DOCSLIB.ORG

Examination of Clinical Trial Costs and Barriers for Drug Development Final

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Examination of Clinical Trial Costs and Barriers for Drug Development Final TASK ORDER NO. HHSP23337007T CONTRACT NO. HHSP23320095634WC EXAMINATION OF CLINICAL TRIAL COSTS AND BARRIERS FOR DRUG DEVELOPMENT FINAL Submitted to: Hui-Hsing Wong Amber Jessup U.S. Department of Health and Human Services Assistant Secretary of Planning and Evaluation (ASPE) 200 Independence Avenue, SW Washington, DC 20201 Submitted by: Aylin Sertkaya Anna Birkenbach Ayesha Berlind John Eyraud Eastern Research Group, Inc. 110 Hartwell Avenue Lexington, MA 02421 www.erg.com July 25, 2014 FINAL JULY 25, 2014 TABLE OF CONTENTS 1 INTRODUCTION AND BACKGROUND ............................................................................... 1-1 2 CLINICAL TRIAL DECISION-MAKING MODEL ............................................................. 2-1 2.1 DATA SOURCES ...................................................................................................................... 2-2 2.1.1 Publically Available Literature ................................................................................. 2-3 2.1.2 Discussions with Experts, FDA Personnel, Drug Sponsors, Contract Research Organizations (CROs), and Academic Clinical Research Centers ........................... 2-3 2.1.3 Medidata Solutions Databases .................................................................................. 2-4 2.2 CONCEPTUAL FRAMEWORK ................................................................................................... 2-5 2.3 OPERATIONAL MODEL ........................................................................................................... 2-8 2.4 MODEL PARAMETERS ............................................................................................................. 2-8 3 ANALYSIS OF COSTS .............................................................................................................. 3-1 3.1 COSTS BY THERAPEUTIC AREA .............................................................................................. 3-2 3.2 COSTS BY TRIAL PHASE ......................................................................................................... 3-4 3.3 COSTS BY COST COMPONENT ................................................................................................. 3-5 3.4 CONCLUSIONS ......................................................................................................................... 3-7 4 BARRIERS TO CLINICAL TRIALS ...................................................................................... 4-1 4.1 HIGH FINANCIAL COST ........................................................................................................... 4-1 4.2 LENGTHY TIMELINES .............................................................................................................. 4-3 4.3 DIFFICULTIES IN RECRUITING AND RETAINING PARTICIPANTS ............................................. 4-5 4.4 INCREASING COMPETITION FOR QUALIFIED INVESTIGATORS AND SITES .............................. 4-6 4.5 REGULATORY AND ADMINISTRATIVE BARRIERS ................................................................... 4-7 4.5.1 Regulations Protecting Human Research Subjects and Their Privacy ..................... 4-8 4.5.2 Safety Reporting Requirements for Investigational New Drugs (INDs) and Biologics (21 CFR 312) ...................................................................................... 4-9 4.5.3 Regulations for Multiple Jurisdictions .................................................................... 4-11 4.5.4 Conservative Regulatory Climate ........................................................................... 4-12 4.5.5 Lack of Clear Regulatory Pathways and Guidance for Some Therapeutic Areas .. 4-13 4.5.6 Barriers Related to the Review Process .................................................................. 4-14 4.6 DRUG SPONSOR-IMPOSED BARRIERS ................................................................................... 4-16 4.6.1 Administrative ......................................................................................................... 4-16 4.6.2 Study Design ............................................................................................................ 4-16 4.6.3 Data and Site Monitoring ........................................................................................ 4-20 4.6.4 Serious Adverse Events (SAEs) Reporting for Investigational New Drugs and Biologics (INDs) (21 CFR 312) ............................................................................... 4-22 4.7 DISCONNECT BETWEEN CLINICAL RESEARCH AND MEDICAL CARE ................................... 4-22 4.8 BARRIERS AT ACADEMIC INSTITUTIONS .............................................................................. 