Patients’ Perspective on HTA and Off-label use David Head MBA Chief Executive RP Fighting Blindness 25th Annual EuroMeeting 4-6 March 2013 RAI, Amsterdam.

Slides:

Advertisements

Similar presentations

1 Chapter 58 - Clinical Syndromes of Metabolic Alkalosis Copyright © 2013 Elsevier Inc. All rights reserved.

Advertisements

1 Copyright © 2013 Elsevier Inc. All rights reserved. Chapter 14.

6th European Patients’ Rights Day The EMA Geriatric Medicines Strategy and the empowered aging patient Francesca Cerreta EMA (European Medicines Agency)

© Safeguarding public health Expert Group on Innovation in the Regulation of Healthcare products Adaptive Licencing workshop at the Wellcome Trust October.

Application for Foundation Trust Status.  What is a Foundation Trust?  Why should we become one?  What the Regulator (Monitor) looks for  How will.

Data Monitoring Models and Adaptive Designs: Some Regulatory Experiences Sue-Jane Wang, Ph.D. Associate Director for Adaptive Design and Pharmacogenomics,

How You Can Be an Advocate for Clinical Trials in Your Community.

Orphan Drugs: Where have we been; Where are we now; Where are we going Marlene E. Haffner, MD, MPH Haffner Associates, LLC 11 October,

Stakeholders In Clinical Research Government and Regulatory Bodies Professor Phil Warner.

Clinical Trials in the Age of Personalized Cancer Medicine: The Evolution of a More Efficient, Patient Focused Clinical Research System Session Chair:

The Correct e- Mixtures (A fully validated EHR, the way forward) Brendan Delaney Guy’s and St Thomas’ Charity Chair in Primary Care Research.

FDA’s Biosimilars Guidance -- Legal and Regulatory Considerations James S. Cohen, Esq. McDermott Will & Emery DIA Webinar April 10, 2012.

HTA cooperation in the EU EPF workshop Jérôme Boehm 18 May 2010.

Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug.

Introduction to Drug Rediscovery John Lisman Attorney-at-law Lisman Legal Life sciences Insert your logo in this area then delete this text box.

How to audit the role of the vendor in the conduct of outsourced studies Kristel Van de Voorde Director Global Quality Regulatory Compliance Bristol-Myers.

Presenter Name Title Organization Twitter Handle Insert your logo here, then delete text.

Adaptive Licensing UK. Content What is adaptive licensing? Draft criteria for pilot candidate selection EMA Road Map 2015 and work programme 2012 UK perspective.

Click to add Presentation Title Arial 32, 5 line max title space line 3, title space line 4, title space line 5 Presenter Title Organization Insert your.

1 Optimal Strategies for Preparing Integrated and Clinical Summaries for a New Drug Application: Making it Work Under Any Circumstance Lisa A. Pierchala,

A. B Founded in 1908, Prevent Blindness America is the nation’s leading volunteer eye health and safety organization dedicated to fighting blindness.

Bayesian approach to equivalence study of medical device 1 1.

Sherri de Coronado Enterprise Vocabulary Services NCI Center for Bioinformatics and Information Technology March 11, 2009 A Terminology.

How to successfully Implement and Leverage the Regulatory Intelligence Function Marianne Koehne EU RI Network Group; Global RI Manager Boehringer Ingelheim.

Making Comments Count for High-Impact Regulations and Guidelines in the US Virginia (Ginny) Beakes-Read Executive Director, Global Regulatory Policy and.

Horst Kastrup September 8, 2015 The “T” in PETD.  The views and opinions expressed in the following PowerPoint slides are those of the individual presenter.

Strategic Analyses and Interpretation: Regulatory Intelligence for Decision Making Amy N. Grant Director, Regulatory Strategy & Science ViroPharma Incorporated.

Good Laboratory Practices and Inspection Readiness Paul Swidersky President/Sr. Consultant Quality Associates, Inc.

Meredith Brown-Tuttle, RAC Director APT Pharmaceuticals, Inc.

Draft White Paper “Protocol Deviations”

DIA ERS SIAC IND CMC eCTD Submissions Part II – IND to NDA

Process-based Metadata From a DIA Presentation: eTMF – Migrating from Paper Trial Master Files to Electronic Eldin Rammell, Managing Director, Rammell.

