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European Biopharmaceutical Review
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One of the biggest obstacles that modern day society faces is the
overwhelming cost of healthcare. Increasing changes are continuously
being introduced, while simultaneously, resources are being diminished.
The concepts of Regenerative Medicine offer cost-effective solutions
Besides the dwindling resources and socio-economic changes introduced by
an ageing population, one of the major challenges in today’s
industrialised world is the overburdening cost of healthcare (1).
Regenerative Medicine (RegenMed) offers a solution to address this
challenge. However, it can be a highly complex approach to treating
patients because it potentially requires the integration of numerous
scientific and clinical disciplines, as well as appropriate
infrastructures (2,3). To bring new RegenMed technologies to patients
effectively, and to further accelerate the developmental pace for
RegenMed therapies, strong and open collaborative efforts are needed.
This article outlines how interregional and open collaboration is an
optimal strategy to address the innate complexities of RegenMed. The
health and financial opportunities that this cutting-edge approach to
medicine represents makes it a natural candidate for driving economic
growth (4). Therefore, it deserves to be addressed by increasing the
capacity of European regions to invest in, and further develop their
commitment to, research-driven translational clusters. Such action could
contribute significantly, not only to an accelerated delivery of new
regenerative therapies to patients, but also to stronger regional
economic development in Europe.
In the future, regional investments will most likely continue to be
matched by funding provided by the EU. The new framework programme for
research, ‘Horizon 2020’ states that the EU will “provide seamless and
coherent funding from idea to market… with a strong focus on creating
business opportunities” for industries that develop products addressing
the socioeconomic challenges in the area of health, demographics and
well-being (5).
The Promises of RegenMed
RegenMed is the process of replacing or regenerating human cells,
tissues or organs to restore or establish normal function (2). It refers
to a group of biomedical and biomaterial approaches to clinical
therapies that may involve the use of small molecule drugs, biologics,
biomaterials, devices and cells (cellbased therapies and tissue
engineering), either alone or in combination. Although it is not a
specific technology, it is a novel approach that is potentially capable
of delivering life-changing, or even curative solutions, to highly
prevalent conditions, such as neurodegenerative conditions, bone or
liver diseases, diabetes, heart or renal failure, which have
traditionally been treated as chronic disorders. These novel disruptive
solutions could have dramatic implications on the quality of life for
the patient, their expected lifespan, her or his productivity, the cost
of the treatment, and on the socioeconomic costs associated with such
medical indications (3).
Both degenerative and chronic diseases, such as Parkinson’s disease
(PD), are prime targets for RegenMed approaches. With increasing life
expectancy and a larger proportion of elderly people in the population,
degenerative diseases are becoming a growing socioeconomic challenge. We
have therefore used PD as an example to illustrate the potential of
RegenMed to alter the current treatment paradigm. Parkinson’s is the
third most common neurodegenerative disorder after dementia and
Alzheimer’s disease (6). The prevalence of PD is about 0.3 per cent of
the whole population in industrialised countries, although it is more
common in the elderly, with prevalence rising from one per cent in those
over 60 years of age, to four per cent in the over 80s. The mean age of
onset is around 60 years (6).
RegenMed in Parkinson’s Disease
In the UK in 2007, the average direct annual cost of healthcare for a PD
patient was calculated to be £5,993, with indirect costs as high as
£27,000 (8). At 38 per cent, the average National Health Service (NHS)
costs represented the highest proportion of direct costs, followed by
social services costs (35 per cent) and private expenditure (27 per
cent) (8). The ratio between primary and secondary care and social and
private cost is not static. The older the patient gets, the higher the
proportion of social and private cost. Based on Findley’s data, we have
estimated the average total costs of a PD patient over 10 years, from a
theoretical initial diagnosis of the disease at the age of 60 still of
working age for example, to a patient age of 70 years (8). Furthermore,
we have assumed that the introduction of a new RegenMed therapy would
increase the NHS costs by 100 per cent, but, will decrease the social
and private costs and indirect costs by 70 per cent.This decrease in
costs would be the consequence of PD patients maintaining an independent
lifestyle. This scenario would result in net savings of more than
£190,000 per patient over a period of 10 years when using the carer
replacement model for indirect cost estimation (see Figure 1).
At present, we do not know how future RegenMed therapeutic products will
compare to present PD therapy schemes, whether it will cost more or
less, and what the life-changing treatment will look like. In addition,
we do not know whether the expected gain in patient independence will
lead to a 50, 70 or 100 per cent reduction of the social and private and
indirect costs associated with PD. However, the estimations in our
example clearly show that the potential savings in societal costs
offered by a curative solution is extremely significant.
