2018 年全球新药研发报告——第2 部分: 趋势与挑战(Ⅴ) Areport Of
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Horizon Scanning Status Report June 2019
Statement of Funding and Purpose This report incorporates data collected during implementation of the Patient-Centered Outcomes Research Institute (PCORI) Health Care Horizon Scanning System, operated by ECRI Institute under contract to PCORI, Washington, DC (Contract No. MSA-HORIZSCAN-ECRI-ENG- 2018.7.12). The findings and conclusions in this document are those of the authors, who are responsible for its content. No statement in this report should be construed as an official position of PCORI. An intervention that potentially meets inclusion criteria might not appear in this report simply because the horizon scanning system has not yet detected it or it does not yet meet inclusion criteria outlined in the PCORI Health Care Horizon Scanning System: Horizon Scanning Protocol and Operations Manual. Inclusion or absence of interventions in the horizon scanning reports will change over time as new information is collected; therefore, inclusion or absence should not be construed as either an endorsement or rejection of specific interventions. A representative from PCORI served as a contracting officer’s technical representative and provided input during the implementation of the horizon scanning system. PCORI does not directly participate in horizon scanning or assessing leads or topics and did not provide opinions regarding potential impact of interventions. Financial Disclosure Statement None of the individuals compiling this information have any affiliations or financial involvement that conflicts with the material presented in this report. Public Domain Notice This document is in the public domain and may be used and reprinted without special permission. Citation of the source is appreciated. All statements, findings, and conclusions in this publication are solely those of the authors and do not necessarily represent the views of the Patient-Centered Outcomes Research Institute (PCORI) or its Board of Governors. -
Volume 37 Number 10
VOLUME 37 NUMBER 10 NOVEMBER 2019 Gates Foundation Plots A Fresh Metric From US To EU: Young Biotechs Early Dialogue: Getting The Most For Market Access: Lives Saved Going It Alone Out Of Advice From HTA Agencies PAGE LEFT BLANK INTENTIONALLY invivo.pharmaintelligence.informa.com STRATEGIC INSIGHTS FOR LIFE SCIENCES DECISION-MAKERS CONTENTS ❚ November 2019 MARKET ACCESS Balancing benefit and value against health care sustainability 10 16 22 In US Drug Pricing Debate, Market Access 2020: Gates Foundation Plots A ICER’s Voice Gets Louder Understanding US Payer Fresh Metric For Market Access: MELANIE SENIOR Expectations Lives Saved The Institute for Clinical and Economic WILLIAM LOONEY WILLIAM LOONEY Review's influence on drug pricing, and Big pharma is facing a difficult US In Vivo visits Gates Medical Research policy, is growing. Spotlighting the worst competitive landscape as its traditional Institute CEO Dr. Penny Heaton to review its drug price rises is one recent example. customers realign to build their own first pipeline of drugs and vaccines to Ten years ago, it would have seemed redoubts of size, scale and reach. attack four of the world’s biggest killers: unthinkable that an independent, Consolidation on the payer side is TB, malaria, enteric diseases and other non-profit organization with no statutory changing the dynamics of success in conditions affecting maternal, newborn power could influence the pricing health care. and child health, as well as highlight the behavior of the US pharmaceutical sector. unique business model of this latest Yet that is what ICER has achieved. 36 addition to the Bill & Melinda Gates Foundation and reveal more about the From US To EU: focus and aims of the Boston, US group. -
The SMA Market: Assessing the Unknowns ❚ MARKET ACCESS: When Disease Dynamics Change the SMA Market: Assessing the Unknowns
