Engaging directly with patients throughout the entire product lifecycle

Selected Case Studies in Patient-Centered Outcomes

Market Analysis

Development Of A New Measurement Instrument To Assess Patients' Acceptance Of Their Medications: The ACCEPT© Questionnaire.

Challenge

Understanding the link between patients’ beliefs and behavior may help explain patients’ attitude with their medication.

Solution

Evaluate acceptance of medication, i.e. how patients rate their medication (advantages and disadvantages) based on their experience and how it results in their willingness to take it. Development of a self-administered questionnaire, based on published articles and interviews with caregivers and patients treated with long-term medications.

Outcomes

ACCEPT© will allow for the identification of medication attributes related to adherence.

Publications

Marant et al., ISPOR, 2008.
Saussier et al., ISPOR, 2008.
Marant et al., ISPOR, 2009.
Chrétin et al., ISPOR, 2010.
Gauchoux et al., Pharmacoepidemio Risk Management Newsletter, 2011. Dias Barbosa et al., ESPACOMP, 2011.
Marant et al., The Patient: Patient-Centered Outcomes Research, 2012. Arnould et al., ISPOR, 2013

 

Development Of The DIVA Questionnaire: Attitudes, Beliefs And Behaviors Of General Practitioners Regarding Vaccination

Challenge

Understanding the factors that influence GPs’ adherence to patient vaccination recommendations in an effort to improve vaccination rates in the general population.

Solution

Develop a self-report tool, which enables the identification of GPs’ attitudes, beliefs and behaviors, to assess the prevalence and determinants of vaccination behaviors, and to identify the modifiable barriers to vaccination.

Outcomes

The DIVA questionnaire: a unique tool, which is appropriate for use in any vaccination and evaluates the modifiable factors that might influence GPs’ attitudes toward a specific vaccination.

Publications

Manuscript in preparation.
Tugaut, B et al., ISPOR Europe, 2013.

Business Contact

Khadra Benmedjahed +33 (0)4 72 13 66 97

Market Authorization

Analysis Of Patient Assessment Of Constipation-Quality Of Life (PAC-QOL) Questionnaire In Resolor Clinical Trials

Challenge

Reluctance of regulatory agencies to deliver PRO labeling claims in constipation based on existing scales.

Solution

Statistical analysis on pooled data from three resolor clinical trials. Confirmation of PAC-QOL psychometric properties. Guidance on interpretation of PAC-QOL changes in scores. Confirmation of meaningful treatment benefits.

Outcomes

EMA Quality of Life and treatment satisfaction claim.

Publications

Dubois et al., ISOQOL, 2008.
Dubois et al., Neurogastroenterol Motil, 2010.

Analysis Of Health-Related Quality Of Life (HRQL) Of Patients With Multiple Myeloma

Challenge

To investigate the impact of the treatment on HRQL in the complex context of oncology clinical trials including multiple, interrelated outcomes: clinical response, death, treatment toxicities.

Solution

Ad-hoc statistical analysis of PRO data from phase III clinical trial: HRQL change, relationships between clinical response and HRQL, quality-adjusted survival, time-to-sustained HRQL improvement.

Outcomes

Evidence supporting the benefit-risk profile of innovative cancer drug.

Publications

Dhawan et al., IMW 2009.
Dhawan et al., ASH 2009.
Delforge et al., European Journal of Haematology 2012.

Development Of The Well-Being Related To Food Questionnaire (Well-BFQ)

Challenge

To screen food products and food-related wellbeing, and reinforce allegation demands to European Food Safety Authority in the general population, the use of appropriate tools is essential – such a tool does not exist.

Solution

Develop and validate a tool that measures the concept of well-being in the specific context of diet, including: grocery shopping, cooking, place where people eat, commensality and eating and drinking habits.

Outcomes

The Well-BFQ is an innovative measure of assessment of food-related well-being and can be used to assess both the short- and long-term benefits of food.

Publications

Guillemin I, et al., ISPOR Dublin, 2013.

Business Contact

Alexia Marrel +33 (0)4 72 35 53 16

Mixed Methods Research In Phase II Clinical Trial Protocol

Challenge

To define measurement of patient benefit in a phase II protocol being finalized, with hypotheses availability limited due to innovative nature of the trial drug.

Solution

Add patient interviews to the end of the trial period to explore patient benefit of treatment.

Outcomes

Through close interaction between Mapi and the clinical and regulatory teams, the study protocol was amended to include qualitative research, and completed within a very short period of time.

Publications

Not for publication.

Business Contact

Alexia Marrel +33 (0)4 72 35 53 16

Market Access

Qualitative Research In Clostridium difficile Infections

Challenge

Reveal the impact of C. difficile infections on healthcare professionals’ work at the hospital and on patients’ everyday lives to increase awareness on condition.

Solution

Qualitative research (phone interviews) with French and US nurses and patients to discuss personal experiences with C. difficile infection episodes.

Outcomes

Published evidence on burden of C. difficile infections on nurses’ work and patients’ everyday lives.

Publications

Guillemin et al., ISPOR Europe, 2012.
Guillemin et al., International Journal of Nursing Practice, 2013 (In press). Guillemin et al., ESCMID, 2013.
Guillemin et al., HISIPS, 2013.
Guillemin et al., Patient. 2014.

Business Contact

Alexia Marrel +33 (0)4 72 35 53 16

Market Adoption

Development And Validation Of A Specific Measure Of Disability Due To Chronic Obstructive Pulmonary Disease (COPD): The Disability Related to COPD Tool (DIRECT)

Challenge

COPD is a major source of disability that is often overlooked, depriving patients of effective treatment. Disability assessment is the missing piece in the clinical assessment of COPD.