4-23 4.9 BARRIERS RELATED TO THE GLOBALIZATION OF CLINICAL RESEARCH ............................. 4-24 5 ANALYSIS OF BARRIERS TO CLINICAL TRIALS ........................................................... 5-1 i FINAL JULY 25, 2014 5.1 USE OF ELECTRONIC HEALTH RECORDS (EHR) .................................................................... 5-3 5.2 RELAXING TRIAL ENROLLMENT RESTRICTIONS ...................................................................... 5-4 5.3 SIMPLIFIED CLINICAL TRIAL PROTOCOLS AND REDUCED AMENDMENTS ............................. 5-5 5.4 REDUCED SOURCE DATA VERIFICATION (SDV) .................................................................... 5-6 5.5 WIDER USE OF MOBILE TECHNOLOGIES SUCH AS ELECTRONIC DATA CAPTURE (EDC) ...... 5-8 5.6 WIDER USE OF LOWER-COST FACILITIES AND/OR AT-HOME TESTING ................................. 5-9 5.7 PRIORITY REVIEW/PRIORITY REVIEW VOUCHERS................................................................ 5-11 5.8 IMPROVEMENTS IN FDA REVIEW PROCESS EFFICIENCY AND MORE FREQUENT AND TIMELY INTERACTIONS WITH FDA .............................................................................. 5-11 5.9 CONCLUSIONS ....................................................................................................................... 5-12 6 REFERENCES ............................................................................................................................ 6-1 APPENDIX A: DRAFT INTERVIEW GUIDE,................................................................................... A-1 APPENDIX B: MEDIDATA DATA ELEMENT DESCRIPTIONS .................................................. B-1 APPENDIX C: FEATURES OF OPERATIONAL MODEL ............................................................. C-1 APPENDIX D: ADDITIONAL DATA CLEANING STEPS .............................................................. D-1 ii FINAL JULY 25, 2014 LIST OF TABLES AND FIGURES FIGURE 1: NEW MOLECULAR ENTITY (NME) AND NEW BIOLOGIC ENTITY (NBE) FILINGS AND APPROVALS ................................................................................................................................... 1-1 FIGURE 2: DRUG DEVELOPMENT DECISION TREE DEPICTING NET PRESENT VALUE (NPV) OF RETURNS AT EACH NODE ........................................................................................................ 2-6 FIGURE 3: CLINICAL TRIAL COSTS (IN $ MILLIONS) BY PHASE AND THERAPEUTIC AREA ....................... 3-3 FIGURE 4: AVERAGE PER-STUDY COSTS BY PHASE (IN $ MILLIONS) ACROSS THERAPEUTIC AREAS ...... 3-4 FIGURE 5: PER-STUDY COSTS ACROSS ALL THERAPEUTIC AREAS, BY COST COMPONENT AND PHASE ... 3-7 FIGURE C - 2: WELCOME SCREEN OF THE CLINICAL TRIALS MODEL ....................................................... C-1 FIGURE C - 1: IMPACT OF REMOVAL OF BARRIERS SCREEN ..................................................................... C-1 FIGURE C - 3: SELECTION OF TYPE OF TRIAL SCREEN FOR EXAMINATION OF COSTS .............................. C-2 FIGURE C - 4: GENERAL QUESTIONS SCREEN ........................................................................................... C-2 FIGURE C - 5: PARAMETER VALUE ENTRY FOR CLINICAL TRIAL STUDY PER TRIAL PHASE SCREEN ...... C-3 FIGURE C - 6: RESULTS SCREEN ................................................................................................................ C-3 TABLE 1: TOTAL PER-STUDY COSTS (IN $ MILLIONS), BY PHASE AND THERAPEUTIC AREA [A] [B] ....... 3-3 TABLE 2: CLINICAL TRIAL COSTS, BY COST COMPONENT, PHASE, AND THERAPEUTIC AREA [A] [B]...... 3-6 TABLE 3: BARRIER MITIGATION MEASURES AND ASSOCIATED MODELING APPROACH FOR ANALYSIS ...................................................................................................................................... 5-2 TABLE 4: PROJECTED IMPACTS OF EHR USE ON CLINICAL TRIAL COSTS (IN $ MILLIONS AND IN PERCENTAGES), BY THERAPEUTIC AREA AND PHASE [A] ............................................................ 5-4 TABLE 5: PROJECTED IMPACTS OF RELAXING TRIAL ENROLLMENT RESTRICTIONS ON CLINICAL TRIAL COSTS (IN $ MILLIONS AND IN PERCENTAGES), BY THERAPEUTIC AREA AND PHASE [A] ........... 5-5 TABLE 6: PROJECTED IMPACTS OF SIMPLIFIED CLINICAL TRIAL PROTOCOLS AND REDUCED AMENDMENTS ON CLINICAL TRIAL COSTS (IN $ MILLIONS AND IN PERCENTAGES), BY THERAPEUTIC AREA AND PHASE [A] ............................................................................................ 5-6 TABLE 7: PROJECTED IMPACTS OF REDUCED SOURCE DATA VERIFICATION (SDV) ON CLINICAL TRIAL COSTS (IN $ MILLIONS AND IN PERCENTAGES), BY THERAPEUTIC AREA AND PHASE [A] ........... 5-8 TABLE 8: PROJECTED IMPACTS OF WIDER USE OF MOBILE TECHNOLOGIES, SUCH AS ELECTRONIC DATA CAPTURE (EDC), ON CLINICAL TRIAL COSTS (IN $ MILLIONS AND IN PERCENTAGES), BY THERAPEUTIC AREA AND PHASE [A] ......................................................................................