The SMA Market: Assessing The Unknowns ❚ MARKET ACCESS: When Disease Dynamics Change The SMA Market: Assessing The Unknowns The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real-world evidence is needed. BY ALESSIA DEGLINCERTI, FRANK he landmark FDA approval of Novartis AG’s Zolgensma (onasemno- BOROWSKY AND MARK RATNER gene abeparvovec-xioi) in May 2019 shook the biopharma world in several ways including its price ($2.1m per dose) and as important, the very small With the approval of two disease data set on which the FDA primarily based its decision – an ongoing open- modifying agents, some patients label single arm trial of 21 infantile-onset patients with spinal muscular with SMA are becoming healthier Tatrophy (SMA) under two years old. Biogen Inc.’s Spinraza (nusinersen) had already and their medical needs are shifting. been approved in SMA in December 2016. As disease-modifying therapies, these compounds are a rarity in the field of neuro- A new natural course of the disease is muscular diseases of genetic origin. They are also at the core of a fascinating, ongo- emerging, with a larger population of ing real-world case study in how the natural course of a disease can change rapidly. individuals having stabilized disease. How companies’ SMA drug development and market access strategies evolve, both in But the extent of residual issues is not yet known – a picture that will only terms of new disease-modifying agents and supportive therapies that address residual come into focus over time. symptoms, could become a blueprint for other neuromuscular diseases like Duchenne’s Muscular Dystrophy (DMD) or Huntington’s Disease. -
Annexes to the Annual Report of the European Medicines Agency 2014
Annexes to the annual report of the European Medicines Agency 2014 Table of contents Annex 1 – Members of the Management Board ............................................................................. 2 Annex 2 – Members of the Committee for Medicinal Products for Human Use ................................... 4 Annex 3 – Members of the Pharmacovigilance Risk Assessment Committee ...................................... 6 Annex 4 – Members of the Committee for Medicinal Products for Veterinary Use ............................... 8 Annex 5 – Members of the Committee on Orphan Medicinal Products ............................................ 10 Annex 6 – Members of the Committee on Herbal Medicinal Products .............................................. 12 Annex 07 – Committee for Advanced Therapies .......................................................................... 14 Annex 8 – Members of the Paediatric Committee ........................................................................ 16 Annex 9 – Working parties and working groups .......................................................................... 18 Annex 10 – CHMP opinions in 2014 on medicinal products for human use ...................................... 22 Annex 11 – CVMP opinions in 2014 on medicinal products for veterinary use .................................. 36 Annex 12 – COMP opinions in 2014 on designation of orphan medicinal products ............................ 41 Annex 13 – HMPC European Union herbal monographs in 2014.................................................... -
Patent Application Publication ( 10 ) Pub . No . : US 2019 / 0192440 A1
US 20190192440A1 (19 ) United States (12 ) Patent Application Publication ( 10) Pub . No. : US 2019 /0192440 A1 LI (43 ) Pub . Date : Jun . 27 , 2019 ( 54 ) ORAL DRUG DOSAGE FORM COMPRISING Publication Classification DRUG IN THE FORM OF NANOPARTICLES (51 ) Int . CI. A61K 9 / 20 (2006 .01 ) ( 71 ) Applicant: Triastek , Inc. , Nanjing ( CN ) A61K 9 /00 ( 2006 . 01) A61K 31/ 192 ( 2006 .01 ) (72 ) Inventor : Xiaoling LI , Dublin , CA (US ) A61K 9 / 24 ( 2006 .01 ) ( 52 ) U . S . CI. ( 21 ) Appl. No. : 16 /289 ,499 CPC . .. .. A61K 9 /2031 (2013 . 01 ) ; A61K 9 /0065 ( 22 ) Filed : Feb . 28 , 2019 (2013 .01 ) ; A61K 9 / 209 ( 2013 .01 ) ; A61K 9 /2027 ( 2013 .01 ) ; A61K 31/ 192 ( 2013. 