Solution

Develop and validate a self-administered questionnaire specifically designed to assess COPD-related disability in the context of clinical practice.

Outcomes

DIRECT is a valid and reliable measure of COPD-related disability and can help enhance the management of COPD patients. Its brevity and excellent measurement performances make it a promising tool for clinicians.

Publications

Arnould B et al. ISOQOL, 2007.
Aguilaniu et al., International Journal of COPD, 2011.

Business Contact

Carla Dias Barbosa +33 (0)4 72 13 66 56

Development Of A Patient Self-Assessment Tool To Improve Patient-Clinician Communication On The Daily Management Of Type 2 Diabetes

Challenge

Concordance between doctor and patient is needed to ensure high adherence and reduce risk of com- plications

Solution

Create a communication tool for clinical practice based on patient needs and clinician priorities.

Outcomes

Pilot version of the OPTIMA questionnaire: a self-assessment tool covering 5 modules: physical activities, treatment, nutrition, knowledge of the disease and knowledge of glycaemia issues. The tool should enhance patient-clinician communication on diabetes management.

Publications

Arnould et al., ISPOR Europe, 2008.

Business Contact

Alexia Marrel +33 (0)4 72 35 53 16

International Development of Questionnaires To Assess The Impact Of Phenylketonuria (PKU) And Its Treatment On Daily Life

Challenge

No instrument available that captures patient-centered outcomes in a rare disease area which dramati- cally demands patient perspective to be taken into account both in clinical research and practice.

Solution

Develop and validate specific questionnaires that enable the measurement of PKU-impact on patients’ and caregivers’ daily lives.

Outcomes

The first PKU-specific Quality of Life questionnaire available and validated in four age groups and seven languages.

Publications

Manuscript in preparation.
Bettiol E et al., SSIEM, Turkey, 2010.
Bettiol E et al., SSIEM, Switzerland, 2011.
Bosch AM et al., SSIEM, Austria, 2014.
Bosch AM et al., ISPOR, Netherlands, 2014

Business Contact

Khadra Benmedjahed +33 (0)4 72 13 66 97

Development And Validation of QUARTIS©, A Clinical Tool To Monitor Allergic Patients During Sublingual Immunotherapy

Challenge

Major limitations in long-term adherence to treatment hinders the efficiency of sublingual immunotherapy.

Solution

To identify patients showing limited motivation to persist with treatment and take appropriate commu- nication steps to restore compliance.

Outcomes

Reinforcement of strategic partnership of Stallergenes with prescribers.

Publications

Arnould, et al., ISPOR, 2005.
Arnould, et al., ISPOR, 2006.
Arnould, et al., ISPOR, 2006.
Arnould, PhD dissertation, 2007.

Business Contact

Khadra Benmedjahed +33 (0)4 72 13 66 97

Somatuline: Acromegaly And SAGIT – Use Of SAGIT In Clinical Practice For Patient Segmentation

Challenge

Specialists of acromegaly share a common framework to assess patient status but lack a common report to describe disease severity and evaluate the control/progression of acromegaly in treated patients. To fill this gap, they developed the SAGIT tool, a standardized clinician-reported outcomes tool for use by endocrinologists in routine practice.

Solution

Support a group of lead specialists in acromegaly in assessing the relevance of the SAGIT tool in their practice, as well as their acceptance and understandability of the tool.

Outcomes

Updated version of the SAGIT tool based on endocrinologist feedback.

Publications

Giustina et al., European ISPOR, 2013.
Giustina et al., Endocrine Society’s 96th Annual Meeting, 2014.
Giustina et al. European ISPOR, 2014.

Business Contact

Carla Dias Barbosa +33 (0)4 72 13 66 56

Development And Psychometric Validation Of The Urinary Symptom Profile (USP)

Challenge

Although efficient treatments are available for urinary disorders only few patients were under medical care for these conditions.

Solution

Provide clinicians with a unique tool assessing symptoms of Stress Urinary Incontinence, Overactive Bladder, and Low Stream.

Outcomes
The USP is the first valid and reliable questionnaire providing comprehensive evaluation of all urinary disorders and their severity, in both men and women. It allows the screening of these symptoms and contributes to the differential diagnosis.

Publications

Arnould B et al., ISOQOL Italy, 2005.
Richard F et al., AFU, France, 2005.
Grise P et al., SIFUD, France, 2005.
Coloby P et al., SIFUD, France, 2006.
Arnould B et al., ISOQOL Hungary, 2007.
Arnould B et al., European SMDM, UK, 2007.
Haab F et al., Urology, 2007.

Business Contact

Khadra Benmedjahed +33 (0)4 72 13 66 97

Development And Validation Of A Specific Questionnaire Measuring Patients’ Satisfaction With Their Anti-TNF Treatment In Severe Crohn’s Disease

Challenge

To demonstrate the potential benefits of anti-TNF treatments and their specificities compared to standard medication perceived by patients suffering from Crohn’s disease.

Solution

Develop and validate a specific questionnaire using a rigorous and scientifically sound methodology to measure patients’ satisfaction with their anti-TNF treatment in terms of expectations, satisfaction and motivation.

Outcomes

Creation of an evaluation criterion and inclusion into an international program to evaluate patients’ satisfaction toward anti-TNF treatments in Crohn’s disease.

Publications

Arnould, et al., ISPOR, 2009.
Marant et al., Clin Exp Gastroenterol, 2011.
Arnould, et al., ISPOR, 2012.
Gilet et al., Patient Prefer Adherence, 2014.

Request a consultation with a Patient-Centered Sciences expert