Introduction to R&D capabilities in Queensland, Australia Introduction March 2009.pdf · Europe vs...

Introduction to R&D capabilities in pQueensland, Australia

Mario PennisiCEOCEO

Outline

Bio-Pharma Landscape in AustraliaBio Pharma Landscape in Australia

Queensland, Life Sciences, QCTN Inc.

Regulatory Environment

A st alia and Clinical T ialsAustralia and Clinical Trials

Final Considerations and Summary

AustraliaAustralia is about the size of the US without Alaska or

32 x the size of UK or 20 x Japan

Queensland: 7x UK

Population of Australia is about 21 million

Northern Territory

~60% of population concentrated in the major cities

Western

Queensland

New Zealand (4 M)

Western Australia Brisbane

SydneyPerth

Adelaide

New South WalesSouth Australia

Victoria

Melbourne

Victoria Tasmania

Pharma sector in Australia in 2007

> 300 Pharma companies> 300 Pharma companies

> 400 Biotech companies

> 600 Medical device companies

Total turnover of Australian Pharma

• approx $9.4 billion ~ incl. Nutra/Compl Med

Only about 1.3% of world market

R & D spend of Pharma: AUD $752 million

Outline






Queensland Life Sciences Approach

QueenslandQueensland rapidly expanding research base

>A$ 1.5 billion invested since 1998 by QueenslandQueensland

Similar investments by other Statesother States and Federal Government

First world medical systems and infrastructure, highly qualified

clinicians & researchers, English, diverse ethnic population …

Queensland Life Sciences Approach

Nutraceuticals/ Ethical Pharma & /Complementary Medicines

Generic Medicines

Biotech Medicines

Medical Devices

R & DPreclinical

TrialsClinical Trials (Phase I II

ry manufacbvvgcvcvcv

Process design, prototyping &

Marketing, sales and distribution

Primary &S dR & D

Preclinical Trials

Clinical Trials (Phase I II



Marketing, sales and distributionR & D

Preclinical Trials

Clinical Trials (Phase I II



Marketing, sales and distribution

Primary &S dR & D Trials (Phase I, II,

III, IV)

gcvcvcvccvcturingprototyping &

scale-upand distribution&Secondary

ManufacturingR & D Trials (Phase I, II,

III, IV)


scale-upand distributionR & D Trials (Phase I, II,

III, IV)


scale-upand distribution&Secondary

Manufacturing

Increase clinical trials in Queensland Facilities

Increase Scale-Up Capability in State

Expand contract manufacturing capability

HOW ?

Queensland Clinical Trials Network Inc.Queensland Clinical Trials Network Inc.

Queensland Government established QCTN in 2005

QCTN i (1) i (2) CROQCTN is: (1) not a government entity, (2) not a CRO

QCTN is an independent industry body with members being preclinical and clinical R&D service providers including hospitals (“umbrella organisation”)

Consultancy Organisations

Regulators AUS: TGA/NHMRC USA: FDA, EU: EMEAJPN: PMDA

Preclinical Research

Sponsor (Pharma, Biotech, Medical Device Companies ) Medical Research Institutes Scale-Up

Clinical Research Organisations (CRO’s)

Analytical & Pathology Labs

Hospitals

Aims of QCTNQ

Matching demand & supply for research services

• Assist national and international bio-pharmaceutical and medical device companies to undertake preclinical and clinical research activities in Australia (one-stop shop concept)concept)

Help build capabilities of QCTN members

• Know-how (Education, Courses, Workshops)

• Process optimization (IRB process, IT systems, Clinical Research)

• Communication and Facilitation (Members, Regulators, Governments, Industry bodies, Networks)

• Marketing & promotion (Conferences, Trade shows)

Facilitate collaboration between public and private p porganisations involved in (pre)clinical research

Research Capabilities of QCTN Members

QCTN Members cover all aspects of drug development• Clinical Network Services CRO• Q-Pharm Phase I-II Unit

l l l• ERA Consulting Regulatory Consultancy• TetraQ Preclinical Drug Profiling• The Wesley Research Institute Clinical Trials Centre• Mater Medical Research Institute Medical Research Institute• Queensland Institute of Medical Research Medical Research Institute• Eskitis Inst. for Cell & Molecular Therapies Discovery Research• Genomic Research Centre Genomics/Med. Res. Centre • IMBcom Commercialisation• IMBcom Commercialisation

