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21st Century Cures Act Improves Funding Outlook for Orphan Drugs

On December 13, 2016, Barack Obama signed the 21st Century Cures Act into law. The Act, says FDA Voice, “builds on FDA’s ongoing efforts to advance medical product innovation and ensure that patients get access to treatments as quickly as possible, with continued assurance from high quality evidence that they are safe and effective.” The new law is a big step forward for orphan drugs, which are used to treat rare diseases such as mesothelioma. 

If you haven’t heard of orphan drugs, they are defined as drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons, but are not expected to recover the costs of developing and marketing a treatment drug. 21st Century Cures amends the Orphan Drug Act to expand the Orphan Drug Grant program so that prospectively planned and designed observational studies and other natural history analysis can qualify, so long as the studies/analyses are used to develop or validate a drug development tool related to a rare disease or condition or understand the full spectrum of disease manifestations, including genotypic and phenotypic variability and identifying and defining distinct subpopulations affected by a rare disease or condition. Previously the Orphan Drug Grant program was limited to clinical and pre-clinical studies in rare diseases.

Around 3,200 mesothelioma cases are diagnosed annually, says the National Institutes of Health (NIH), and the disease is one of the toughest forms of cancer to treat. Because mesothelioma is a unique disease, doctors rely heavily on innovative, investigational drugs, used alone or in combination with traditional treatments, to treat the condition. With grant funding now available for earlier stage research, the hope is that the focus on lesser understood rare diseases such as mesothelioma will increase, and more orphan therapies will be developed in the future—therapies like Ofev (nintedanib).

Just one day after the Act was signed, Boehringer Ingelheim announced that the U.S. Food and Drug Administration (FDA) had granted orphan drug designation to nintedanib for the treatment of mesothelioma. “The drug has shown promise as a potential treatment for malignant pleural mesothelioma in clinical trials to-date,” says Martina Flammer, Vice President of Clinical Development & Medical Affairs Specialty Care at Boehringer Ingelheim. “This designation is a validating milestone in its development.”

Researchers at Boehringer Ingelheim are now recruiting participants for a large meso-nintedanib study. They plan to randomize 450 patients at 137 sites in 27 countries.



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Henley, S. Jane, Theodore C. Larson, Manxia Wu, Vinicius C S Antao, Mary Lewis, Germania A. Pinheiro, and Christie Eheman. "Mesothelioma Incidence in 50 States and the District of Columbia, United States, 2003-2008." International Journal of Occupational and Environmental Health 19.1 (2013): 1-10. Web. 25 Feb. 2017.

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Upton, Fred. "Text - H.R.6 - 114th Congress (2015-2016): 21st Century Cures Act." U.S. Library of Congress, 13 July 2015. Web. 25 Feb. 2017.