Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical Manufacturing Hurdles Early
A presentation by Chief Operating Officer, Dr Stephen Ward
Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical Manufacturing Hurdles Early
A presentation by Chief Operating Officer, Dr Stephen Ward
The biotech industry is characterised by constant change and fast-paced growth. Keeping up with the rapid evolution is close to impossible. Nevertheless, that’s exactly what Niels Guldager, Senior Technology Partner within biopharmaceuticals, does best. In this slide series, he presents the top ten biotech trends as he sees them.
Over the last 8 years Scotland has made a very significant investment in building the human and physical infrastructure required to leverage our leading position in the field of stem cell biology to support the development of a thriving Scottish Regenerative Medicine industrial sector. This initial investment is nearing completion, has substantially delivered and has started to make a real impact. However its full value will only be realised as the world-wide regenerative medicine sector matures and near term scientific, technical and industrial impediments are overcome. Our competitors in other parts of the UK and across the world are continuing to invest in this space in order to imbue their nascent industries with comparative advantage.
With these considerations in mind the community has undertaken a review of the current state of play, in order to determine what now needs to be done in order to drive the growth of the Regenerative Medicine industry in Scotland and embed significant long-term benefits to the economy and health of the nation.
This document sets out a strategy which has been developed in collaboration with the academic, business and clinical communities. It is intended for high-level discussion and to provide a framework for future collective direction and investment.
Business Transformation and Partnering Strategies to Accelerate Commercialisa...Oxford Global
Magda Papadaki PhD, former Head of Manufacturing & Innovation, Association of the British Pharmaceutical Industry (ABPI) and current Associate Director, MSD shares her recent presentation at London's Cell & Gene Therapy Congress 2018, “Business Transformation and Partnering Strategies to Accelerate Commercialisation of Cell & Gene Therapies.”
The presentation includes key information and insights into:
- New Pathways to CGT commercialisation and clinical use
- System Readiness: New Pathways & Collaborations
- Innovating how we Innovate to Deliver Cures.
More information about the 2019 Cell & Gene Therapy Congress can be found here: www.celltherapy-congress.com
Pharma IQ brings you Clinical Trial Supply Europe Conference Profit. Successfully cutting costs and overages whilst increasing the flexibility and reactivity of your clinical supply network to support global clinical trials.
Prof Angela Timoney
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Dr Carol Clugston from the University of Glasgow presents at the University's Commonwealth Future Cities Business Networking event on the 24th July 2014
Carole Jones & Gopalan Narayanan share their insights on commercialization of Advanced Therapy Medicinal Products (ATMPs) at the Market Access for Cell and Gene Therapies Conference on October 19th 2017.
The biotech industry is characterised by constant change and fast-paced growth. Keeping up with the rapid evolution is close to impossible. Nevertheless, that’s exactly what Niels Guldager, Senior Technology Partner within biopharmaceuticals, does best. In this slide series, he presents the top ten biotech trends as he sees them.
Over the last 8 years Scotland has made a very significant investment in building the human and physical infrastructure required to leverage our leading position in the field of stem cell biology to support the development of a thriving Scottish Regenerative Medicine industrial sector. This initial investment is nearing completion, has substantially delivered and has started to make a real impact. However its full value will only be realised as the world-wide regenerative medicine sector matures and near term scientific, technical and industrial impediments are overcome. Our competitors in other parts of the UK and across the world are continuing to invest in this space in order to imbue their nascent industries with comparative advantage.
With these considerations in mind the community has undertaken a review of the current state of play, in order to determine what now needs to be done in order to drive the growth of the Regenerative Medicine industry in Scotland and embed significant long-term benefits to the economy and health of the nation.
This document sets out a strategy which has been developed in collaboration with the academic, business and clinical communities. It is intended for high-level discussion and to provide a framework for future collective direction and investment.
Business Transformation and Partnering Strategies to Accelerate Commercialisa...Oxford Global
Magda Papadaki PhD, former Head of Manufacturing & Innovation, Association of the British Pharmaceutical Industry (ABPI) and current Associate Director, MSD shares her recent presentation at London's Cell & Gene Therapy Congress 2018, “Business Transformation and Partnering Strategies to Accelerate Commercialisation of Cell & Gene Therapies.”
