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20 How toOvercome theValley ofDeath fromBasic Science toClinical Trials
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219
is currently raising a Series F round, with a $1
billion Series E round led by Google
(Crunchbase)).
20.11 Commercialization Process
Technology represents devices and processes that
can be made for nancial gain. This concept of
bench to bedside or the commercialization of
biotechnology is the valley of death that one as
inventor tries to hurdle (Fig.20.1). To progress to
market, the scientic entity must dene customers and users. Most inventions will potentially
fail in their rush to commercialization and most
patents are not commercialized. This is the reality of the biomedical commercialization process.
Challenges to technological commercialization can take the form of recognition of the device
potential, technology push, market pull, regulatory hurdles, access to capital, and overall entity
management. The commercialization process is
akin to the garden metaphor where one plants a
seed, nurtures the product, and then pulls the
weeds or harvests. Overlapped with this metaphor
is discovering the technology (planting the seed),
developing (nurturing), and market development
(weed or harvest). Commercial entities develop
options when to access these different time points.
Identifying the product, testing and developing
the product, and then marketing the product coincides in parallel with regulatory phases depending
on what country the product is developed in.
Accessing this value-chain is critical for commercialization success. The value begins by
dening the product and its clinical need.
Otherwise known as discovery, this phase will
use research funds granted by federal, state, and
outside investors. Moving forward into product
development, which will be tethered to phase 1, 2
and 3 trials, commercialization exits will need to
be decided upon. Licensing the product or selling
the company must be considered. A license will
maximize the likelihood of short-term success.
However, the entity will lose the ability to be
involved with discussion for future innovation, as
the larger corporate entity will control innovation
and direction of the technology. Selfcommercialization can be a path taken but is not
always feasible secondary to increased nancial
risk. Joint-commercialization can be a considered option. This enables the rm to acquire
experience that can enable self- commercialization
over the long term.
Early StageSeed Capital Later Stage
Friends,
Family,
& Founders
Fig. 20.1 Idea discovery, development, and market launch highlighting the role of capital infusions both pre and post
regulatory approval
Angels SBIR
Proof of concept
Te chnology transfer
NIH
DoD
State Funding
VALLEY OF DEATH
Early feasibility
studies
PUBLIC SECTOR PRIVATE SECTOR
Early Venture
Captial
Technical
validation
Industry Venture
Capital
AB CDE
FDA
approval
Product
launch
HURDLES
IP / FTO hurdles
Regulatory hurdles
Reimbursement hurdles
Early market
tensions
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K. Bridgham et al.
Taking factors into consideration such as
amount of nancing required to move through
regulatory hurdles must be balanced along with
the cost of capital raises. Dilution equity events
for stakeholders must be considered. Risks that
must also be factored when to commercialize are
based upon scientic risk, operational risk, and
whether there is a true market for the product.
Financial return for the original inventors and
developers must be considered versus incorporating and raising capital from outside sources such
as venture capital, professional investors, and
corporations.
Commercialization activities should be integrated into development plans. Market analysis,
commercial planning, market cultivation, prelaunch, launch, and commercialization are all
overlapped with phase 1, 2, and 3 development of
regulatory development. These activities all
begin the same time when conception of idea is
formulated, and the path to customer is realized.
20.12 Conclusions
The success of any innovation is measured by
whether it achieves its desired impact. In the case
of a new biotechnology, pharmaceutical, or medical device company, the end goal is for the product to reach routine clinical implementation.
Taking a basic science idea and turning it into a
novel treatment or therapy for patients is a complicated process that requires a substantial
amount of time, effort, and money. Not every
idea or innovation can overcome the hurdles
associated with the translational process. Those
that are well suited to overcome these hurdles
face a long journey ahead, however, the rewards
of the journey could affect the lives of millions.
Particularly high expectations surround the eld
of regenerative medicine. It is essential that the
breakthroughs experienced in the laboratory
reach our clinics. With the appropriate knowledge and planning, the potential clinical impacts
of regenerative medicine are endless.
References
1. Gehr S, Garner CC.Rescuing the lost in translation.
Cell. 2016;165(4):765–70.
2. Milne CP, Kaitin KI.Translational medicine: an engine
of change for bringing new technology to community
health. Sci Translational Med. 2009;1(5):5cm5.
3. Fernandez-Moure JS. Lost in translation: the gap
in scientic advancements and clinical application.
Front Bioeng Biotechnol. 2016;4:43.
4. Butler D. Translational research: crossing the valley
of death. Nature. 2008;453(7197):840–2.
5. Siegel DS, Veugelers R, Wright M. Technology
transfer ofces and commercialization of university intellectual property. Oxford Rev Econ Pol.
2007;23(4):640–60.
6. Tajonar A.How to start a biotech company. Mol Biol
Cell. 2014;25(21):3280–3.
7. Mas JP, Hsueh AB.An investor perspective on forming and funding your medical device start-up. Tech
Vasc Intervent Radiol. 2017;20(2):101–8.
8. Yock PG, Zenios S, Makower J, Brinton TJ, Kumar
UN, Watkins FTJ, Denend L, Krummel TM, Kurihara
CQ. Biodesign: the process of innovating medical technologies. 2nd ed. Cambridge: Cambridge
University Press; 2015.
9. Kolchinsky P. Entrepreneur’s guide to a biotech
startup. https://www.ldeming.com/s/Kolchinsky.pdf.