Besides the societal interest in allocating existing financial resources
appropriately, the most important question for the individual patient
is: ‘How does a new therapy affect my quality of life?’ In health
economic studies, incremental health benefits are expressed in
quality-adjusted life years (QALYs) (10). Life years are weighted by
quality of life (QoL), where 1 signifies perfect health and 0 represents
death. The total incremental QALY gain for a new therapeutic solution,
in simplified form, is a function of improvement in QoL (ΔQoL) and the
duration over which this improvement is sustained. The formula to
calculate incremental QALYs (under the assumption that the therapy leads
to QoL improvements only, and not to concurrent gains in life
expectancy) is shown in Equation 1:
ΔQALY = (ΔQoL) x time (in years) (10)
Incremental QALYs can be converted into monetary terms to offset the
financial costs of the treatment, to arrive at a net health benefit for
each treatment (10). If the societal willingness to pay is assumed to be
£30,000 per QALY (11), the ‘headroom’ – that is, the maximally
acceptable additional cost of the new therapy – can be calculated as
shown in Equation 2:
ΔCosts (headroom) =ΔQALY x £30,000 (10) The headroom definition can thus
be used to quantify the gain in quality of life introduced by a new
therapy in monetary terms. If we assume that a new RegenMed therapy
would result in a 20 per cent improvement in quality of life (as
determined by the ‘time trade-off method’) over a period of 10 years,
this therapy would have an added value (headroom) to society of up to
£60,000 for each individual PD patient.
Prerequisites needed to turn RegenMed concepts into successful products
So how do we turn these regenerative approaches into successful
products? There are a number of challenges that will need to be
addressed, as well as a number of stakeholders that may choose to
partner with each other in order to drive the process of RegenMed
development.
Clusters of Knowledge and Translation Centres
Efficient and cost-effective interdisciplinary work should be conducted
in close physical proximity, ideally in one location. Therefore, in the
past, numerous regional clusters and translation centres for
regenerative medicine have been established (12). Below is a list of the
bottlenecks that have started to be addressed by the various clusters
and translation centres and have since turned from challenges and
requirements into successful attributes:
● Close proximity to large clinics that provide access to patients,
doctors and opinion leaders articulating the medical need, as well as
access to clinical material
● Availability to form functional development teams comprised of
doctors, scientists and engineers who collaborate in an integrated
manner
● Ability to develop and cost-effectively produce medical-grade biomaterials and other materials
● Ability to ‘opportunity-check’ new therapeutic concepts (technical
visibility, market analyses, intellectual property (IP) analyses, early
dialog with potential commercialisation partners)
● Ability to develop Good Laboratory Practice and Good Manufacturing
Practice (GMP), and produce potential new therapeutic solutions
● Availability of disease-relevant and therapeutic platform-relevant animal models
● Ability to finance and perform clinical studies, including the
opportunity for integrated interaction with contract research
organisations
● Ability to adopt new healthcare cost models and business models ●●
Availability of an established regional entrepreneurial culture and
network
● Availability of entrepreneurial education programmes and management capacities
Many of these essential characteristics are now being adopted in a
number of translational centres and clusters for regenerative medicine
that have been founded throughout Europe, North America and Asia over
the last few years (13, 14). They present themselves as innovation and
de-risking factories for new RegenMed solutions. Some of the necessary
commercialisation partners for the later phases of clinical development
and distribution are already appreciating the benefits from interacting
with these translation centres and clusters.
Important manufacturing issues that are currently addressed by translational centres and RegenMed clusters are (15):
● Lack of standards for (cell-based) production and quality control (16)
● Scale-up issues in manufacturing
● Availability of medical-grade materials
● Sterilisation methods for biomaterials and combination materials
● Economisation of GMP-facility usage
● Regulatory hurdles and lack of harmonisation of regulatory requirements
● Opportunity for integrated interaction with contract manufacturing organisations
All of the above bottlenecks need to be successfully addressed in order
to generate a convincing pipeline of RegenMed candidate products and to
attract a sufficient amount of sustainable investment to drive the field
towards achieving its goals. To further de-risk their projects and to
accelerate the development process, leading translational centres have
recently formed a consortium, the ‘Regenerative Medicine Coalition’
(17).
Spin-Off and Start-Up Companies
Important stakeholders that help drive the process of RegenMed
development are spin-off companies. Small but experienced management
teams that participate in the foundation of spinoff companies, and
interact closely with translational centres and clusters, comprise
another important component of a capital-efficient technology
development model. By developing the business model and an appropriate
intellectual property (IP) portfolio, and by raising the capital needed
for generating comprehensive pre-clinical and clinical proof-of-concept
data, these teams can further de-risk a RegenMed therapeutic solution up
to the stage where it will be taken over by a strategic partner (large
pharma, biotech or medical device companies) or attract substantial
investment for final development (typically Phase 3 trials) and market
introduction.
