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Title: Leveraging the Value of Clinical Outcomes in DMD for Decision Making

Date: Wednesday, December 4, 2024
11:00AM EST |3:00PM UTC | 5:00PM CEST

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Description:

Duchenne Muscular Dystrophy (DMD) is a rare, progressive, fatal, genetic neuromuscular disorder caused by mutations in the DMD gene that are located in the X chromosome. DMD is characterized by progressive muscle atrophy, resulting in the loss of ambulation, decreased upper body function, and impaired cardiorespiratory function. Clinical trials in DMD face several challenges, including:

• Selection of endpoints: choosing responsive, reliable, and meaningful endpoints that capture the heterogenous disease progression and that are appropriate for each phase of the disease
• Adequate sample sizes for trials to support decision making with enough power is challenging in setting of a rare disease
• Control groups: decision makers often desire placebo control groups in clinical trials, in contrast with the community’s desire to minimize placebo exposure and maximize the use of historical natural history controls to evaluate the long-term treatment effect.

These challenges highlight the need for appropriate trial design from a patient-centric approach.

In this webinar, speakers representing the clinician, patient/caregiver and industry perspective will explain how DMD clinical trials are designed, which endpoints are often used, and the meaningfulness of these endpoints and its relevance to inform decision making.

The session will cover relevant aspects for a range of stakeholders with an interest in DMD and other rare diseases. We would recommend attendance from health economists, payers, policymakers, regulators, and patient advocates.

Learning Objectives:

• Understand DMD disease course to accurately assess disease progression; Understand disease milestones and their relevance and meaningfulness from the clinical and patient perspective.
• Appreciate the complexity of clinical trial design including outcomes, eligibility criteria, timing, and control groups.
• Explore how patient advocates, clinicians, and industry collaborate to support patient access to DMD drugs.

Moderator:

Francois Meyer, MD, Independent consultant on HTA and Public Health, Paris, France

Speakers:

Valeria A Sansone, MD, PhD, Clinical-Scientific Director, Centro Clinico NeMO Milano, Milan, Italy

Conny Pelicaen, BSc, Director, Belgium Duchenne Parent Project, Wespelaar, Leuven, Belgium

Paolo Bettica, MD, PhD, Chief Medical Officer, Italfarmaco S.p.A, Cinisello Balsamo, Milan, Italy

Brought to you by: Italfarmaco S.p.A.

Please note: On the day of the scheduled webinar, the first 1000 registered participants will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the ISPOR Educational Webinar Series webpage approximately 2 days after the scheduled Webinar. Reservations are on a first-come, first-served basis.