business of biosimilars & biobetters 2011 le… · 18.09.2011 · chief regulatory science...
TRANSCRIPT
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Dr. Nigel A.S. HernandezPresident & Founder
Lessons Learned from Biosimilar Development in the Established European Marketplace
Business of Biosimilars & Biobetters 2011Seaport World Trade Center, Boston, MASeptember 2011
© 2011 Center for the Study of Regulatory Affairs Inc.
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Keys Areas• Lessons Learned from Biosimilar Development in the Established
European Marketplace• European countries are furthest ahead in biosimilar development,
with Germany and the UK leading the way. • Hear what CenSRA takes into account when evaluating biosimilars• Estimate the effect a growing marketplace will have on biosimilar
pricing strategies• Discuss the long term benefits of creating biosimilars in a highly
regulated market, including the opportunity to easily move to less-regulated marketplaces
• Analyze regional-specific opportunities for commercializing biosimilars
© 2011 Center for the Study of Regulatory Affairs 2
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Outlines • Introduction to CenSRA• Drug Development Economics• ‘The Birth’ of the Legal Framework for Biosimilar Medicines in
the European Union • When Can Biosimilar Medicines Applications Take Place? • What are the Data Requirements? • INN/International Non-proprietary Name • Post-Authorization • Biosimilar Medicines in the Market Place • CenSRA Vision
© 2011 Center for the Study of Regulatory Affairs 3
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Acronyms• EU European Union• EMEA (EMA) European Medicines Agency• CHMP Committee for Medicinal Products for Human Use• EC European Commission• WHO World Health Organization• MS Member State• MAA Marketing Authorization Application• MAH Marketing Authorization Holder• DE Data Exclusivity• ME Market Exclusivity• INN International Non-Proprietary Name• ADR Adverse Drug Reaction• RMP Risk Management Plan• PASS Post Authorization Safety Study
© 2011 Center for the Study of Regulatory Affairs 4
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Speaker BiographyEXPERIENCE:• Founder and Chairman, Center for the Study of Regulatory Affairs• Chief Regulatory Science, Quality & Food Safety Officer, GloboFruit, LLC• Chief Regulatory Science Officer, GMP Regulatory Science Partners• Academic Appointments: University of Maryland University College,
Previous: West Virginia University & Northeastern University
NORTHEASTERN UNIVERSITY• Doctor of International Biomedical & Food Regulatory Science.• MSc., Regulatory Affairs for Drugs, Biologics & Medical Devices• Masters Certificate, International Biopharmaceutical Regulatory Affairs• Masters Certificate, Medical Devices Regulatory Affairs (US, EU, Japan)
BOSTON UNIVERSITY• Master of Science, Advertising
EDINBURGH BUSINESS SCHOOL• Master of Business Administration
SYRACUSE UNIVERSITY• BA/ BS
REGULATORY AFFAIRS CERTIFIED (RAC)EMEA – PHARMACOVIGILANCE CERTIFIED (RPPV)
© 2011 Center for the Study of Regulatory Affairs 5
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ABOUT CENSRAAbout the Center for the Study of Regulatory Affairs
© 2011 Center for the Study of Regulatory Affairs 6
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Introduction• CenSRA - The Center for the Study of Regulatory
Affairs addresses regulatory affairs within each category of regulated agents: drugs, biologics, medical devices and combination products.
• An innovative regulatory center that will serve all constituencies in the medical products community –industry, regulators, researchers, clinicians, patients, associations and academic centers – to share knowledge to accelerate the regulatory paradigms and best practices for the discovery and commercialization of new medical products for improving patients lives and outcomes.
© 2011 Center for the Study of Regulatory Affairs Inc. 7
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Partnership BuildingLegislators & Policy
MakersNIH, NSF
Centers & Labs
Science Organizations
Schools, Colleges, Universities
Informal ScienceEducation Institutions
Business, Industry,Foundations
National and Int’lOrganizations
Patient and DiseaseGroups
Federal, State & LocalAgencies
Center for the Study of Regulatory Affairs
© 2011 Center for the Study of Regulatory Affairs Inc. 8
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DecisionsData Knowledge Impact
The CenSRA Framework
• Regulatory Agencies
• Industry Associations
• Databases/Internet• People• Institutes/Centers
• Research Requests• RGRA (Regulatory
Strategy and Risk Assessments)
• Regulatory Workshops
• Roundtables• Regulatory training
• Regulatory/ registration strategies
• Clinical trial designs
• Comments• Position Papers
• Faster approvals• Compliance• Competitive
labeling• Favorable
legislation• Industry
leadership• Policy leadership
• Web 2.0 framework
• Newsletters• Newsflashes• Reports
Inform Analyze Distribute Recommend Impact
© 2011 Center for the Study of Regulatory Affairs Inc. 9
Information
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DRUG DEVELOPMENT ECONOMICS
The Business of Drug Development is Approval
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© 2011 Center for the Study of Regulatory Affairs
Target
Qualified lead
Qualified candidate
Product in human
lead generation
candidate generation
Milestone: high affinity/specificity
Milestone: acceptable PK/production characteristics and efficacy in animal disease models
Milestone: acceptable safety, established production process
The Discovery Value Chain
11
lead compound
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The Economics?Consequences of weak
drug regulatory capacity
Irrational consumption and prescription,
substandard, counterfeit, harmful, useless drugs on
sale
Consequences of regulatory inaction
Death to industry, patients, loss income,
increased health costs, market failure?