The presentation includes key information and insights into:
- New Pathways to CGT commercialisation and clinical use
- System Readiness: New Pathways & Collaborations
- Innovating how we Innovate to Deliver Cures.
More information about the 2019 Cell & Gene Therapy Congress can be found here: www.celltherapy-congress.com
Pharma IQ brings you Clinical Trial Supply Europe Conference Profit. Successfully cutting costs and overages whilst increasing the flexibility and reactivity of your clinical supply network to support global clinical trials.
Prof Angela Timoney
Presentation at EIPG - Royal Pharmaceutical Society Scientific Symposium "Advances in Technology Impacting the Pharmaceutical Industry" at the University of Strathclyde, Glasgow 2015.
Dr Carol Clugston from the University of Glasgow presents at the University's Commonwealth Future Cities Business Networking event on the 24th July 2014
Carole Jones & Gopalan Narayanan share their insights on commercialization of Advanced Therapy Medicinal Products (ATMPs) at the Market Access for Cell and Gene Therapies Conference on October 19th 2017.
CAR-T (Cell Therapy) Nomenclature Review & Brand Equity Study. April 15, 2015Bill Smith
CAR-T (Cell Therapy) Nomenclature Review & Brand Equity Study. April 15, 2015.
Brand Acumen. The Global Leader in Pharmaceutical Name Development and Submission Strategy.
Process development considerations for quality and safety of vaccinesDr. Priyabrata Pattnaik
"New Technologies Symposium" presentation at Annual General Meeting of Developing Country Vaccine Manufacturers Network (DCVMN), 5th-7th October 2015, Bangkok, Thailand.
Production and purification of Viral vectors for gene and cell therapy appli...Dr. Priyabrata Pattnaik
Presentation at "2016 Osong BioExcellence - Renaissance in Immunotherapy" at South Korea, an event jointly hosted by Kbio Health and Merck on 6th October 2016.
Chris Cleary, Vice President of Corporate Development, Medtronic presented at MEDTECH 2014 on Fulfilling the Mission: Medtronic Embraces Change to Respond to an Evolving Healthcare Landscape.
About UCI Applied Innovation:
UCI Applied Innovation is a dynamic, innovative central platform for the UCI campus, entrepreneurs, inventors, the business community and investors to collaborate and move UCI research from lab to market.
About the Cove @ UCI:
To accelerate collaboration by better connecting innovation partners in Orange County, UCI Applied Innovation created the Cove, a physical, state-of-the-art hub for entrepreneurs to gather and navigate the resources available both on and off campus. The Cove is headquarters for UCI Applied Innovation, as well as houses several ecosystem partners including incubators, accelerators, angel investors, venture capitalists, mentors and legal experts.
Follow us on social media:
Facebook: @UCICove
Twitter: @UCICove
Instagram: @UCICove
LinkedIn: @UCIAppliedInnovation
For more information:
cove@uci.edu
http://innovation.uci.edu/
R&D, Competition and Diffusion of Innovation in EU: The Case of Direct Acting...Office of Health Economics
An OHE research studies the role of R&D incentives and market competition in facilitating the diffusion of pharmaceutical innovation across European countries. The research studies the case of the DAA treatments for hepatitis C. Methods and results were presented and discussed at EUHEA conference 2018 held in Maastricht, The Netherlands, 11-14 July. The analysis concludes that (i) R&D incentives may have encouraged in-patent competition of DAAs and (ii) competition had a positive impact on uptake and adoption of DAA treatments in the countries studied (top-5 European plus Portugal).
Author(s) and affiliation(s): Mikel Berdud (OHE), Phill O'Neill (OHE), Martina Garau (OHE), Adrian Towse (OHE)
Conference/meeting: EUHEA Conference 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
Karen Livingstone - ECO 17: Transforming care through digital healthInnovation Agency
Presentation by Karen Livingstone, Director of Innovation Exchange and SBRI Healthcare, NHS England: Transforming Health and Social Care Services - The Innovation Exchange and SBRI Healthcare at ECO 17: Transforming care through digital health on Tuesday 4 December at Lancaster University, Lancaster
R&D, Competition and Diffusion of Innovation in EU: The Case of Direct Acting...Office of Health Economics
Mikel Berdud, PhD (Office of Health Economics) discussed on the role of incentives for Pharmaceutical innovation (with especial attention to IP rights) and in-patent competition on facilitating access to DAAs across European countries. Other factors affecting access to DAAs in European countries beyond incentives for R&D and market competition were also discussed in the presentation.