Load more

Recommended publications
  • 2017 Sustainability Report
    2017 SUSTAINABILITY REPORT CREATING A BETTER WORLD CONTENTS WHO WE ARE CREATING A BETTER WORLD 4 About this Report 10 Business Overview 24 Our Goals 6 Letter from our CEO 13 Our Products 29 Our Planet 8 About Medidata OUR APPROACH GRI INDEX 14 Stalkholders 30-32 GRI Index 16 Materials References: PEOPLE FIRST Priority Issue: An issue deemed material to the company through our sustainability issue prioritization process. 18 Ethics Global Reporting Initiative (GRI): The GRI Sustainability Reporting Standards are the first and 20 Our People most widely adopted global standards for sustainability reporting. 22 Diversity Matters UN Global Compact: The UNGC is a voluntary initiative based on CEO commitments to implement universal sustainability principles and to understand partnerships in support of UN Goals. Sustainable Development Goals: The SDGs are a universal call to action to end poverty, protect the planet, and ensure that all people enjoy peace and prosperity. 2 2017 MEDIDATA SUSTAINABILITY REPORT 2017 MEDIDATA SUSTAINABILITY REPORT 3 CREATING A CREATING A WHO WE ARE OUR APPROACH PEOPLE FIRST BETTER WORLD GRI INDEX WHO WE ARE OUR APPROACH PEOPLE FIRST BETTER WORLD GRI INDEX About this Report Letter from our CEO About Medidata Business Overview Our Products About this Report Letter from our CEO About Medidata Business Overview Our Products ABOUT THIS REPORT As we reflect on the last 18 years, the spirit of No information contained on our website is innovation—one that has truly paved the way intended to be included as part of, or incorporated for better solutions, higher quality treatments, by reference into, this Annual Report on Form and quicker go-to-market times—remains our 10-k.
    [Show full text]
  • Expression Profiling / Microarray / Data Analysis at Amgen
    Expression Profiling / Microarray / Data Analysis at Amgen Dan Fitzpatrick, PhD Expression Profiling & Pharmacogenomics Department Amgen, Inc. Changes in Drug Development: Feeding the Beast Dan Fitzpatrick, PhD Expression Profiling & Pharmacogenomics Department Amgen, Inc. Seminar Outline The current state of affairs, and where we want to go Instruments of change The Amgen experience The High Stakes in Pharmaceuticals •Global R&D increased 14% in 1999 to 24 billion. •Top 11 geographical markets grew 9% to $202 billion in sales. •U.S. market valued at $83 billion. …And High Pressure •U.S. FDA approved 35 new molecular entities in 1999 (30 in 1998, 39 in 1997). •36 branded pharmaceuticals ($1.9 billion in sales) came off patent protection in 1999. •Between 2000 & 2005, 173 products representing $30 billion in sales to lose patent protection. C&E News, Jan. 17, 2000 The Gap Between R&D and New Drugs New U.S. R&D molecular entities Year (Billions) FDA approved 1993 $10.5 25 1994 11.1 22 1995 11.9 28 1996 13.6 53 1997 15.5 39 1998 17.2 30 1999 20.1 35 Top Five Drug Targets 56 (11%) brands 29% of prescription sales HMG CoA reductase (hypercholesterolemia) Proton pump (ulcers) Serotonin transporter (depression) Calcium channel (hypertension) Angiotensin converting enzyme (hypertension) A Piece of the Pie Drug Company Zocor Merck & Co. HMG-CoA Lipitor Warner-Lambert/Pfizer Reductase Pravachol Bristol-Myers Squibb Inhibitors Mevalotin Sankyo Mevacor Merck & Co. Lescol Novartis Baycol Bayer/SKB Lodales Sanofi Why Pharmaceutical Executives Sleep like Babies at Night. •‘Return’ on R&D diminishing •Potential for reduced government subsidization and/or HMO reimbursement •Investor pressure •Biologics Biologics Come of Age Drug Company Target Indication 1.
