Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary (2015)
Chapter: Appendix A: References
Abbott, A. 2011. Novartis to shut brain research facility. Nature News 480(7376):161−162.
Bloom, D. E., E. Cafiero, E. Jane-Llopis, S. Abrahams-Gessel, L. R. Bloom, S. Fathmia, A. B. Feigl, T. Gaziano, A. Hamandi, M. Mowafi, D. O’Farrell, E. Ozaltin, A. Pandya, K. Prettner, L. Rosenberg, B. Seligman, A. Z. Stein, C. Weinstein, and J. Weiss. 2011. The global economic burden of noncommunicable diseases. Geneva, Switzerland: World Economic Forum.
Choi, D. W., R. Armitage, L. S. Brady, T. Coetzee, W. Fisher, S. Hyman, A. Pande, S. Paul, W. Potter, B. Roin, and T. Sherer. 2014. Medicines for the mind: Policy-based “pull” incentives for creating breakthrough CNS drugs. Neuron 84(3):554−563.
Delavande, A., M. D. Hurd, F. Martorell, and K. M. Langa. 2013. Dementia and out-of-pocket spending on health care services. Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association 9(1):19−29.
DiMasi, J. A., L. Feldman, A. Seckler, and A. Wilson. 2010. Trends in risks associated with new drug development: Success rates for investigational drugs. Clinical Pharmacology and Therapeutics 87(3):272−277.
Eichler, H. G., K. Oye, L.G. Baird, E. Abadie, J. Brown, C. L. Drum, J. Ferguson, S. Garner, P. Honig, M. Hukkelhoven, J. C. W. Lim, M. M. Lumpkin, G. Neil, B. O’Rourke, E. Pezalla, D. Shoda, V. Seyfert-Margolis, E. V. Sigal, J. Sobotka, D. Tan, T. F. Unger, and G. Hirsch. 2012. Adaptive licensing: taking the next step in the evolution of drug approval. Clinical Pharmacology & Therapeutics 9(3):426-437.
FDA (Food and Drug Administration). 2010. Adaptive design clinical trials for drugs and biologics. Washington, DC: FDA.
FDA. 2013. Alzheimer’s disease: Developing drugs for the treatment of early stage disease. Washington, DC: FDA.
FDA. 2015. Frequently asked questions: Breakthrough therapies. http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/fdasia/ucm341027.htm (accessed May 7, 2015).
Frias, Z. 2013. Data exclusivity, market protection and paediatric rewards. Paper presented at the European Medicines Agency Workshop for Micro, Small and Medium Sized Enterprises, London, UK, April 26.
Grabowski, H., G. Long, and R. Mortimer. 2011. Data exclusivity for biologics. Nature Reviews Drug Discovery 10(1):15–16.
Guha, R., and M. Salgado. 2013. The economics of irreparable harm in pharmaceutical patent litigation. Washington, DC: Cornerstone Research.
Hoe, J., G. Hancock, G. Livingston, B. Woods, D. Challis, and M. Orrell. 2009. Changes in the quality of life of people with dementia living in care homes. Alzheimer Disease & Associated Disorders 23(3):285−290.
Hounsome, N., M. Orrell, and R. T. Edwards. 2011. Eq-5d as a quality of life measure in people with dementia and their carers: Evidence and key issues. Value in Health 14(2):390–399.
Hurd, M. D., P. Martorell, A. Delavande, K. J. Mullen, and K. M. Langa. 2013. Monetary costs of dementia in the United States. New England Journal of Medicine 368(14):1326–1334.
Hyman, S. E. 2013. Psychiatric drug development: Diagnosing a crisis. Cerebrum: The Dana Forum on Brain Science 2013:5.
IOM (Institute of Medicine). 2011. Glutamate-related biomarkers in drug development for disorders of the nervous system: Workshop summary. Washington, DC: The National Academies Press.
IOM. 2013a. Improving and accelerating therapeutic development for nervous system disorders: Workshop summary. Washington, DC: The National Academies Press.
IOM. 2013b. Improving the utility and translation of animal models for nervous system disorders: Workshop summary. Washington, DC: The National Academies Press.
IOM. 2015. Sharing clinical trial data: Maximizing benefits, minimizing risk. Washington, DC: The National Academies Press.
Korieth, K. 2014. Tracking major changes in the R&D pipeline. CenterWatch 21(2):1−6.
Lund, C., A. Breen, A. J. Flisher, R. Kakuma, J. Corrigall, J. A. Joska, L. Swartz, and V. Patel. 2010. Poverty and common mental disorders in low and middle income countries: A systematic review. Social Science & Medicine 71(3):517−528.