Author(s) and affiliation(s): Mikel Berdud (Office of Health Economics) Martina Garau (Office of Health Economics) Margherita Neri (Office of Health Economics) Phill O’Neill (Office of Health Economics) Chris Sampson (Office of Health Economics) Adrian Towse (Office of Health Economics)
Project funded by: Gilead Sciences Ltd.
Conference meeting: XXXVII Jornadas de Economía de la Salud
Location: Auditorio Alfredo Kraus, Las Palmas de Gran Canaria, Canarias, Spain
Date: 20/06/2018
Integrate RWE into clinical developmentIMSHealthRWES
With greater application of RWE throughout the pharmaceutical
lifecycle, learnings are emerging that offer guidance for
approaches to derive the maximum value. This article captures
the author’s experience at a leading international biotech, with
insights for smoothing RWE assimilation into clinical
development and realizing the benefits it brings.
Commercial considerations in early drug developmentSunil Ramkali
It is important in the drug development process that marketers and researchers collaborate early to ensure that products being developed are truly innovative and deliver brand value to the different end users in a way that the product and the subsequent brand messaging is relevant, compelling and differentiating compared to the competition. T
In the market place that is heavily cost constraint, innovation is no longer about a unique mode of action or a new formulation, but more about the incremental brand value offered by new pharmaceutical products over existing treatments (standard of care) and how much healthcare systems are prepared to pay for these incremental benefits. My lecture at the Department of Innovation, Lund University, Sweden explored the importance of R&D functions getter closer to external stakeholders to really understand their needs, how they define brand value and the importance of considering this early in the drug development process.
Making Value-Based Healthcare in Cataract a Reality Insights from VBHCAT Pr...Alexandre Lourenço
Alexandre Lourenço's keynote on "Making Value-Based Healthcare in Cataract a Reality - Insights from VBHCAT Project in Portugal", at the 44th World Hospital Congress organized by the International Hospital Federation, in November 8th 2021.
Ethanol (CH3CH2OH), or beverage alcohol, is a two-carbon alcohol
that is rapidly distributed in the body and brain. Ethanol alters many
neurochemical systems and has rewarding and addictive properties. It
is the oldest recreational drug and likely contributes to more morbidity,
mortality, and public health costs than all illicit drugs combined. The
5th edition of the Diagnostic and Statistical Manual of Mental Disorders
(DSM-5) integrates alcohol abuse and alcohol dependence into a single
disorder called alcohol use disorder (AUD), with mild, moderate,
and severe subclassifications (American Psychiatric Association, 2013).
In the DSM-5, all types of substance abuse and dependence have been
combined into a single substance use disorder (SUD) on a continuum
from mild to severe. A diagnosis of AUD requires that at least two of
the 11 DSM-5 behaviors be present within a 12-month period (mild
AUD: 2–3 criteria; moderate AUD: 4–5 criteria; severe AUD: 6–11 criteria).
The four main behavioral effects of AUD are impaired control over
drinking, negative social consequences, risky use, and altered physiological
effects (tolerance, withdrawal). This chapter presents an overview
of the prevalence and harmful consequences of AUD in the U.S.,
the systemic nature of the disease, neurocircuitry and stages of AUD,
comorbidities, fetal alcohol spectrum disorders, genetic risk factors, and
pharmacotherapies for AUD.
Flu Vaccine Alert in Bangalore Karnatakaaddon Scans
As flu season approaches, health officials in Bangalore, Karnataka, are urging residents to get their flu vaccinations. The seasonal flu, while common, can lead to severe health complications, particularly for vulnerable populations such as young children, the elderly, and those with underlying health conditions.
Dr. Vidisha Kumari, a leading epidemiologist in Bangalore, emphasizes the importance of getting vaccinated. "The flu vaccine is our best defense against the influenza virus. It not only protects individuals but also helps prevent the spread of the virus in our communities," he says.
