Breakthrough Therapy For Patients With Chronic Lymphocytic...

Introduction Breakthrough Therapy Designation is introduced as a new designation with the passage of the FDA Safety and Innovation Act of 2012 (FDASIA) [1]. According to section 902 of the FDASIA, the general criteria according to which this new designation can be applied are (1) serious or life-threatening disease or condition and (2) the drug also demonstrates substantial improvement over existing therapies on one or more clinically significant endpoints. Once a drug is designated as a Breakthrough Therapy, the FDA and the drug sponsor work closely to determine the most efficient pathway for generating additional evidence about safety and efficacy. As of March 31, 2015, a total of 293 requests for Breakthrough Therapy designation have been submitted, out of which 82 requests are granted [2]. This document outlines the application process for Breakthrough Therapy designation for novel drugs. It then proceeds with outlining the supporting data needed to apply for a Breakthrough Therapy designation for a novel anti-cancer molecule that has evidence of efficacy to treat patients with Chronic Lymphocytic Leukemia. When to apply for designation request The Breakthrough Therapy designation is granted on the basis of evaluating preliminary clinical evidence obtained from clinical trial data in humans. The data from animal studies or studies conducted in vitro are insufficient to defend this Breakthrough Therapy designation [1] . Therefore, a sponsor usually submits a BreakthroughShow MoreRelatedLeukemia1235 Words   |  5 PagesLeukemia Description: Leukemia is a type of blood cancer that begins in the bone marrow. Types of leukemia- acute or chronic: Acute leukemia gets worse very fast and may make you feel sick right away. Chronic leukemia gets worse slowly and may not cause symptoms for years. And Lymphocytic or myelogenous: lymphocytic affects the white blood cells called lymphocytes and myelogenous affects the white blood cells called myelocytes. Sign and symptoms: fever/ night sweats, headaches, bruising/bleedingRead MoreEssay about Leukemia and Its Treatment843 Words   |  4 Pages Leukemia is something that we hear about a lot. I believe that people do not understand exactly what leukemia is and how dangerous it can be, I chose this topic to try to help them understand. In this paper I will explain what leukemia is and what treatments you can choose from to treat the cancer. Leukemia is a type of cancer. Cancer is a group of more than 100 diseases that have two or more important things in common. One is that certain cells in the body become abnormal. Another is that theRead MoreReview of Cancer Treatments2177 Words   |  9 Pages and Gleevec. They all have a different tasks and developments that take affects in the body but each remarkable in its own way. INTRODUCTION Cancer in one way or another touches all of us, whether as a patient or through the diagnosis of the people you love around you. Millions of patients who are faced with cancer are depending on oncologists everywhere to cure cancer so others will not suffer like they had to. Optimistically, sooner rather than later this international problem will come toRead MoreCauses And Consequences Of Cancer Essay2258 Words   |  10 Pagesare expected to be diagnosed, out of which 589,430 patients are expected to lose their battle against cancer in 2015.3 Currently, cancer is the second most common cause of death in the US after heart diseases, causing the deaths of about 1,620 people per day and is expected to become number one within next few years.4 The significant increase in a number of cancer patients every year has propelled the interest of scientists to hunt for novel therapies to treat cancer. As a result, many new advancesRead MorePediatric Nursing Study Guide -- Covering Communicable Diseases/ Blood Disorders/Childhood Cancers/Integumentary5211 Words   |  21 Pages* Varicella pneumonia (rare in normal children) * Hemorrhagic Varicella (tiny hemorrhages in vesicles and numerous petechiae in skin) * Chronic or transient thrombocytopenia * Nursing Care Management * Maintain standard, airborne and contact precautions until ALL lesions are crusted; for immunized child w mild breakthrough Varicella, isolate until no new lesions are seen. * Keep child in home away from susceptible individuals until vesicles have dried (usually 1 wk