The 21st Century Cures Act was signed into law on December 13, 2016. The Act is a step towards more funding and faster access to innovative treatments that can treat rare diseases such as mesothelioma. The Act addresses orphan drugs, which are 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.
Previously, the Orphan Drug Grant program was limited to clinical and pre-clinical studies in rare diseases. With the new law, the Orphan Drug Act and the Orphan Drug Grant program have been expanded so that prospectively planned and designed observational studies and other natural history analysis can qualify. The studies/analyses must be 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.
More than 3,000 mesothelioma cases are diagnosed each year and it is one of the most difficult forms of cancer to treat. Because mesothelioma is so distinct, doctors often turn to innovative, investigational drugs to treat the condition. These drugs may be used alone or in combination with traditional treatment such as chemotherapy, radiation, and surgery. With greater access to funding for earlier stage research, the hope is that the study of lesser understood rare diseases such as mesothelioma will increase and more orphan therapies will be developed in the future.
Califf, Robert M., MD. "21st Century Cures Act: Making Progress on Shared Goals for Patients." FDA Voice. U.S. Food & Drug Administration (FDA), 13 Dec. 2016. Web. 25 Feb. 2017.
"Developing Products for Rare Diseases & Conditions." U.S. Food and Drug Administration (FDA). Office of the Commissioner, n.d. Web. 25 Feb. 2017.
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.
"National Institutes of Health (NIH)." National Institutes of Health. U.S. Department of Health and Human Services, n.d. Web. 25 Feb. 2017.
Upton, Fred. "Text - H.R.6 - 114th Congress (2015-2016): 21st Century Cures Act." Congress.gov. U.S. Library of Congress, 13 July 2015. Web. 25 Feb. 2017.