Have FDA Expedited Programs Shortened Drug Development Timelines? An analysis of newly approved therapies and how FDA expedited programs impacted drug.

Slides:



Advertisements
Similar presentations
Want to SAVE a Few Million Dollars?. Give Us the OPPORTUNITY Do Just That and Well.
Advertisements

Rare Diseases and FDASIA
Agenda Overview: the Bio-Pharma industry Major challenges for developers Genzyme: Innovations for unmet medical needs.
Targeted Cancer Therapeutics, LLC Investor Presentation.
1 August 2013 WASHINGTON DC AUSTIN SAN FRANCISCO Corporate Biography.
Clinical Trials — A Closer Look. The Food and Drug Administration (FDA) is the main consumer watchdog for numerous products: Drugs and biologics (prescription.
About OMICS Group OMICS Group International is an amalgamation of Open Access publications and worldwide international science conferences and events.
Wednesday, December 17, :00pm-4:30pm EST National Coalition for Cancer Survivorship Post-Training Webinar ©2014 National Coalition for Cancer Survivorship.
CADTH Therapeutic Reviews
The Health Care Sector Analysts: Heather Hund and Emily Butler.
Biomedical research methods. What are biomedical research methods? An integrated approach using chemical, mathematical and computer simulations, in vitro.
Clinical Pharmacy Basma Y. Kentab MSc..
Entrepreneurship in Biotechnology Columbia University Graduate School of Arts and Sciences BIOT 4180 Columbia University GSAS BIOT 4180.
DOCUMENTATION GUIDELINES FOR E/M SERVICES
Stages of drug development
Healing Hands Clinical Research Services is a Site Management organization with broad spectrum of activities.
David A H Whiteman MD FAAP FACMG Global Clinical Sciences Leader Shire Pharmaceuticals.
FDA Regulatory Considerations in Launching Products Michael A. Swit, Esq. Vice President, Life Sciences WITI (Women In Technology International) San Diego.
Science, research and developmentEuropean Commission Chile-EC S&T Agreement Brussels, 24 September 2002 Life Sciences, Genomics and Biotechnology for Health.
Presented by: Jason Brown, CPA Date: May 10, 2010 Qualified Therapeutic Discovery Project Tax Credit.
Program Collaboration and Service Integration: An NCHHSTP Green paper Kevin Fenton, M.D., Ph.D., F.F.P.H. Director National Center for HIV/AIDS, Viral.
1 Value Assessment of Development-Stage Assets Pharmaceutical Products, Medical Devices, and Related Intellectual Property Frank S. Castellana, M.D., Eng.Sc.D.
NCI Review of the Clinical Trials Process 6 th Annual National Forum on Biomedical Imaging in Oncology James H. Doroshow M.D. April 7, 2005 Bethesda, Maryland.
1 Safety Pharmacology for Oncology Pharmaceuticals at CDER John K. Leighton Associate Director for Pharmacology CDER/OND/OODP.
The Business Research Company Healthcare. Typical Healthcare Projects Copyright TBRC Business Research. All Rights Reserved. 2 Market Analysis Product.
Biomedical Research Objective 2 Biomedical Research Methods.
SIPLAS RO is a full service contract research organization CRO, offering nanotechnology, biopharmaceutical and medical device companies comprehensive.
UNCLASSIFIED10/12/ :41 AM Slide 1 Division of Regulated Activities and Compliance.
Update From FDA: Office of the Commissioner and Center for Drug Evaluation and Research Janet Woodcock, M.D. Acting Deputy Commissioner for Operations.
National Health Expenditures as a Share of Gross Domestic Product (GDP) FIGURE 7.1 Between 2001 and 2011, health spending is projected to grow 2.5 percent.
Supporting Informed Formulary Decision Making: CADTH’s Common Drug Review Denis Bélanger, Director, CADTH New Brunswick Stroke Summit November 27, 2010,
Single Patient Use of Investigational Anticancer Agents: An Industry Perspective Gerard T. Kennealey, MD Vice President, Clinical Research, Oncology AstraZeneca.