    [Show full text]
  • Evidence Regarding Research and Development Investments in Innovative and Non-Innovative Medicines James Love Consumer Project on Technology September 22, 2003
    Evidence Regarding Research and Development Investments in Innovative and Non-Innovative Medicines James Love Consumer Project on Technology September 22, 2003 1. Introduction.......................................................................................................... 3 2. Empirical Estimates of Drug Development Costs........................................... 3 2.1 The Tufts Study ......................................................................................... 3 Table RND 2.1-1: Tufts study estimate of costs of development of self- originated New Chemical Entity .................................................................... 4 2.2 The TB Alliance Study of Drug Development Costs........................................ 6 2.3 Orphan Drug Development ........................................................................ 7 Table 2.3-1: Pre-Tax Cost of Trials for Orphan Products: FDA approvals of Orphan Indications and Orphan NMEs (Millions of USD)............................. 7 2.4 Parexel Analysis of Size of Clinical Trials ................................................. 9 Table RND 2.4-1 : Mean and median number of patents in clinical trials: FDA NME approvals 1998 - 2001 .......................................................................... 9 2.5 October 2001 FDA Study of Clinical Trials. .................................................... 9 2.6 The PERI Survey of development costs ........................................................... 9 Table RND 2.6-1: Average Time and Spending per project..........................10
    [Show full text]
  • Semi-Annual Market Review
    Semi-Annual Market Review HEALTH IT & HEALTH INFORMATION SERVICES JULY 2019 www.hgp.com TABLE OF CONTENTS 1 Health IT Executive Summary 3 2 Health IT Market Trends 6 3 HIT M&A (Including Buyout) 9 4 Health IT Capital Raises (Non-Buyout) 14 5 Healthcare Capital Markets 15 6 Macroeconomics 19 7 Health IT Headlines 21 8 About Healthcare Growth Partners 24 9 HGP Transaction Experience 25 10 Appendix A – M&A Highlights 28 11 Appendix B – Buyout Highlights 31 12 Appendix C – Investment Highlights 34 Copyright© 2019 Healthcare Growth Partners 2 HEALTH IT EXECUTIVE SUMMARY 1 An Accumulating Backlog of Disciplined Sellers Let’s chat about fireside chats. The term first used to describe a series of evening radio addresses given by U.S. President Franklin D. Roosevelt during the Great Depression and World War II is now investment banker speak for “soft launches” of sell-side and capital raise transactions. Every company has a price, and given a market of healthy valuations, more companies are testing the waters to find out whether they can achieve that price. That process now looks a little more informal, or how you might envision a fireside chat. Price (or valuation) discovery for a company can range from a single conversation with an individual buyer to a full-blown auction with hundreds of buyers and everything in between, including a fireside chat. Given the increasing share of informal conversations, the reality is that more companies are for sale than meets the eye. While the healthy valuations publicized and press-released are encouraging more and more companies to price shop, there is a simultaneous statistical phenomenon in perceived valuations that often goes unmentioned: survivorship bias.