Please visit:Please visit:www.qctn.com.au/

members

Example: BIO 2008 – June – San Diego

10 QCTN Members attended• BioAssayLink, BPA, CNS, InvestBrisbane, Emphron,

Perkins, QIMR, Q-Pharm, TetraQ, and WRIThree QCTN Sessions• Clinical & Regulatory sessions: 7 speakersg y p• QCTN session at trade floor: 4 speakers• Speakers from AUS, Taiwan, China, Japan (PMDA)

Outline






Regulatory Pathways

Regulatory processes in the major markets are well-defined for starting clinical trials

g y y

g

USA – the investigational new drug (IND) application

Europe – the clinical trial application (CTA)

Good Clinical Practice standards for conducting clinical research under ICH are appliedresearch under ICH are applied

However, the requirements and timeframes for applications to start trials are not the same in Europe vs the USA vs Australia vs JapanEurope vs. the USA vs. Australia vs. Japan

Comparison of the regulatory requirements for data and time needed for review of documentation is important i d fi i li i l d l t t t d h iin defining a clinical development strategy and choosing clinical sites

Australian Regulation and Clinical Trials

Australian Regulatory Authority for regulating Medicines and Medical Devices is called TGA• Therapeutics Goods Administration (TGA)p ( )• Australian regulations consistent with ICH

There are two pathways or schemes for gettingThere are two pathways or schemes for getting Regulatory Approval to start Clinical Trials:• CTN: Clinical Trial Notification

CTX Cli i l T i l E ti• CTX: Clinical Trial Exemption

Australian Clinical Trial Approvals (1)

Clinical Trial Notification Scheme (CTN)

Applies to > 95% of clinical studies in Australia

pp ( )

Applies to > 95% of clinical studies in Australia

Based on approval by ethics committee, supported by scientific advisory groups

Agency (TGA) notified, may request protocol, no formal review

F d t ti l ( t bl ) i k tFor products representing low (acceptable) risk to patients, including small molecules and some biologicals

Can cover studies from Phase I – Phase III

Available to INDUSTRY, not just academia

No similar pathway exists in the USA or in Europe, where every clinical trial must undergo regulatory review

Australian Clinical Trial Approvals (2)

Clinical Trial Exemption Scheme (CTX)

Requires TGA approval of application containing

pp ( )

Requires TGA approval of application containing summary CMC, non-clinical and clinical data

50 working days for TGA review, questions can be k dasked

For products representing high risk to patients, including many high-tech products such as cell or g y g pgene therapies

Requires ethics committee approval

Data requirements and submission content similar to the CTA in Europe, where review times vary from about 30-180+ days

CTN and CTX schemes for getting Regulatory Approval to start Clinical TrialsApproval to start Clinical Trials

Features CTN CTX

TGA Review Not required Yes

Ethics/IRB approval Yes Yes

Timelines 7 to 10 * 50Timelines(working days)

7 to 10 * 50

Fees $260 (AUD) $15,300 (AUD)

Approval Process Notification of HREC (=IRB)

approval needs to be obtained and

TGA reviews and issues letter of no

objection

submitted to TGA

* This means that from a regulatory standpoint a trial can in principle start 7-10 working days after the approval of the HREC ( IRB) t t th TGA Th CTN h i f ll d f(~ IRB) was sent to the TGA. The CTN scheme is followed for >95% of all trials because it is fast!

Comparing Clinical Trial Applications

Data requirements for an application to start a trial –

p g pp

sufficient for an Investigator’s Brochure, at minimum

Approximate order for the amount of information and data required:data equ ed

CTN << CTA (Europe) < CTX < IND

Timeframes favour the CTN process which depends onTimeframes favour the CTN process, which depends on the ethics committee supported by its scientific advisory groups

Regulatory review of an IND submission is 30 calendarRegulatory review of an IND submission is 30 calendar days, therefore less than a CTA or CTX, but a deficient IND application is put on HOLD (which has other disadvantages)g )

Outline






Australia was ranked first for Clinical Trials*

Singapore

USA

Australia Aggregate Benchmark Scores

India

UK

Singapore

Japan

Germany

India

0 2 4 6 8 10• Low average costs of clinical trials

Large amount of qualified trial sites• Large amount of qualified trial sites• High % of clinical trials completed within allocated time• Highly skilled population

Si ifi t t ti l f li i l t i l• Significant potential for more clinical trials* Source: The Economist Intelligence Unit Report “Benchmarking Study of the Characteristics of the Australian and International Pharmaceutical Industries”, September 2005

The Australian Phase 1 Industry (1/2)

Revenue

AUD 50 Milli• > AUD 50 Million p.a.