This year, the flu season is expected to coincide with a potential increase in other respiratory illnesses. The Karnataka Health Department has launched an awareness campaign highlighting the significance of flu vaccinations. They have set up multiple vaccination centers across Bangalore, making it convenient for residents to receive their shots.
To encourage widespread vaccination, the government is also collaborating with local schools, workplaces, and community centers to facilitate vaccination drives. Special attention is being given to ensuring that the vaccine is accessible to all, including marginalized communities who may have limited access to healthcare.
Residents are reminded that the flu vaccine is safe and effective. Common side effects are mild and may include soreness at the injection site, mild fever, or muscle aches. These side effects are generally short-lived and far less severe than the flu itself.
Healthcare providers are also stressing the importance of continuing COVID-19 precautions. Wearing masks, practicing good hand hygiene, and maintaining social distancing are still crucial, especially in crowded places.
Protect yourself and your loved ones by getting vaccinated. Together, we can help keep Bangalore healthy and safe this flu season. For more information on vaccination centers and schedules, residents can visit the Karnataka Health Department’s official website or follow their social media pages.
Stay informed, stay safe, and get your flu shot today!
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdfAnujkumaranit
Artificial intelligence (AI) refers to the simulation of human intelligence processes by machines, especially computer systems. It encompasses tasks such as learning, reasoning, problem-solving, perception, and language understanding. AI technologies are revolutionizing various fields, from healthcare to finance, by enabling machines to perform tasks that typically require human intelligence.
Lung Cancer: Artificial Intelligence, Synergetics, Complex System Analysis, S...Oleg Kshivets
RESULTS: Overall life span (LS) was 2252.1±1742.5 days and cumulative 5-year survival (5YS) reached 73.2%, 10 years – 64.8%, 20 years – 42.5%. 513 LCP lived more than 5 years (LS=3124.6±1525.6 days), 148 LCP – more than 10 years (LS=5054.4±1504.1 days).199 LCP died because of LC (LS=562.7±374.5 days). 5YS of LCP after bi/lobectomies was significantly superior in comparison with LCP after pneumonectomies (78.1% vs.63.7%, P=0.00001 by log-rank test). AT significantly improved 5YS (66.3% vs. 34.8%) (P=0.00000 by log-rank test) only for LCP with N1-2. Cox modeling displayed that 5YS of LCP significantly depended on: phase transition (PT) early-invasive LC in terms of synergetics, PT N0—N12, cell ratio factors (ratio between cancer cells- CC and blood cells subpopulations), G1-3, histology, glucose, AT, blood cell circuit, prothrombin index, heparin tolerance, recalcification time (P=0.000-0.038). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and PT early-invasive LC (rank=1), PT N0—N12 (rank=2), thrombocytes/CC (3), erythrocytes/CC (4), eosinophils/CC (5), healthy cells/CC (6), lymphocytes/CC (7), segmented neutrophils/CC (8), stick neutrophils/CC (9), monocytes/CC (10); leucocytes/CC (11). Correct prediction of 5YS was 100% by neural networks computing (area under ROC curve=1.0; error=0.0).
CONCLUSIONS: 5YS of LCP after radical procedures significantly depended on: 1) PT early-invasive cancer; 2) PT N0--N12; 3) cell ratio factors; 4) blood cell circuit; 5) biochemical factors; 6) hemostasis system; 7) AT; 8) LC characteristics; 9) LC cell dynamics; 10) surgery type: lobectomy/pneumonectomy; 11) anthropometric data. Optimal diagnosis and treatment strategies for LC are: 1) screening and early detection of LC; 2) availability of experienced thoracic surgeons because of complexity of radical procedures; 3) aggressive en block surgery and adequate lymph node dissection for completeness; 4) precise prediction; 5) adjuvant chemoimmunoradiotherapy for LCP with unfavorable prognosis.
Report Back from SGO 2024: What’s the Latest in Cervical Cancer?bkling
Are you curious about what’s new in cervical cancer research or unsure what the findings mean? Join Dr. Emily Ko, a gynecologic oncologist at Penn Medicine, to learn about the latest updates from the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer. Dr. Ko will discuss what the research presented at the conference means for you and answer your questions about the new developments.