Experiences from building a lessons- learned database for regulatory interactions Åsa Rembratt Sr Reg Intelligence Manager Novo Nordisk A/S 26th Annual.

Group Sequential Tests for Delayed Responses Christopher Jennison Department of Mathematical Sciences University of Bath Lisa Hampson Department of Mathematics.

Making Comments Count for High-Impact Regulations and Guidelines in Canada, EU, Japan, and US Chairperson: Amy N. Grant Director, Regulatory Strategy &

Gabor Fari Life Sciences Solution Strategist Microsoft Corporation

Session Title Date ǀ Time

- A “Portable” Implementation

A capacity building programme for patient representatives

Mutagenic Impurities: Guidances Update w/ CMC Perspectives

Efficacy and Safety of Medicines

Presenter Title Organization

Being told you have an Inherited Retinal Dystrophy… and that there is no treatment Tina Houlihan Chief Executive, RP Fighting Blindness.

Track 11 Symposium 27 June :30 – 3:00 PM

Clinical Trial Disclosure:

Perspective on GCP Warning Letters

Implementing a Successful Corrective and Preventative Action Program

Molly Butler Auditor II Quality Associates, Inc.

Interactive Session: Presentation of Scenarios and Q&A

Community-Based and Cluster-Randomized Studies –‘Pragmatic’ Approaches for Life Cycle Evidence? Florian Eichmann, PhD Principal Scientific Affairs and.

FirstPoint and FirstDoc Application of the DIA EDM Reference Model

Presenter Name Title Organization.

1 Topic Title First slide 2 line 3 line 4 line

1 Topic Title First slide 2 line 3 line 4 line

PMI® Leadership Institute Meeting 2018―EMEA

PMI® EMEA Congress 2018 Session Title Session Date | Time

Significant (greater than $10K)

Presenter Name Title Organization.

Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug.

1 Session Title 2 line 3 line 4 line

ATTN Presenters – please review and remove this slide from your PowerPoint deck before uploading.

Session Title Date ǀ Time

Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to.

1 Session Title 2 line 3 line 4 line

1 Topic Title First slide 2 line 3 line 4 line

1 Topic Title First slide 2 line 3 line 4 line

Regulatory Perspective of the Use of EHRs in RCTs

Dr. Ilona Große-Michaelis for the CIOMS MLG Exploratory Team

William W. Gregory for the CIOMS MLG Exploratory Team Pfizer Inc

SESSION TITLE Session # <insert number here> Date Month Year

Presentation transcript:

Patients’ Perspective on HTA and Off-label use David Head MBA Chief Executive RP Fighting Blindness 25th Annual EuroMeeting 4-6 March 2013 RAI, Amsterdam Netherlands

Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. (“DIA”), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners. 2

Context: What is RP Fighting Blindness? UK Charity Patient Organisation Research Funder Provider of Patient Information Provider of Patient Support 3 RP Fighting Blindness

Context: What is RP? Retinitis pigmentosa – a genetic condition Degeneration of retinal cells Progressive sight loss Currently not preventable or curable Orphan Disease affects 2 – 3m worldwide Off label prospects Devices and drugs in clinical trials 4 Retinitis Pigmentosa

5

Not very scientific but …. Have you ever heard of Healthcare Technology Assessments? 93% said no Have you heard the term "Off Label Use“ 84% said no 6 Straw Poll

Industry Stakeholders 7 Pharma Patients Patient Organisations Regulators

Trust in the Doctor Prognosis Family Pain Trust in the Healthcare System Friends Fears Media Patient Decision Making 8

Patient Perspective 9 Video embedded here

Patient Perspective 10

The Obvious Suspects Expediting trials and approvals Access to therapies Safety Risk benefit decisions Real life and quality of life issues 11 Patient Organisation Priorities

No patient population perception on HTAs and off-label use Patient Organisation and Experts are a different kettle of fish These are self-selecting groups Work is needed to build trust in pharma and regulators, but patients trust their healthcare professionals Note: My perspective is inevitably tainted 12 Summary

PharmaPatientsRegulators Old Stakeholder Map

Pharma Patients Regulators We are all people first

15

Patients’ Perspective on HTA and Off-label use David Head MBA Chief Executive RP Fighting Blindness 25th Annual EuroMeeting 4-6 March 2013 RAI, Amsterdam Netherlands