CRA is a proud sponsor of the conference. Come visit us at booth #41!
Bhavesh Patel and Owen Male present the session titled “Maximizing Value & Positioning in the Emerging Competitive Rare Disease Arena.”
With increasing numbers of rare diseases served by multiple approved therapies, we are seeing the return on investment for Orphan Drug incentives and the emergence of competitive arenas. Given the unique challenges in rare diseases, how could policies evolve and where should industry focus their efforts to meet payer evidence expectations and effectively position in the evolving environment?
Michele Pistollato and Charlotte Poon host the roundtable discussion on “How can we manage the tension between immediate access to innovation and affordability? The future of rare disease policy poses a crucial question.”
There has been a step-change in the number of orphan medicines over recent years and the pipeline suggests this will continue to the foreseeable future. This is the result of patient advocacy, government policies and improved understanding of rare diseases. As spending increases, there will pressure to control healthcare. However, given the number of diseases without effective treatment the need to support the development of orphan medicines from a societal perspective is as important as ever. We will explore the cost containing measures emerging across countries, identify the potential implications for rare diseases and finally, how to balance these competing objectives.
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