Research in Your Backyard

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1 Research in Your Backyard Developing Cures, Creating Jobs Pharmaceutical Clinical Trials in South Carolina Dots show locations of clinical trials in the state.

2 Executive Summary Clinical Trials in South Carolina Biopharmaceutical research companies are conducting or have conducted more than 3,200 clinical trials of new medicines in collaboration with the state s clinical research centers, university medical schools and hospitals (1999 to present). Of the more than 3,200 clinical trials, 1,679 target the nation s six most debilitating chronic diseases asthma, cancer, diabetes, heart disease, mental illnesses and stroke. Economic Benefits of Clinical Trials in South Carolina Biopharmaceutical research companies have been an important source of jobs, tax revenue and research spending in South Carolina. A study by Battelle Technology Partnership Practice found that in 2011 the industry supported more than 18,000 jobs throughout the state. Wages and benefits for employees working directly for the biopharmaceutical sector were estimated to be $352 million in 2011, resulting in about $51 million in federal taxation and $5.3 million in state taxation. Biopharmaceutical research companies directly generated $2.4 billion in economic output in the state and supported another $1.8 billion in products and services through its vendors and suppliers. Biopharmaceutical companies have conducted more than 3,200 clinical trials of new medicines in South Carolina since 1999 and that has provided a steady source of revenue for such local institutions as the Medical University of South Carolina Hospitals and Clinics in Charleston, McLeod Regional Medical Center in Florence, Bon Secours St. Francis Hospital in Greenville and the Cancer Centers of the Carolinas in Easley, Greenville and Spartanburg. Equally as important, the trials have given local researchers an opportunity to be engaged in cutting-edge biomedical science research. The biopharmaceutical companies are collaborating on these clinical tests of new medications with the state s university medical schools and science centers, local hospitals and private clinical research facilities. Wayne Roper President SCBIO (South Carolina Biotechnology Industry Organization) Company employees in South Carolina include life sciences researchers, management executives, office and administrative support workers, production workers, engineers, architects, computer and math experts and sales representatives. 2 PhRMA Pharmaceutical Clinical Trials in South Carolina

3 The trials conducted in South Carolina have benefited patients because biopharmaceutical companies and their local research collaborators have targeted disease wisely more than 1,600 of their trials conducted over the last 13 years have been aimed at the nation s six most debilitating chronic diseases asthma, heart disease, stroke, cancer, diabetes and mental illnesses. These are the medical conditions that account for more than 75 percent of all health care costs throughout the United States. These diseases have certainly been devastating in the Palmetto State diabetes alone afflicts nearly 10 percent of South Carolinians and heart disease and stroke are the second and fourth leading causes of death in the state. Otis Rawl President and CEO South Carolina Chamber of Commerce About Clinical Trials In the development of new medicines, clinical trials are conducted to prove therapeutic safety and effectiveness and compile the evidence needed for the Food and Drug Administration (FDA) to approve treatments. Clinical tests of new drugs are conducted in three phases and account for an average of seven of the 10 to 15 years it takes to bring a new drug from development to patients. Clinical trials for a given drug or treatment involve thousands of volunteer patient participants, and the generation of tens of thousands of pages of technical and scientific data. Clinical trials are responsible for 45 to 75 percent of the $1.2 billion average cost of developing one new cutting-edge biotechnology medicine. For patients, the trials offer another potential therapeutic option. Clinical tests may provide a new avenue of care for some chronic disease sufferers who are still searching for the medicines that are best for them. Some trials are also conducted to compare existing treatments and some are done to learn if a drug is appropriate for a different patient population, such as children. Still others are conducted to find ways to make existing approved drugs more effective and easier to use with fewer side effects. All clinical trials must be reviewed and approved by an Institutional Review Board (IRB), an independent committee of physicians, statisticians, local community advocates and others to ensure a trial is ethically conducted and patient rights are protected. Clinical trial progress reports must be submitted at least annually to the FDA and IRB. All facilities that conduct or support biomedical research involving patients must comply with federal regulations and have an IRB. Clinical Trials in South Carolina since 1999 Completed and Active All Clinical Trials Six Major Chronic Diseases 3,247 1,679 Source: Note: Search criteria = South Carolina, United States; Phase 0, 1, 2, 3; industry only. Search performed 7/23/2013. Research in Your Backyard Developing Cures, Creating Jobs 3

4 We need to make more patients and doctors in South Carolina aware of local community clinical trials of new medicines. The fact is, we know that some chronic disease sufferers are still seeking treatments that are best for them and a clinical trial of a new drug, for some, could be a good therapeutic option to explore and discuss with their doctors. It s important, very important, that nearly 300 trials of new chronic disease treatments in the state are still active and seeking patient recruits. Those trials include 178 tests of new cancer medicines, 31 tests of new diabetes drugs and 23 of new heart disease medications. Paul Jeter Executive Director Leukemia & Lymphoma Society, South Carolina Chapter Clinical Trials and Chronic Diseases Chronic diseases pose the greatest threats to our nation s health and our ability to treat and prevent medical conditions. According to the Centers for Disease Control and Prevention, today, in the United States: Patients with chronic diseases account for 75 cents of every dollar spent on health care. Chronic diseases are the leading cause of death and disability. Clinical Trials in South Carolina Communities Location Asthma Cancer Diabetes Heart Disease Mental Illness Stroke Anderson Charleston Columbia Easley Greenville Greer Hilton Head 3 Mt. Pleasant Myrtle Beach Orangeburg Spartanburg Summerville 4 1 Sumter 3 3 Source: Note: Search criteria = South Carolina, United States; Phase 0, 1, 2, 3; industry only. Search performed 7/23/2013. See Appendix for detailed information about these clinical trials. Disease columns will not match totals in the Appendix because some clinical trials are recruiting in more than one city. This list of cities and towns is representative and not a complete list of where clinical trials are taking place in South Carolina. 4 PhRMA Pharmaceutical Clinical Trials in South Carolina