New Drug Discovery and Development .....NEHA GUPTA
The "New Drug Discovery and Development" process involves the identification, design, testing, and manufacturing of novel pharmaceutical compounds with the aim of introducing new and improved treatments for various medical conditions. This comprehensive endeavor encompasses various stages, including target identification, preclinical studies, clinical trials, regulatory approval, and post-market surveillance. It involves multidisciplinary collaboration among scientists, researchers, clinicians, regulatory experts, and pharmaceutical companies to bring innovative therapies to market and address unmet medical needs.
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
263778731218 Abortion Clinic /Pills In Harare ,sisternakatoto
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Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
Title: Sense of Taste
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the structure and function of taste buds.
Describe the relationship between the taste threshold and taste index of common substances.
Explain the chemical basis and signal transduction of taste perception for each type of primary taste sensation.
Recognize different abnormalities of taste perception and their causes.
Key Topics:
Significance of Taste Sensation:
Differentiation between pleasant and harmful food
Influence on behavior
Selection of food based on metabolic needs
Receptors of Taste:
Taste buds on the tongue
Influence of sense of smell, texture of food, and pain stimulation (e.g., by pepper)
Primary and Secondary Taste Sensations:
Primary taste sensations: Sweet, Sour, Salty, Bitter, Umami
Chemical basis and signal transduction mechanisms for each taste
Taste Threshold and Index:
Taste threshold values for Sweet (sucrose), Salty (NaCl), Sour (HCl), and Bitter (Quinine)
Taste index relationship: Inversely proportional to taste threshold
Taste Blindness:
Inability to taste certain substances, particularly thiourea compounds
Example: Phenylthiocarbamide
Structure and Function of Taste Buds:
Composition: Epithelial cells, Sustentacular/Supporting cells, Taste cells, Basal cells
Features: Taste pores, Taste hairs/microvilli, and Taste nerve fibers
Location of Taste Buds:
Found in papillae of the tongue (Fungiform, Circumvallate, Foliate)
Also present on the palate, tonsillar pillars, epiglottis, and proximal esophagus
Mechanism of Taste Stimulation:
Interaction of taste substances with receptors on microvilli
Signal transduction pathways for Umami, Sweet, Bitter, Sour, and Salty tastes
Taste Sensitivity and Adaptation:
Decrease in sensitivity with age
Rapid adaptation of taste sensation
Role of Saliva in Taste:
Dissolution of tastants to reach receptors
Washing away the stimulus
Taste Preferences and Aversions:
Mechanisms behind taste preference and aversion
Influence of receptors and neural pathways
Impact of Sensory Nerve Damage:
Degeneration of taste buds if the sensory nerve fiber is cut
Abnormalities of Taste Detection:
Conditions: Ageusia, Hypogeusia, Dysgeusia (parageusia)
Causes: Nerve damage, neurological disorders, infections, poor oral hygiene, adverse drug effects, deficiencies, aging, tobacco use, altered neurotransmitter levels
Neurotransmitters and Taste Threshold:
Effects of serotonin (5-HT) and norepinephrine (NE) on taste sensitivity
Supertasters:
25% of the population with heightened sensitivity to taste, especially bitterness
Increased number of fungiform papillae
Business-led Translation A presentation by CEO, Keith Thompson, about growing a UK cell therapy industry. Oct 2013
1. Business-led Translation
Growing a UK cell therapy industry
delivering
health and wealth
Keith Thompson CEO
Terrapin
Stem Cell, Cord Blood and Cell Culture Conference,
Cambridge MA.
