients. The other co-authors are affiliated with Allergan, Albert Einstein College of Medicine and the Mayo Clinic.

23rd Annual ISPOR meeting in Baltimore on May 19th-23rd

Shayna Egan, Martha Gauthier, Aisling Ryan and Mary Lynn Cala will be presenting two posters at the 23rd Annual ISPOR meeting in Baltimore in May.  Both posters highlight methodological work we have been conducting in hopes of contributing to advancements in the field of clinical outcomes assessment research. The topics include:

  • Do words matter? Patient perspectives on conceptually similar concepts
  • Patient definitions of improvement across four disease areas

More information about the posters will be included on the Publications section of the website following the conference in May.

Welcome to Endpoint Outcomes

Endpoint Outcomes is pleased to welcome Michelle Borges to our office in Boston as a senior research coordinator.  Prior to joining Endpoint Outcomes, Michelle worked as a Research Technician at Boston Medical Center on a clinical trial testing the effectiveness of delivering diabetes self-management education using virtual world technology versus a face-to-face medical group visit format.  Michelle also has research experience on studies identifying methods to advance transitional care services for patients and caregivers, reduce hospital readmission rates among patients with depression, and feasibility testing of a cognitive behavioral therapy intervention delivered by relational agent technology.  Michelle holds a Bachelor of Science degree in Biological Anthropology and a Bachelor of Arts degree in Spanish from the Pennsylvania State University and a Master of Arts in Medical Anthropology and Cross Cultural Practice from Boston University School of Medicine.

Time Up for Traditional Bladder Diaries?

A recent publication co-authored by Nathan Johnson, Zoe Kopp, Shayna Egan and Chris Evans details our international collaboration to find a potential replacement for burdensome daily diaries in overactive bladder trials: Kelleher C, Chapple C, Johnson N, Payne C, Homma Y, Hakimi Z, Siddiqui E, Evans C, Egan S, Kopp Z. Development of an overactive bladder assessment tool (BAT): a potential improvement to the standard bladder diary.  Neurology and Urodynamics 2018; 1-10.

2017 Year End Wrap Up

2017 was a busy year for Endpoint Outcomes.  We continued our extensive instrument development activities to support sponsors in developing labeling claims with COAs. We cruised through our 2,500th patient interview and are well on our way to our 3,000th interview.  One of the most rewarding aspects to our work in 2017 was our research in rare/orphan diseases where we hear directly from patients and their caregivers on the major impacts that their diseases have on their daily lives.  We have also been active in innovative psychometric research and continue to expand our work in consensus generating techniques through Delphi panels to develop consensus recommendations and screening tools. Finally, it was our pleasure to work with Grace Cares and Project Hearts to help improve public health in the Dominican Republic.

Moving forward in 2018 we are looking at expanding our operations in Boston again and building up our psychometric team under the leadership of Dave Andrae. We look forward to seeing everyone at ISPOR, ISOQOL and C-Path in 2018.

Drug Development Tool Qualification Continues in Idiopathic Pulmonary Fibrosis

Nathan Johnson, Chris Evans and Aisling Ryan continue their work with their colleagues to advance the Living with Idiopathic Pulmonary Fibrosis (L-IPF) tool through the FDA’s qualification process.  The most recent results of that research were presented as an Encore presentation at the Pulmonary Fibrosis Foundation Summit in Nashville on November 9, 2017.