innovation benefits and risks - pearrl
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Innovation – Benefits and
Risks
Regulatory Science Symposium – PEARRL Annual Meeting
21st June 2017
Una Moore,
Pharmaceutical Assessment Manager (Acting)
Health Products Regulatory Authority
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits and
Risks
Regulatory Science Symposium – PEARRL Annual Meeting
21st June 2017
Una Moore,
Pharmaceutical Assessment Manager (Acting)
Health Products Regulatory Authority
The contents of this presentation are personal opinions
and are not necessarily representative of the HPRA.
presented at the PEARRL Regulatory symposium 2017 – for personal use only
What is Innovation?
21/06/2017 3
Creates
value
Something
new
Brings constant
value to customer’s
business or life
New useful,
bettering
people’s lives
Turning an idea
into a solution
that adds value
Executing an idea -
addresses a specific
challenge and achieves
value for the company
and customer
presented at the PEARRL Regulatory symposium 2017 – for personal use only
What is Innovation?
21/06/2017 4
Creates
value
Something
new
Brings constant
value to customer’s
business or life
New, useful,
bettering
people’s lives
Turning an idea
into a solution
that adds value
Executing an idea -
addresses a specific
challenge and achieves
value for the company
and customer
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits and
Risks
5presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation - Benefits &
Risks
6
1950 - Polio vaccines - live attenuated vaccines (Koprowski).
1953 – Mass vaccination with inactivated vaccine (John Elders, Salk and Sabin)
1963 - Measles vaccine (John
Enders)
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation - Benefits &
Risks
7
1950 - Polio vaccines - live attenuated vaccines (Koprowski).
1953 – mass vaccination with inactivated vaccine (John Elders, Salk and Sabin)
1963 - Measles vaccine (John
Enders)
Vaccination – most effective global
public health intervention after
provision of clean water.
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits &
Risks
8
• 1955 – polio vaccines contained live virus – 250 cases of polio
• 1955 – 1963 – polio vaccine contaminated with SV40.
• Theoretical cancer risk
• Swine flu pandemic (2009)
• Associated with the H1N1 virus
• Second pandemic withthis virus.
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits &
Risks
9
• Swine flu pandemic (2009)
Pandemic declared 11th June 2009presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits &
Risks
10
• Swine flu pandemic (2009)
Pandemic declared 11th June 2009
Mortality estimates
Initially 18,000…
Revised to 284,500
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation Benefits &
Risks
Biotech Products
11presented at the PEARRL Regulatory symposium 2017 – for personal use only
12presented at the PEARRL Regulatory symposium 2017 – for personal use only
Immunogenicity
• Immune responses to therapeutic protein products
may pose problems for both patient safety and
product efficacy.
• Anaphylaxis - can have a fatal outcome unless
treated
• Cytokine release syndrome
• Neutralisation of product
13
Innovation Benefits &
Risks
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation Benefits &
Risks• Cetuximab (Erbitux):
• Chimeric IgG antibody; produced in the mouse cell line Sp2/0
• Indications:
• EGFR receptor expressing colorectal cancer
• Squamous cell cancer of head and neck
• Anaphylaxis in some patients – about 1 to 3%
• Severe anaphylaxis in Southern
U.S as high as 22%
14presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation Benefits &
Risks
• Identified that the reaction was IgE-mediated.
• Anaphylaxis due to pre-existing IgE to Galactose-
alpha-1,3-galactose [Alpha-gal]
• Sugar is not found in humans; present on
cetuximab
• How?