October 2nd 2013
info@ct.catapult.org.uk
Catapult is a Technology Strategy Board programme
2. The Launch of Catapults
Hauser Report
• Creating new manufacturing industries for the UK
• Better exploiting the UK science base
• “Grow and stick”
Technology Strategy Board
• £440m for innovation in 2013/14
• £250m+ assigned to TSB for 7 Catapults 2012
•
High Value Manufacturing, Cell Therapy, Offshore Renewables, CDE, Satellite
Applications, Transport Systems, future Cities
• 2 new ones announced August 2013
•
Stratified Diagnostics, Energy Storage
• Mandated to bridge the gap between research and
commercialisation through industry collaboration
2
3. Cell Therapy
-Mind the (translational funding) gap
3
•
Limited evidence yet that new cell
therapies can be developed, licensed and
adopted successfully
•
Limited commercial investment
•
Limited precedents for valuable exits via
IPO or acquisition
•
Large corporates mostly watching and
waiting
•
Operational SME’s lack finance and
breadth of resources for rapid advance
Catapult
4. Industry Barriers
Business
Manufacturing
and
Supply Chain
Clinical
And
Regulatory
• Health Economics
• Business Models
• Reimbursement
• Partnering
• Robustness & Reliability
• COGS & Scale up
• Characterisation & Analytical
• GMP
• CMC
• Delivery
• Complex Regulatory Landscape
• Pre Clinical Packages
• Clinical trial design
• NHS partnering
4
5. Approach
Project led
• Pathfind therapies into and through the Clinic and
out and into the market
Ø Core projects with direct investment
Ø Rounded phase II data package
Ø Investible propositions
• Assist industry to progress to commercialisation
Ø Collaboration
Ø Contract research
• Technology platforms
Ø Develop novel technologies and license to industry
• Train professionals though immersion
5
6. Stage Gated
6
Proof of Principle
• Scientific, clinical, regulatory, commercial
Non-clinical
• Safety, toxicology, GMP proving, assays
Suitability
• Commercial
Development
Plan
Clinical
• Safety and efficacy, investible data
Platform
• Generic issues and large collaborations
7. Projects: Successful Path to Commercialisation
7
Regulatory agency dialogue on
plans at each stage
-start with the end product in mind and deliver an integrated plan
Science
•
•
Efficacy and safety hypothesis and evidence
Definition and characterisation of cellular product
Clinical
•
•
•
•
Patient population, unmet medical need, differentiation
Safety
Robust evidence of efficacy
Dose and dosing regimen
•
•
•
GMP manufacturing process; release; comparability assays
Supply logistics
Scale-up / scale –out; Control of cost
•
•
•
Pricing and reimbursement plan
Defensibility (IP, know-how…)
Commercialisation partner
Manufacturing
Business
8. Working models and finance
8
Nature of project Paid for by: Carried out by:
Ownership
of outputs
Finance
Core Project
Catapult
Catapult
Catapult
£70m/
5 years
Contracted
Development
Client
Catapult
Client
£10m/
Per year
Industry
Collaboration
Grant or
Catapult
Catapult
and
and partner partner
Outputs
shared
£10m/
Per year
9. Funding for Cell Therapy in the UK
Govt.
Support
Medical
Charities
Commercial
Development
Plan
Financial
Investment
Corporate
Venturing
9
10. Assets
10
-Facilities and Teams
Facilities
•
•
•
•
Central London location
1200 sq m on 12th floor Guys Tower
Clinical research cluster
Capacity for 100 people
Teams
• Business
•
•
Business development
Business models
•
Health economics
• Manufacturing and Supply
•
Process development
•
Analytical development
•
•
GMP process proving
Supply Chain
• Clinical Trial and Regulatory
•
•
•
Regulatory
Clinical trial sponsor
Clinical operations
12. Lab - Pilot - Scale
12
Catapult Multi Functional development Pod
Commercial Scale
Catapult GMP Proving Lab
T
e
c
h
t
r
a
n
s
f
e
r
13. Product release criteria
Non clinical
development
Phase I
13
Phase II
Market
Approval
Phase III
•
Cell therapy characterisation requirements change depending upon where a
product is in the commercialisation process
•
Goal is to define and quantify the critical quality attributes for a particular
product (release criteria)
•
Need to develop appropriate assays for product release which can be
incorporated into the manufacturing process (rapid, simple)
Pilot GMP
Process
Development
Process
Commercial
Process
Scale Up/
Out
14. Cell therapy characterisation
Non clinical
development
Phase I
Phase II
14
Phase III
Market
Approval
Products in pre-clinical development often require more thorough/complex
characterisation using more advanced analytical approaches to understand the product
MOA
Cell markers
Potential
potency markers
Identification
markers
Direct Surrogate
Purity assay
process qualification
Pilot GMP
Process
Process Comparability
Development
Process
Commercial
Process
Scale Up/
Out
15. Cell therapy characterisation
Non clinical
development
Phase I
Phase II
15
Phase III
Market
Approval
MOA
Cell markers
Raw Materials
Stability
Potential
potency markers
Identification
markers
Direct Surrogate
Purity assay
Sterility testing
Mycoplasma
Virus testing
Endotoxins
Shelf life
Genetic stability
Viability
the aim is to develop assays that are reliable, reproducible with low variance,
Pilot GMP
Commercial
Scale Up/
Development
inexpensive, simple, andProcess with appropriate reference standards — that
rapid,
Process
Process
Out
also have relevance to the intended clinical activity.