© 2011 Center for the Study of Regulatory Affairs 12
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Challenges faced by Pharma/BIO
• Competitive and market changes• Turnover • New scientific knowledge and discoveries • Regulatory changes/ new standards • New disease knowledge • Potential indications• Financial & Capital Markets
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Biosimilars: Europe’s New Opportunity ?
“Biosimilars offer new opportunities both for the growth of our generic industry and for the control of our national health expenditure.”
© 2011 Center for the Study of Regulatory Affairs
Günter Verheugen, Vice-President EU Commission April 2006
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Chance favorsonly the
prepared mind
(Pasteur)
Serendipity
Science, Management, Predictability
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TIME & COST: 8-12 YEARS & $800M-1.2B
Source: Page 70 Scientific American April 2000 Understanding Clinical Trials
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REGULATORY LANDSCAPEThe Regulatory Landscape, what it maters….
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Legislation has publicly stated goals, but• Are they well resourced?• Are they well planned? • Are they independent?• Are the interests aligned?• Are they informed by all
relevant stakeholders?• Are they free of political or
other bias?
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FEEDBACK
PERSPECTIVESDevelopmentImplementationEvaluation
EVALUATION STAGESPlanning & designConducting the evaluationInstitutionalizing change
POLITICS
LAW
TACTICSFramingInterpersonal influenceTimingInformation and analysis
STANDARDSUtilityFeasibilityProprietyAccuracy
PUBLIC POLICY
The Policy Connection
CHANGE AGENTSEventsKnowledgeInterest GroupsIndividuals
INFLUENCERS
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The Biosimilar Regulatory Landscape
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The EU Trinity
© 2011 Center for the Study of Regulatory Affairs
EUROPEAN COMMISSION
COUNCIL OF MINISTERSEUROPEAN PARLIAMENT
• Guardian• Protectors• Sole Right
• Decision Taker• Representer
• Co-legislator;• Representer
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Sounds easy right?
© 2011 Center for the Study of Regulatory Affairs
EU Legislation
EP
EC
Council 27
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© 2011 Center for the Study of Regulatory Affairs 23
Careful development…over timePharmaReview
2001
Directive 2003/63/EC“Annex I” published:
recognition of ‘biosimilars’
Directive 2001/83/ECas amended published
AdoptedDirective
takes effect
Legal
Regulatory
2001 2002 2003 2004 2005 2006
2001 2002 2003 2004 2005 2006
EMEA’s biosimilar ‘quality issues’and ‘non-clinical and clinical
issues’ draft guidelines published
EMEA’s Revised ‘comparability’
guidelines published
EMEAreleases
biosimilar‘conceptpapers’
EMEA’s product specific Draft guidelines released for hGH, insulin, EPO and
G-CSF.
EMEAworkshop
Firstbiosimilarapproved
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EU Framework for Biosimilars:
Legal certainty• Clear legal basis• Abbreviated data package
Flexibility• Case by case approach• General and product
specific guidelines
© 2011 Center for the Study of Regulatory Affairs
key forinvestments
adjustments of datarequirements (science, technology, experience)
• Art.10.4./Directive 2001/83/EC as amended (31 March 2004)
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BIOSIMILAR APPLICATIONS?When Can Biosimilar Medicines Applications Take Place?
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© 2011 Center for the Study of Regulatory Affairs 26
15 Years Maximum Market Exclusivity for Reference Product:
e.g. the marketingauthorization is granted tooriginator in year 12
Maximum 5 years extension ofSupplementary ProtectionCertificate (SPC)
10 12 20 25
10 Years
(new: 8+2+1)
Data exclusivity (DE) period of reference product
Biosimilar application,registration andauthorization
NO Patent LinkageSubmission of biosimilar applications only possible after DE expiry
Patent Duration
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WHAT’S COVERED?Which Products are Covered?
