The importance of regulatory science in a societal and industrial perspective Annual Conference CORS 24 November 2016

Size: px
Start display at page:

Download "The importance of regulatory science in a societal and industrial perspective Annual Conference CORS 24 November 2016"

Transcription

1 The importance of regulatory science in a societal and industrial perspective Annual Conference CORS 24 November 2016 Marianne Kock, Head of Global Regulatory Affairs & Managing Director, Ferring Pharmaceuticals A/S

2 In my office..at Ferring 1

3 Copenhagen Center for Regulatory Science (CORS) Regulatory Science: The science of developing new tools standards approaches to evaluate the efficacy, safety, quality and performance of medical products in order to assess benefit/risk and facilitate a sound and transparent regulatory decision making. 1985: A literature search would not find regulatory science 2014: A search for regulatory science resulted in 200,000 hits 2

4 Regulatory Science vs Regulatory Affairs Regulatory Science focuses on the scientific underpinnings and concerns. Regulatory science is an emerging area of interest within pharmaceutical medicine as the shaping and implementation of legislation and guidelines Regulatory affairs and regulatory law refer to the administrative and legal aspects including implementation, compliance, or enforcement of existing legislations/laws 3

5 Regulatory Science: ideas for the future Regulatory Science must keep pace with biomedical research to develop safe and effective medicines/treatments to serve the needs of the patients and the society and which are available at reasonable cost to patients New and innovative regulatory pathway is needed for drug approvals, otherwise the pharmaceutical industry investment will dry out New and more clinically meaningful endpoint with patient involvement needs to be continuously developed Understanding the biology of diseases will help to better define patient population resulting in increased responder rate; improving benefit/risk Patients may have different perceptions of risk than regulators Improve the science of benefit/risk assessment by engaging the patients 4

6 Industry perspective: example Acceptable Non-acceptable Quality Pharmacology Non-clinical Clinical Phase 1 Clinical Phase 2 Clinical Phase 3 Risk Management Plan Patients reported Outcomes (PROs) 5 Full risk / benefit evaluation by Agency: Might be approval Final label Experience or Science?

7 Industry perspective: example Quality Pharmacology Non-clinical Clinical Phase 1 V 6 Clinical Phase 2 Clinical Phase 3 Risk Management Plan Price dossier Reimbursement Label NICE / insurance comp. etc. Launch and a new product for patient Not enough! Regulatory Science

8 Drug development in the future The process of drug development must be more efficient and serve a medical need Must be beneficial for patients, doctors and society Biomarkers may become one of the key tools in improving the drug development. International consensus on the qualification of biomarkers must be agreed upon (Regulatory science) Similarly, companion diagnostics allow to better target treatments for patients 7

9 Regulatory dossiers should be global R&D is global in nature Significant differences in the approval process for clinical trials and products in different countries/regions In spite of harmonisation, there are significant regional/national differences in regulatory requirements Significant differences between approved labels in the different regions Identical regulatory dossier may lead to approval or rejection by the FDA and the EU 8

10 Transparency of data and its use Rolling evaluation of new products Transparency of data will help regulatory science (companies / universities) by identifying assumptions, judgements, and related processes during the regulatory evaluation process Those involved in development of regulations & legislations 9

11 Paradigme shifts have started and will impact the evaluation of new medicinal products From Programmatic to Cognitive/AI ( Augmented IBM Watson Intelligence ) Health 1 - Broad collaboration with pharmaceutical companies, Data Driven Solutions hospitals and research institutions - Analytical access to +200 data sources 2 - Centricity Oncology: and 95% Solutions of accuracy in diagnosis, 3 treatment recommendations - Analysis of 10 years research documentation: 6 new From Product to Platforms molecules in 10 weeks 4 Kaiser Permanente Innovation - Integrated 5 patient records, including behavioural data - Seamless management of customers, personalized Patients Physicians management and treatments Insurance companies Hospitals Universities Biotech / VC From Product Centricity to Customer From Closed to Open and Networked From Selective Data Analysis to Big 10

12 From new products to accessing new technologies and the review of these. In today s Pharma, internal research is partly replaced by access to external innovation from universities, biotechs and different other companies New opportunities Other indications New products for patients 11 New way of capture data to give added value: APPs, instruments etc. Henry W. Chesbrough, Why Companies Should Have Open Business Models, MIT Sloan Management Review, Winter 2007, Volume 48, Np. 2, p.27.

13 Where can regulatory science act in a societal and industrial perspective Be part of making regulations/ guidance's based on regulatory science on how and what to be included in future applications/dossiers Find way to embrace global development and find ways to make R&D global Speed up evaluations of new medicinal products. Agencies direct access to data as they are made ( rolling submissions) Be part of evaluate biomarkers which can be used Combination products: products with, pen, apps, instruments, databases.. CORS Regulatory Science : "The science of developing new tools, standards and approaches to evaluate the efficacy, safety, quality and performance of medical products in order to assess benefit-risk and facilitate a sound and transparent regulatory decision-making 12

14 Vision, future, collaboration Thank you for your Pharma companies Universities attention. Agencies Customers/ patients Looking forward to the collaboration.. 13

Setting the Scene - Industry. Regulatory Science. Merete Schmiegelow Senior Director, Regulatory Policy