MATERIALS AND METHODS Tracking Women’s Participation and Sex Analyses in Late-Phase Clinical Trials of New Molecular Entity (NME) Drugs and Biologics Approved.
Developing medicines for the future and why it is challenging Angela Milne.
PUTTING THE PATIENT AT THE CENTER OF HEALTHCARE RESEARCH Towards a more inclusive model.
RITA M. HIRIART, MOT NOVARTIS PHARMACEUTICALS RARE DISEASES BUSINESS PRESENTATION.
Current Plan for Critical Path Initiative Janet Woodcock, M.D. Acting Deputy Commissioner For Operations November 5, 2004.
Protection of Intellectual Property in Canada George Jackowski, Ph.D., KCTJ October 19 th, 2015 Chairman of NAVA Corp.
 Treats a population of < 200,000 in the US  Same review and development standards as for a non- orphan product  Numbers of patients in clinical trials.
SEEK is a drug-discovery group that uses a pioneering scientific and commercially-driven approach to create breakthrough medicines which address major.
Who we are 2© 2015 INC Research, LLC Therapeutically specialized teams The complete range of Phase I to IV clinical development services Helping you develop.
Biosimilar (Insulin) – Competitive Landscape and Market & Pipeline Analysis, 2016 DelveInsight’s, “Biosimilar (Insulin) – Competitive Landscape and Market. Request for sample of this research report:
Published Date : 3 August 2015 World Specialty Enzymes Market.
© Copyright 2004 Frost & Sullivan. All Rights Reserved. Strategic Analysis of the U.S. Overactive Bladder and Stress Urinary Incontinence Emerging Markets.
Date of download: 6/23/2016 Copyright © The American College of Cardiology. All rights reserved. From: Cardiovascular Drug Development: Is it Dead or Just.
MarketsandMarkets Presents Pain Management Devices Market worth 4.64 Billion USD by 2021
If you can't see this message pleas e us e this linkpleas e us e this link FDAs Expedited Programs for Serious Conditions - Drugs and Biologics Friday,
INVESTIGATOR PORTAL A TRANSLATIONAL RESEARCH KNOWLEDGE BASE DAVID HORKY COUNTRY MANAGER – CENTRAL AND EASTERN EUROPE SEPTEMBER.
1 Promotion in Management and Research Tracks in Industry Magdalena Alonso-Galicia, PhD Cardiovascular Diseases Department Merck Research Laboratories.
Rigid Endoscopes : Medical Device Industry Research And Analysis
Endoscopy Visualization Systems - Medical Devices Pipeline Assessment
Positron Emission Tomography (PET) Systems - Medical Devices Pipeline Assessment, 2016
FDA DRUG APPROVAL FDA’s Lengthy Drug Approval Process in Twelve Steps Overview of the FDA Drug Approval Process Drug Developed June 13, 2016 | Emilia Varrone.
© Coherent market Insights. All Rights Reserved PEDIATRIC CLINICAL TRIALS MARKET Global Industry Insights, Outlook Size, Share and Opportunity Analysis,
© 2016 Global Market Insights, Inc. USA. All Rights Reserved Precision Medicine Market share to hit $87.7bn by 2023
The Impact of Accountable Care Organizations in Radiology
Industry Perspective: Expanded Access Programs
Clinical Trials — A Closer Look
Expedited Drug Approval Programs
FDA Perspective on Cardiovascular Device Development
NASs approval time by therapeutic area:
Finland, a Global Testbed for Personalized Cancer Research?
Changing the Game for Sjögren's Patients
Positive Impacts of Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials* SPECIFIC BENEFITS   SHORT-TERM MEDIUM-TERM LONG-TERM.
Speeding access to therapies
Median submission gap, median approval time and percentage approved as expedited for new active substances (NASs) approved by six authorities:
Suzanne M. Sensabaugh, MS, MBA
Phase 2 to phase 3 clinical trial transitions: Reasons for success and failure in immunologic diseases  Dhavalkumar D. Patel, MD, PhD, Christian Antoni,
Objective 2 Biomedical Research Methods
Global Specialty Pharmaceuticals Market.
Presentation transcript:

Have FDA Expedited Programs Shortened Drug Development Timelines? An analysis of newly approved therapies and how FDA expedited programs impacted drug development timelines September 8, 2014 Artisan Healthcare Consulting, Inc phone

We collected the following dates for each NME approved from 2009 to 2014: IND(investigational new drug) submission date of the first approved indication Date of the FDA end of phase 2 (EOP2) meeting where available or the start date for the pivotal trials NDA (new drug application)/BLA (Biologics License Application) filing dates NDA/BLA approval dates We stratified the NMEs by year of approval, and type of FDA expedited program used. We categorized each NME by therapeutic area: Allergy, Autoimmune, Cardiovascular, Central Nervous System (CNS), Diabetes/ Metabolism, Diagnostic, Infectious disease, Musculoskeletal, Oncology, Ophthalmology, Rare diseases, Respiratory system, Sexual Dysfunction, Skin, Supportive care, and Women’s Health. We collected the following dates for each NME approved from 2009 to 2014: IND(investigational new drug) submission date of the first approved indication Date of the FDA end of phase 2 (EOP2) meeting where available or the start date for the pivotal trials NDA (new drug application)/BLA (Biologics License Application) filing dates NDA/BLA approval dates We stratified the NMEs by year of approval, and type of FDA expedited program used. We categorized each NME by therapeutic area: Allergy, Autoimmune, Cardiovascular, Central Nervous System (CNS), Diabetes/ Metabolism, Diagnostic, Infectious disease, Musculoskeletal, Oncology, Ophthalmology, Rare diseases, Respiratory system, Sexual Dysfunction, Skin, Supportive care, and Women’s Health. 1. We collected key dates for new molecular entities (NME) and biologics approved over the past 6 years. Methodology (1 of 2) 3. We compiled and analyzed the findings to determine the effect of FDA expedited programs on drug development timelines. Fast Track is introduced to accelerate the approval for medications treating serious conditions with unmet medical needs or a drug that has been designated as a qualified infectious disease product. Accelerated Approval allows for approval of medication treating serious conditions based on surrogate endpoints. Priority Review is for medications that offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Breakthrough therapy designation is for medications that treat serious or life threatening disease and the medications may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.. Fast Track is introduced to accelerate the approval for medications treating serious conditions with unmet medical needs or a drug that has been designated as a qualified infectious disease product. Accelerated Approval allows for approval of medication treating serious conditions based on surrogate endpoints. Priority Review is for medications that offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Breakthrough therapy designation is for medications that treat serious or life threatening disease and the medications may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.. 2. We looked at the 4 major FDA programs used to expedite clinical development to understand impact on time to market

Methodology (2 of 2) *Sources: “Approved drugs 2013”, “2011 Novel New Drugs”, “2012 Novel New Drugs Summary”  IND filing: As reported in FDA filings - where information not available dates extracted from the Federal Register  End of Phase 2 meeting/beginning of pivotal trial -As reported in FDA filings – where the information was not available start dates of the pivotal trial extracted from  NDA/BLA filing: As reported in FDA filings  NDA/BLA approval: As reported in FDA filings Excluded NMEs from the analysis  Lumizyme and Corifact: Excluded entirely from the analysis as IND filings and EOP2 meeting data are not available  2014 approved NMEs Belsomra, Cerdelga, Jardiance, Jublia, Orbativ, Otezla, Pledridy, Sivextro, Striverdi Respimat, Tanzeum, Zydelig and Zykadia: Excluded as full FDA documentation is not published Sources used