5 Chronic diseases are a leading driver of rising health care costs with expenses totaling billions of dollars every year. Biopharmaceutical research companies are developing new medicines to help treat those conditions that are taking an unprecedented toll on American lives, and many of these medicines are being tested today in clinical trials throughout South Carolina. Since 1999, biopharmaceutical research companies are sponsoring or have sponsored 1,679 clinical trials of potential new medicines in South Carolina alone for asthma, cancer, heart disease, stroke, diabetes and mental illnesses. Of these trials, 290 are either not yet recruiting or are just now seeking South Carolina patients. Many of the state s clinical tests involve collaborations with such respected local institutions as the Medical University of South Carolina, the Charleston VA Medical Center, Bon Secours St. Francis Hospital, Spartanburg Regional Medical Center, Sisters of Charity Providence Hospital, McLeod Health and the Wm. Jennings Bryan Dorn VA Medical Center. Many of the medicines being clinically tested here are new-generation biotechnology treatments. Clinical Trials for Top Chronic Diseases Chronic Disease All Clinical Trials Clinical Trials Still Recruiting Asthma Cancer Diabetes Heart Disease Mental Illness Stroke 29 7 Total 1, Source: Note: Search criteria = South Carolina, United States; Phase 0, 1, 2, 3; industry only. Search performed 7/23/2013. Some clinical trials appear in more than one disease category. Research in Your Backyard Developing Cures, Creating Jobs 5

6 Clinical Trials in South Carolina Clinical tests of new medicines are a vitally important part of the drug development and approval process they account for 45 to 75 percent of the $1.2 billion average cost of developing a new drug and are conducted to determine the safety and effectiveness of that treatment in patients. Some trials are also conducted to compare existing treatments and some are done to learn if a drug is appropriate for a different patient population, such as children. Still others are conducted to find ways to make existing approved drugs more effective and easier to use with fewer side effects. It s essential that trials be conducted properly so that clinicians and drug reviewers can develop accurate assessments of the efficacy and safety of medicines when used by patients. The FDA is a vigilant regulatory agency and its pharmaceutical review officers are effective in detecting flawed information. Questionable or confusing data can lead to lengthy delays in product approval or outright FDA rejection of a new drug. Biopharmaceutical research companies are looking for the best physicians and research institutions to meticulously help design and conduct their clinical trials to determine whether a medicine is safe and effective. Side effects must be painstakingly documented and a determination made as to whether they occur too often and are dangerous. Clinical tests involve three phases and thousands of volunteer patients and are often conducted at multiple sites around the country. In South Carolina, biopharmaceutical Clinical Trials for Top Chronic Diseases Chronic Disease All Clinical Trials Clinical Trials Still Recruiting Asthma Cancer Diabetes Heart Disease Mental Illness Stroke 29 7 Total 1, Source: Note: Search criteria = South Carolina, United States; Phase 0, 1, 2, 3; industry only. Search performed 7/23/2013. Some clinical trials appear in more than one disease category. companies are providing funds to have trials conducted at the states well-respected university medical schools and science centers, local hospitals and clinical research organizations. According to U.S. News and World Report, the Medical University of South Carolina ranked 59th and the University of South Carolina ranked 85th among this year s top 100 research-oriented medical schools in the United States. 6 PhRMA Pharmaceutical Clinical Trials in South Carolina