Setting the Scene - Industry. Regulatory Science. Merete Schmiegelow Senior Director, Regulatory Policy CORS-Industry-241116 1 Regulatory Science Setting the Scene - Industry Merete Schmiegelow Senior Director, Regulatory Policy Honorary Industrial Ambassador in Regulatory Science at the University of Copenhagen

More information

Type of Activity. Universal Activity Number L04-P

Type of Activity. Universal Activity Number L04-P Below are the pharmacy designated Universal Activity Numbers (UANs) and type of activity that is applicable for each of the following program offerings Session # Title 104 Impact of Biologics, Vaccines,

More information

Developing Drugs for Rare Diseases: Patient Advocacy s Perspective. Kristina Bowyer Executive Director, Patient Advocacy

Developing Drugs for Rare Diseases: Patient Advocacy s Perspective. Kristina Bowyer Executive Director, Patient Advocacy Developing Drugs for Rare Diseases: Patient Advocacy s Perspective Kristina Bowyer Executive Director, Patient Advocacy February 5, 2018 An Advocacy Perspective Why develop drugs for Rare Disease? What

More information

Regulatory Science «A Ferring perspective» Sukhwinder S. Jossan, PhD Associate Vice President, Global Regulatory Affairs

Regulatory Science «A Ferring perspective» Sukhwinder S. Jossan, PhD Associate Vice President, Global Regulatory Affairs Regulatory Science «A Ferring perspective» Sukhwinder S. Jossan, PhD Associate Vice President, Global Regulatory Affairs Regulatory Science: Introduction Literature search in1985 did not find the phrase

More information

RWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact.

RWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact. Real Real World World Data Data Across Across the the Product Product Lifecycle: Lifecycle: RWE from pre-clinical to launch RWE from pre-clinical to launch Standard of care Unmet needs Disease burden Budget

More information

Precision Medicine & Health Insurance Business Model Disruption? A Data& Evidence perspective

Precision Medicine & Health Insurance Business Model Disruption? A Data& Evidence perspective Precision Medicine & Health Insurance Business Model Disruption? A Data& Evidence perspective December 12 th, 2017 Dr. Tim Wintermantel Real World Insights Lead, Switzerland Copyright 2017 IQVIA. All rights

More information

Making the case for Personalised Medicine

Making the case for Personalised Medicine Making the case for Personalised Medicine The biopharmaceutical industry perspective Barbara Freischem Executive Director European Biopharmaceutical Enterprises Who we are EBE represents the voice of biopharmaceutical

More information

EUROPEAN COMMISSION DIRECTORATE-GENERAL FOR HEALTH AND FOOD SAFETY

EUROPEAN COMMISSION DIRECTORATE-GENERAL FOR HEALTH AND FOOD SAFETY EUROPEAN COMMISSION DIRECTORATE-GENERAL FOR HEALTH AND FOOD SAFETY Health systems, medical products and innovation Medicines: policy, authorisation and monitoring STAMP 4/24 Record STAMP Commission Expert

More information

GENERAL PAYER CONSIDERATIONS FOR NEW HEALTH TECHNOLOGIES (INCLUDING DIAGNOSTICS)

GENERAL PAYER CONSIDERATIONS FOR NEW HEALTH TECHNOLOGIES (INCLUDING DIAGNOSTICS) FORUM Payer perspectives and actions impacting diagnostics in the US and Europe Eric Faulkner, MPH Director, Quintiles Global Market Access Consulting; Executive Director, Genomics Biotech Emerging Medical

More information

Critical Path to TB Drug Regimens (CPTR)

Critical Path to TB Drug Regimens (CPTR) Critical Path to TB Drug Regimens (CPTR) Why do we need a CPTR initiative? We finally have a pipeline of TB drug candidates Today, we test drugs same way we did 20 years ago Need better tests for drug

More information

2016 Harvard vs. MIT Case Competition. Harvard Team 6 Vladislava Chalei Kathryn McKeough Jonathan Xu Zishu Chen

2016 Harvard vs. MIT Case Competition. Harvard Team 6 Vladislava Chalei Kathryn McKeough Jonathan Xu Zishu Chen 2016 Harvard vs. MIT Case Competition Harvard Team 6 Vladislava Chalei Kathryn McKeough Jonathan Xu Zishu Chen Purpose of Case The purpose of this case was to help a Boston-based pharmaceutical company

More information

Docket #: FDA-2018-D-3268

Docket #: FDA-2018-D-3268 Subject: Comment on FDA Draft Guidance for Industry Titled Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings Docket #: FDA-2018-D-3268 ARM is an international

More information

Defining the true market

Defining the true market Defining the true market Case study: How real-world data and outcomes improved a major pharmaceutical company s launch strategy Cardinal Health Specialty Solutions Competing in a crowded marketplace A

More information

MARKET ACCESS & PUBLIC AFFAIRS MANAGER BELUX

MARKET ACCESS & PUBLIC AFFAIRS MANAGER BELUX MARKET ACCESS & PUBLIC AFFAIRS MANAGER BELUX Sofie Paeps Valerie Van Sannen Senior Executive Search Consultant Executive Search Consultant 0032.478.93.15.11 0032.485.97.52.32 sofie.paeps@hronegroup.com

More information

ehealth-strategien im Wettbewerb: Erfolgsfaktor oder schmückendes Beiwerk? ehealth Kongress Darmstadt, 17 Oktober 2014

ehealth-strategien im Wettbewerb: Erfolgsfaktor oder schmückendes Beiwerk? ehealth Kongress Darmstadt, 17 Oktober 2014 ehealth-strategien im Wettbewerb: Erfolgsfaktor oder schmückendes Beiwerk? ehealth Kongress Darmstadt, 17 Oktober 2014 Health care is in the middle of a perfect storm Changing demographics Unmet needs

