Patient-Reported Outcomes

Our studies assess the value of pharmaceutical products and devices from the patient’s perspective. We evaluate patient-reported outcomes using a variety of data sources, including patient survey data, focus group data, interviews, and clinical trials. Our experience includes: 

  • Developing and validating patient-reported outcomes (PRO) instruments
  • Analyzing clinical trial data to demonstrate treatment efficacy using PRO instruments
  • Developing PRO evidence for dossiers in connection with label consideration
  • Establishing the minimal clinically important difference (MCID) for PRO instruments
  • Developing interactive tools to communicate the health-related quality of life (HRQL) burden of a disease
  • Designing patient surveys and conducting primary data collection to assess the HRQL of a disease
  • Designing and carrying out discrete choice experiment (DCE) studies to investigate patients’ preferences and willingness to pay for medical products

Selected Examples of Our Work

  • Webb SM, Ware JE Jr, Forsythe A, Yang M, Badia X, Nelson LM, Signorovitch JE, McLeod L, Maldonado M, Zgliczynski W, de Block C, Portocarrero-Ortiz L, Gadelha M. Treatment Effectiveness of Pasireotide on Health-Related Quality of Life in Patients with Cushing’s Disease. Eur J Endocrinol. 2014;171:89–98.
  • Victorson D, Carlozzi NE, Frank S, Beaumont JL, Cheng W, Gorin B, Duh MS, Samuelson D, Tulsky D, Gutierrez S, Nowinski CJ, Mueller A, Shen V, Sung V. Identifying Motor, Emotional-Behavioral, and Cognitive Deficits that Comprise the Triad of HD Symptoms from Patient, Caregiver, and Provider Perspectives. Tremor Other Hyperkinet Mov. 2014; 4:224 doi: 10.7916/D8JW8BWS.
  • Hoch J, Yang M, Yan S, van den Broek N, Xie J, Warusavitarne J. A Health-Related Quality of Life Study Comparing Vitala™ Continence Control Device Versus Traditional Pouch System Only in Patients with End Colostomy. Eur J Gastroenterol Hepatol. 2013;25:739–747. 
  • Signorovitch J, Brainsky A, Grotzinger KM. Validation of the FACIT-Fatigue Subscale, Selected Items from FACT-Thrombocytopenia, and the SF-36v2 in Patients with Chronic Immune Thrombocytopenia. Qual Life Res. 2011;20:1737–1744.
  • Cella D, Pickard AS, Duh MS, Guérin A, Mishagina N, Antràs L, Neary MP, McCann L, Hodge R, Sternberg CN. Health-Related Quality of Life in Patients with Advanced Renal Cell Carcinoma Receiving Pazopanib or Placebo in a Randomised Phase III Trial. Eur J Cancer. 2012;48:311–323.
Selected Examples of Our Work