7 Asthma is a debilitating condition for more than 25 million Americans, including 7.1 million children under the age of 18. The toll is also severe in South Carolina, with 293,200 adults and 90,005 children suffering from asthma, according to the South Carolina Department of Health and Environmental Control. Currently, 22 clinical trials of new asthma medicines are recruiting patients in South Carolina. Trials are being conducted at Greenville Pharmaceutical Research, Inc. in Greenville and CU Pharmaceutical Research in Rock Hill. Cancer, the second leading cause of death in the United States, now afflicts nearly 14 million Americans, according to the National Cancer Institute. In South Carolina, nearly 28,000 new cancer cases will be diagnosed this year and 9,800 victims in the state will die, according to the American Cancer Society. Currently, 176 clinical trials of new cancer medicines are recruiting patients in South Carolina. Biopharmaceutical companies are collaborating on the tests with such prominent institutions as the Hollings Cancer Center at the Medical University of South Carolina in Charleston, the Greenville Hospital System in Greenville, Cancer Centers of the Carolinas in Easley, the Carolina Urologic Research Center in Myrtle Beach, the Institute of Translational Oncology Research (ITOR) in Greenville, the Spartanburg Regional Medical Center in Spartanburg and the Wm. Jennings Bryan Dorn VA Medical Center in Columbia. Diabetes affects more than 25 million Americans more than 8 percent of the U.S. population including 7 million people who are unaware they have the disease. In South Carolina, nearly 10 percent of residents have been diagnosed with diabetes, according to the South Carolina Department of Health and Environmental Control. In 2011, 1,085 South Carolinians died from diabetes. Currently, 35 diabetes clinical tests are seeking patients in South Carolina. The trials are being conducted at the Carolina Musculoskeletal Institute in Aiken, Palmetto Clinical Trial Services in Simpsonville, Southeastern Research Associates in Anderson and TLM Medical Services in Columbia. Heart disease and stroke are the first and fourth leading disease causes of death in the United States and the second and fourth in South Carolina. According to the American Heart Association, more than 82 million Americans are affected by these diseases. In South Carolina, in 2011, more than 9,000 residents died from some form of heart disease and 2,285 died from a stroke, according to the South Carolina Department of Health and Environmental Control. Currently, 21 heart disease and seven stroke clinical tests are seeking patients in South Carolina. The trials are being conducted at AnMed Health in Anderson, Greenville Hospital System in Greenville, McLeod Health/Pee Dee Cardiology in Florence, the Medical University of South Carolina in Charleston, Sisters of Charity Providence Hospital in Columbia and the South Carolina Heart Center in Columbia. Mental illness affects nearly 60 million Americans who suffer from some form of the disease from anxiety to depression to schizophrenia to eating disorders. In South Carolina, about 170,000 adults live with serious mental illness and about 48,000 children live with serious mental health conditions, according to the National Alliance on Mental Illness. Currently, 29 clinical trials for mental illness are recruiting patients in South Carolina. The trials are taking place at the Charleston VA Medical Center in Charleston, the Coastal Carolina Research Center in Mount Pleasant and Radiant Research in Greer. Physicians and patients can find out about clinical trials being conducted all over the state in collaboration with local institutions by accessing a database sponsored by the National Institutes of Health. Information on medicines in development is also available on the website of the Pharmaceutical Research and Manufacturers of America (PhRMA), under Related Content in the Innovation section. Research in Your Backyard Developing Cures, Creating Jobs 7

8 What is the Clinical Trial Experience? Clinical trials are research studies which grant participants early access to new drugs, treatments and therapies that are being developed to help combat chronic, serious and life-threatening diseases. By volunteering for a clinical trial, patients take an active role in their healthcare by helping researchers test new medical treatments, and helping to find better ways of using existing treatments so they will be more effective, easier to use and result in fewer or more tolerable side effects. In South Carolina alone, thousands of clinical trials are being conducted to study diseases like asthma, cancer, diabetes, heart disease, mental illness, and stroke. Phases of Clinical Trials There are three phases of testing used to evaluate new drugs and treatments: Phase I This phase is designed to test the safety of a new drug or treatment. Researchers test the drug on a small group of people (20-80) and evaluate safety aspects of the drug, such as safe dosage range, the best way of administering the treatment (pill form vs. a shot, for example) and identifying what, if any, side effects present themselves. Phase II This phase is designed to test efficacy and to further measure safety. The treatment is given to a larger group of people ( ) to make sure the treatment works correctly and to try to identify any less-common side effects, which may appear when more people are tested. This phase is usually placebo-controlled and double-blinded; meaning neither the patient nor the doctor knows whether the patient is getting the placebo or the real treatment. Phase III This phase is meant to confirm efficacy and safety information, monitor known side effects and compare the experimental treatment to commonly used ones to see which work better. A large group (1,000-3,000) receives this treatment, and like Phase II, it is usually placebo-controlled and double-blinded. Learning About and Accessing Clinical Trials There are several ways patients can access information about clinical trials. Healthcare providers are aware of clinical trials being conducted at hospitals, universities and other leading healthcare facilities, and these institutions can be valuable sources of information for patients looking to participate. Patients can also turn to hospitals and universities websites to see what studies are being conducted in their area, and what the eligibility criteria are for each trial. More information about clinical trials and volunteering can be found at a PhRMA-recommended website. 8 PhRMA Pharmaceutical Clinical Trials in South Carolina

9 What to Expect Treatments for clinical trials usually occur in a doctor s office. Patients may need to devote more time to doctor s visits and physical exams than they would normally. They may also have additional responsibilities, like keeping a daily log of their health. All prospective participants must sign an informed consent document saying they understand that the clinical trial is research, and that they can leave the trial at any time. Once they have consulted with their healthcare providers, patients can reach out via phone or to express interest in participating, at which point a pre-screening interview will take place. If the patient matches the trial s criteria, they will then be able to enroll in the study. Patient Expenses Patients should always ask during their pre-screening interviews what it will cost them to participate in a clinical trial. Clinical trial sponsors usually pay for all researchrelated costs and any additional testing or doctors visits the trial requires. Patients or their insurance companies may be asked to pay for any routine treatments that they would normally undergo for their disease. However, some health plans do not pay for these costs once a patient joins the trial. Patients should be sure to check with the clinic conducting the trial to find out if they or their insurance companies will be charged with any fees, and should make sure their insurance companies will cover the costs of routine exams if they join a trial. Non-local patients should be sure to look into the sponsoring clinic s policy on patient living arrangements. The National Cancer Institute, for example, makes patients responsible for their own travel costs for the initial screening visits. Once a patient is enrolled, the Institute will pay for transportation costs for all subsequent trial-related visits. These patients will receive a small per diem for food and lodging. The policy will differ from clinic to clinic. Research in Your Backyard Developing Cures, Creating Jobs 9

