Emerging Therapies for Orphan Diseases: Ethics and Economics

Friday, March 25, 2022 10:00to13:00
online event

Emerging Therapies for Orphan Diseases: Ethics and Economics

March 25th, 2022
Virtual Event


The last decade has witnessed significant advances in regenerative medicines, particularly in the context of rare and hereditary diseases. Many such regenerative medicines are expensive and unlikely to fail traditional cost-utility analyses, thus placing heavy financial pressures on healthcare systems.

Until now, healthcare systems have been able to accommodate the costs of treating orphan diseases, given the small number of treatments available and the rarity of disease. However, as regenerative treatment approaches advance and precision medicine qualifies more and more patients as having orphan diseases, healthcare systems will face increasing challenges balancing the needs of patients with orphan diseases with those with more prevalent diseases.

This half-day symposium will bring together philosophers, health economists, clinicians, patients and policy-makers to address the ethics of resource allocation for rare diseases, particularly in the context of expensive treatment approaches like gene therapy. The event will involve four 20-minute presentations from diverse stakeholders, followed by a 1-hour panel discussion addressing whether- and how much- fair healthcare systems ought to budget for new, expensive regenerative medicines.

Please note that simultaneous translation will be available for this event.

This event is sponsored by the Department of Equity, Ethics and Policy (DEEP) and the Class of Med ‘70.

More information and registration on the website.


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