01 ) ; Related U . S . Application Data A61K 9 /2072 ( 2013 .01 ) (63 ) Continuation of application No. 16 /028 ,305 , filed on Jul. 5 , 2018 , now Pat . No . 10 , 258 ,575 , which is a (57 ) ABSTRACT continuation of application No . 15 / 173 ,596 , filed on The present disclosure provides a stable solid pharmaceuti Jun . 3 , 2016 . cal dosage form for oral administration . The dosage form (60 ) Provisional application No . 62 /313 ,092 , filed on Mar. includes a substrate that forms at least one compartment and 24 , 2016 , provisional application No . 62 / 296 , 087 , a drug content loaded into the compartment. The dosage filed on Feb . 17 , 2016 , provisional application No . form is so designed that the active pharmaceutical ingredient 62 / 170, 645 , filed on Jun . 3 , 2015 . of the drug content is released in a controlled manner. Patent Application Publication Jun . 27 , 2019 Sheet 1 of 20 US 2019 /0192440 A1 FIG . -
Innovationsreport 2020 Kurzfassung
Innovationsreport 2020 Auswertungsergebnisse von Routinedaten der Techniker Krankenkasse aus den Jahren 2017 bis 2018 Herausgeber: Gerd Glaeske Erstellt mit freundlicher Unterstützung der Techniker Krankenkasse (TK) 3 Herausgeber Prof. Dr. Gerd Glaeske Experten für ausgewählte Kapitel Prof. Dr. med. Janbernd Kirschner, Bonn Prof. Dr. med. Dieter Ukena, Bremen Prof. Dr. med. Barbara Schmalfeldt, Hamburg Prof. Dr. med. Wolfgang Schramm, München Autoren Prof. Dr. med. Karl Broich, Dr. Stanislava Dicheva‐Radev, Dörte Fuchs, Prof. Dr. Gerd Glaeske, Dr. Marion Haberkamp, Dr. Iris Hinneburg, Friederike Höfel, Prof. Dr. Janbernd Kirschner, Dr. Wiebke Löbker, Anja Lübs, Dr. André S. Morawetz, Lutz Muth, Dr. Frauke Naumann‐Winter, Linda Richter, Saskia Ritter, Dr. Kristin Sauer, Dr. Birgit Schindler unter Mitarbeit von Esra Aksoy, Friederike Höfel, Berit Marquardt, Linda Richter, Marle Wilhelm Anschrift: Universität Bremen, SOCIUM, Mary‐Somerville‐Str. 5, 28359 Bremen Aus Gründen der besseren Lesbarkeit wurde auf die Nennung beider geschlechtsspezifischer Formen verzichtet. Im Allgemeinen ist aber das jeweils andere Geschlecht ebenfalls gemeint. 2 Glossar .......................................................................................... 7 Vorwort zum Innovationsreport 2020 ...........................................15 Vorwort des Herausgebers ............................................................17 1 Einleitung ................................................................................19 2 Ziele und Methodik..................................................................33 -
2016 Medicines in Development for Rare Diseases a LIST of ORPHAN DRUGS in the PIPELINE
2016 Medicines in Development for Rare Diseases A LIST OF ORPHAN DRUGS IN THE PIPELINE Autoimmune Diseases Product Name Sponsor Official FDA Designation* Development Status Actemra® Genentech treatment of systemic sclerosis Phase III tocilizumab South San Francisco, CA www.gene.com Adempas® Bayer HealthCare Pharmaceuticals treatment of systemic sclerosis Phase II riociguat Whippany, NJ www.pharma.bayer.com ARA 290 Araim Pharmaceuticals treatment of neuropathic pain in patients Phase II Tarrytown, NY with sarcoidosis www.ariampharma.com ARG201 arGentis Pharmaceuticals treatment of diffuse systemic sclerosis Phase II (type 1 native bovine skin Collierville, TN www.argentisrx.com collagen) BYM338 Novartis Pharmaceuticals treatment of inclusion body myositis Phase III (bimagrumab) East Hanover, NJ www.novartis.com CCX168 ChemoCentryx treatment of anti-neutrophil cytoplasmic Phase II (5a receptor antagonist) Mountain View, CA auto-antibodies associated vasculitides www.chemocentryx.com (granulomatosis with polyangitis or Wegener's granulomatosis), microscopic polyangitis, and Churg-Strauss syndrome * This designation is issued by the FDA's Office of Orphan Products Development while the drug is still in development. The designation makes the sponsor of the drug eligible for entitlements under the Orphan Drug Act of 1983. The entitlements include seven years of marketing exclusivity following FDA approval of the drug for the designated use. Medicines in Development: Rare Diseases | 2016 1 Autoimmune Diseases Product Name Sponsor Official FDA -