10 New Generation Medicines in Development Some of the medicines that have been tested in South Carolina are cutting-edge biotechnology drugs. America s biopharmaceutical research companies are using biotechnology to develop hundreds of new medicines and vaccines today. And South Carolina is one of the states where this research and development work is being done. Through biotechnology, new ways are being developed to not only more effectively treat disease, but also to predict it and even prevent it. Biotechnology medicines are developed through biological processes using living cells or organisms, rather than traditional chemical synthesis, the mainstay of pharmaceutical development for decades. Such novel treatments use a variety of new approaches to treat disease. For example, a monoclonal antibody is a laboratory-made version of the naturally occurring immune system protein that binds to and neutralizes foreign invaders. Interferons are proteins that interfere with the ability of a cell to reproduce. Antisense drugs, meanwhile, are medicines that interfere with the communication process that tells a cell to produce an unwanted protein. In addition, nanotechnology is being used in biotechnology research to provide drugdelivery systems, new treatments and diagnostics. Many of the medicines in clinical testing at South Carolina medical schools and research centers feature these technologies. For example: A genetically-modified virus-based vaccine to treat melanoma, with clinical trials conducted at the Medical University of South Carolina in Charleston and the Institute for Translational Oncology Research in Greenville. A recombinant fusion protein to treat diabetic macular edema and other types of macular edema with trials taking place at the Carolina Retina Center and the Palmetto Retina Center, both in Columbia. Clinicians at research facilities in Charleston, Myrtle Beach, and Simpsonville are testing a monoclonal antibody that targets lupus and different types of cancer. A therapeutic vaccine, designed to jump-start the immune system to fight disease, is in development for lung cancer and melanoma, with clinical trials underway in Charleston and Columbia. A gene therapy for the treatment of Alzheimer s disease is being studied at the Medical University of South Carolina in Charleston. An engineered human antibody to reduce inflammation in psoriasis is in clinical trials in Anderson and Greer. A monoclonal antibody for rheumatoid arthritis that may block the inflammatory process is being studied in Charleston and Columbia. These are only a portion of the examples of new ways the nation s biopharmaceutical companies and South Carolina research institutions are working together to attack disease. The biotechnology medicines and vaccines in development are helping to expand the frontiers of science and potentially bring more and better treatments to patients. 10 PhRMA Pharmaceutical Clinical Trials in South Carolina

11 Conclusion Biopharmaceutical companies close collaboration with clinicians and research institutions in South Carolina benefits patients, the state s economy and the advancement of science and patient care. Clinical trials provide stimulating biopharmaceutical research work and a reliable source of revenue for the states medical schools, hospitals and local contract research organizations, and the medicines being tested are often cutting-edge cell and protein treatments with the potential to be safer and more effective than older chemical compound drugs. What s more, South Carolinians considering participation in clinical trials have a wide range of choices, including 290 tests of new medicines for the six most debilitating chronic diseases. Research in Your Backyard Developing Cures, Creating Jobs 11

12 The Drug Discovery, Development and Approval Process It takes years on average for an experimental drug to travel from the lab to U.S. patients. Only five in 5,000 compounds that enter preclinical testing make it to human testing. One of these five tested in people is approved. Clinical Trials Discovery/ Preclinical Testing Phase I Phase II Phase III FDA Phase IV Years Test Population Purpose Laboratory and animal studies Assess safety, biological activity and formulations File IND at FDA 20 to 80 healthy volunteers Determine safety and dosage 100 to 300 patient volunteers Evaluate effectiveness, look for side effects 1,000 to 3,000 patient volunteers Confirm effectiveness, monitor adverse reactions from long-term use File NDA/BLA at FDA Review process/ approval Additional postmarketing testing required by FDA Success Rate 5,000 compounds evaluated 5 enter trials 1 approved The Drug Development and Approval Process The U.S. system of new drug approvals is perhaps the most rigorous in the world. It takes years, on average, for an experimental drug to travel from lab to U.S. patients, according to the Tufts Center for the Study of Drug Development. Only five in 5,000 compounds that enter preclinical testing make it to human testing. And only one of those five is approved for sale. On average, it costs a company $1.2 billion, including the cost of failures, to get one new medicine from the laboratory to U.S. patients, according to a 2007 study by the Tufts Center for the Study of Drug Development. Once a new compound has been identified in the laboratory, medicines are usually developed as follows: Preclinical Testing. A pharmaceutical company conducts laboratory and animal studies to show biological activity of the compound against the targeted disease, and the compound is evaluated for safety. Investigational New Drug Application (IND). After completing preclinical testing, a company files an IND with the U.S. Food and Drug Administration (FDA) to begin to test the drug in people. The IND shows results of previous experiments; how, where and by whom the new studies will be conducted; the chemical structure of the compound; how it is thought to work in the body; any toxic effects found in the animal studies; and how the compound is manufactured. All clinical trials must be reviewed and approved by the Institutional Review Board (IRB) where the trials will be conducted. Progress reports on clinical trials must be submitted at least annually to FDA and the IRB. Clinical Trials, Phase I Researchers test the drug in a small group of people, usually between 20 and 80 healthy adult volunteers, to evaluate its initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects. Clinical Trials, Phase II The drug is given to volunteer patients, usually between 100 and 300, to see if it is effective, identify an optimal dose, and further evaluate its shortterm safety. Clinical Trials, Phase III The drug is given to a larger, more diverse patient population, often involving between 1,000 and 3,000 patients (but sometime many more thousands), to generate statistically significant evidence to confirm its safety and effectiveness. They are the longest studies, and usually take place in multiple sites around the world. New Drug Application (NDA)/Biologic License Application (BLA). Following the completion of all three phases of clinical trials, a company analyzes all of the data and files an NDA or BLA with FDA if the data successfully demonstrate both safety and effectiveness. The applications contain all of the scientific information that the company has gathered. Applications typically run 100,000 pages or more. Approval. Once FDA approves an NDA or BLA, the new medicine becomes available for physicians to prescribe. A company must continue to submit periodic reports to FDA, including any cases of adverse reactions and appropriate quality-control records. For some medicines, FDA requires additional trials (Phase IV) to evaluate long-term effects. Discovering and developing safe and effective new medicines is a long, difficult, and expensive process. PhRMA member companies invested an estimated $48.5 billion in research and development in PhRMA Pharmaceutical Clinical Trials in South Carolina