More information

PharmaPerspectiveonCDx. DrGillian Ellison

PharmaPerspectiveonCDx. DrGillian Ellison PharmaPerspectiveonCDx DrGillian Ellison Pharma Perspective Need for CDx Partnering with Dx company Co- development Launch readiness & LCM Need for CDx Drug development Drug development is a challenging

More information

Maximizing Market Access: THE 5 MOST CRITICAL QUESTIONS TO ASK WHEN LAUNCHING A SPECIALTY DRUGS

Maximizing Market Access: THE 5 MOST CRITICAL QUESTIONS TO ASK WHEN LAUNCHING A SPECIALTY DRUGS Maximizing Market Access: THE 5 MOST CRITICAL QUESTIONS TO ASK WHEN LAUNCHING A SPECIALTY DRUGS by Jan Nielsen, division president, Access & Patient Support Are You Asking the Right Questions? We ve all

More information

Reimbursement Strategy for Companion Diagnostics:

Reimbursement Strategy for Companion Diagnostics: Reimbursement Strategy for Companion Diagnostics: Emerging Models and Requirements Edward E. Berger, Ph.D. Larchmont Strategic Advisors Definition Companion diagnostic A diagnostic test used to predict

More information

IMI2: Strategic research agenda and overall approach Hugh Laverty Senior Scientific Project Manager

IMI2: Strategic research agenda and overall approach Hugh Laverty Senior Scientific Project Manager IMI2: Strategic research agenda and overall approach Hugh Laverty Senior Scientific Project Manager Introducing IMI2: Vienna 16 July 2014 The way in which pharmaceutical companies develop new medicines

More information

EBE White Paper on Personalised Medicine

EBE White Paper on Personalised Medicine EBE White Paper on Personalised Medicine EBE is a specialised group of European Federation of Pharmaceutical Industries and Associations, EFPIA Content p3 p4 p6 p6 p7 p9 p10 p11 p13 p14 Executive Summary

More information

Issues in Cancer Drug Development of the Future. Janet Woodcock M.D. Deputy Commissioner/Chief Medical Officer, FDA October 5, 2007

Issues in Cancer Drug Development of the Future. Janet Woodcock M.D. Deputy Commissioner/Chief Medical Officer, FDA October 5, 2007 Issues in Cancer Drug Development of the Future Janet Woodcock M.D. Deputy Commissioner/Chief Medical Officer, FDA October 5, 2007 Agenda: Scientific Issues n Why improve the quality of cancer clinical

More information

Coordination and Support Action: Enabling platform on medicines adaptive pathways to patients

Coordination and Support Action: Enabling platform on medicines adaptive pathways to patients Coordination and Support Action: Enabling platform on medicines adaptive pathways to patients Solange Rohou (AZ) & Luk Maes (BMS) 17.12.2014 IMI webinar Content Background information CSA project proposal

More information

Jordi Aparici Market Intelligence Consultant

Jordi Aparici Market Intelligence Consultant Jordi Aparici Market Intelligence Consultant Helping pharma companies make robust decisions based on market, competitors and patient knowledge Market Intelligence Strategy Marketing Market Research Data

More information

EU Big Data Initiatives

EU Big Data Initiatives European Network of Excellence for Big Data in Hematology, consisting of 51 partners from 11 countries. Aliki Taylor, HARMONY Alliance Partner Director Global Health Outcomes, Takeda EU Big Data Initiatives

More information

CDRH Device Approval

CDRH Device Approval CDRH Device Approval Mary S. Pastel, ScD Deputy Director for Radiological Health Office of In Vitro Diagnostics and Radiological Health (OIR) Center for Devices and Radiological Health (CDRH) US Food and

More information

REIMAGINING DRUG DEVELOPMENT:

REIMAGINING DRUG DEVELOPMENT: Biology Reconstructed REIMAGINING DRUG DEVELOPMENT: Accurate Disease Modeling To Drive Successful Therapies Julia Kirshner, CEO julia@zpredicta.com 1 SUCCESS RATES OF DRUG DEVELOPMENT ARE LOW, " PARTICULARLY

More information

The Future of Market Access A FirstWord ExpertViews Dossier Report

The Future of Market Access A FirstWord ExpertViews Dossier Report AM PL E PA G ES S A G ES S A FirstWord ExpertViews Dossier Report Published Copyright 2016 Doctor s Guide Publishing Limited All rights reserved. No part of this publication may be reproduced or used in

More information

MODEL-BASED DRUG DEVELOPMENT: A FDA CRITICAL PATH OPPORTUNITY

MODEL-BASED DRUG DEVELOPMENT: A FDA CRITICAL PATH OPPORTUNITY MODEL-BASED DRUG DEVELOPMENT: A FDA CRITICAL PATH OPPORTUNITY Donald R. Stanski MD Scientific Advisor, Office of the Commissioner, FDA Professor of Anesthesia Stanford University, CA THE OPINIONS EXPRESSED

More information

Delivering on the promise: the clinical application of new diagnoses and treatments for RD K A T E B U S H B Y N E W C A S T L E U N I V E R S I T Y