JULY 2019 Mrx Pipeline a View Into Upcoming Specialty and Traditional Drugs TABLE of CONTENTS
JULY 2019 MRx Pipeline A view into upcoming specialty and traditional drugs TABLE OF CONTENTS EDITORIAL STAFF Introduction Maryam Tabatabai, PharmD Editor in Chief Senior Director, Drug Information Pipeline Deep Dive Carole Kerzic, RPh Executive Editor Drug Information Pharmacist Keep on Your Radar Consultant Panel Michelle Booth, Pharm D Director, Medical Pharmacy Strategy Becky Borgert, PharmD, BCOP Pipeline Drug List Director, Clinical Oncology Product Development Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Glossary Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs YuQian Liu, PharmD Manager, Specialty Clinical Programs Troy Phelps Senior Director, Analytics Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management. 1 | magellanrx.com INTRODUCTION Welcome to the MRx Pipeline. In its third year of publication, this quarterly report offers clinical insights and competitive intelligence on anticipated drugs in development. Our universal forecast addresses trends applicable across market segments. Traditional and specialty drugs, agents under the pharmacy and medical benefits, new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars are profiled in the report. -
RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges
1521-0081/72/4/862–898$35.00 https://doi.org/10.1124/pr.120.019554 PHARMACOLOGICAL REVIEWS Pharmacol Rev 72:862–898, October 2020 Copyright © 2020 by The Author(s) This is an open access article distributed under the CC BY-NC Attribution 4.0 International license. ASSOCIATE EDITOR: RHIAN M. TOUYZ RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges Ai-Ming Yu, Young Hee Choi, and Mei-Juan Tu Department of Biochemistry and Molecular Medicine, UC Davis School of Medicine, Sacramento, California (A.-M.Y., Y.H.C., M.-J.T.) and College of Pharmacy and Integrated Research Institute for Drug Development, Dongguk University-Seoul, Goyang-si, Gyonggi-do, Republic of Korea (Y.H.C.) Abstract. ....................................................................................863 Significance Statement ......................................................................863 I. Introduction. ..............................................................................863 II. Classification and General Features of RNA-Based Therapeutics .............................864 III. RNAs as Therapeutic Drugs .................................................................865 A. The Rise and Promise of RNA Therapeutics ..............................................865 B. Types of RNA Drugs and Mechanisms of Action ..........................................866 1. Antisense Oligonucleotides ...........................................................866 Downloaded from 2. Small Interfering RNAs . ............................................................868 -
Der Einfluss Der VO (EG) 1901/2006 Auf Den Off-Label Use in Der Neonatologie“
DIPLOMARBEIT / DIPLOMA THESIS Titel der Diplomarbeit / Title of the Diploma Thesis „Der Einfluss der VO (EG) 1901/2006 auf den Off-Label Use in der Neonatologie“ verfasst von / submitted by Stefanie Bub angestrebter akademischer Grad / in partial fulfilment of the requirements for the degree of Magistra der Pharmazie (Mag.pharm.) Wien, 2016 / Vienna, 2016 Studienkennzahl lt. Studienblatt / A 449 degree programme code as it appears on the student record sheet: Studienrichtung lt. Studienblatt / Diplomstudium Pharmazie degree programme as it appears on the student record sheet: Betreut von / Supervisor: Ao. Univ.