13 The Good News Many Clinical Trials are Still Recruiting There are 290 clinical trials of new chronic disease drugs recruiting patients in South Carolina. These trials target the top most debilitating chronic conditions cancer, heart disease, stroke, asthma, diabetes and mental illness. Clinical Trials in South Carolina Communities Location Asthma Cancer Diabetes Heart Disease Mental Illness Stroke Anderson Charleston Columbia Easley Greenville Greer Hilton Head 3 Mt. Pleasant Myrtle Beach Orangeburg Spartanburg Summerville 4 1 Sumter 3 3 Source: Note: Search criteria = South Carolina, United States; Phase 0, 1, 2, 3; industry only. Search performed 7/23/2013. See Appendix for detailed information about these clinical trials. Disease columns will not match totals in the Appendix because some clinical trials are recruiting in more than one city. This list of cities and towns is representative and not a complete list of where clinical trials are taking place in South Carolina. Research in Your Backyard Developing Cures, Creating Jobs 13

14 The Good News Many Clinical Trials are Still Recruiting (continued) Asthma Leading Institutions Conducting Clinical Trials CU Pharmaceutical Research, Rock Hill Greenville Pharmaceutical Research, Inc., Greenville Cancer Leading Institutions Conducting Clinical Trials AnMed Health Cancer Center, Anderson Bon Secours St. Francis Hospital, Greenville Cancer Centers of the Carolinas, Easley, Greenville, Spartanburg Carolina Hematology Oncology, Sumter Carolina Urologic Research Center, Myrtle Beach Charleston Hematology Oncology Associates PA, Charleston Greenville Hospital System, Greenville Gynecologic Oncology Research and Development, LLC, Greenville Hollings Cancer Center at the Medical University of South Carolina, Charleston Institute of Translational Oncology Research (ITOR), Greenville Liberty Hematology Oncology, Columbia McLeod Regional Medical Center, Florence Medical University of South Carolina Hospitals and Clinics, Charleston Roper Hospital and Medical Associates, Charleston Saint Francis Hospital, Greenville Santee Hematology/Oncology, Sumter South Carolina Cancer Specialists, Hardeeville, Hilton Head South Carolina Oncology Associates, Columbia Spartanburg Regional Medical Center, Spartanburg Upstate Carolina-Community Clinical Oncology Program, Spartanburg Upstate Oncology Associates, Greenville Wm. Jennings Bryan Dorn VA Medical Center, Columbia Diabetes Leading Institutions Conducting Clinical Trials Carolina Musculoskeletal Institute, Aiken Palmetto Clinical Trial Services, LLC, Simpsonville Southeastern Research Associates, Inc., Anderson, Taylors TLM Medical Services, Columbia Heart Disease Leading Institutions Conducting Clinical Trials AnMed Health, Anderson Columbia Cardiology Consultants, Columbia Greenville Hospital System, Greenville McLeod Health/Pee Dee Cardiology, Florence 14 PhRMA Pharmaceutical Clinical Trials in South Carolina

15 Medical University of South Carolina, Charleston Sisters of Charity Providence Hospital, Columbia South Carolina Heart Center, Columbia Spartanburg Regional Medical Center, Spartanburg Coastal Carolina Research Center, Mount Pleasant Medical University of South Carolina, Charleston Radiant Research, Greer SleepMed of South Carolina, Columbia Upstate Cardiology, Greenville Mental Illness Leading Institutions Conducting Clinical Trials Carolina Clinical Research Services, Columbia Stroke Leading Institutions Conducting Clinical Trials Medical University of South Carolina, Charleston Palmetto Health Richland, Columbia Charleston VA Medical Center, Charleston Research in Your Backyard Developing Cures, Creating Jobs 15