Delivering on the promise: the clinical application of new diagnoses and treatments for RD K A T E B U S H B Y N E W C A S T L E U N I V E R S I T Y Delivering on the promise: the clinical application of new diagnoses and treatments for RD K A T E B U S H B Y N E W C A S T L E U N I V E R S I T Y PERSPECTIVE/ DISCLAIMERS Doctor with >24 years experience

More information

Biopharma in the coming era of connected health

Biopharma in the coming era of connected health 14 Health-care focus: Perspective Celia Johnson Biopharma in the coming era of connected health Disruptive technologies are already reshaping the industry. Here s how executives can meet the resulting

More information

INNOVATION ACTION PLAN

INNOVATION ACTION PLAN Digital Health INNOVATION ACTION PLAN Digitization Across the Health Care Continuum Moving health care from the Clinic to the Patient. Understanding patient s behavior and physiology In the wild. Focusing

More information

6th EUROPEAN CONFERENCE ON RARE DISEASES & ORPHAN PRODUCTS ECRD 2012 Brussels

6th EUROPEAN CONFERENCE ON RARE DISEASES & ORPHAN PRODUCTS ECRD 2012 Brussels www.eurordis.org 6th EUROPEAN CONFERENCE ON RARE DISEASES & ORPHAN PRODUCTS ECRD 2012 Brussels Brussels, 24-25 May 2012 www.eurordis.org THE NEW PARADIGMS OF RARE DISEASES TREATMENT DEVELOPMENT & ACCESS

More information

Clinical Trial Simulations

Clinical Trial Simulations Clinical Trial Simulations Clinical Pharmacology meeting 07.10. 2014 Flora T Musuamba Outline Introduction Estimation vs Simulations Simulations as a diagnostic tool Simulation as a tool for quantitative

More information

The Move Toward Patient Centricity. LIFE SCIENCES Patient Connect Accelerator ebook

The Move Toward Patient Centricity. LIFE SCIENCES Patient Connect Accelerator ebook The Move Toward Patient Centricity LIFE SCIENCES Patient Connect Accelerator ebook are in a unique position to drive value and they must to successfully compete with new competitors (e.g. large consumer

More information

Personalised Healthcare Solutions (PHCS) Excellence for your Biomarker-driven Strategies

Personalised Healthcare Solutions (PHCS) Excellence for your Biomarker-driven Strategies Personalised Healthcare Solutions (PHCS) Excellence for your Biomarker-driven Strategies The value of In Vitro Diagnostics The value of diagnostics as an integral part in the healthcare value chain Diagnostics

More information

May Patient Reported Outcomes: Strategies for gaining or expanding market access ES SAMPLE PAGES SA. A FirstWord ExpertViews Dossier Report

May Patient Reported Outcomes: Strategies for gaining or expanding market access ES SAMPLE PAGES SA. A FirstWord ExpertViews Dossier Report A FirstWord ExpertViews Dossier Report Published Copyright 2016 Doctor s Guide Publishing Limited All rights reserved. No part of this publication may be reproduced or used in any form or by any means

More information

Innovative Medicines Initiative

Innovative Medicines Initiative Innovative Medicines Initiative EMA - EBE Regulatory Conference on ATMPs Salah-Dine Chibout, Novartis Global Head Discovery & Investigative Safety/ Global Head Preclinical Safety Therapeutic Areas IMI

More information

1201 Maryland Avenue SW, Suite 900, Washington, DC ,

1201 Maryland Avenue SW, Suite 900, Washington, DC , 1201 Maryland Avenue SW, Suite 900, Washington, DC 20024 202-962-9200, www.bio.org May 31, 2012 Dockets Management Branch (HFA-305) Food and Drug Administration 5600 Fishers Lane, Rm. 1061 Rockville, MD

More information

ARIKAYCE U.S. FDA Approval

ARIKAYCE U.S. FDA Approval S E P T E M B E R 2 8, 2 0 1 8 ARIKAYCE U.S. FDA Approval Forward Looking Statements This presentation contains forward-looking statements. "Forward-looking statements," as that term is defined in the

More information

HTA and managed entry practices in Europe - Pharmaceutical industry perspective. Edith FRENOY Director Market Access EFPIA

HTA and managed entry practices in Europe - Pharmaceutical industry perspective. Edith FRENOY Director Market Access EFPIA HTA and managed entry practices in Europe - Pharmaceutical industry perspective Edith FRENOY Director Market Access EFPIA Piperska workshop 19 March 2011 EFPIA members are committed to demonstrating the

More information

European Medicines Agency Perspective

European Medicines Agency Perspective European Medicines Agency Perspective WMA Workshop on revision of DoH, 4-6 Dec 2012, Capetown, South Africa. Presented by: Fergus Sweeney, PhD Head of Sector, Compliance and Inspection, European Medicines

More information

Public private partnerships in support to regulatory sciences

Public private partnerships in support to regulatory sciences Public private partnerships in support to regulatory sciences The Innovative Medicines Initiative example M. Chlebus 3 October 2017 www.efpia.eu Summary: Partnering on regulatory sciences Progress much

More information

Clinical Trial Design, Approval Process and Trial Conduct

Clinical Trial Design, Approval Process and Trial Conduct Clinical Trial Design, Approval Process and Trial Conduct Topic: Special Considerations in Gene Transfer: Surrogate Endpoints Michael Kalos, Ph.D. University of Pennsylvania School of Medicine May 18,