    [Show full text]
  • Amgen's Complaint 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19
    Case3:14-cv-04741 Document1 Filed10/24/14 Page1 of 39 1 SIDLEY AUSTIN LLP Vernon M. Winters (SBN 130128) 2 555 California Street, Suite 2000 San Francisco, CA 94104-1503 3 Telephone: (415) 772-1200 4 Facsimile: (415) 772-7400 [email protected] 5 PAUL, WEISS, RIFKIND, WHARTON 6 & GARRISON LLP Nicholas Groombridge (pro hac vice application to be filed) 7 Jennifer Gordon Peter Sandel (pro hac vice application to be filed) 8 Jennifer H. Wu (pro hac vice application to be filed) 9 Michael T. Wu (pro hac vice application to be filed) 1285 Avenue of the Americas 10 New York, NY 10019-6064 Telephone: (212) 373-3000 11 Facsimile: (212) 757-3990 [email protected] 12 13 AMGEN INC. Wendy A. Whiteford 14 Lois M. Kwasigroch Kimberlin L. Morley 15 One Amgen Center Drive Thousand Oaks, CA 91320-1789 16 Telephone: (805) 447-1000 17 Facsimile: (805) 447-1010 [email protected] 18 Attorneys for Plaintiffs Amgen Inc. 19 and Amgen Manufacturing, Limited 20 UNITED STATES DISTRICT COURT 21 NORTHERN DISTRICT OF CALIFORNIA 22 AMGEN INC. and Case No. ____________ AMGEN MANUFACTURING, LIMITED, 23 24 Plaintiffs, COMPLAINT FOR PATENT vs. INFRINGEMENT, CONVERSION, 25 AND UNFAIR COMPETITION SANDOZ INC., SANDOZ (CAL. BUS. & PROF. CODE § 17200) 26 INTERNATIONAL GMBH, and SANDOZ GMBH, JURY TRIAL DEMANDED 27 28 Defendants. AMGEN’S COMPLAINT Case3:14-cv-04741 Document1 Filed10/24/14 Page2 of 39 1 Plaintiffs Amgen Inc. and Amgen Manufacturing, Limited (collectively, “Plaintiffs”), 2 by and through their undersigned attorneys, for their Complaint against Defendants Sandoz 3 Inc., Sandoz International GmbH, and Sandoz GmbH (collectively, “Defendants”) hereby 4 allege as follows: 5 NATURE OF THE ACTION 6 1.
    [Show full text]
  • Setting Fair Prices for Life-Saving Drugs by Bruce A
    Virtual Mentor American Medical Association Journal of Ethics January 2007, Volume 9, Number 1: 38-43. Policy forum Setting fair prices for life-saving drugs by Bruce A. Chabner, MD, and Thomas G. Roberts Jr., MD, MSocSci Cancer drugs are big business. Worldwide sales are projected to reach $25 billion in 2006 and to increase to almost $50 billion by 2010 [1]. This represents a startling growth in a segment of the drug industry once shunned by major pharmaceutical manufacturers as too high-risk and unprofitable. While a few drug companies, notably Bristol-Myers Squibb (BMS) and Pharmatalia, made significant profits on cancer drugs between 1970 and 1990 when the first effective combination therapies came into common practice, the turning point in this industrial segment occurred in 1992 with the approval of Bristol-Myers Squibb’s paclitaxel, which became a multibillion-dollar-per-year product by 1998. To understand our current concerns with cancer drug costs and their potential effect on medical care financing and access, one needs to be familiar with the paclitaxel experience. The story of paclitaxel’s discovery and commercial development reflects both the lack of interest that industry had in cancer drugs at that time and the sudden emergence of drug cost as a social justice issue. In 1964 Monroe Wall and associates, working at the Research Triangle Institute under a National Cancer Institute (NCI) contract, isolated the active compound in paclitaxel from the bark of the common yew tree [2]. Its tortuous development, complicated by difficulties in material procurement, compound purification and formulation, delayed its entry into clinical trials until 1983, and its efficacy in treating ovarian cancer was not demonstrated until 1987 [3].
    [Show full text]
  • Digital R&D: the Next Frontier for Biopharmaceuticals
    Digital R&D │ The Next Frontier for Biopharmaceuticals The Next Frontier Digital R&D The Next Frontier for Biopharmaceuticals Pharmaceuticals and Medical Products Practice 2017 Designed by the US Design Center Copyright © McKinsey & Company, Inc. www.mckinsey.com Digital R&D The Next Frontier for Biopharmaceuticals 2017 Editors Sastry Chilukuri Ann Westra For more info please contact [email protected] Contents 1 Digital in R&D—the $100 billion opportunity Sastry Chilukuri, Edd Fleming, and Ann Westra R&D in the age of analytics 13 Real-world evidence: From activity to impact Olivia Cavlan, Sastry Chilukuri, Matthias Evers, and Ann Westra 31 Randomized pragmatic trials: Can they fulfill their promise? Arnaub Chatterjee, Sastry Chilukuri, Michael Pencina, Eric Peterson, Saif Rathore, and Vijay Vaidya 35 The next generation in clinical operations performance Sastry Chilukuri, Edd Fleming, Eoin Leydon, Fareed Melhem, and Michael Steinmann 47 Moving beyond serendipity in drug discovery Sastry Chilukuri, Leeland Ekstrom, Jonathan Usuka, and Ann Westra 61 Digital vigilance: Building the backbone for insight-driven safety Kate Chavez and Brandon Parry 69 How big data can revolutionize pharmaceutical R&D Jamie Cattell, Sastry Chilukuri, and Michael Levy Connecting with the individual customer 85 Medical affairs: Key imperatives for engaging and educating physicians in a digital world Matthias Evers, Ivan Ostojic, Brindan Suresh, Josh Weiner, and Ann Westra 101 Engaging patients during clinical trials Montana Cherney, Amit Paley, Leslie Ruckman,