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Legal Perspective
• In principle the concept of “similar biological medicinal (biosimilar) products” applies to any biological medicine
• ‘Generic approach’ is legally not excluded
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Spectrum of Complexity
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(source: Dr. John Purves/EMEA)
Science
Chemicals Recombinant DNAtechnology
Blood derived Immunologicals Advancedtherapy
FullDossierBiosimilarGeneric
(essentially similar)
*
*Legislation
* Future Developments ?
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DATA REQUIREMENTS?What are the Data Requirements?
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Regulatory Perspective• For the type and quantity of data to be
provided, the EU legislative framework refers to detailed guidelines
• But guidelines are NOT mandatory before submission and approval
• Guidance follows science– Guidance is built on experience gained through
scientific advice procedures and assessment of marketing authorization application
– Guidelines do not have legal force – but a justification for non compliance must be provided
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Current EU Biosimilar Guidelines
© 2011 Center for the Study of Regulatory Affairs
http://www.emea.europa.eu/htms/human/humanguidelines/multidiscipline.htm
Overarching Guideline (CHMP/437/04).“Guideline on Similar Biological Medicinal Products”
Biotechnology- derived proteins
Quality
Non-clinical
------------Clinical
Non-Clinical
----------------------------
Clinical
Non-Clinical
----------------------------
Clinical
Non-Clinical
----------------------------
Clinical
Non-Clinical
----------------------------
Clinical
Non-Clinical
----------------------------
Clinical
Insulin Somatropin GCSF Epoetinunder review
LMMHIFN- α
Defines philosophyand principles
General guidelines
Annex guidelines -specific datarequirements
Non-Clinical
----------------------------
Clinical
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Example: Omnitrope® Approved before Somatropin Guidance• Omnitrope Positive
Scientific Opinion 26 Jan 2006
• Approval of Omnitrope 18 April 2006
• Adoption of guideline22 Feb 2006
• Came into effect1 June 2006
© 2011 Center for the Study of Regulatory Affairs
EMEA/CHMP/BMWP/94528/2005
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High Level Scientific Evaluationby EMEA and EC Approval
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INNINN International Non-proprietary Name
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Purpose of INN
• Classification / nomenclature– Identification of pharmaceutical substances
• WHO Guidance on INN – broad scope– Pharmacopoeia, labelling, product
information, drug regulation, basis for product names (generics)…
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INN is NOT…
• Identification of a drug product or its impurities
• Statement of therapeutic equivalence and/or substitution
• Means of managing the practice of medicine
• Traceability and pharmacovigilance apply to drug products
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Current INN Policy
• INNs for glycosylated rDNA proteins– Core name based on Amino Acid sequence– Greek letter added: ‘differences in
glycosylation’• Difficult to define ‘differences’
– eg, batch to batch differences
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INNs for Biologicals
• EU consideration at WHO level:– INNs should have “high level” utility –
identification– Up to Regulatory Authorities to assess
Benefit / Risk– AA sequence basis for INN– Presence/absence of glycosylation
compared to native protein• Discussions continue
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Nomenclature: Same INNDecision lies with RegulatorsWith regard to the nomenclature of biosimilars, we do not endorse the idea that any difference in glycosylation automatically leads to a different INN. We have serious doubts that this stance could be scientifically justified. We are also concerned that the WHO is promoting a double-standard policy on biosimilars.
© 2011 Center for the Study of Regulatory Affairs 40
G.Lalis/Director General EC -Statement on WHO INN Policy at EGA Annual Conference Paris 2 Junehttp://www.egagenerics.com/doc/GLalis_speech_egaconf2008.pdf
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POST-AUTHORIZATIONAfter Market – So What?
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Pharmacovigilance
• Company Risk Management System must be in place– Risk Management Plan
• Pharmacovigilance activitiesRoutine pharmacovigilancePASS
– Risk minimization activitiesLabelling (counter-indication, advice, warning)
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Traceability of ALL Biologics
All biological drugs need to be monitored• An adverse reaction report for any biological
drug should always include– full name of the biological drug– batch number
• Where information is missing, Member States/MAHs should ensure that reports are followed up for completion
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G. Lalis/Director General ECon Pharmacovigilance for BiosimilarsEGA Annual Conference Paris 6/08
Furthermore we have written to Member States' regulatory authorities to take necessary measures to ensure • a method to link suspected adverse reaction
reports to specific products (such as a unique product identifier) and
• to ensure that prescribing doctors know which glycoprotein has been given to their patient.
