Developing Cures, Creating Jobs Pharmaceutical clinical trials in NEBRASKA Executive Summary This report shows how biopharmaceutical research companies continue to be vitally important to the economy and patient health in Nebraska. Since 2004, biopharmaceutical research companies have conducted or are conducting nearly 3,000 clinical trials of new medicines in Nebraska in collaboration with clinical research centers and hospitals. These clinical trials have investigated or are investigating some of Nebraska’s biggest health care challenges, including asthma, arthritis, cancer, diabetes, cardiovascular disease and Alzheimer’s disease. Executive SummaryClinical trials in NEBRASKA CLINICAL TRIALS IN NEBRASKA ARE A VITAL PART OF THE FDA DRUG APPROVAL PROCESS In the development of new medicines, clinical trials are conducted to prove therapeutic safety and effectiveness and compile the evidence needed for the U.S. Food and Drug Administration (FDA) to approve new treatments. Clinical tests of new drugs are conducted in three phases and, on average, account for nearly seven of the more than 10 years it takes to bring a new drug from development to patients. Clinical trials are responsible for more than half of the $2.6 billion average cost of developing one new innovative medicine. All clinical trials must be reviewed and approved by an Institutional Review Board (IRB) in advance; an independent committee of physicians, statisticians, local community advocates and others to ensure a trial is ethically conducted and patient rights are protected. Clinical Trials in Nebraska since 2004— Completed and Open All Clinical Trials Open Clinical Trials Source: www.clinicaltrials.gov. Search criteria: Nebraska, United States; Phase: early 1, 1, 2, 3; Industry only, first posted on or after 1/1/2004. Search performed 10/1/2019. Open clinical trials are recruiting, not yet recruiting or expanded access available. 2,978 326 RESEARCH IN YOUR BACKYARD / DEVELOPING CURES, CREATING JOBS 1 Executive Summary (cont.) Company employees in Nebraska include life CLINICAL TRIALS OFFER science researchers, management executives, IMPORTANT THERAPEUTIC office and administrative support workers, production workers, engineers, architects, OPTIONS FOR PATIENTS computer and math experts, and sales representatives. Biopharmaceutical companies For patients, clinical trials offer the potential for also supported the jobs of their vendors and another therapeutic option. Clinical tests may suppliers, including construction and IT firms. And provide a new avenue of care for some chronic the employees of biopharmaceutical companies disease sufferers who are still searching for the help to support local restaurants, day care centers medicines that are best for them. and other community businesses. Some clinical trials are conducted to compare existing treatments and some are done to explore ECONOMIC IMPACT OF whether a drug is appropriate for a different patient population, such as children or the elderly. CLINICAL TRIALS IN Still others are conducted to find ways to make NEBRASKA existing approved drugs more effective and easier to use with fewer side effects. A separate study by TEConomy Partners found that in 2017 alone, there were 403 active industry-sponsored, site-based clinical trials in ECONOMIC IMPACT OF Nebraska, with an estimated enrollment of 8,403 Nebraska residents. Infectious diseases and THE BIOPHARMACEUTICAL virology had the leading clinical trial enrollment SECTOR IN NEBRASKA in the state. The investment of these site-based clinical trials Biopharmaceutical research companies have was more than $153 million and the estimated been and continue to be a good source of jobs, total economic impact was more than $394 tax revenue and research spending in Nebraska. million. A study by TEConomy Partners found that in 2015, the industry supported more than 17,900 “This report illustrates one segment of jobs throughout Nebraska. Wages and benefits for employees whose jobs were supported by the life sciences—clinical trials—and gives us biopharmaceutical sector resulted in more than $233 million in state and federal taxes paid. yet another opportunity to publicize the Biopharmaceutical research companies supported impact this industry makes on our state’s the generation of $5.4 billion in economic activity economy and, more importantly, the role in the state, including the direct economic output of the sector itself, the output of the sector’s this industry has to impact patient care. vendors and suppliers and the output generated by the buying power of its workforce The clinical trials ongoing in our state— further our knowledge about disease and help to improve lives of Nebraskans.” Rob Owen, Executive Director, Bio Nebraska Life Sciences Association 