• Linked to bites from the Lone
Star Tick
15presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits & Risks
TeGenero mAB (TNG1412) trial
• Immunomodulatory drug – treatment of B
cell leukaemia
• CD28 Agonist
• <500 times the dose found safe in non-
human primates
• Phase I studies - Induced severe inflammatory
reactions
• Catastrophic systemic organ failure (cytokine
storm)
16
• Unforeseen biological action in humans - no obvious errors
• Several proposals e.g. Extracellular domain only 96%
homology, lower CD28 expression on the CD4+ memory T-
cells in non-human primates.presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation - Benefits &
Risks
• Advancements in Oncology
• Cancer Immunotherapy – check point targets
17presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation - Benefits &
Risks
• Cancer Immunotherapies – check point targets
18presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation – Benefits &
Risks
19
Immunotherapy
PD-L =programmed cell death ligand;
Supresses the immune system -presented at the PEARRL Regulatory symposium 2017 – for personal use only
Innovation - Benefits &
Risks
20
Immunotherapy
PD-L =programmed cell death ligand;
Supresses the immune system -Sznol M., Chen L., Clin. Cancer Res.
2013; 1021 - 1034
presented at the PEARRL Regulatory symposium 2017 – for personal use only
EU Approved Checkpoint
Immunotherapies
21
2011:
Yervoy (Ipilimumab) – advanced melanoma
Binds to the CTLA-4 receptor
2015:
Opdivo (nivolumab) – melanoma, NSCLC, renal carcinoma
and Hodgkin’s lymphoma
Keytruda (Pembrolizumab) - melanoma, NSCLC and
Hodgkin’s lymphoma
Bind to the PD-1 receptor
2016:
Avelumab – indicated for gastric cancer
Binds to the PD-L1 receptor
Additional
Monitoring
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Ultra-rare disease
22
Gene therapy
Microbiome therapies
Immunotherapy and
cancer vaccines
Personalised medicine
Drug/device combinations
Gene editing
Veterinary
biologicals
Regenerative medicine
presented at the PEARRL Regulatory symposium 2017 – for personal use only
How does HPRA support
Innovation in the Biological Area?
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Developing the HPRA
Knowledge Base in Biopharma
24
• HPRA - a centre of excellence within the EU in the
biological/ advanced therapy space
• Why?
• Supporting innovation through assessment
work at the EMA network and preparation of
guidance documents
• Supporting the Biotech industry within Ireland
which is one of Ireland’s major growth
industries
• Providing expert guidance in the area of
biological medicines to Irish healthcare
providers and the Irish public
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Developing the HPRA
Knowledge Base in Biopharma
• Established a cross
organisational WG
• Identified areas of existing expertise
• Horizon scanning/liaising other
government organisations –
identified innovative areas of the
future
• Identified what actions required
• Training
• External experts
• What collaborations required with Irish
organisation and other EU agencies
25
Biological
strategy
Compliance
Quality
(Human
and Vet)
PV
Clinical/
Preclinical
(Human
and Vet)
presented at the PEARRL Regulatory symposium 2017 – for personal use only
The Biological Strategy
26
ATMPs
Antibody Drug
Conjugates
Botulinum toxins
mAbs
Heparins
Biosimilars
Veterinary
biologicals
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Progress to date
• HPRA acting as Rapp/Co-Rapp for new product applications
• HPRA contributing to the assessment of scientific advice
applications
• HPRA acting as Ref-NCA for clinical trials assessments
• HPRA assessed the first heparin biosimilar via Centralised
Procedure
• HPRA takes a lead EU role in assessment of botulinum products
• HPRA assessed the centralised application for the first antibody
for veterinary use
• HPRA is actively involved in revising the EMA clinical trial
guideline ‘Requirements for quality documentation concerning
biological investigational medicinal products in clinical trials’
• HPRA is recognised as expert in assessment of biosimilars
27presented at the PEARRL Regulatory symposium 2017 – for personal use only
Conclusion
• Innovative technologies - huge benefits
• Safe and timely access to patients
• Benefits > Risks
• HPRA is supporting innovation in the biological
space
• HPRA is planning to extend our assessment
activities into the ATMP (gene, cell and
regenerative therapies)
21/06/2017 28
Innovation – Benefits &
Risks
presented at the PEARRL Regulatory symposium 2017 – for personal use only
21/06/2017 29presented at the PEARRL Regulatory symposium 2017 – for personal use only
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