More information

TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT

TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT Specialist in tissue analysis by Histology, Immunohistochemistry and In Situ Hybridization TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT CONTENTS Histalim: who we are Our areas of expertise

More information

The Yale Open Data Access (YODA) Project: Repurposing Data for Researchers

The Yale Open Data Access (YODA) Project: Repurposing Data for Researchers The Yale Open Data Access (YODA) Project: Repurposing Data for Researchers American Library Association Annual Meeting June 27, 2016 Jessica Ritchie Klein, MPH, PMP YODA Project Manager Center for Outcomes

More information

Career Growth Areas in Physiology / Pharmacology

Career Growth Areas in Physiology / Pharmacology Career Growth Areas in Physiology / Pharmacology Magdalena Alonso-Galicia, PhD Pharmacology Department Forest Research Institute, Inc. Jersey City, NJ 1 Career Growth Areas in BioPharma n Pharmaceutical

More information

Certification in Pharmaceutical Medicine and Clinical Research The vision of regulators

Certification in Pharmaceutical Medicine and Clinical Research The vision of regulators Certification in Pharmaceutical Medicine and Clinical Research The vision of regulators Presented by Sergio Bonini Professor of Medicine, Second University of Naples Expert-on-Secondment, European Medicine

More information

Start With the End in Mind A Diagnostic Company s Perspective on Companion Diagnostic Development. Paul Docherty PhD Relationship Management

Start With the End in Mind A Diagnostic Company s Perspective on Companion Diagnostic Development. Paul Docherty PhD Relationship Management Start With the End in Mind A Diagnostic Company s Perspective on Companion Diagnostic Development Paul Docherty PhD Relationship Management Disclaimer This presentation contains my personal views and research

More information

Section I: Pharmaceuticals and Medical Devices

Section I: Pharmaceuticals and Medical Devices SUPPLEMENT on HEALTHCARE INNOVATION Visionary Goals and Recommendations 51th Japan-U.S. Business Conference Japan-U.S. Business Council / U.S.-Japan Business Council November 14, 2014 The R&D-based pharmaceutical

More information

DEMONSTRATING YOUR MEDICINE S VALUE TO ALL STAKEHOLDERS TRUSTED COMMERCIALIZATION AND MARKET ACCESS EXPERTISE

DEMONSTRATING YOUR MEDICINE S VALUE TO ALL STAKEHOLDERS TRUSTED COMMERCIALIZATION AND MARKET ACCESS EXPERTISE CATALYSTS DRIVING SUCCESSFUL DECISIONS IN LIFE SCIENCES DEMONSTRATING YOUR MEDICINE S VALUE TO ALL STAKEHOLDERS TRUSTED COMMERCIALIZATION AND MARKET ACCESS EXPERTISE WHEN COMMERCIALIZING A MEDICINE, IT

More information

Moving HTA forward: The challenges of incorporating real world evidence into Health Technology Assessment

Moving HTA forward: The challenges of incorporating real world evidence into Health Technology Assessment Moving HTA forward: The challenges of incorporating real world evidence into Health Technology Assessment Matthias Egger Mike Chambers Univ. of Berne, Switzerland GSK, UK Slide 1 Increasingly complex external

More information

Fast Track Drug Development The Clinical (Caleidoscopic) Perspective

Fast Track Drug Development The Clinical (Caleidoscopic) Perspective 20 Years AGAH, Annual Meeting, Hamburg 21.-23. February 2010 Fast Track Drug Development The Clinical (Caleidoscopic) Perspective Fritz R. Bühler, MD ECPM, University of Basel European Innovative Medicine

More information

GUIDE HEA

GUIDE HEA Companies are often overwhelmed by the field of digital health. From digital and med device companies to biotechnology and pharmaceutical companies, there is a struggle to cut through the noise of numerous

More information

Perspective: Convergence of CLIA and FDA Requirements A Rational Shift in the Regulatory Paradigm

Perspective: Convergence of CLIA and FDA Requirements A Rational Shift in the Regulatory Paradigm Perspective: Convergence of CLIA and FDA Requirements A Rational Shift in the Regulatory Paradigm Planning for Efficiencies of Data, Resources, and Timelines A PRECISION BRIEF Introduction As precision

More information

Life Sciences Debashis Ghosh. 1 Copyright 2016 Tata Consultancy Services Limited

Life Sciences Debashis Ghosh. 1 Copyright 2016 Tata Consultancy Services Limited Life Sciences Debashis Ghosh 1 Copyright 2016 Tata Consultancy Services Limited From biology, chemistry and anatomy to data, analytics and technology Analytics driven Genomics Research We identify disease

More information

January Summary

January Summary The Academy of Medical Sciences and the Royal Academy of Engineering joint response to the Medicine and Healthcare products Regulatory Agency s public consultation on the revision of European legislation

More information

Personalised medicine towards the market and patients: the approval process

Personalised medicine towards the market and patients: the approval process An agency of the European Union Personalised medicine towards the market and patients: the approval process European Medicines Agency s perspective Presented by: Marisa Papaluca Amati Human Medicines Development

More information

The Integration of Market Access and Advocacy: The Changing Landscape OCTOBER 2016

The Integration of Market Access and Advocacy: The Changing Landscape OCTOBER 2016 The Integration of Market Access and Advocacy: The Changing Landscape OCTOBER 2016 The Integration of Market Access and Advocacy: The Changing Landscape Introduction Healthcare market access and advocacy