Small and Medium Enterprises (SMEs)
The process of product development can repeat itself many times, for
example by adapting a new therapeutic solution to various indications.
In addition, spin-off companies may have the potential to grow further
and solidify their position by offering manufacturing contract services
(such as cell production) to biopharma partners, thereby further
leveraging their know-how advantage. As a result, small spin-off
companies have the ability to develop into bigger operations (SMEs)
employing significant workforces and outgrowing their roots in the
capital-efficient translation centre model (18).
In RegenMed, there are autologous therapy options currently being
developed that will require new business models, for example
servicebased operations and point of care bioprocessing devices. These
are personalised approaches to medicine that offer the option to
distribute a new therapy through regional cell-production facilities
and/or new specialised regional clinics. These business models do not
necessarily need financial partners because they can be realised by an
adequately financed SME alone. Such SMEs can generate significant new
employment in their regions.
Larger Multinational Life Sciences Industry Companies
The role of the Big Pharma, biotech and medical device companies in
translation centres is essentially as strategic partners for late
clinical development, regulatory approval and distribution. They provide
best practice experience to spin-offs and SMEs in collaborative
projects.Often they employ a significant part of the region’s workforce
and therefore it is important for the regions to maintain an environment
that is driven by cuttingedge technology development.
Interregional Collaborations Accelerate Technology Development
Regional clusters have an advantage in that people with different
experiences, skill sets and networks can work together in close
proximity, communicate regularly and with ease, and most importantly,
can develop trust in one another. Together, the members of such a
cluster should ideally comprise a skill set portfolio that is highly
comprehensive and complementary, serving as an integrated horizontal
supply chain such as that seen in Silicon Valley for IT (19). The
biotech cluster in the bay area of San Francisco, California, is one
prominent example for such a successful industry cluster in the life
sciences field. Members of regional clusters operate in the same
cultural environment and time zone. Thus regional clusters enable
incremental steps to be processed in an efficient integrated
multidisciplinary manner in order to have a greater chance of effective
innovation and the development of commercial products and services.
Research-driven regional clusters typically comprise universities, both
research and translational centres, enterprises and regional
authorities. It should also be noted that translation centres comprise
the extreme form of clusters, since many of the stakeholders essential
for translation are located in one organisation.
Despite all the advantages of a regional ‘working network’,
interregional collaborations between research-driven clusters provide
additional aspects that can further increase the output efficiency of
such networks and lead to greater acceleration of technology
development.
Regular and structured communication between regional RegenMed clusters could lead to:
● Sharing of experience and accelerating learning curves
● Sharing of current interregional IP with a focus on developing further IP
● Joint definition of the global situation and shared visualisation of
the future in order to better define the local ‘niche’ and thereby
increase regional competitiveness
● Meaningful and coordinated regional specialisation focusing in the area of RegenMed
● Sharing of infrastructures (for example, expensive instrumentation and GMP production capacities)
● Ability to perform multicentre, largescale clinical verification studies
In essence, interregional collaboration can speed up technology
development by broadening the common knowledge base for each region,
freeing up resources to be allocated to developing new IP. In addition,
developing a shared view on the future, and putting in place a joint
strategy or action plan, will allow each regional working network to
better prioritise its objectives.
The above aspects could potentially strengthen the research potential of
European regions, boost economic development and accelerate the
delivery of new RegenMed therapies to patients. The European Commission
(EC) is making great efforts to support regional clusters and
interregional collaborations – for example, with the initiation of the
‘Regions of Knowledge’ programme in 2007 with ‘the overarching objective
to promote European competitiveness and regional development through
smart specialisation of regions in a globalised world’ (20). One of the
projects funded in the programme is Tissue Engineering and Regenerative
Medicine (TERM). TERM links research organisations in the field of
cell-based therapies through European cooperation between regional
research clusters. The TERM project was commenced at the end of 2010 and
will generate a joint action plan for 11 regional European RegenMed
clusters (21).
Conclusion
We believe that interregional collaboration between clusters focused on
RegenMed will lead to acceleration in the technology development and
regional economic growth. In order for this to happen, EU member states
and regional authorities need to take stock of the different areas where
investment would overcome existing market failure. Solutions include
communication platforms that foster interregional communication between
stakeholders; exchange programmes for students, researchers, engineers,
clinicians and technology transfer experts; regularly scheduled
interregional conferences to promote collaboration; implementation and
development of translation centres; and the implementation of
infrastructure that supports spinoff companies and SMEs. If regional
authorities invest in these areas now, such action will contribute
significantly to the accelerated delivery of new RegenMed therapies to
patients, as well as contributing to the regeneration and future growth
of the European economy. So why wait?
