European Parliament Advanced Therapies Hearing May 11, 2006 Advanced Therapies The proposed EU...

European Parliament Advanced Therapies Hearing May 11, 2006

Advanced TherapiesThe proposed EU regulation from the viewpoint of a Pharmaceutical company with experience in the field of Tissue Engineering

Priv.Doz.Dr,med.Detlef Niese Novartis Pharma AG, Basel CH European Biopharmaceutical Enterprises (EBE)Member of EuropaBio Advanced Therapies Working Group


About EBE

EBE – European Biopharmaceutical Enterprises is a specialised group of EFPIA created in 2000

EBE has 65 corporate members engaged in research in Europe involving the application of emerging bioscience technologies with the aim of launching new healthcare products


Advanced Therapy Products and Pharmaceutical Companies Academic institutions, small biotech

companies and some large pharmaceutical companies have been involved and are still involved in research and development of cell therapy, tissue engineering and gene therapy products.

Regulatory uncertainty and development risks associated with these products prove to be significant hurdles

As a pharmaceutical company, Novartis has gathered development experience in all sectors of „advanced therapies“


A Real Life Case: Apligraf

What it looks like Histology Apligraf Normal Skin


Apligraf® A Long Clinical & Regulatory Path

1979

E. BellE. BellM.I.T.M.I.T.

1985

OrganogenesisOrganogenesisformedformed

1995 1998 2000 2003

VSU-001VSU-001 DUS-001DUS-001

FDA FDA ApprovalApproval

Venous Leg Venous Leg UlcersUlcers

2004

FDA FDA ApprovalApproval

Diabetic Foot Diabetic Foot UlcersUlcers

1992 1996

FDA FDA Designated asDesignated as

device device class III class III

interactive interactive wound dressingwound dressing

I.D.E. I.D.E. submittedsubmitted

E.M.E.A.E.M.E.A.SubmissionSubmission

E.M.E.A.E.M.E.A.WithdrawalWithdrawal


A Diverse Regulatory Environment

Initial Regulatory Classifications

EventualRegulatory Classification

MedicinalProduct

Apligraf Regulatory

Device Medicinal Other Reg Unregulated

USA

Finland

Germany

Sweden

Norway

Spain

Portugal

Denmark

France (Cell Therapy)Belgium(TissueBank)

Greece

Switzerland (Transplant)

UK

Italy

Ireland

Austria

Netherlands

Canada

Apligraf

Device Medicinal Unregulated

USA

Finland

Germany

Sweden

Norway

Spain

Portugal

Denmark

France (Cell Therapy)Belgium(TissueBank)

Greece

Switzerland (Transplant)

UK

Italy

Ireland

Austria

Netherlands

Canada


Lessons Learned A harmonized regulatory framework for all

cell based products in Europe is urgently needed to address Quality assurance Development and Marketing authorisation

Address unique safety & efficacy issues Include efficacy assessments based on trial designs that

address ethical and practical aspects of surgical research Benefits of harmonised regulations

Protection of patient safety Encourage investment by industry and successful

global commercialisation through “level playing field”

Provide patients access to innovative & valuable therapies


How We See the Proposed Regulation – What We Welcome A single, harmonised regulatory framework for

all advanced therapy products Use of the Centralised Procedure through

EMEA ensuring harmonised efficacy, quality and safety requirements for all products

The creation of a Committee for Advanced Therapies consisting of experts in the field

The use of subsidiarity for ethical aspects such as embryonic stem cells


How We See the Proposed Regulation – Our Concerns To ensure safety and effectiveness of AT

products the regulation should apply to all products produced following a standardised process (no exemptions)

Instead of exemptions from the regulation, consider providing administrative support or regulatory expertise as an additional incentive for all manufacturers who may require it


How We See the Proposed Regulation – Our Concerns The formation of a specialised committee

(CAT) at EMEA is welcomed but Expertise of members should reflect the broad

spectrum of products and technologies Expertise of members should prevail over national

representation (which is provided by CHMP) Existing legislation needs adaptation to (e.g.

2001/20, GMP)


Conclusion The new regulation is principally welcome Assurance of safety and efficacy through

review by regulatory authorities supported by an expertise based CAT should drive development of high quality products and support access for patients

Instead of excluding specific products from the regulation, administrative hurdles for hospital based products or SMEs should be minimised and may require additional incentives

Careful adaptation of all affected existing legislation (like Directive 2001/20 EC) is a crucial success factor

European Parliament Advanced Therapies Hearing May 11, 2006 Advanced Therapies The proposed EU...

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Transcript of European Parliament Advanced Therapies Hearing May 11, 2006 Advanced Therapies The proposed EU...