    [Show full text]
  • Estimated Research and Development Investment Needed to Bring a New
    Estimated research and development investment needed to bring a new medicine to market, 2009-2018 LSE Research Online URL for this paper: http://eprints.lse.ac.uk/103688/ Version: Accepted Version Article: Wouters, Olivier J., McKee, Martin and Luyten, Jeroen (2020) Estimated research and development investment needed to bring a new medicine to market, 2009- 2018. JAMA, 323 (9). pp. 844-853. ISSN 0098-7484 https://doi.org/10.1001/jama.2020.1166 Reuse Items deposited in LSE Research Online are protected by copyright, with all rights reserved unless indicated otherwise. They may be downloaded and/or printed for private study, or other acts as permitted by national copyright laws. The publisher or other rights holders may allow further reproduction and re-use of the full text version. This is indicated by the licence information on the LSE Research Online record for the item. [email protected] https://eprints.lse.ac.uk/ Estimated research and development investment needed to bring a new medicine to market, 2009-2018 Olivier J. Wouters, PhD1*; Martin McKee, MD, DSc2; Jeroen Luyten, PhD3 1 Department of Health Policy, London School of Economics and Political Science, London, UK 2 Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, UK 3 Leuven Institute for Healthcare Policy, Department of Public Health and Primary Care, KU Leuven, Belgium Olivier J. Wouters, PhD Assistant Professor of Health Policy Department of Health Policy London School of Economics and Political Science Houghton Street London WC2A 2AE United Kingdom [email protected] * Corresponding author Prof.
    [Show full text]
  • Drug Pricing and Pharmaceutical Patenting Practices
    Drug Pricing and Pharmaceutical Patenting Practices February 11, 2020 Congressional Research Service https://crsreports.congress.gov R46221 SUMMARY R46221 Drug Pricing and Pharmaceutical Patenting February 11, 2020 Practices Kevin T. Richards, Intellectual property (IP) rights in pharmaceuticals are typically justified as necessary to allow Coordinator manufacturers to recoup their substantial investments in research, development, and regulatory Legislative Attorney approval. IP law provides exclusive rights in a particular invention or product for a certain time period, potentially enabling the rights holder (e.g., a brand-name drug manufacturer) to charge Kevin J. Hickey higher-than-competitive prices. If rights holders are able to charge such prices, they have an Legislative Attorney incentive to lengthen the period of exclusive rights as much as possible. Indeed, some commentators allege that pharmaceutical manufacturers have engaged in patenting practices that unduly extend the period of exclusivity. These critics argue that these patenting practices are used Erin H. Ward to keep drug prices high, without any benefit for consumers or innovation. Criticisms center on Legislative Attorney four such practices: “Evergreening”: So-called patent “evergreening” is the practice of filing for new patents on secondary features of a particular product as earlier patents expire, thereby extending patent exclusivity past the original twenty-year term. Later-filed patents may delay or prevent entry by competitors, thereby allowing the brand-name
    [Show full text]
  • Check out Our White Paper
    Welcome to Accumulus Synergy We are developing a global information exchange platform to transform how drug innovators and health regulators interact to bring safe and effective medicines to patients faster and more efficiently. i It’s time for a moon shot. M ultiple worlds have moved online in the past three decades, from entertainment to commerce and the arts. The world of information and data exchange between biopharmaceuticals and regulators has lagged behind in some respects. Filing a New Drug Application may have shifted from driving a truckload of paper to the relevant regulatory authority, to FedEx-ing a CD-ROM, to uploading a set of PDFs through the Electronic Submissions Gateway, but the documents themselves, and the underlying processes, remain little changed. It’s long past time to play catchup — it’s time for a moon shot. At Accumulus Synergy, our vision is to accelerate the delivery of critical therapies to the citizens of the world. We should all want to accelerate these critical thera- pies to citizens globally in a safe and effective way. We use the word “citizens” intentionally, to include patients, caregivers, providers, and family members. At some point in our lives, we all benefit from medicines and vaccines. We envision information exchange between a biopharma company and its regula- tors changing from the traditional dispatch of static information and documents, to an invitation to access a cloud environment to view up-to-date data as it emerges. Of course, the environment has to be access based, ensure data privacy and meet or exceed current cyber security parameters.
