The advent of the 1983 Orphan Drug Act brought about hundreds of drugs for once-neglected rare diseases, and has offered life-saving treatments for patients who once had little hope. There's no arguing that the law put in place incentives which allowed this market to thrive.
Yet, more than thirty years later, out-of-control pricing, abuse of the system and new technologies have critics wondering if changes need to be made. Meanwhile, the industry continues to benefit from the continually untapped market, as both small biotechs and big pharmas capitalize on the high unmet need in thousands of still-untreated orphan diseases.
In this Spotlight edition, BioPharma Dive explores how the law has impacted drug development, the changing pricing dynamics for rare disease drugs, trends shaping the space and the challenges remaining.
We hope you enjoy reading and please provide us with any feedback. You can reach us at BioPharma Dive by Twitter or via email.
Lisa LaMotta
Editor, BioPharma Dive
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News and Trends
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Feature Story
The 1983 law opened the doors to a flood of treatments for rare diseases, but rising costs and hundreds of still-untreated conditions have raised questions about whether changes are needed.
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Feature Story
Sales of orphan drugs have grown rapidly, prompting drugmakers to invest in rare diseases. But with that growth has come new scrutiny.
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Learn successful strategies for planning and executing a clinical study for an orphan indication with a unique set of challenges and considerations. Watch now
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Feature Story
Despite small patient populations and historically high prices, the pricing model for rare disease drugs has reached a turning point, prompting companies and insurers to push for new innovative models.
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Feature Story
It can be a long shot to find patients for some rare disease clinical trials. Recruiting challenges, however, often make those odds even smaller.
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Feature Story
The annual Rare Disease Day is designed to connect the rare disease community, build awareness and raise research funds.
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Featured Resources
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The need is great and time is of the essence in rare disease studies. Learn about the future of rare disease in this whitepaper. Download now →
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Learn how Medpace applies lessons learned, innovative thinking, and creative problem solving to rare disease clinical research. Download now →
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Rare disease and orphan indication studies have unique regulatory needs. Learn about their global regulatory environments. Read more →
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What We're Reading
Kaiser Health News
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The New York Times
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Harvard Business Review
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MM&M
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