Executive Summary The U.S. innovative biopharmaceutical industry leads the world in the development of new medicines: over the past decade some 300 new prescription medicines have been approved for use by the U.S. Food and Drug Administration (FDA). Together, these innovations have contributed to a range of new treatments resulting in improvements in the length and quality of life and reduced disease burden for individuals and society. However, the need for innovative new therapies for some of the most costly and challenging diseases and conditions has never been greater. This study presents data on two types of potential new treatments in the research and development pipeline:  New medicines in development, or new molecular entities (NMEs) – data for which are referred to in this report as new “products”; and  New molecule-indication combinations in development (which may be NMEs or new indications for medicines previously approved by the FDA) – data for these unique molecule-indication combinations are referred to in this report as “projects.” In both cases, we have focused our review on those activities that have advanced to the clinical testing stage in human volunteers, except where otherwise noted. In addition to excluding preclinical research, several other types of innovative activities were beyond the scope of this report. While new and enhanced methods of delivery and new formulations also represent new treatment options for patients and their health care providers, the study’s scope was limited to new molecules and new molecule-indication combinations. Similarly, post-approval research and Phase IV trials were beyond the scope of the analysis. The study is based on a review of data from 1986 onward from EvaluatePharma, a proprietary commercial database containing information on over 4,500 companies and 50,000 products, complemented by data on clinical trials found on ClinicalTrials.gov, and on orphan drug designations in the FDA Orphan Drug Product database. Developing a new medicine is a long and complex process, with risk of failure at each step. While thousands of new medicine candidates are screened in the laboratory, only one may eventually result in an FDA-approved medicine, after 10 to 15 years of testing, development, and review. It is impossible to predict which of the specific products or projects described in this report will eventually proceed all the way to FDA approval and ultimately to patients. Key findings from the report include the following:  The pipeline of drugs contained over 5,400 products in clinical development (i.e., those which have advanced to clinical testing in human volunteers, but have not yet been launched).  Taking into account the fact that a single molecule may be undergoing or have undergone clinical trials in more than one indication, there were nearly 8,000 projects in clinical development (that is, unique molecule-indication combinations; e.g., a particular drug in clinical trials for use in Alzheimer’s disease and schizophrenia would be counted as one product and two projects). Development projects were distributed across many therapeutic areas, from cancer to cardiovascular disease and diabetes, to neurology. For example, more than 1,600 projects were under way in neurology alone. INNOVATION IN THE BIOPHARMACEUTICAL PIPELINE A high percentage of NME-focused development activities were potentially first-in-class (i.e., those described by a unique pharmacological class distinct from those of any other marketed products): 78 percent of projects in Phase I, 69 percent in Phase II, and 45 percent in Phase III were potentially first-in-class. Only one molecule in a given class can eventually win first-in-class designation; however, it cannot be known in advance which molecule will proceed through clinical testing and be approved first. There were particularly high percentages of potential firstin-class medicines in neurology (84 percent), psychiatry (80 percent), cancer (80 percent), and diabetes (79 percent).  As of October 2011, 1,795 projects with an orphan disease designation by the FDA were in development. These activities address a broad range of diseases and conditions from enzyme storage disorders to rare cancers. Orphan diseases individually affect fewer than 200,000 people in the U.S., but taken together are estimated to affect some 25 million people.  A number of potential medicines in development would provide new clinical options for patients with diseases for which no new therapies have been approved for many years. An analysis of 15 selected diseases with no approvals in the past 10 years identified over 400 projects in development.  Personalized medicine approaches are receiving a growing emphasis in development. A separate analysis of data on only Phase III and Phase IV U.S. clinical trials involving the use of molecular biomarkers (i.e., characteristics that can guide treatment and diagnosis and are integral to personalized medicine) identified 155 personalized medicine trials that were initiated on or before January 2009.  A range of novel scientific approaches to address various diseases and conditions were being pursued. Broad classes of scientific “platforms” readily identifiable in the dataset revealed: – 245 projects using cell therapy; – 127 projects using antisense RNA interference therapy (an approach that targets RNA, which carries genetic information that creates proteins, rather than proteins themselves); – 102 projects using monoclonal antibodies joined to cytotoxic agents to target tumor cells while sparing nearby healthy cells; and – 99 projects using gene therapy.

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