    [Show full text]
  • 4Th DIA China Annual Meeting
    ײࣷනٷ ˖࿋ڇࢇӸ 35(/,0,1$5< 352*5$0 ዐࡔᅅᄱࡔा঍ୁዐ႐ ',$ ዐࡔ౎ࣷ ຺াᄱ࿿႑တၹࣷڼ ႎظዐࡔᇑ๘হüüࢇፕᇑ WK',$&KLQD$QQXDO0HHWLQJ &ROODERUDWLRQ ,QQRYDWLRQLQ&KLQD ዐࡔฉ࡛ࡔाࣷᅱዐ႐ 6KDQJKDL,QWHUQDWLRQDO&RQYHQWLRQ&HQWHU&KLQD ౎ሆනȍȍጆ༶ಢჟ ౎ሆනȍȍࣷᅱԒߢĂ༪ஃĂቛબࢅஃ࿔ՄԒ 0D\3UHFRQIHUHQFH:RUNVKRSV 0D\&RQIHUHQFH([KLELWLRQDQG3RVWHUV WWW.DIACHINA.ORG John J. HU, PhD Vice President and General Manager, United States Pharmacopeia-China Welcome to 4th DIA China Annual Meeting in Shanghai! This year’s theme, “Collaboration and Innovation in China”, speaks to the fact that future pharmaceutical innovation is about collaboration, and China is an important member of the global innovation community that will contribute to this partnership. Thanks to the outstanding hard work of the program committee and DIA-China staff, we have added exciting new events to this year’s program. We are delighted and hon- ored to have Dr. Yin Li, the commissioner of SFDA, and two distinguished international thought leaders, Dr. David Ho and Dr. Alberto Grignolo as keynote speakers. For the first time, SFDA’s Center for Drug Evaluation (CDE), has agreed to conduct a town hall meeting that will allow meeting participants have an open and direct dialog with CDE. Working with China’s SFDA, US FDA and US Department of Commerce, we have also added two new API regulatory workshops at the preconference session. In addition, we are fortunate to be able to invite Japan’s PMDA to provide a special session focus- ing on recent regulatory policy development in the world’s second largest pharmaceu- tical market. New to this year’s program are dedicated tracks on biological and vac- cine development, CRO collaborations and China’s various high-tech parks that offer government incentives for pharmaceutical R&D activities.
    [Show full text]
  • COVID-19 and Clinical Trials: the Medidata Perspective Release 7.0
    MAYJULY 18, 13, 2020 2020 COVID-19 and Clinical Trials: The Medidata Perspective Release 7.0 Copyright 2020 Medidata Solutions, Inc., a Dassault Systèmes company JULY 13, 2020 2 COVID-19 AND CLINICAL TRIALS: THE MEDIDATA PERSPECTIVE Table of Contents What’s New/What’s Significantly Updated in Release 7.0 3 Insights to Ongoing Data Capture in Clinical Trials 3 Regulatory Response 5 Impact to Medidata Customers, Patients and Trials 6 The Race for a Vaccine 7 Medidata Solutions to Assist Sponsors/Partners, Patients and Trials 10 Details on New and Adapted Medidata Solutions 11 Summary 18 Copyright 2020 Medidata Solutions, Inc., a Dassault Systèmes company JULY 13, 2020 3 COVID-19 AND CLINICAL TRIALS: THE MEDIDATA PERSPECTIVE What’s New/What’s Significantly Updated in Release 7.0 ā New: Metrics on new patients entering trials by country/region and therapeutic area by month ā Updated: Regulatory Response ā Updated:The Race for a Vaccine ā Updated: Summary table of the current vaccine clinical trials for COVID-19 ā Updated: Graphical Representation of COVID-19 Vaccination Trials ā Updated: Summary Insights to Ongoing Data Capture in Clinical Trials Medidata is continuously monitoring the global impact of COVID-19 on clinical trials. Our first data and insights impact report was released on March 23, with subsequent releases on April 3, April 17, May 4, May 18, June 15 and now July 13. At the beginning of the pandemic, we were looking at year-over-year changes to understand and grasp the magnitude of COVID-19 on the impact on clinical trials in terms of trial activity, across geographies and therapeutic areas (TAs).
    [Show full text]