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causes of disease yields

causes of disease yields new opportunities while also changing numerous aspects of the drug development process. For example, personalized medicine offers enormous potential to revolutionize the treatment paradigm, but the complex nature of the development process for these exceptionally precise treatments and diagnostic tests requires changes in how medicines are identified, studied, and manufactured. • Research on Complex Diseases: Investigators continue to explore potential new treatment options for more complex diseases such as neurological disorders, cancer, and many rare diseases for which few or no treatments exist. For example, the number of Alzheimer’s disease medicines in development jumped from 26 in 2003 to 82 today. 17,18 Science has always been, and always will be, about exploration, but with exploration comes the inevitable setbacks inherent in the research of complex diseases. • Regulatory Environment: The regulatory requirements and growing complexity of clinical trials translates into more numerous and more complex eligibility criteria for study enrollment, increased site visits and required procedures, longer study duration, and more rigorous data collection requirements. Recruiting patients for clinical trials can also be challenging, especially as science reveals molecular identifiers of various diseases that allow researchers to focus on increasingly narrow and specific patient populations. The form researchers use to collect data from each trial participant more than doubled in length between 2000 and 2011, underscoring the increased efforts required from the clinical trial research community (see Figure 19). 19 FIGURE 19: The Complexity of Clinical Trials Has Increased During the last decade, clinical trial designs and procedures have become much more complex, demanding more staff time and effort, and discouraging patient enrollment and retention. TRENDS IN CLINICAL TRIAL PROTOCOL COMPLEXITY 2000–2003 2008–2011 Increase in Complexity Total procedures per trial protocol (median) (eg, bloodwork, routine exams, x-rays) 105.9 166.6 57% Total investigative site work burden (median units) 28.9 47.5 64% Total eligibility criteria 31 46 48% Clinical trial treatment period (median days)* 140 175 25% Number of case report form pages per protocol (median) 55 171 211% *These numbers reflect the “treatment duration” of the protocol only. Sources: Getz KA, Campo RA, Kaitin KI. Variability in protocol design complexity by phase and therapeutic area. Drug Inf J. 2011;45(4):413-420; updated data provided through correspondence with Tufts Center for the Study of Drug Development. 54 Research and Clinical Trials

• Incorporating the Patient Perspective: Biopharmaceutical companies are working on integrating patient perspectives into the drug development process. Incorporating patient views on the outcomes that matter most to them, in terms of quality of life, day-to-day impact, and new therapy’s benefits and risks, allows researchers to develop medicines that RESEARCHING ALZHEIMER’S MEDICINES: SETBACKS AND STEPPING STONES Although a number of recent scientific advances have led to innovative treatments for patients, for many disease areas, the more we learn about a disease the greater our appreciation for a disease’s complexity and mystery. Such has been the case with Alzheimer’s disease, where just four new medicines were approved during the same period where there were 123 socalled “failures,” where medicines did not make it through the development process (see Figure 20). Although frustrating, these setbacks give researchers important insights that give them a better understanding of the disease and inform research on other medicines in development. This is an inherent part of biopharmaceutical research. A PhRMA report exploring these crucial stepping stones is available at http://www.phrma.org/sites/default/files/pdf/alzheimersetbacksreportfinal912.pdf. FIGURE 20: Setbacks in Alzheimer's Disease Research Provide Stepping Stones for Future Innovation Since 1998, 123 medicines in development for the treatment of Alzheimer’s disease have not made it through clinical trials, with only 4 gaining FDA approval. These setbacks highlight the complexity of the R&D process. Though disappointing, they provide important knowledge to fuel future research. Number of Alzheimer’s Drugs No Longer Under Development 15.0 12.5 10.0 7.5 5.0 2.5 0.0 1998 7 1999 1 NEW 1 APPROVAL NEW APPROVAL 2000 2001 2002 Unsuccessful Alzheimer’s Drugs in Development 1998-2014 10 9 9 5 1 5 NEW APPROVAL 2 2 2 2003 3 2004 2005 2006 2007 13 2008 14 2009 12 2010 8 2011 5 2012 10 2013 1 NEW APPROVAL 7 2014 Source: Pharmaceutical Research and Manufacturers of America (PhRMA). Researching Alzheimer’s medicines: setbacks and stepping stones. http://phrma.org/sites/default/files/pdf/ alzheimers-setbacks-and-stepping-stones.pdf. Published 2015. Accessed April 2016. Research and Clinical Trials 55

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