More information

KPI Definition Comment Relates to Baseline Target

KPI Definition Comment Relates to Baseline Target IMI2 Key performance indicators (KPIs) Reporting on measuring and outcomes on the ten following Key Performance Indicators will be provided yearly as part of the IMI2 JU Annual Activity Reports for year

More information

The International Consortium for Personalised Medicine

The International Consortium for Personalised Medicine The International Consortium for Personalised Medicine Mairead O Driscoll Alliance for Biomedical Research in Europe, 8 November 2017 Personalised Medicine A definition Characterisation of individuals

More information

EU health policy. Strategy for the pharmaceutical industry and biosimilars. Salvatore D'Acunto. DG Research. DG Internal Market. DG Health & Consumers

EU health policy. Strategy for the pharmaceutical industry and biosimilars. Salvatore D'Acunto. DG Research. DG Internal Market. DG Health & Consumers Strategy for the pharmaceutical industry and biosimilars Salvatore D'Acunto European Commission Enterprise and Industry Directorate-General London, 3 April 2014 EU health policy DG Research R&D, innovation

More information

Unpacking Expanded Access to Investigational Drugs and Biologics

Unpacking Expanded Access to Investigational Drugs and Biologics Unpacking Expanded Access to Investigational Drugs and Biologics When All Else Fails Richard Klein Office of Health and Constituent Affairs Food and Drug Administration September 13, 2016 FDA Drug Approval

More information

Subgroups along the development axis

Subgroups along the development axis Paper PD03 A dedicated Programming Group working in a pharmaceutical Modeling & Simulation organization Vincent Buchheit, Novartis, Basel, Switzerland 1. ABSTRACT Modeling and Simulation (M&S) emerges

More information

Real World Evidence, Digital Health and Market Access

Real World Evidence, Digital Health and Market Access Real World Evidence, Digital Health and Market Access Dr. Kyle E. Dolbow Oncology Market Access Market Access It s going to be a fact of life that we have to accept a slower uptake, Mr. Jimenez told the

More information

Strategy resets to patient outcomes. The state of life sciences

Strategy resets to patient outcomes. The state of life sciences Strategy resets to patient outcomes The state of life sciences New innovative products, even at a high cost, can be a great deal for society and the health care system if they cure a chronic disease. Expensive

More information

CENTRAL NERVOUS SYSTEM (CNS) BIOMARKERS: TECHNOLOGIES AND GLOBAL MARKETS

CENTRAL NERVOUS SYSTEM (CNS) BIOMARKERS: TECHNOLOGIES AND GLOBAL MARKETS CENTRAL NERVOUS SYSTEM (CNS) : TECHNOLOGIES AND GLOBAL MARKETS BIO074C March 2016 Jackson Highsmith Project Analyst ISBN: 1-62296-246-X BCC Research 49 Walnut Park, Building 2 Wellesley, MA 02481 USA 866-285-7215

More information

Personalized Healthcare Diagnostik und Therapie aus einer Hand

Personalized Healthcare Diagnostik und Therapie aus einer Hand Personalized Healthcare Diagnostik und Therapie aus einer Hand Priv.-Doz. Dr. med. Christian Meisel DVFA Life Science Conference, 17 June 2009 1 The Challenge Personalised healthcare Roche s unique position

More information

Reimagining Life Sciences With AI-Enabled Digital Transformation. Abstract

Reimagining Life Sciences With AI-Enabled Digital Transformation. Abstract Reimagining Life Sciences With AI-Enabled Digital Transformation Abstract Technological, regulatory and environmental changes, along with an increasingly active involvement of patients in their treatment

More information

Regulatory and Reimbursement Obstacles Clinical Update of Molecular Diagnostics ASU

Regulatory and Reimbursement Obstacles Clinical Update of Molecular Diagnostics ASU Regulatory and Reimbursement Obstacles Clinical Update of Molecular Diagnostics ASU Bruce Quinn MD PhD Bruce. Quinn @ faegrebd. com 323 839 8637 mobile April 8, 2016 Sky Song / Scottsdale Digital Health

More information

New Health Data Economy: Strategic Partnerships with Life Sciences Company

New Health Data Economy: Strategic Partnerships with Life Sciences Company New Health Data Economy: Strategic Partnerships with Life Sciences Company Pravin R Jadhav Sr. Director, Innovative Data Solutions Otsuka Data Sciences Otsuka Pharmaceutical Development and Commercialization

More information

FDA s Proposed Regulatory Changes to Laboratory-Developed Tests (LDTs) By Roz Sweeney, Ph.D., Nerac Analyst

FDA s Proposed Regulatory Changes to Laboratory-Developed Tests (LDTs) By Roz Sweeney, Ph.D., Nerac Analyst FDA s Proposed Regulatory Changes to Laboratory-Developed Tests (LDTs) By Roz Sweeney, Ph.D., Nerac Analyst Introduction The FDA recently issued updated draft guidance for proposed regulatory changes affecting

More information

Challenges in developing Biomarker Assays for patient selection and Companion Diagnostic (CDx) Assays in early and late stage of drug development

Challenges in developing Biomarker Assays for patient selection and Companion Diagnostic (CDx) Assays in early and late stage of drug development Challenges in developing Biomarker Assays for patient selection and Companion Diagnostic (CDx) Assays in early and late stage of drug development The 9 th JBF Symposium February 8 th, 2018 Kenji Nakamaru

