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How to Capture the Patients and Caregivers Perspectives in Rare Diseases? A Challenge for Orphan Drug Developers

December 13, 2016 @ 10:00 - 11:00 UTC+0


Webinar Topic:

How to Capture the Patients and Caregivers Perspectives in Rare Diseases? A Challenge for Orphan Drug Developers

Webinar Date/Time:

Tuesday, December 13, 2016 at

  • 10:00 AM EST (NYC, USA)
  • 3:00 PM GMT (London, UK)
  • 4:00 PM CET (Paris, FR)

Webinar Host:

Benoit Arnould, PhD

Benoit Arnould - Senior Director of Patient-Centered Outcomes, Mapi Group

Dr. Benoit Arnould has been conducting studies to develop and validate Patient-Reported Outcome (PRO) measures for 17 years. In recent years, Benoit has increasingly been asked by Industry clients to assist in their Endpoint strategy definition.  Benoit has sound knowledge of clinical and epidemiological trial design, data analysis and interpretation.  Benoit is a Health Economics graduate with a specialty in statistics, and has completed, under the supervision of Professor Gerard Duru, his PhD on tools for clinical decision making, a subject on which he publishes regularly.  Benoit leads the Mapi Global Patient- Centered Outcomes research team.

Webinar Description:

The cumulated burden of the many rare diseases on the society is such that numerous countries have developed specific policies to support research and encourage the emergence of innovative therapeutics. However, to support their decisions, regulators, payors and prescribers frequently miss adequate assessments measuring the perspective of the patients and their caregivers. Such assessments are critical to capture, communicate and compare the burden of each specific disease, as well as the outcomes of treatments.

In this session, we will present two routes to address this need: the development of specific measures, and the conduct of Mixed-Methods Research.

We will first tell how the PKU-QoL was internationally developed, validated and used to assess the impact of Phenylketonuria on patient’s and caregivers daily lives. This example will show how challenges such as cross-cultural variations, working across different age groups, and optimizing research in small samples, can be addressed. Every step has been published in congresses and peer-reviewed journals, supporting the use of PKU-QoL to validly assess the burden of diseases, as well as the outcomes of treatments. With the e-version of PKU-QoL made available for clinical practice, all stakeholders now share a common metric, and this metric is patient-centered. As frequently in rare diseases, the success results from three key features: first, the collaborative work of an international group of leaders and an experienced team of questionnaire specialists, second, the engagement of patients and their clinicians, and third, the strategic effort of a sponsor.

We will then explain why the traditional approach to Patient-Centered Outcomes research is not always the ideal route in orphan drug development, and will identify emerging solutions. We will discuss the paradox of rare diseases, where patient burden is the alpha and the omega of all stakeholders concerns whereas Patient-Reported Outcomes are rarely used to support regulatory claims, payors decisions, or drug prescription. We will show how innovative approaches such as Mixed Methods Research, can improve the knowledge, save time and costs. We will finally give an overview of several current initiatives aiming at providing adapted guidance to the conduct of Patient-Centered Outcomes Research in the context of Orphan Drug Development.

As a conclusion, we will give a flavor of what tomorrow could look like for assessing the patient benefit in rare diseases: a secured, efficient combination of traditional approaches and innovative solutions.

>> Register to attend this upcoming webinar now:

Have you registered to attend our November 15, 2016 workshop based in Brussels, Belgium yet?

  • Don't miss out on seeing Mapi's Senior Direct of Patient-Centered Outcomes Dr. Benoit Arnould, PhD present his slides as seen at WODC USA 2016 on the topic of How to Capture the Patients and Caregivers Perspectives in Rare Diseases? A Challenge for Orphan Drug Developers -- Click here to learn more and register to attend today



December 13, 2016
10:00 - 11:00
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