17. For healthcare products, price, reimbursement and
demand are interlinked determinants of profit
All these have to be effectively addressed to reach
commercial goals…
PRICE
PROFIT
REIMBURSEMENT
17
DEMAND
18. Successful commercialization depends on both
regulatory approval and optimal market access
Quality
Safety
Efficacy
REGULATORY APPROVAL
18
Comparative Clinical & CostEffectiveness; Budget Impact
MARKET ACCESS
19. Payers have a common aim: to achieve the greatest
health care value for the money they spend
Payers are key market access decision-makers
(with input from clinical and economic advisors)
Is it
worth it?
Does it add
value
over SOC?
Cost-effectiveness
Price comparison
Can we
afford it?
Budget impact
Should we
control
its use?
Comparative
clinical
effectiveness
Restrictions
Do we need
to fund it?
Is the product
needed?
Unmet need
Political imperative
The emphasis on these elements differs
across markets, most notably the use of
cost-effectiveness in decision-making
19
20. Pricing approaches in healthcare are shifting
towards value-based models
Cost-based
Competitor-based
Value-based
What is it?
• Price is set by
assumptions on
costs, expected
sales volumes and
margins
• Price is driven by
the pricing of
competitor
products
• Price is based
upon
therapeutic /
economic value to
the customer
Examples
• Cost-plus pricing
• ROI based pricing
(e.g. PPRS in UK)
• Penetration
pricing
• Reference group
pricing
• Value-based
pricing
Comments
20
• Enforced by many
reimbursement
• Becoming obsolete;
no longer resonates
systems for
“undifferentiated”
with payers
products
• Typical approach
for differentiated
products
21. Value-based pricing relies on the quantification of the
added-value that a new technology delivers over SOC
• Reference Value of Standard of Care (SOC)
o Comparative data against the SOC is required:
V = RV + PDV - NDV
H2H comparative data demonstrating superiority or
non-inferiority of Product X against the SOC is preferred
Ø Indirect comparisons of high methodological standards
(NMA) sufficient for non-inferiority claims
Ø
PDV
Negative
Differentiation
Value (NDV)
Positive
Differentiation
Value
V
RV
21
Reference
Value (SOC)
NDV
• Differentiating Value
o Clinical effectiveness
o Economic impact: budget impact, cost-minimization,
cost-effectiveness, cost-utility
• Value (V)
o For a given indication “V” varies depending on the
intervention’s positioning in the treatment algorithm
& the target patient profile
22. In markets like UK, cost-effectiveness is the measure of
value and the determinant of reimbursed price
• QALYs are the measure of clinical effectiveness
Ø QALYs = Life expectancy (life years) x Quality of life (utility)
Ø
Ø
•
Utility ranges from 0 (death) to 1 (full health)
Utility determined by HRQoL instruments; incorporation in clinical trials is key for market access
Costs
Ø
Direct (healthcare) and indirect (social care) costs
o
Incremental Cost Effectiveness Ratio (ICER)
Ø
QoL (utility score)
•
Societal costs (e.g. time off work) not yet accounted by NICE
22
1.0
Cost B-Cost A / QALY B- QUALY A
NICE ICER thresholds:
• Set at £20K/QALY; up to £30K justifiable
if:
Treatment A
Ø
Treatment B
0.5
Ø
•
QALYs gained (B vs A)
•
Orphans: No defined threshold
Under VBP additional factors are likely to
impact ICER threshold e.g.