More information

CPTR 2013 Workshop September 30 th - October 3 rd. CPTR Roadmap: Accomplishments and Future Direction

CPTR 2013 Workshop September 30 th - October 3 rd. CPTR Roadmap: Accomplishments and Future Direction CPTR 2013 Workshop September 30 th - October 3 rd CPTR Roadmap: Accomplishments and Future Direction CPTR Mission & Purpose Accelerate the development of new, safe, and highly effective regimens for TB

More information

Insights into the Evolving Pricing & Market Access Environment

Insights into the Evolving Pricing & Market Access Environment Insights into the Evolving Pricing & Market Access Environment UBC Global Market Access Survey July 2012 JULY 2012 GLOBAL MARKET ACCESS SURVEY UBC s Global Market Access Survey: Setting the Stage For those

More information

Solving the Problem of New Uses

Solving the Problem of New Uses Solving the Problem of New Uses Benjamin Roin, Harvard Law School benroin@law.harvard.edu Presented at Duke Law School Center for Innovation Policy s New Approaches and Incentives in Drug Development,

More information

Enterprise Systems in Healthcare - Blessing or Curse?

Enterprise Systems in Healthcare - Blessing or Curse? Enterprise Systems in Healthcare - Blessing or Curse? Research topics that interest me... Three agendas: n Evolution and assessment of information systems Evolution of information systems Business value

More information

Introduction to Drug Development

Introduction to Drug Development Introduction to Drug Development Yves Geysels, PhD Head Clinical Research Operations, Belgium Past President of the Associoation of Clinical Research Professionals (ACRP) Board Member of the European Forum

More information

White paper The Polish pharmaceutical market has undergone many changes as a result of the new Reimbursement Act

White paper The Polish pharmaceutical market has undergone many changes as a result of the new Reimbursement Act White paper The Polish pharmaceutical market has undergone many changes as a result of the new Reimbursement Act Published: July 2014 Author: Anantharaman Kavassery Viswanthan Anantharaman Kavassery Viswanathan

More information

E&T objectives in evolving pharma business models. Magda Chlebus Director Science Policy Brussels, 2 March 2015

E&T objectives in evolving pharma business models. Magda Chlebus Director Science Policy Brussels, 2 March 2015 E&T objectives in evolving pharma business models Magda Chlebus Director Science Policy Brussels, 2 March 2015 Education adapted to demand? Declining number of students choosing science Education still

More information

VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT?

VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT? VIVEK VIKRAM SINGH, SUBJECT MATTER EXPERT, RESEARCH & ANALYTICS CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG DEVELOPMENT? wns wns CAN COMPETITIVE INTELLIGENCE STRATEGIES HELP IN ORPHAN DRUG

More information

Mapping the Patient Journey: Harnessing the power of big data and analytics A FirstWord ExpertViews Dossier Report

Mapping the Patient Journey: Harnessing the power of big data and analytics A FirstWord ExpertViews Dossier Report AM PL E PA G ES S A S G ES data and analytics A FirstWord ExpertViews Dossier Report Published Copyright 2016 Doctor s Guide Publishing Limited All rights reserved. No part of this publication may be reproduced

More information

Innovative Medicines Initiative:

Innovative Medicines Initiative: Innovative Medicines Initiative: Opportunities for local players to become important parts of multicountry pharma innovation Nathalie Serigneuret 4 March 2015 Clinical Trials Conference 2015, Budapest

More information

OASIS DITA Pharma. Initial Meeting 30 July 2009

OASIS DITA Pharma. Initial Meeting 30 July 2009 OASIS DITA Pharma Initial Meeting 30 July 2009 Agenda DITA PCSC Welcome Objectives of today s meeting Introductions Round Robin Expectations of the Group Round Robin Story Boards of Opportunity DITA in

More information

Advertising and Marketing Genetic Tests New Pathways or Old Roads?

Advertising and Marketing Genetic Tests New Pathways or Old Roads? Advertising and Marketing Genetic Tests New Pathways or Old Roads? Kathleen M. Sanzo, Morgan Lewis FDLI Advertising & Promotion Conference Wednesday, September 28, 2016 Context for Use of Genetic Tests

More information

FDA Critical Path Initiative: Closing Productivity Gap in Medical Product Development. October 4, 2004

FDA Critical Path Initiative: Closing Productivity Gap in Medical Product Development. October 4, 2004 FDA Critical Path Initiative: Closing Productivity Gap in Medical Product Development October 4, 2004 1 What is the problem? Trends in Biomedical Research Spending R&D Spending Has Accelerated Since 1990

More information

VALUE OF COMPANION DIAGNOSTICS IN PERSONALISED MEDICINE

VALUE OF COMPANION DIAGNOSTICS IN PERSONALISED MEDICINE VALUE OF COMPANION DIAGNOSTICS IN PERSONALISED MEDICINE Stimulating innovation for improving health through companion diagnostics 5 March 2015 I. Introduction Personalised healthcare provides targeted

More information

Overview of Biomarker Qualification. Marc K Walton MD, PhD Office of Translational Sciences CDER-FDA

Overview of Biomarker Qualification. Marc K Walton MD, PhD Office of Translational Sciences CDER-FDA Overview of Biomarker Qualification Marc K Walton MD, PhD Office of Translational Sciences CDER-FDA Categories of Biomarkers Prognostic Predictive Pharmacodynamic Including adverse response Efficacy-response

