Topics in Evaluating the Cost-Effectiveness of Gene Therapies

May 29, 2019
1:00PM EDT | 6:00PM GMT | 7:00PM CET - 1 hour in duration
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Speakers: Scott J. Johnson, MHA, PhD

Several gene therapies are approved or will be approved in the near future by the FDA, and several hundred more are in development. These therapies are characterized by being developed to treat severe disease where there is a high unmet need. They have high treatment efficacy, are expected to be one-time treatments, and are associated with high treatment costs. About two thirds are being developed for indications other than cancer, often in diseases that are rare or ultra-rare, in some cases for pediatric populations. In this webinar, we will discuss topics in assessing the cost-effectiveness of gene therapies, including modeling technique, assessing the durability of the treatment effect in lieu of limited data, discounting, the model time horizon, the importance of indirect costs and ISPOR’s recent work in refining value frameworks.

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ISPOR’s Mid-west Regional Chapter


Scott J. Johnson, MHA, PhD
Medicus Economics LLC

Please note: On the day of the scheduled webinar, the first 500 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 weeks after the scheduled Webinar.

Reservations are on a first-come, first-served basis for all ISPOR members.

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