-Prof. Mag. Dr. Rosa Lemmens Danksagung Als erstes möchte ich mich bei Frau Prof. Dr. Rosa Lemmens für die Betreuung meiner Diplomarbeit bedanken. Durch sie war es mir erst möglich, dieses Thema zu behandeln und meine Interessen zu verwirklichen. Ganz besonders möchte ich auch Dr. Andreas Doschek für seine jahrelange Unterstützung in diesem Studium danken. Abgesehen von den abertausenden Kopien und Ausdrucken, die er mir und meinen MitstudentInnen zur Verfügung gestellt hat, hat er mir vor jeder Prüfung den Rücken freigehalten, mich abgeprüft und mir so das Studium sehr erleichtert. Ihm habe ich auch das Interesse für die Materie des Arzneimittelrechts zu verdanken, weswegen ich mich für dieses Thema in meiner Abschlußarbeit entschieden habe. Meiner Familie und meinen Freunden danke ich für die Motivation und die guten Ratschläge und meinen MitstudentInnen für eine wundervolle Zeit. Danke! Wien, September 2016 I II Inhaltsverzeichnis Seite 1. Einführung 1 2. Grundlagen der zulassungsüberschreitenden Arzneimittelanwendung 4 2.1. Begriffsdefinitionen 4 2.1.1. Random controlled trial (RCT) 4 2.1.2. Pharmakokinetik-Studien 4 2.1.3. -
WHO Drug Information Vol
WHO Drug Information Vol. 34, No. 2, 2020 WHO Drug Information Contents Publication News 143 Publication of the 54th report of the World Health Organization (WHO) Expert Committee on Specifications for Pharmaceutical Preparations (ECSPP) (WHO Technical Series, N° 1025) Consultation Documents 146 Points to consider on the different approaches – including HBEL – to establish carryover limits in cleaning validation for identification of contamination risks when manufacturing in shared facilities 164 World Health Organization/United Nations Population Fund recommendations for condom storage and shipping 170 World Health Organization/United Nations Population Fund Guidance on conducting post-market surveillance of condoms 179 World Health Organization/United Nations Population Fund Guidance on testing of male latex condoms 201 Good reliance practices in regulatory decision-making: high-level principles and recommendations 231 Guideline on data integrity ATC/DDD Classification 255 ATC/DDD Classification (Temporary) 261 ATC/DDD Classification (Final) International Nonproprietary Names (INN) 263 List No. 123 of Proposed International Nonproprietary Names (INN) for Pharmaceutical Substances Abbreviations and websites CHMP Committee for Medicinal Products for Human Use (EMA) EMA European Medicines Agency (www.ema.europa.eu) EU European Union FDA U.S. Food and Drug Administration (www.fda.gov) Health Canada Federal department responsible for health product regulation in Canada (www.hc-sc.gc.ca) HPRA Health Products Regulatory Authority, Ireland -
Stembook 2018.Pdf
The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances FORMER DOCUMENT NUMBER: WHO/PHARM S/NOM 15 WHO/EMP/RHT/TSN/2018.1 © World Health Organization 2018 Some rights reserved. This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence (CC BY-NC-SA 3.0 IGO; https://creativecommons.org/licenses/by-nc-sa/3.0/igo). Under the terms of this licence, you may copy, redistribute and adapt the work for non-commercial purposes, provided the work is appropriately cited, as indicated below. In any use of this work, there should be no suggestion that WHO endorses any specific organization, products or services. The use of the WHO logo is not permitted. If you adapt the work, then you must license your work under the same or equivalent Creative Commons licence. If you create a translation of this work, you should add the following disclaimer along with the suggested citation: “This translation was not created by the World Health Organization (WHO). WHO is not responsible for the content or accuracy of this translation. The original English edition shall be the binding and authentic edition”. Any mediation relating to disputes arising under the licence shall be conducted in accordance with the mediation rules of the World Intellectual Property Organization. Suggested citation. The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances. Geneva: World Health Organization; 2018 (WHO/EMP/RHT/TSN/2018.1). Licence: CC BY-NC-SA 3.0 IGO. Cataloguing-in-Publication (CIP) data.