More information

Successful and Faster Drug Development through Data Mining Dirk Belmans, Ph.D. SAS Belgium

Successful and Faster Drug Development through Data Mining Dirk Belmans, Ph.D. SAS Belgium Successful and Faster Drug Development through Data Mining Dirk Belmans, Ph.D. SAS Belgium The Life-Cycle of a Successful Pharmaceutical Product Sales Costs Patent Filing R&D time to launch is decreasing

More information

Regulatory challenges and opportunities for the use of Real World Evidence for drug registration and labelling

Regulatory challenges and opportunities for the use of Real World Evidence for drug registration and labelling Presentation title Date 1 Regulatory challenges and opportunities for the use of Real World Evidence for drug registration and labelling Marianne Bork Samuelsen, Msc Pharm Regulatory Affairs Novo Nordisk

More information

While individually rare, orphan diseases are actually collectively common, with an OF ORPHAN DRUG DEVELOPMENT MEETING THE UNIQUE CHALLENGES

While individually rare, orphan diseases are actually collectively common, with an OF ORPHAN DRUG DEVELOPMENT MEETING THE UNIQUE CHALLENGES ELECTRONICALLY REPRINTED FROM JUNE 2017 CLINICAL TRIALS Rare Diseases: MEETING THE UNIQUE CHALLENGES OF ORPHAN DRUG DEVELOPMENT BY MICHAEL F. MURPHY, MD, Ph D While individually rare, orphan diseases are

More information

Clinical trial applications in the EU and US

Clinical trial applications in the EU and US Clinical trial applications in the EU and US Alain Patat, M.D. Translational Development Wyeth Research Paris, France AGAH-Club Phase 1 1 st Symposium Strasbourg 17-18 March 2005 Overview of the presentation

More information

Konica Minolta to Acquire Invicro (US)

Konica Minolta to Acquire Invicro (US) Konica Minolta to Acquire Invicro (US) Acceleration of expansion of precision medicine business Offering new value for drug discovery and development in immuno-oncology and neurodegenerative disease September

More information

THE BIOSIMILARS LANDSCAPE: STRATEGIES FOR CLINICAL AND COMMERCIAL SUCCESS

THE BIOSIMILARS LANDSCAPE: STRATEGIES FOR CLINICAL AND COMMERCIAL SUCCESS THE BIOSIMILARS LANDSCAPE: STRATEGIES FOR CLINICAL AND COMMERCIAL SUCCESS What all developers need to know Alicia Baker Director, Global Regulatory Affairs Strategy, Covance John Carlsen, MHA Vice President,

More information

MAXIMIZING THE VALUE OF CLINICAL TRIALS DATA: A COLLABORATIVE FRAMEWORK FOR DATA STANDARDS GOVERNANCE FROM DATA DEFINITION TO KNOWLEDGE MANAGEMENT

MAXIMIZING THE VALUE OF CLINICAL TRIALS DATA: A COLLABORATIVE FRAMEWORK FOR DATA STANDARDS GOVERNANCE FROM DATA DEFINITION TO KNOWLEDGE MANAGEMENT MAXIMIZING THE VALUE OF CLINICAL TRIALS DATA: A COLLABORATIVE FRAMEWORK FOR DATA STANDARDS GOVERNANCE FROM DATA DEFINITION TO KNOWLEDGE MANAGEMENT PharmaSUG Single Day Event Using Standards, Even for Non-Standard

More information

CAREER DEVELOPMENT FOR EXPERIENCED REGULATORY PROFESSIONALS

CAREER DEVELOPMENT FOR EXPERIENCED REGULATORY PROFESSIONALS CAREER DEVELOPMENT FOR EXPERIENCED REGULATORY PROFESSIONALS How TOPRA can support you as you drive your regulatory career forward ABOUT TOPRA TOPRA (The Organisation for Professionals in Regulatory Affairs)

More information

EXPANDED ACCESS PROGRAMS

EXPANDED ACCESS PROGRAMS EXPANDED ACCESS PROGRAMS PRACTICAL OPERATIONAL CONSIDERATIONS While patient demand for expanded access is growing, a clear consensus has yet to emerge on the best way to address this demand. Debate continues

More information

Dr. Earl Dye CMC/GMP Considerations for Expedited Development Programs

Dr. Earl Dye CMC/GMP Considerations for Expedited Development Programs Dr. Earl Dye CMC/GMP Considerations for Expedited Development Programs Earl Dye is Director of Technical Regulatory Policy in Genentech s Washington, DC Regulatory Affairs Office, and is the FDA Liaison

More information

FDA Driving Biomedical Product Innovation

FDA Driving Biomedical Product Innovation FDA Driving Biomedical Product Innovation Vicki Seyfert-Margolis, Ph.D. Senior Advisor, Science Innovation and Policy Office of the Commissioner November 12, 2012 1 FDA Mission FDA is charged with protecting

More information

Pediatric Drug Development:

Pediatric Drug Development: Pediatric Drug Development: Where Have We Been and Where Are We Going? Barry Mangum, PharmD, FCP Director Clinical Pharmacology Duke Clinical Research Center Duke University Medical Center Specialty Pediatric

More information

Update on Real World Evidence Data Collection

Update on Real World Evidence Data Collection Update on Real World Evidence Data Collection STAMP, 10 March 2016 Presented by Dr Peter Arlett EMA An agency of the European Union What is Real-world evidence? Big data = umbrella term describing large

More information