Melek, S. P., D. T. Norris, and J. Paulus. 2014. Economic impact of integrated medical−behavioral healthcare—implications for psychiatry. Denver, CO: Milliman, Inc. P. 20.
Miller, G. 2010. Is pharma running out of brainy ideas? Science 329(5991):502–504.
Mullard, A. 2013. Learning from the 2012–2013 class of breakthrough therapies. Nature Reviews Drug Discovery 12(12):891–893.
NIH (National Institutes of Health). 2013. Advisory committee to the NIH Director interim report: Brain research through advancing innovative neurotechnologies (brain) working group. Bethesda, MD: NIH. P. 9.
NMSS (National Multiple Sclerosis Society). 2015. The MS disease-modifying medications. New York: National Multiple Sclerosis Society.
Pankevich, D. E., B. M. Altevogt, J. Dunlop, F. Gage, and S. E. Hyman. 2014. Improving and accelerating drug development for nervous system disorders. Neuron 84(3):546−553.
Paul, S. M., D. S. Mytelka, C. T. Dunwiddie, C. C. Persinger, B. H. Munos, S. R. Lindborg, and A. L. Schacht. 2010. How to improve R&D productivity: The pharmaceutical industry’s grand challenge. Nature Reviews Drug Discovery 9(3):203−214.
PhRMA (Pharmaceutical Research and Manufacturers of America). 2013. Rare diseases: A report on orphan drugs in the pipeline. Washington, DC: PhRMA.
PhRMA. 2015. Chart pack: Biopharmaceuticals in perspective. Washington, DC: PhRMA
Prince, M., M. Guerchet, and M. Prina. 2013. Policy brief for heads of government: The global impact of dementia 2013−2050. London, UK: Alzheimer’s Disease International.
Prince, M., M. Knapp, M. Guerchet, P. McCrone, M. Prina, A. Comas-Herrera, R. Wittenberg, B. Adelaja, B. Hu, D. King, A. Rehill, and D. Salimkumar. 2014. Dementia U.K.: Update. London, UK: Alzheimer’s Society.
Stovall, S. 2011. R&D cuts curb brain−drug pipeline: Development of new medicines for brain disorders could be threatened as major drug makers scale back research. http://www.wsj.com/articles/SB100014240527487SB10001424052748704474804576222463927753954 (accessed May 11, 2015).
TCSDD (Tufts Center for the Study of Drug Development). 2015. Biosimilars entering the U.S. market are likely to face multiple challenges. Boston, MA: TCSDD.
Thomas, D., and C. Wessel. 2015. Venture funding of therapeutic innovation: A comprehensive look at a decade of venture funding of drug R&D. Washington, DC: Biotechnology Industry Organization. https://www.bio.org/sites/default/files/BIO-Whitepaper-FINAL.PDF (accessed June 12, 2015).
Thraves, L. 2014. Financial cost of dementia. http://www.alzheimers.org.uk/site/scripts/documents_info.php?documentID=418 (accessed May 7, 2015).
UnitedHealth Group. 2014. The growth of specialty pharmacy: Current trends and future opportunities. Minnetonka, MN: UnitedHealth Group.
US Burden of Disease Collaborators. 2013. The state of U.S. health, 1990–2010: Burden of diseases, injuries, and risk factors. JAMA 310(6):591−606.
USHOR (U.S. House of Representatives). 2015a. 21st Century Cures Act discussion document. Washington, DC: U.S. House of Representatives. http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion-Document.pdf (accessed June 9, 2015).
USHOR. 2015b. The 21st Century Cures discussion document. Washington, DC: U.S. House of Representatives. http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/files/114/FINAL%20Cures%20Discussion%20Document%20White%20Paper.pdf (accessed June 9, 2015).
Vernon, J. A., J. H. Golec, and J. A. Dimasi. 2010. Drug development costs when financial risk is measured using the Fama–French three-factor model. Health Economics 19(8):1002–1005.
Vos, T., A. D. Flaxman, M. Naghavi, R. Lozano, C. Michaud, M. Ezzati, C. J. L. Murray. 2012. Years lived with disability (YLDS) for 1160 sequelae of 289 diseases and injuries 1990–2010: A systematic analysis for the Global Burden of Disease Study 2010. Lancet 380(9859):2163−2196.
Wegener, G., and D. Rujescu. 2013. The current development of CNS drug research. International Journal of Neuropsychopharmacology 16(7): 1687−1963.
Zuliani, G., M. Galvani, F. Sioulis, F. Bonetti, S. Prandini, B. Boari, F. Guerzoni, and M. Gallerani. 2012. Discharge diagnosis and comorbidity profile in hospitalized older patients with dementia. International Journal of Geriatric Psychiatry 27(3):313−320.
