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Title: Challenges in Defining Elements of Value in DMD for Decision Making

Tuesday, March 25, 2025

11:00am EDT |4:00pm UTC | 6:00pm CEST

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Description:

Duchenne Muscular Dystrophy (DMD) is a rare, progressive, and lethal X-linked recessive disorder. DMD is characterized by early progressive muscle injury, resulting in the loss of ambulation (LOA), loss of upper limb function, wheelchair dependency and premature death due to respiratory failure or cardiac insufficiency. The decline in motor function significantly impacts patients quality of life, but also significantly increases caregivers’ burden.

Decision makers use value assessment frameworks to allocate resources. However, traditional value assessment frameworks in DMD are often a challenge, as they do not adequately capture the broader value of innovation.

As potential innovative treatments for DMD enter the market, understanding these elements of value that influence society’s willingness and ability to pay will be crucial.

Even if these elements of value are not easy to quantify, and not formally incorporated into traditional frameworks, they should be considered for a more holistic approach to value innovation.

This educational webinar will describe the factors that affect the assessment of the clinical and economic value of medical innovations in DMD, highlighting specific issues related to the societal value, and placing these issues in the context of future health technology assessments for DMD treatments.

The session will cover an important topic for a range of stakeholders with an interest in value assessments in rare diseases, and in particular in DMD. We would recommend attendance from health economists, payers, policymakers, and patient advocates.

Learning Objectives:

1. Introduce the current challenges with traditional value assessment frameworks, with a focus on rare diseases and DMD; and understand the factors influencing societal willingness and ability to pay for treatments.
2. Understand the relevance of caregiver burden and why it should be considered an element of decision making for the assessment of new DMD treatments, from a patient and payer perspective including impact on costs and well-being.
3. Describe potential methods for capturing these factors in the HTA context for DMD, including impact on caregiver costs and well-being.

Moderator:

Meindert Boysen, PharmD, MSc, Independent HTA Expert, The Netherlands

Speakers:

Meindert Boysen, PharmD, MSc, Independent HTA Expert, The Netherlands

Fleur Chandler, Patient Access, United Kingdom

Erik Landfeldt, PhD, Senior Epidemiologist, Associate Editor, Journal of Neuromuscular Diseases, Sage Publishing, Stockholm, Sweden

Brought to you by: Italfarmaco

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.