2 PhRMA / Pharmaceutical Clinical Trials in NEBRASKA “Clinical research is critical to the mission of our academic medical center, because it is critical to the development of cutting edge care for our patients. Through clinical research we strive to improve the health of individuals throughout Nebraska, and throughout the World.” Christopher J. Kratochvil, M.D. Associate Vice Chancellor for Clinical Research, University of Nebraska Medical Center Vice President for Research, Nebraska Medicine Executive Director of Clinical Research, Global Center for Health Security Chief Medical Officer, UNeHealth Open Clinical Trials in Nebraska by Disease Disease Number of Trials Alzheimer’s Disease 3 Arthritis/Musculoskeletal Disorders 10 Autoimmune Diseases 3 Bladder Conditions 2 Cancer 141 Cardiovascular Diseases 22 Diabetes 5 Gastrointestinal/Esophageal Diseases 19 Genetic Disorders 5 Infectious Diseases 35 Kidney Diseases 5 Liver Diseases 5 Mental Disorders 8 Neurological Disorders 9 Respiratory Diseases 10 Skin Diseases 27 Transplantation-Related 3 Other Diseases 14 Total 326 Source: www.clinicaltrials.gov. Search criteria: Nebraska, United States; Phase: early 1, 1, 2, 3; Industry only, first posted on or after 1/1/2004. Search performed 10/1/2019. Open clinical trials are recruiting, not yet recruiting or are expanded access available. RESEARCH IN YOUR BACKYARD / DEVELOPING CURES, CREATING JOBS 3 Patient Resources & Directory WHAT IS THE CLINICAL TRIAL EXPERIENCE? Clinical trials are research studies that generate data to support FDA approval of a new medicine or a new indication for an existing medication. They also grant participants early access to new medicines, which are being developed to help combat chronic and serious diseases. By volunteering for a clinical trial, patients take an active role in their health care by helping researchers test new treatments. In Nebraska, 2,978 clinical trials since 2004 have targeted diseases and conditions like asthma, arthritis, cancer, diabetes, cardiovascular disease and Alzheimer’s disease. Phases of Learning About Clinical Trials and Accessing There are three phases of clinical testing used to Clinical Trials evaluate potential new medicines: Patients can learn about clinical trials in several PHASE I—Researchers test the drug in a small ways. Health care providers are aware of clinical group of people, usually between 20 and 100 trials being conducted at hospitals, universities, healthy adult volunteers, to evaluate its initial safety and other leading health care facilities, and these and tolerability profile, determine a safe dosage institutions can be valuable sources of information range and identify potential side effects. for patients looking to participate. Patients can also use hospital and university websites to find the PHASE II—The drug is given to volunteer patients, trials being conducted in their area. For instance, usually between 100 and 500 people, to study its for clinical trials at the University of Nebraska efficacy, identify an optimal dose and to further Medical Center visit www.unmc.edu/cctr/for- evaluate its short-term safety. public/clinical-trials/index.html. PHASE III—The drug is provided to a larger, More information about clinical trials in Nebraska more diverse patient population, often involving and how to volunteer for one can be found at between 1,000 and 5,000 patients (but sometimes www.centerwatch.com, a PhRMA-recommended many more thousands), to generate statistically website. significant evidence to confirm its safety and effectiveness. They are the longest studies and usually take place in multiple sites around the world. 4 PhRMA / Pharmaceutical Clinical Trials in NEBRASKA What to Expect Expanded Access Since clinical trials are often conducted Successful completion of the clinical trials is in a doctor’s office, patients may need to required to demonstrate to the FDA that an devote more time to physician visits and investigational drug is safe and effective, so physical examinations. They may also have that it can be approved and made available additional responsibilities, like keeping a to a broad patient population. Clinical trials daily log of their health. All prospective are the primary route by which patients can participants must sign an informed consent participate in the drug development process, document saying they understand that the receive access to unapproved investigational clinical trial is research, and that they can drugs and contribute to the collection of leave the trial at any time. After consulting safety and efficacy data necessary for FDA with their health care providers, patients can approval. volunteer to participate, leading to a pre- screening interview. If they fit the criteria and For patients with