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BIOSIMILARS MARKETPLACEBiosimilar Medicines in the Market Place
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Biosimilar Drugs in the EU Market• National pricing approvals and reimbursement
have been agreed without any major barriers• Market in-roads are made• Patients and healthcare professionals are• catching up• But scare tactics still ongoing by certain
interested parties
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Economics
© 2011 Center for the Study of Regulatory Affairs
Bio similar Market
Supply side
policies
Demand side
policies
Regulatory Pathway
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Economics
© 2011 Center for the Study of Regulatory Affairs
Bio similar MarketSupply
side policies
Demand side
policies
Regulatory Pathway
Hospital/Pharmacists Patients Doctors
Regulators
Ministriesof Health
Price &reimbursement
Authorities
Manufacturers
Wholesalers &Pharmacies
Hospitals
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Message from the European Commission• ………….’we are confident that if a
product goes through all the steps and meets all the requirements and gets at the end an approval through a Commission Decision, it means that this product is as safe and efficacious as any other product authorised by the European Commission in the EU’.
© 2011 Center for the Study of Regulatory Affairs
N. Rossignol at 6th EGA symposium on biosimilar medicines 2008
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WHAT IS CRITICAL?The Regulatory Science Function is Critical
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The RA Function Is Critical
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PharmacyNursingEngineeringLaboratory ScienceTechnical WritingMedicine
StatisticsLaw
RA ProfessionalsAre Not Born, They are Made
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GLOBAL DEVELOPMENTIs Global Development a Myth or Reality?
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Global Development for Biosimilars• Streamlining the development process• Avoids repetition of unnecessary trials• Increases access through affordable medicines• Increases competition
– Market competition from biosimilar drugs willdrive the biotechnology industry to do what itdoes best – discover new drugs that enhance,sustain, and save lives.
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Market Share Value by Country in Q2
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COUNTRY Human Growth Hormone
Erythropoetin Granulocytehormone colony stimulating factor
US 6.6% 0 0Japan 0 0 0France 12.5% 2.0% 3.8%Germany 5.1% 52.1% 31.0%Italy 15.3% 0.2% 18.5%Spain 1.4% 1.6% 7.1%UK 1.0% 0.9% 24.0%
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Leaders & Numbers• Sandoz (Teva and Hospira right behind)
• Global Biologics Market Sales in 2009 exceeded $125 Billion
• 2011 Biosimiliars accounted for only $89 million in sales (Sandoz Omnitrope acounts for 33%)
• Germany – greatest market share in Europe and Worldwide.
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SWOTSTRENGTHS
• Lower cost structure• Development is more calculable versus
research on new drug leads• Biosimilars are a reality
WEAKNESS• Lack of credibility in the prescribing
community• Lack of credibility in the policy forming
community• Lack of Credibility as an industry
OPPORTUNITIES• Favorable pricing and reimbursement
policies• Driving further innovation• Continued growth of developing, non-
saturated markets
THREATS• Evolving regulatory environment not
always aligned with industry interests• Technology improvement and shifts• Intellectual property domain
BIOSIMILARS
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SWOT Analysis• Timing Matters • Dare to be different• Keep it simple• Don’t play on IP• Prepare deep pockets for financing
the expected
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STRATEGIES?What does this all mean?
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Strategies for Success• Analyze new regulatory impacts - build and use the links that
create rnetworks.• Collaborate internally/externally and work together to share
information and expertise.• Align behavior with dynamic strategy - understand and deliver
regulatory intelligence that affect business outcomes across the network (partners, customers, collaborators, industry).
• Leverage competency - focus on core competencies and collaboration, entrusting your network to do more with less.
• Integrate partners and collaborators proactively.• Operate at net speed - respond quickly and decisively to rapidly
changing regulatory policy and market dynamics.
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Strategies for Success• Provide a vital link by working outside the framework
of government and industry to ensure informed analysis and independent guidance.
• Reach globally and virtually - Collaborate or exchange knowledge regardless of time or regulatory diversity.
• Manage Knowledge dynamically - leverage data from internal and external sources to create competitive advantage.
• Build strong market presence and credibility.• Develop brand awareness and create a need for why
your Biosimilar should be approved.
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The Benefit/Risk Evaluation Fallacy
Benefits
Risks
Reduce Preventable and Quality Risks
Reg. Agency
PROVIDER
PATIENT
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What is safety? Safety is not Absolute• ≠ zero risk (absence of injuries and deaths)• Involves overall assessment of benefit/risk
Safety is not Exclusive• ≠ only experience with the drug• Involves comparison across
drugs/interventions
Safety is not Static• A safe drug/device today may be “unsafe”
and outmoded tomorrow• Experimental risk versus Situational risk
Safety is Evolutionary…only the real world can tell….What about efficacy?
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© 2011 Center for the Study of Regulatory Affairs Inc.
THANK YOU
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Dr. Nigel A.S. HernandezPresident & Founder
Lessons Learned from Biosimilar Development in the Established European Marketplace
Business of Biosmiliars & Biobetters 2001Seaport World Trade Center, Boston, MASeptember 2011
© 2011 Center for the Study of Regulatory Affairs Inc.