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Abstract

The business model under which the pharmaceutical industry has operated in the recent past has become untenable. The era that has seen a consistent pipeline of blockbuster medicines for common chronic diseases is waning. New paradigms for more efficient and more economical drug development are being sought and implemented. Recent growth both in the repurposing of existing drugs and in the orphan product market has signaled the new hope for success and profit. New technology and the promise of personalized medicine augment the sense of optimism in this time of complementary transition in the wider healthcare industry. Yet, are the new paradigms sustainable within the framework of the Orphan Drug Act as implemented today? What is the cost of rare disease and orphan product development to the relevant stakeholders: government, private payers, patients and families, caregivers, research universities, biotechnology firms, and pharmaceutical companies? This article considers the perpetual debate: what is the appropriate balance between access and innovation, specifically in the context of incentivizing therapies for rare disease?

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