NMEs That Used Any of the FDA Expedited Programs 4 Therapeutic area Autoimmune Cardiovascular CNS Diabetes/Metabolism Diagnostics Infectious diseases Oncology Rare diseases Respiratory system Supportive care Ophthalmology *2014 FDA approvals by August 2014

Which Programs Are Used the Most? 5 Accelerated programs Fast track Accelerated approval Breakthrough therapy Priority review Any program 1 of 25 (4%) 0 of 20 (0%) 15 of 30 (50%) 14 of 39 (36%) 10 of 27 (37%) 1 of 26 (45%) 2 of 25 (8%) 2 of 20 (10%) 3 of 30 (10%) 4 of 39 (10%) 2 of 27 (7%) 4 of 26 (15%) -- 3 of 27 (11%) 2 of 26 (8%) 6 of 25 (24%) 6 of 20 (30%) 15 of 30 (50%) 16 of 39 (41%) 10 of 27 (37%) 8 of 26 (31%) 7 of 25 **(28%) 8 of 20** (40%) 17 of 30**(57%) 22 of 39 **(56%) 13 of 27 **(48%) 12 of 26** (46%) *2014 FDA approvals by August 2014 Since 2011, ~50% Of All Approved Therapies Have Benefitted From An Expedited Pathway, With Priority Review and Fast Track Being the Most Heavily Utilized. **Number of drugs approved in particular year

Which Therapeutic Areas Use the Expedited Programs the Most? 6 Therapeutic area/ All programs Oncology Rare diseases Supportive care Diagnostics Infectious diseases Diabetes/Metabolism Cardiovascular Autoimmune Ophthalmology CNS Respiratory system Other Total 3 of 52 of 27 of 710 of 118 of 84 of 434 of 37 (92%) 0 of 11 of 102 of 20 5 of 6 (84%) 002 of 31 Of 1003 of 4 (75%) 001 of 12 of 21 of 30 of 14 of 7 (60%) 1 of 41 Of 12 of 43 of 33 of 43 of 613 of 22 (60%) 0 of 10 of 20 of 11 of 70 of 20 of 41 of 17 (59%) 2 of 52 of 31 of 41 of 3 0 of17 of 19 (37%) 0 of 32 of 43 of 30 of 301 of 46 of 17 (35%) 0001 of 3001 of 3 (33%) 1 of 40 0 of 10 of 32 of 34 of 16 (25%) 000 of 21 of 20 of 20 of 11 of 7 (14%) of 8 (0%) 7 of 25 (28%) 8 of 20 (40%) 17 of 30 (57%) 22 of 39 (56%) 13 of 27 (48%) 12 of 26 (46%) 79 of 167 (47%) In Oncology, Rare Diseases and Supportive Care over 75% of Approved Therapies Utilized an Expedited Program.

Which Expedited Programs Are Used the Most by Individual Therapeutic Areas? Fast track Priority review Accelerated approval Breakthrough therapy Any single Program 2 programs 3 programs All 4 programs Autoimmune 3 of 175 of of 172 of 1700 Cardiovascular 1 of 197 of of 1900 CNS 1 of 162 of 161 of 1604 of Diabetes/Meta bolism 01 of Diagnostics 04 of Infectious diseases 9 of 2211 of 222 of 221 of 2213 of 228 of 221 of 220 Oncology 20 of 3724 of 3712 of 374 of 3734 of 3716 of 372 of 371 of 37 Rare diseases 2 of 63 of 61 of 605 of 61 of 600 Respiratory system 1 of Supportive care 3 of 4 1 of 403 of 42 of 41 of 40 Ophthalmology 01 of Other Total 40 of 167 (23%) 61 of 167 (37%) 20 of 167 (12%) 3 of 167 (18%) 79 of 167 (47%) 31 of 167 (19%) 3 of 167(18%) 1 of 167 (1%) In Oncology, Infectious Diseases and Supportive Care, over 35% of NMEs Use Multiple Expedited Programs.