16 Appendix The clinical trials listed here involve tests that have not yet started recruiting patients or are just now seeking volunteers to participate. This information is potentially valuable to patients still seeking effective treatments for their chronic diseases. It provides a new therapeutic option to discuss with physicians. Those interested in obtaining more information about certain trials can use the URL code listed for each test to log onto the clinical tests database of the National Institutes of Health. Asthma (22 clinical trials recruiting) Study 1: A 12-week Study to Compare the Efficacy and Safety of Albuterol Spiromax Versus a Placebo in People 12 Years and Older With Persistent Asthma Study 2: Study of Efficacy and Safety of Brodalumab Compared With Placebo in Inadequately Controlled Asthma Subjects With High Bronchodilator Reversibility Study 3: A Study to Establish the Efficacy of QBX258 in Patients With Moderate to Severe Asthma Study 4: A Dose-ranging Study of Fluticasone Furoate (FF) Study 5: A Study of Lebrikizumab in Patients With Uncontrolled Asthma on Inhaled Corticosteroids and a Second Controller Medication Study 6: A Study of MEMP1972A in Patients With Allergic Asthma Inadequately Controlled on Inhaled Steroids And A Second Controller (COSTA) Study 7: Evaluate Safety of T/I on Diabetic Subjects With Mild Obstructive Pulmonary Disease 16 PhRMA Pharmaceutical Clinical Trials in South Carolina

17 Study 8: Open-Label Assessment of the Albuterol Spiromax Dry Powder Inhaler (DPI) Study 9: A Study of the Effectiveness and Safety of Different Doses of Fluticasone Propionate Taken From a Dry Powder Inhaler (Puffer) in Adolescents and Adults Who Have Asthma That is Not Controlled by High Dose Inhaled Corticosteroid Asthma Medications Study 10: Efficacy and Safety of 2 Doses of Tiotropium Respimat Compared to Placebo in Children With Severe Persistent Asthma Study 11: Evaluate the Safety, Efficacy and Dose Response of GSK in Combination With Fluticasone Furoate in Subjects With Asthma Study 12: An Efficacy and Safety Study of Fluticasone Furoate/ Vilanterol (FF/VI) 200/25 Microgram (Mcg), FF/VI 100/25 Mcg, and FF 100 Mcg in Adults and Adolescents With Persistent Asthma Study 13: Efficacy and Safety of 2 Doses of Tiotropium Respimat Compared to Placebo in Children With Moderate Persistent Asthma Study 14: Efficacy and Safety of 2 Doses of Tiotropium Respimat Compared to Placebo in Adolescents With Severe Persistent Asthma Study 15: Evaluation of Tiotropium 2.5 and 5 Mcg Once Daily Delivered Via the Respimat Inhaler Compared to Placebo in 1 to 5 Year Old Patients With Persistent Asthma Study 16: Efficacy and Safety of QGE031 versus Placebo and Omalizumab in Patients Aged Years With Asthma Study 17: Efficacy of Inhaled Albuterol Spiromax in Subjects With Persistent Asthma Study 18: Dose Finding Study for QAW039 in Asthma Study 19: 12-Week Study in Adult Subjects With Asthma Study 20: A 12-Week Study in Adult Subjects With Asthma Study 21: Dose-ranging Study of Vilanterol (VI) Inhalation Powder in Children Research in Your Backyard Developing Cures, Creating Jobs 17

18 Study 22: Long-Term Efficacy and Safety Study of SCH / MK-8237 in Children and Adults With House Dust Mite-Induced Allergic Rhinitis/Rhinoconjunctivitis (P05607) Study 5: A Study of Perjeta (Pertuzumab) in Combination With Herceptin (Trastuzumab) and Chemotherapy in Patients With HER2-Positive Metastatic Gastroesophageal Junction or Gastric Cancer Study 6: Cancer (176 clinical trials recruiting) Study 1: Efficacy and Safety of Zoledronic Acid (Every 4 Weeks vs. Every 12 Weeks) in Patients With Documented Bone Metastases From Bone Cancer Study 2: Study of Denosumab in Subjects With Giant Cell Tumor of Bone Study 3: Phase 1 Study of PLX7486 as Single Agent and With Gemcitabine Plus Nab-Paclitaxel in Patients With Advanced Solid Tumors Study 4: A Study of Trastuzumab Emtansine Versus Trastuzumab as Adjuvant Therapy in Patients With HER2-Positive Breast Cancer Who Have Residual Tumor in the Breast or Axillary Lymph Nodes Following Preoperative Therapy (KATHERINE) Study of GDC-0068 Or GDC-0980 With Abiraterone Acetate Versus Abiraterone Acetate in Patients With Castration-Resistant Prostate Cancer Previously Treated With Docetaxel Chemotherapy Study 7: FOLFOX6m Plus SIR-Spheres Microspheres vs. FOLFOX6m Alone in Patients With Liver Mets From Primary Colorectal Cancer Study 8: Comparison of Neoadjuvant Chemotherapy With Weekly Paclitaxel or Eribulin Followed by A/C in Women With Locally Advanced HER2-Negative Breast Cancer Study 9: Phase Ib Study of PI3 (Phosphoinositol 3)-Kinase Inhibitor BAY With MEK (Mitogen-activated Protein Kinase) Inhibitor BAY in Patients With Advanced Cancer Study 10: Everolimus Plus Best Supportive Care vs. Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin) 18 PhRMA Pharmaceutical Clinical Trials in South Carolina

