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Challenges and Solutions for Budget Impact Analysis of Gene Therapies

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Pharmaceutical innovation is a key driver of improvement in health outcomes, quality of life and productivity for people with acute and chronic conditions. This progress has been exemplified in the context of haemophilia, where iterative and incremental innovation has added significant improvements for patients and health systems. Yet, despite an impressive array of treatments, there is a remaining unmet need. People with haemophilia may experience pain, joint damage, and mental burden due to their condition, necessitating further innovation and new treatments. Treatment developments have increased the life expectancy of people living with haemophilia which in turn has presented further challenges related to ageing with haemophilia.
The OHE has recently published a report “Investing in Innovation: A Spotlight on Haemophilia Therapies” which outlines the evolution of the treatment landscape for haemophilia, discusses recent and upcoming treatment developments and the remaining unmet needs of people living with haemophila.
Based on key recommendations highlighted in the report , the webinar will draw from a diverse panel of stakeholders, representing the perspective of haemophilia patients, physicians, health economists and policymakers to reflect on recent progress, explore the remaining unmet need and set out the required steps for continued innovation in this space.
This webinar is intended to be useful for a wide range of stakeholders that are interested in health technology assessment, including (but not limited to) payers, patient advocacy groups, regulators, health data scientists, academics, and industry.
This is a Pfizer commissioned and funded, non-promotional meeting facilitated by OHE. Pfizer has been involved in the initiation, development, and production of the agenda and content of this webinar.
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