References
1. Secretary General of the OECD, Health at a Glance: OECD Indicators, OECD Publishing, pp1-204, 2011
2. Mason C and Dunhill P, A brief description of regenerative medicine, Regen Med 3(1): pp1-5, 2008
3. Mason C and Dunhill P, The strong financial case for regenerative
medicine and the regen industry, Regen Med 3(3): pp351-363, 2008
4. Mason C, Brindley DA, Culme-Seymour EJ and Davie NL, Cell therapy
industry: billion dollar global business with unlimited potential, Regen
Med 6(3): pp265-272, 2011
5. Horizon 2020, The Framework Programme for Research and Innovation,
European Commission, 2011 Visit: http://ec.europa.eu/research/
horizon2020/index_en.cfm
6. DeLau LM and Brezeler MM, Epidemiology of Parkinson’s disease, Lancet Neurol 5(6): pp525-535, 2006
7. Huse DM, Schuman K, Orsini L, Castelli- Haley J, Kennedy S and
Lenhard C, Burden of illness in Parkinson’s disease, Mov Disord 20,
pp1,449-1,454, 2005
8. Findley LJ, The economic impact of Parkinson’s disease, Parkinsonism and Related Disorders 13: pp8-12, 2007
9. Brown G, The Bitter End, New Scientist 196: pp42-43, 2007
10. McAteer H, Cosh E, Freeman G, Pandit A, Wood P and Lilford R,
Cost-effectiveness analysis at the development phase of a potential
health technology, J Tissue Eng Regen Med 1: pp343-349, 2007
11. Devlin N and Parkin D, Does NICE have a cost-effectiveness threshold
and what other factors infl uence its decisions? A binary choice
analysis, Health Econ 13: pp437-452, 2004
12. World stem cell report, Regen Med 6(5) Suppl 2011
13. Lauter FR and Wirsching S, Global update Germany in ‘World Stem Cell report 2011’, Regen Med 6(5) Suppl 2011
14. Brindley DA, Reeve BC, Sahlman WA, Bonfi glio GA, Davie NL,
Culme-Seymour EJ and Mason C, The impact of market volatility on the
cell therapy industry, Cell Stem Cell 9: pp397-401, 2011
15. Davie NL, Brindley DA, Culme-Seymour EJ and Mason C, Streamlining
cell therapy manufacture – from clinical to commercial scale, Bioprocess
Intl 10(3) Supplement, 2012
16. PAS 84 Cell therapy and regenerative medicine glossary, Regen Med Supplement 2012
17. Visit : www.the-rmc.org
18. REMEDiE: Regenerative medicine in Europe, Emerging needs and
challenges in a global context. Visit: www.york. ac.uk/satsu/remedie/
19. Saxenian A, Regional Advantage: Culture and Competition in Silicon Valley and Route 128, Harvard University Press, 1996
20. Website of European Commission programme ‘Regions of Knowledge’.
Visit: http://ec.europa.eu/research/fp7/ index_en.cfm?pg=know
21. Visit: www.termproject.eu
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Frank-Roman Lauter serves as Secretary General of the Regenerative Medicine Coalition and Head of Business Development at the Berlin-Brandenburg Centre for Regenerative Therapies (RMC). He co-organises strategy development for regenerative medicine clusters from seven European countries, and has initiated a collaboration between the California Institute for RegenMed and the German Federal Ministry for Education and Research, resulting in a joint funding programme. His interest lies in the organisation of academic infrastructures to promote effi cient translation of research findings into new therapies. He is employed by the Center for Biomaterial Development Helmholtz Center Geesthacht, Germany.
Boo Edgar, Dr Med Sci(Clin Pharm) is the Associate Professor of Innovation and Entrepreneurship at the Institute of Medicine, University of Gothenburg and at Chalmers. Boo has a career in research and marketing within the pharmaceutical industry and is the CEO of a venture seed fund, as well as several entrepreneurial start-ups.
Sylvie Ponchaut is the Scientific Director of BioWin, the Health Competitiveness Cluster of Wallonia in Belgium. She has qualified as a Pharmacist and completed a doctoral thesis at the Christian de Duve Institute in Brussels. Sylvie is the founder and manager of IPinnova sprl.
Chris Mason is the Professor of Regenerative Medicine Bioprocessing at the Advanced Centre for Biochemical Engineering, University College, London. Chris is working on the translation and commercialisation of cell and gene therapies. His other roles include Senior Editor of Regenerative Medicine, Chair of BIA Cell Therapy Industry Group, Trustee of the UK Stem Cell Foundation and Director of the London Regenerative Medicine Network.
Greg Bonfiglio is the Founder and Managing Partner of Proteus LLC, an investment and advisory fi rm focused solely on regenerative medicine. He was an early investor in the fi eld and has been on the board of VistaGen Therapeutics, StemCyte Inc and California Stem Cells Inc. He is a member of the ISSCR and ISCT.
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