Facilities• ~ 170 beds in specialized, dedicated Phase 1 units p ,• All located within or adjacent to major tertiary

teaching hospitals • Most with access to specialized services andMost with access to specialized services and

emergency support

Business Sources75 80% b i f US E J• 75-80% business from US, Europe, Japan

• ~50% repeat business• Diverse client base

• Major Pharma – both innovator and generic• Small to Mid-Pharma and Biotech

The Australian Phase 1 Industry (2/2)Services• Phase 1 to Phase 3 but mainly Phase 1-2A• Full and niche service providers: Planning/ Reg.Affairs/ p g g

Study Conduct/Analytical Services/Reports• Special services

• Biomarkersd l d• Medical Imaging, PET studies

• Ethno pharmacology• Ambulatory cardiac telemetry

Conduct 140 clinical studies per year (90% Phase 1 2)• Conduct ~140 clinical studies per year (90% Phase 1-2)

Competitive AdvantageC t l t t (CTN/CTX)• Current regulatory system (CTN/CTX)• Open, safe, transparent, efficient

• Quality of workSt d d f d di l i f t t• Standards of care and medical infrastructure

• Cost effective compared to US/Western Europe

TetraQ Profile

Highly skilled scientific & commercial team

Long track record in providing tailored preclinical

Q

• Long track record in providing tailored preclinical solutions for biotechnology clients

Aim to provide interpretation and guidance, not just data

Quality science behind all services (no short-cuts)

Leaders have wealth of experience in all 4 preclinical d d l t di i lidrug development disciplines

Drug discovery

Preclinical drug

developmentPhase I

clinical trialsPhase II

clinical trialsPhase III

clinical trials

Efficacy ToxicologyIntegrated Integrated t d

ADME Pharmaceutics

reports and advice

reports and advice

TetraQ Profile: ServicesQ

ADME Pharmaceutics• bioanalytical services • In vitro metabolism

studies & metabolite identification

• Physicochemical characterization

• Formulation studies • Biowaiver studiesidentification

• plasma protein binding• Pharmacokinetic &

biodistribution studies

• Biowaiver studies

Toxicology• Cytotoxicity

Efficacy• Specialize in ‘proof-of-

concept’ studies in

y yassessment

• Genotoxicity• Acute & repeat dose

toxicity studiesconcept studies in animal models of human disease:

• Pain (specialism), CNS

toxicity studies• Safety pharmacology

Program Management models, diabetes, obesity

g gServices

Q-Pharm - LocationBrisbane Queensland Specialists in early phase clinical trialsBrisbane, Queensland- Royal Brisbane & Women’s Hospital Complex

Co located within

Specialists in early phase clinical trials• Successful FDA Inspection 2007 &

other inspections by Regulators and Sponsors

- Co-located within Queensland Institute of Medical Research

• Phase I/II, Pharmacokinetic and Pharmacodynamic studies

• Proof of Concept studies• Bio-Equivalence/Bio-Availability• Bio Equivalence/Bio Availability• Phase 0/Microdosing capability• Japanese-Caucasian bridging studies

New building under constructionNew building under construction

Clinic ( D Floor)

Mervyn Eadie Clinic, Outpatients Clinic

Q-Pharm – Early Phase Clinics

• 48 bedsH it l ICU/E A• Hospital ICU/Emergency Access

• Secure Pharmacy• PC2 Laboratory• Telemetry• Telemetry• Freezer/Fridge room• 4 Medical Officers• 50 Clinic/Lab/Data personnel50 Clinic/Lab/Data personnel • >70 Casual Nurses• Volunteer Database

CNS Profile(Clinical Network Services Pty Ltd Founded in 1998)(Clinical Network Services Pty Ltd – Founded in 1998)

Clinical Management support services • Full-service CRO for Australia and New Zealand• Early phase clinical development of products is >70% of

work• Late phase clinical development for big Pharma (incl.

Japanese pharma)• Queensland’s leading educator of research professionalsQ g p

National and international customersh d l & h ( )• Biotech, Medical Device & Pharma (US, EU, AUS, Japan)

• Nutraceutical (China & AUS)

Experienced, multinational team

ERA Profile

ERA offers strategic, scientific and regulatory consulting for human medicinesconsulting for human medicines

Due diligence for regulatory milestones

Clinical trial applications for Australia, Europe and the USA

Product development advicep

Review of quality (CMC), non-clinical and clinical documents for regulatory compliance

Regulatory Agency meetings

Experience with over 300 biotech/high-tech products

QCTN has observed a trend among (VC-backed) American & European Biotechs

Asia JPEU USA

AUS

Politically stable, safe, legal and regulatory robustPolitically stable, safe, legal and regulatory robustHigh quality, experienced western medical expertise Quick start-up of clinical trials is possible

