Transcript Document

Pat O’Mahony
Chief Executive
Irish Medicines Board
Bringing the best to healthcare
through partnership and innovation
IPHA, 1st December 2011
Encouraging innovation: the
regulators perspective
Slide 1
Some Key Challenges/Opportunities and Current
Shared Topics
 Balance of benefit and risk (harm) – Pharmacovigilance
Regulations
 New science and new therapies and Need to improve
research and development and regulatory processes
 Globalisation of the market – International regulatory
collaboration
 Demographics, disease burden and unmet needs Scarce resources for health budgets – HTA?
 Medicines Availability
 Transparency, communication & information –
Patient Engagement - Prescription Status
Consultative Panel
Slide 2
New Science and New Therapies
 New science and new therapies and Need to improve
research and development and regulatory processes
 Biomarkers – Critical Markers of Disease
 Surrogate endpoints
 Personalised Medicines
 Nanotechnology - EU has decided on a definition of a
nanoparticle - first region to do so
Slide 3
EMA Qualification of Novel
Methodologies for Drug Development (1/8)
 Common understanding important
•
Scientists in universities, consortia and companies
need to discuss with regulators early to achieve a
common understanding on what is needed to qualify a
novel methodology also in terms of regulatory
approval
•
The regulatory requirements should be up to date with
the progress in science
•
Question: Can this novel methodology be used as
inclusion criterion or as an endpoint in a clinical trial?
Slide 4
EMA Qualification of Novel
Methodologies for Drug Development (2/8)
 Biomarkers – Briefing meetings since 2001 with EMA
Innovation Task Force and the CHMP
Pharmacogenomics Working Party
 Novel methodologies – CHMP qualification
•
June 2008: draft EMA guidance published for
consultation on the qualification of novel
methodologies including biomarkers, imaging
techniques, new non-clinical models, new statistical
approaches via the Scientific Advice Working party of
the CHMP
• January 2009: Final guidance published
Slide 5
EMA Qualification of Novel
Methodologies for Drug Development (3/8)
 Qualification Advice and Qualification Opinion
•
CHMP Qualification Advice on future studies and
methodologies for further method development
towards qualification, based on the evaluation of the
scientific rationale and on preliminary data submitted.
•
CHMP Qualification Opinion on the acceptability of a
specific use of the proposed novel method in
non-clinical or clinical studies, based on the
assessment of submitted data obtained in studies
already performed, not necessarily specific to one
product.
Slide 6
EMA Qualification of Novel
Methodologies for Drug Development (4/8)
 Qualification team
•
•
One Coordinator (SAWP or CHMP member)
•
One EMA Scientific Administrator
Minimum 4 experts, one a statistician (not bound to
Coordinator country). We target inclusion of at least
one external to the regulatory system, a key opinion
leader
Slide 7
EMA Qualification of Novel
Methodologies for Drug Development (5/8)
 Other regulatory agencies
•
Applicant is encouraged to apply in parallel to the EMA
and FDA. The confidentiality agreement between the
FDA and EMA makes it possible to have the procedure
ongoing at the same time in both agencies.
•
The agencies will communicate the assessment and
meet with the Applicant together.
•
It is envisaged to reach the same conclusions and
experience so far confirms this.
• Applicant still has the choice to apply to one
agency only.
Slide 8
EMA Qualification of Novel
Methodologies for Drug Development (6/8)
 Today and the future
•
•
21 procedures started
2 Qualification Opinions finalised (public after
agreement with the sponsor at www.ema.europa.eu)
 One non-clinical: biomarkers for nephrotoxicity.
 One clinical: biomarkers in the cerebrospinal fluid
as inclusion criteria for clinical trials in early
Alzheimer’s disease.
•
4 Qualification Advices finalised (confidential to
Applicants).
Slide 9
EMA Qualification of Novel
Methodologies for Drug Development (7/8)
 What is new? – What is the ultimate goal?
•
Scientific Advice Working Party / CHMP early involvement
in the design of the strategy towards qualification of novel
methodologies.
•
Scientific Advice Working Party / CHMP commitment to
evaluate the data obtained from the agreed studies and to
provide a Qualification Opinion regarding the use of the
method in R&D.
•
Qualification Opinion part of the future Marketing
Authorisation Application to support use of the novel
methodology in the context of the development of a new
product.
•
Goal: speed up drug development, contribute to
public health.
Slide 10
EMA Qualification of Novel
Methodologies for Drug Development (8/8)
 Reference document:
•
Review titled New pathway for qualification of novel
methodologies in the European Medicines Agency
Authors:
Efthymios Manolis, Spiros Vamvakas and Maria Issac (European
Medicines Agency, London)
Published in Journal Proteomics Clinical Applications. 2011, 5 ,1-8
Slide 11
Conference
Critical Markers of Disease
(CMOD)
May 1st and 2nd 2012
Slide 12
New Science and New Therapies
• Advanced Therapies
• Much happening on regulatory front
• IMB is running a joint scientific conference with PDA
(Parenteral Drug Association) Ireland Chapter, on Advanced
Therapy Medicinal Products in July 2012, entitled “Making
gene and cell therapy a reality” (subtitle “An efficient
partnership between authorities and industry in Europe”)
• The conference will run for two days from July 10th -11th
2012, and will form a satellite event immediately before the
prestigious ESOF 2012 multidisciplinary scientific congress
on 11th - 15th July
• ESOF( European Science Open Forum) is the
centerpiece of Dublin being designated as
European City of Science for 2012
Slide 13
Some Key Challenges/Opportunities and Current
Shared Topics
 Globalisation of the market - International regulatory
collaboration
 How to ensure both strength and depth of expertise in
regulators and efficient processes
 Various actions - ICH, Summit Themes (2011 - Performance,
Community Expectations, Transparency, Medical Devices,
HTA engagement)
 Demographics, disease burden and unmet needs Scarce resources for health budgets – HTA?
 Personalised Medicines and Drug device combination
products
 Medicines Availability
 Transparency, communication & information – Patient
Engagement - Prescription Status Consultative Panel
Slide 14
Pat O’Mahony
Chief Executive
Irish Medicines Board
Bringing the best to healthcare
through partnership and innovation
IPHA, 1st December 2011
Encouraging innovation: the
regulators perspective
THANK YOU!
Slide 15