19 Study 11: S0820, Adenoma and Second Primary Prevention Trial Study 12: Phase I Study to Evaluate the Effect of LDE225 on the Pharmacokinetics of Bupropion and Warfarin in Patients Study 13: Phase II, Open Label, Single Arm Study of SAR In Myelofibrosis Patients Previously Treated With Ruxolitinib Study 14: A Phase 2 of GS-9973 in Combination With Idelalisib in Subjects With Relapsed or Refractory Hematologic Malignancies Study 15: Fosaprepitant + 5HT3 Receptor Antagonists + Dexamethasone in Germ Cell Tumors Study 16: DN24-02 as Adjuvant Therapy in Subjects With High Risk HER2+ Urothelial Carcinoma Study 17: Nilotinib Treatment-free Remission Study in CML (Chronic Myeloid Leukemia) Patients Study 18: Safety and Efficacy of Pomalidomide, Bortezomib and Low-dose Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma Study 19: First-line Everolimus +/- Paclitaxel for Cisplatin-ineligible Patients With Advanced Urothelial Carcinoma Study 20: ADAM-Afatinib Diarrhea Assessment and Management Study 21: Phase 3 Study of Nivolumab or Nivolumab Plus Ipilimumab Versus Ipilimumab Alone in Previously Untreated Advanced Melanoma Study 22: Study to Compare the Effect of Ipilimumab Retreatment With Chemotherapy in Advanced Melanoma Study 23: A Study of Two Vismodegib Regimens in Patients With Multiple Basal Cell Carcinomas Study 24: A Study Of Inotuzumab Ozogamicin Versus Investigator s Choice Of Chemotherapy In Patients With Relapsed Or Refractory Acute Lymphoblastic Leukemia Research in Your Backyard Developing Cures, Creating Jobs 19

20 Study 25: A Randomized, Controlled Study Evaluating the Efficacy and Safety of GS-1101 (CAL-101) in Combination With Ofatumumab for Previously Treated Chronic Lymphocytic Leukemia Study 26: Study of Brentuximab Vedotin Combined With Bendamustine in Patients With Hodgkin Lymphoma Study 27: CNDO-109-AANK for AML in First Complete Remission (CR1) Study 28: A Study of the Bruton s Tyrosine Kinase Inhibitor Ibrutinib Given in Combination With Bendamustine and Rituximab in Patients With Newly Diagnosed Mantle Cell Lymphoma Study 29: Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia Study 30: Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia in Complete Remission Study 31: Phase II Phosphatidylinositol 3-Kinase (PI3K) Inhibitor in Relapsed, Indolent or Aggressive Non-Hodgkin s Lymphomas (NHL) Study 32: Safety and Efficacy of CML Patients Who Switch to Nilotinib and Stop Treatment After Achieving and Sustaining MR4.5 Study 33: Phase III Study of CPX-351 Versus 7+3 in Patients Years Old With Untreated High Risk (Secondary) Acute Myeloid Leukemia Study 34: Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome Study 35: Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome Study 36: Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis Study 37: Anemia Treatment for Advanced Non-Small Cell Lung Cancer (NSCLC) Patients Receiving Chemotherapy Study 38: TRINOVA-3: A Study of AMG 386 or AMG 386 Placebo in Combination With Paclitaxel and Carboplatin to Treat Ovarian Cancer 20 PhRMA Pharmaceutical Clinical Trials in South Carolina

21 Study 39: Comparison of Cabazitaxel/Prednisone Alone or in Combination With Custirsen for 2nd Line Chemotherapy in Prostate Cancer Study 40: Safety and Efficacy Study of Enzalutamide Versus Bicalutamide in Men With Prostate Cancer Study 41: Denosumab Compared to Zoledronic Acid in the Treatment of Bone Disease in Subjects With Multiple Myeloma Study 42: A Phase 1 Study Evaluating AMG 232 in Advanced Solid Tumors Study 43: A Study of Onartuzumab (MetMAb) in Combination With Tarceva (Erlotinib) in Patients With Met Diagnostic-Positive Non-Small Cell Lung Cancer Who Have Received Chemotherapy For Advanced or Metastatic Disease (MetLung) Study 44: A Study of Pertuzumab in Addition to Chemotherapy and Herceptin (Trastuzumab) as Adjuvant Therapy in Patients With HER2-Positive Primary Breast Cancer Study 46: Study for Women With Platinum Resistant Ovarian Cancer Evaluating EC145 in Combination With Doxil (PROCEED) Study 47: IMAAGEN: Impact of Abiraterone Acetate in Prostate-Specific Antigen Study 48: A Trial of Preoperative MM-121 With Paclitaxel in HER2-negative Breast Cancer Study 49: A Study of Necitumumab and Chemotherapy in Participants With Stage IV Squamous Non-Small Cell Lung Cancer Study 50: A Study to Test Safety and Efficacy of IMGN901 in Combination With Carboplatin/Etoposide in Patients With Advanced Solid Tumors and Extensive Stage Small Cell Lung Cancer Study 51: Trial in Squamous Non Small Cell Lung Cancer Subjects Comparing Ipilimumab Plus Paclitaxel and Carboplatin Versus Placebo Plus Paclitaxel and Carboplatin Study 45: A Phase 3 Efficacy Study of a Recombinant Vaccinia Virus Vaccine to Treat Metastatic Prostate Cancer Research in Your Backyard Developing Cures, Creating Jobs 21