QCTN has observed a trend among (VC-backed) American & European Biotechs G t E l R&D R lt i A t liGet Early R&D Results via Australia

Enables Early Go/No Go Decision-Making

Asia JPEU USA

Quality Data/Quality Data/Speed

AUSPre-clinical & Phase I / II Program

SpeedSpeed

Pre-clinical & Phase I / II Program g

Politically stable, safe, legal and regulatory robust

Program

Politically stable, safe, legal and regulatory robustHigh quality, experienced western medical expertise Quick start-up of clinical trials is possible

Also an Opportunity for Asian Bio-Pharmaceutical Companies:

G t E l R&D R lt i A t liGet Early R&D Results via Australia Enables Early Go/No Go Decision-Making

Asia JPEU USA

Quality Data/Pre-clinical & Phase I / II Program

AUS

SpeedProgram

Politically stable, safe, legal and regulatory robustPolitically stable, safe, legal and regulatory robustHigh quality, experienced western medical expertise Quick start-up of clinical trials is possible

Example how QCTN and its members can assist a Bio Pharma Companyassist a Bio-Pharma Company

Contract Manufacture

Preclinical drug Phase I Phase II Phase III R&D

GLP/GMP drug development clinical trials clinical trials clinical trials

QCTN’s role is

EskitisBPA

TetraQ

Q-Pharm

WRI AKTN d th

QCTN s role is that of facilitator during the exploratory stage between Ph d WRI, AKTN and other

QCTN-membersPharma and QCTN’s members and therefore QCTN provides a “window

CNS: Act as sponsor on behalf of Pharma and as CRO focusing on Project Management, Interaction with IRB/HREC and TGA, Clinical Monitoring, Window person for Pharma; Additional services: Protocol Writing CRF

person” until the time a contract is in place

person for Pharma; Additional services: Protocol Writing, CRF design, Data Management, Biostatistics, Report writing

ERA: Regulatory advice concerning Australian trial program in relation to accessing USA and EU markets

Outline






Final ConsiderationsFast start - for most trials no need for an approval process (i.e. via the CTN process)

Rigorous review of gene therapy and related th t di b A t li th iti

Use Australia for early phase clinical trials as a stepping stone for starting trials in US/EU

therapy studies by Australian authorities

Highly skilled clinical staff (ICH-GCP) + Very modern facilities + Strong IP protection + Safe environment

Use Australia as part of Multinational clinical trials for US or EU markets

ICH-GCP mandatory for all trials Necessary to arrange local (=Australian)ICH GCP mandatory for all trials Necessary to arrange local (=Australian) legal representation (“sponsorship”) and insurance

Cost-effective as compared to US, EU, JPN Consider doing Bridging trials in Australia for Asian markets

High % on-time completion of trials and there is capacity to do more trials

Need for multiple ethics committee approvals for multi-centre trials in some states in Australia

Multicultural population = 21 million, mainly Caucasian and Asian

Possibility of continuing seasonal studies in AUS during off-season in the Northern Caucas a a d s a US du g o seaso t e o t eHemisphere

Excellent network of preclinical, genomic & bioinformatics and early phase clinical trials service providers in Queensland (Brisbane)

Manage perceived distance and time differences (for EU and US) via good project management

Ready access to Comparator drugs, Background therapy, Standards of care, Disease tracking programs for notifiable disease outbreaks and Flu monitoring network

Medical-technical documentation such as Case Report Forms, Informed Consent Forms, etc. are all in English + recognizable units & abbreviations

SummaryAustralia represents a small bio pharmaceutical marketAustralia represents a small bio-pharmaceutical market from a sales perspective, but a global player in the product development pipeline• Robust but efficient regulatory environment• Robust but efficient regulatory environment

• Cost effective

In the past decade, Government policies and funding in basic research, commercialisation have created an entrepreneurial climate & attracted venture-capital => Results:Results:• A very active biopharma/medical device industry

• A modern Australian contract research services industry

hi h li ff i i• A high quality, cost effective, stepping stone

QCTN can introduce your company to organisations providing world-class preclinical and clinical research services in Australia and assist in accelerating new product development

Meet us at:

DIA Euromeeting – Berlin - Next Week

BIO – Atlanta -May

CALAsia – San Diego – June

DIA – San Diego – June

Bio Korea – Seoul – Septemberp

Bio Japan – Yokohama – October

TRX-09 – Brisbane – OctoberTRX-09 – Brisbane – October

AusBiotech – Melbourne –October

S l bAAPS – Los Angeles - November

Visit: www.qctn.com.au/members

Introduction to R&D capabilities in Queensland, Australia Introduction March 2009.pdf · Europe vs...

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