Ø
0
1
Life Years
There is confidence in results
Technology is highly innovative
Ø
Ø
Disease burden
Level of unmet need
Size of target population
23. Main commercial preparations to optimize market access
Enviromental
Reviews
Qualitative
Research with Key
Market Access
Stakeholders
Suitability assessment
• Assess
• Generate insights
opportunity (e.g. on reaction to
epidemiology,
“Target Product
disease burden,
Profile”, key value
unmet need,
drivers, likely
Objectives clinical /pricing
positioning,
benchmarks,
pricing,
funding &
reimbursement,
supporting data
uptake,
requirements,
supporting data
competition)
requirements
23
Value Story
Development
Development
Quantitative
Pricing
Research
Value Dossier
Development
Out-licensing or Launch
• Development of • Identify revenue
clinical value
maximising
arguments and
target price
economic models • Inform asset
• Test value story
valuation and
with key market
market access
access
strategy
stakeholders
• Enhance market
• Identify areas to
strengthen story
and data
access potential
and strategic
partnering
• Compile in a
single document
clinical and
economic value
proposition and
corresponding
evidence to
support
negotiations
25. Databases of UK preclinical (<2 yrs from clinic)
and clinical stage cell therapies
Category
Preclinical (<2 yrs from
clinic)
Number
Comparision
More allogeneic therapies in
preclinical stage
37
Larger variety of cell types in
preclinical stages
Larger range of indications for
preclinical projects
Clinical (UK trial ongoing)
34
Total
71
http://ct.catapult.org.uk/
Few commercially sponsored
projects in both pre clinical and
clinical stages
25
26. Regulation………
• Interact with regulators to improve
clarity and speed
• Clinical access to NHS
• Clinical Trial Sponsor
• Use the existing flexibility
26
27. Clinical trials for cell therapies
27
Safety
•
•
•
Risk mitigation
Monitoring
Duration follow-up
Efficacy
•
Patient population
•
•
•
Risk vs benefit
Disease stage
Paediatrics
Logistics
•
Very close integration of
clinical and
manufacturing teams
Dose selection
•
•
Recognised
endpoints
• Head to head
comparison
• HRQoL
Placebo and
blinding
• In study vs
• historical /parallel
Single vs multiple
Dose escalation
Feasibility
•
•
Accompanying
device / surgical
technique
Manufacturing success
rate
29. Identifying projects
• Pre-clinical and clinical databases
• Technology transfer offices
• Intermediaries
• Grant Funders
• Industry Groups
• Charities
• Investors
• Direct contact
•
•
Inward investors
EU entrants
31. Project Examples
• Share of expertise
• Support for in house projects
• New delivery device to reduce injection
pain
• Phase 2 clinical trials
• Scale up, Assays, Freezing and distribution
of cells
Large Cap
Company
• Manufacturing partner, Regulatory,
Clinical trial design and delivery
• Immunomodulation
• Regulatory, Clinical trial design, business
models
31
32. Platform Project Example
-Build a GMP iPS bank with Roslin Cells
Biopsy
Reprogramming
Existing registry
of volunteers
for platelet and
BM donation
Known subpopulation of
triple HLA-A, B
and DR
homozygotes
Cell therapy
GMP iPS cell bank
Differentiated cells/
tissues of chosen
types and origins
Phase 1: Generate 6 cell lines: £2m
•
•
Generate highly specified Master and Working Cell banks
Generate Research grade equivalents and distribute to bona fide researcher
Phase 2: Expand to 20 cell lines: £3m
• Milestone and success driven
Phase 3: Expand to 100 cell lines:
• Self financing dependent upon early clinical trial results
33. INVESTABILITY
Market
sen-ment
for
cell
therapy
investments
improving
• Looking
for
rounded
data
package
Clinical
Data,
Health
Economics,
Business
Models,
Market
Access,
Reimbursement,
Defensibility
• Pricing
target
and
COGS
drive
long
term
margin
towards
valuaHon
• Clinical
Data
incorporaHng
H2H
and
HRQoL
improves
valuaHon
Health
economics,
pricing
and
value
story
built
to
support
commercialisaHon
Transac-ons
• Financial
investment
–
Venture
Capital
• Corporate
Venturing
• Direct
Corporate
TransacHons
34. Strategic Goals
Pipeline
• Increased cell therapies in UK clinical trial and clinical use
Value
• Investible propositions created leading to cell therapy companies
that succeed and stay in the UK
Attractiveness
• Demonstrating that the UK is the place to do this work, with
increased inward investment
Goals
• Build a £10bn industry
34