Have Expedited Programs Shortened the Time from IND to NDA/BLA Approval? 8 # of years from IND to NDA/BLA Approval (average) Year of NDA/BLA approval When Considering the Time from IND to NDA Approval, Expedited Programs Do not Appear to Reduce Time to NDA/BLA Approval.

Have Expedited Programs Shortened the Time from the EOP2 Meeting to NDA/BLA Approval? 9 On Average, NMEs Using Expedited Programs Reduced the Time from EOP2 to NDA/BLA Approval by 1.2 Years.

Have Expedited Programs Shortened the Time from IND to NDA/ BLA Approval? 10 Average time: 7.4 years Average time: 4.5 years Average time: 8.6 years # of years from IND to NDA/BLA Approval (average) Average time: 9.0 years Average time: 8.8 years Breakthrough Therapy Has the Greatest Impact on Time to Approval of All Accelerated Programs; Accelerated Approval Also Provides Benefit.

Have Expedited Programs Shortened the Time from EOP2 meeting to NDA/ BLA Approval? 11 Average time: 4.7 years Average time: 2.5 years Average time: 4.5 years # of years from IND to NDA/BLA Approval (average) Average time: 4.4 years Average time: 4.7 years When Considering Time from EOP2 Meeting to NDA/BLA Approval, Only Breakthrough Therapy Significantly Improves Time to Approval Compared to Other Expedited Programs.

In Which Therapeutic Areas Have the Expedited Programs Shortened the Time from IND to NDA/BLA Approval? 12 Reduction in Time From IND Submission to NDA/BLA Approval Is not Consistent Across Therapeutic Areas.

In Which Therapeutic Areas Have the Expedited Programs Shortened the Time from Time from EOP2 meting to NDA/BLA approval? 13 For Most of the Therapeutic Areas Using Expedited Program Has Shorten the Time from EOP2 to NDA /BLA Approval.

Conclusions 14 Since 2011, ~50% Of All Approved Therapies Have Benefitted From An Expedited Pathway, With Priority Review and Fast Track Being the Most Heavily Utilized. ✔ When Considering Time from EOP2 meeting to NDA Approval FDA Expedited Programs Reduced the Time from EOP2 to Approval by 1.2 Years. The Expedited Programs Do not Shorten the Time from IND Submission to NDA/BLA Approval. ✔ ✔ Breakthrough Therapy Was introduced the Latest but Seems to Have the Biggest Impact on Approval Time Compared to Other Expedited Programs. In Oncology, Rare Diseases and Supportive Care over 75% of Approved Therapies Utilized an Expedited Program. In Oncology, Infectious Diseases and Supportive Care, over 35% of NMEs Use Multiple Expedited Programs. ✔

Brad Payne - Bio Brad Payne is a Team Leader for Artisan Healthcare Consulting. He has over 5 years experience in the healthcare consulting space, with expertise in domestic and international reimbursement and market access. Lead of the global market access practice Extensive knowledge of national and regional payers in Europe, Canada, Latin America, and Asia/Pacific Advanced understanding of U.S. market access, with a proven track record of developing strategy that influenced national and regional payer coverage and payment decisions Proven ability to develop market access strategies utilizing experience across a number of therapeutic areas including oncology, immunology, infectious diseases, rare diseases, and devices Understanding of health economics, with extensive expertise in Health Technology Assessment (HTA) analyses Prior to joining Artisan, Brad was a consultant at Trinity Partners, a pharmaceutical and biotech consulting firm, where he provided insight on forecasting projects, licensing and acquisition opportunities, and provided recommendations for managed care contracting strategies. Brad graduated with a degree in economics Cum Laude from Harvard University and received an MBA from the W.P. Carey School of Business at Arizona State University. 15 Brad Payne Team Leader office cell Brad Payne Team Leader office cell