22 Study 52: VTX-2337 and Pegylated Liposomal Doxorubicin (PLD) in Patients With Recurrent or Persistent Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer Study 53: Study of Cabozantinib (XL184) Versus Prednisone in Men With Metastatic Castration-resistant Prostate Cancer Previously Treated With Docetaxel and Abiraterone or MDV Study 54: A Phase II Study to Evaluate the Efficacy of TKI258 for the Treatment of Patients With FGFR2 Mutated or Wild-type Advanced and/or Metastatic Endometrial Cancer Study 55: A Phase 1b/2 Study of OMP-59R5 in Combination With Gemcitabine in Subjects With Previously Untreated Stage IV Pancreatic Cancer Study 56: Tivozanib Hydrochloride in Combination With Paclitaxel Versus Placebo With Paclitaxel in Patients With Locally Recurrent or Metastatic Triple Negative Breast Cancer Study 57: Study of PX-866 and Docetaxel in Solid Tumors Study 58: The BEACON Study (Breast Cancer Outcomes With NKTR-102) Study 59: Dose Finding Study of RAD001 (Everolimus, Afinitor ) in Combination With BEZ235 in Patients With Advanced Solid Tumors Study 60: Study of a Drug [DCVax -L] to Treat Newly Diagnosed GBM Brain Cancer Study 61: A Study of AMG 820 in Subjects With Advanced Solid Tumors Study 62: M402 and Gemcitabine in Pancreatic Cancer Part A Study 63: Phase 1b/2 Trial of AMG386 With Pemetrexed and Carboplatin in Non-Small Cell Lung Cancer Study 64: A Study That Provides Long-term Safety Follow-up and Examines Long-term Exposure to Abiraterone Acetate 22 PhRMA Pharmaceutical Clinical Trials in South Carolina

23 Study 65: Study of Imprime PGG in Combination With Cetuximab in Subjects With Recurrent or Progressive KRAS Wild Type Colorectal Cancer Study 66: Two-Dose Level Evaluation of NX-1207 for the Treatment of Low Risk, Localized (T1c) Prostate Cancer Study 67: Study With TMX-101 in Patients With Carcinoma In Situ (CIS) Bladder Cancer Study 68: Study Assessing Safety, Pharmacokinetics and Efficacy of CC-223 With Either Erlotinib or Oral Azacitidine in Advanced Non-Small Cell Lung Cancer Study 69: Study of MM-398 With or Without 5-Fluorouracil and Leucovorin, Versus 5-Fluorouracil and Leucovorin in Patients With Metastatic Pancreatic Cancer Study 70: Immunotherapy Study for Surgically Resected Pancreatic Cancer Study 71: Intravesical Administration of INSTILADRIN (rad-ifn With Syn3) in Patients With Bladder Cancer Study 72: A 2 Part Phase 2 Trial of Galeterone in the Treatment of Castration Resistant Prostate Cancer Study 73: A Study in Second Line Metastatic Colorectal Cancer Study 74: Sativex for Relieving Persistent Pain in Patients With Advanced Cancer Study 75: AZD2014 and Fulvestrant in Patients With ER+ Advanced Metastatic Breast Cancer Study 76: The Study Evaluating Efficacy And Tolerability Of Veliparib in Combination With Temozolomide or In Combination With Carboplatin and Paclitaxel Versus Placebo in Subjects With BRCA1 and BRCA2 Mutation and Metastatic Breast Cancer Study 77: Efficacy & Safety of ODSH (2-0, 3-0 Desulfated Heparin) in Patients With Metastatic Pancreatic Cancer Treated With Gemcitabine & Abraxane Study 78: Trial Evaluating Dovitinib Combined With Fulvestrant, in Postmenopausal Patients With HER2- and HER+ Breast Cancer Research in Your Backyard Developing Cures, Creating Jobs 23

24 Study 79: A Phase 2 Diagnostic Imaging Study With 99mTc-MIP-1404 in Men With High-Risk Prostate Cancer Scheduled for Radical Prostatectomy (RP) and Extended Pelvic Lymph Node Dissection (EPLND) Compared to Histopathology Study 80: Phase III Study of BKM120/Placebo With Fulvestrant in Postmenopausal Patients With Hormone Receptor Positive HER2-negative Locally Advanced or Metastatic Breast Cancer Refractory to Aromatase Inhibitor Study 81: Early Switch From First-Line Docetaxel/Prednisone to Cabazitaxel/Prednisone and the Opposite Sequence, Exploring Molecular Markers in Men With Metastatic Castration-Resistant Prostate Cancer (mcrpc) Study 82: A Phase II Clinical Study of BBI608 in Adult Patients With Advanced Colorectal Cancer Study 83: A Study of PSMA ADC in Subjects With Metastatic Castration-resistant Prostate Cancer (mcrpc) Study 84: Safety and Efficacy of BKM120 in Patients With Metastatic Non-small Cell Lung Cancer Study 85: Erlotinib Plus Tivantinib (ARQ 197) Versus Single Agent Chemotherapy in Locally Advanced or Metastatic Non-Small Cell Lung Cancer Study 86: A Study of Paclitaxel With GDC-0941 Versus Paclitaxel With Placebo in Patients With Locally Recurrent or Metastatic Breast Cancer Study 87: Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): An Extension Study (ROMANA 3) Study 88: Safety and Efficacy of Anamorelin HCl in Patients With Non-Small Cell Lung Cancer-Cachexia (ROMANA 2) Study 89: Study of BMS Compared to Docetaxel in Previously Treated Advanced or Metastatic Squamous Cell Non-small Cell Lung Cancer (NSCLC) Study 90: A Phase I Clinical Study of BBI503 in Adult Patients With Advanced Solid Tumors Study 91: A Phase I Clinical Study of BBI608 in Adult Patients With Advanced Malignancies 24 PhRMA Pharmaceutical Clinical Trials in South Carolina