Narcolepsy and Cataplexy in Children

Endpoint Outcomes has recently published a qualitative study that examined the concepts important for incorporation into a daily diary of cataplexy frequency and the content validity of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD).

New Senior Director of Statistics and Psychometrics

Endpoint Outcomes is pleased to welcome Dave Andrae as Senior Director of Statistics and Psychometrics.  Dave joins Endpoint Outcomes from Allergan where he was previously Executive Director, Centers of Excellence for Real World Evidence and Economic Modeling, Global Value and Evidence Development.  Previous work experience includes Senior Director of Biostatistics and Psychometrics at Furiex Pharmaceuticals and various roles, with increasing responsibility, at PPD.  Dave is a graduate of the University of Iowa (BS in psychology) and the University of Nebraska (MA and PhD with a focus on cognitive psychology and quantitative research methods). Dave has published and presented widely in the field of outcomes research.

New Itch Measure for Observers to Support Product Labeling

Martha, Nathan and co-authors recently reported on a qualitative study they worked on to develop a measure of itching in pediatric cholestasis: Kamath BM, Abetz-Webb L, Kennedy C, Hepburn B, Gauthier M, Johnson N, Medendorp S, Dorenbaum A, Todorova L, Schneider BL.  Development of a novel tool to assess the impact of itching in pediatric cholestasis. Patient 2017 July 14. Doi: 10.1007/s40271-017-0266-4.

ISPOR Conference

Shayna Egan, Mary Lynn Cala, Alp Tutuncu, Aisling Ryan, Brooke Witherspoon, Daniel Serrano and Chris Evans will be presenting several posters at the 22nd Annual ISPOR meeting in Boston in May.  For this ISPOR we decided to skip the traditional posters, presentations and workshops on instrument development and validation work and present some methodological work we have been conducting which we hope will advance the field some. The topics included:


  • Proximal versus distal impacts of disease: drivers of bothersomeness ratings in patient-reported outcomes studies.
  • What is being measured in patient-reported outcome measures? Symptom questions may often reveal impact answers.
  • Are patients’ responses to five point verbal descriptor scales and visual analogue scales replicate measures? An experimental manipulation.
  • Pain assessment: a myriad of techniques to measure pain severity – does it matter which one is used?

Delphi Panel to Develop Consensus Recommendations for the Treatment of Diabetic Macular Edema

In 2016, Nathan Johnson, Nicole Lyn and Chris Evans worked on conducting a Delphi panel with leading retina experts. The Delphi panel was somewhat unique in that the first round was conducted on SurveyMonkey and the second round was conducted in “real time” utilizing an electronic audience response system (while still preserving the anonymity of responses).  The results of panel were recently published in Ophthalmic Surgery Lasers and Imaging Retina:

Patients in Early Stage Research and Development

Chris Evans was pleased to support Grunenthal in their commitment to bring the patient’s voice in early drug development in a patient summit meeting held in Amsterdam in March.

As part of the summit meeting, breakout sessions were held to discuss pain descriptors, communication needs of patients and the need for a PRO in vulvodynia.  Chris participated in a session that looked at patient complaints in the area of vulvodynia where questions were asked of patients and patient advocates and an illustrator drew the main concepts described. Photo with permission of Grunenthal.  Illustration by


Bladder Assessment Tool

Nathan Johnson, Zoe Kopp and Chris Evans will be showcasing the results of our on-going work to develop a new, more comprehensive bladder assessment tool to be used in clinical trials at the EAU17 Conference in March in London.

Endpoint Outcomes, GRACE Cares and Project Hearts

The staff from Endpoint Outcomes was pleased to participate in a service project with Grace Cares and Project Hearts in the Dominican Republic.  Following a company meeting, the staff travelled to Baitoa to work with the staff of Project Hearts in installing water tanks, gutters and a foundation to a building addition.  Many thanks to Rueben Ottenwalder and Anel Adea for their guidance, encouragement and hospitality.  For more information on these non-profits please go to and

Feeling Sleepy?

Steve Hwang and Chris Evans, with our former colleagues at Mapi, recently published a review of measures used to assess cataplexy and sleepiness in a pediatric population:  Benmedjahed K, Wang G, Lambert J, Evans C, Hwang S, Black J, Johns MW.  Assessing sleepiness and cataplexy in children and adolescents with narcolepsy: a review of current patient-reported measures. Sleep Medicine 2017; 32:143-149.

Welcome to Endpoint Outcomes

Endpoint Outcomes is pleased to welcome Alp Tutuncu to our office in Long Beach.  Alp previously worked in project management at the Conrad Prebys Center for Chemical Genomics at the Sanford Burnham Prebys Medical Discovery Institute in La Jolla, California and as a research assistant at Elevation Pharmaceuticals.