Accessed 2 April 2018.
10. Naghshineh N, Brown S, Cederna PS, Levi B,
Lisiecki J, D'Amico RA, Hume KM, Seward W,
Rubin JP. Demystifying the U.S. Food and Drug
Administration: understanding regulatory pathways.
Plast Reconstr Surg. 2014;13(3):559–69.
11. Abou-El-Enein M, Duda GN, Gruskin EA, Grainger
DW. Strategies for derisking translational processes
for biomedical technologies. Trends Biotechnol.
2016;35(2):100–8.
12. Gompers P, Gornall W, Kaplan SN, Strebulaev
IA.How do venture capitalists make decisons? NBER
Working Paper Series, No. 22587 2016. https://
medium.com/vcdium/venture-capital-decision-making-c3258bc1b09c
. Accessed 2 April 2018.
Moving Your Results fromBench
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toBedside: Protecting Scientic
Findings
DominikThor
21
21.1 Introduction
Scientic research, especially in natural sciences, can be an exceptionally expensive and
time- consuming process requiring the investment of millions of dollars before any potential
prot and often before a technology’s later aptitude for monetization can be guaranteed. This
happens to be the case in most if not all medical
research, where a plethora of different aspects
inuence the actual suitability of new scientic
ndings for clinical use. There are multiple challenges in the drug design process [1], such as
selectivity and potency optimization for the
intended target through changes in the physicochemical properties of the drug or the modication of functional groups, or drug formulation.
This, together with the extensive pre-clinical and
clinical testing required for the identication of
the likelihood for metabolic interactions and signicant side- effects in toxicology studies and
thus a comprehensive benet-to-risk evaluation
of a new drug, result in huge nancial efforts and
therefore entrepreneurial risk. Additional efforts
are linked to the obligatory review of new drugs
by a regulatory agency, which is required before
D. Thor (*)
Vienna University of Economics and Business,
Institute for Small Business Management
and Entrepreneurship, Vienna, Austria
e-mail: dominik@thor.at
a new substance can be marketed [2]. The
European Medicines Agency (EMA), the Food
and Drug Administration (FDA) and the Japanese
Pharmaceuticals and Medical Devices Agency
(PMDA) are the biggest regulatory agencies,
which are responsible for the regulation,
approval, and oversight of drug products. The
complexity of interacting with the regulatory
agencies is signicant and the challenges of fullling their specic requirements force pharmaceutical companies to dedicate funds and human
resources entirely to dealing with regulatory
affairs, thus increasing the costs and nancial
risks for them further.
For companies to accept such risks, the eventual monetization of their scientic research,
most often in the successful launch of a new
drug, has to offer an accordingly high potential
income, and the prerequisite for that comes in
the form of exclusive legal rights to sell the new
drug for at least some years after it enters the
market. This may be different for basic research,
which requires less investment, and is often
inspired by the scientist’s wish to further the
understanding of a certain aspect of a scientic
eld rather than monetary motivation or simply
does not offer an obvious or feasible potential
for monetization. As such basic research is often
a domain of academic efforts [3], nonetheless,
reaching the goals of both commercial and academic research requires the protection of scientic results or, in more general terms, intellectual
© Springer Nature Switzerland AG 2019
D. Duscher, M. A. Shiffman (eds.), Regenerative Medicine and Plastic Surgery,
https://doi.org/10.1007/978-3-030-19958-6_21
221
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D. Thor
property. This unfortunately still happens to be
an insufciently understood and inconsequently
applied principle by many scientists especially
in the academic sector.
21.2 Patents fortheProtection
ofIntellectual Property (IP)
Intellectual property (IP) is a term used to describe
a plethora of creations of the mind, such as inventions of all kinds, artistic and literary works, as
well as symbols, names, and images. While some
kinds of intellectual property are automatically
safeguarded by copyrights and thereby belong to
their creator, other types of intellectual property
require legal protection that must be applied for.
This legal protection comes in the form of intellectual property rights such as patents, trademarks,
and copyrights, which are essential for monetizing
or protecting innovation. The importance of intellectual property was ofcially recognized in two
treaties, namely the Paris Convention for the
Protection of Industrial Property (1883) and the
Berne Convention for the Protection of Literary
and Artistic Works (1886). Today, they are administered by the World Intellectual Property
Organization (WIPO). These conventions recognized several reasons why intellectual property
rights are vital to progress, and in today’s world of
quickly evolving technologies they are of increasing importance. Further progress of humanity
requires the capacity to create and invent new
works in the areas of technology and culture. Only
legal protection, through its options for recognition or nancial benet, can sufciently encourage
the pursuit of innovation − which in turn spurs
economic growth, creates new jobs, industries,
enriches our cultural lives, enables us to lead
healthier lives with increased life expectancy, and
enhances the general quality of life [4].
From the perspective of the innovator, intel-
lectual property rights offer many advantages:
1. They give control over a creation’s commer-
cial production, use, distribution, or sale.
2. They ensure that a superior product or process
cannot be copied by competitors.
3. They can be sold or licensed, and thus generate revenue.
4. They are proof of a pioneering role in
science.
5. They can be an essential part of marketing.
6. They are assets of economic value and can be
used as collateral.
Intellectual property falls into two groups.
First, types of IP that are under automatic protection and for which the author automatically has
the copyright. This category consists of original
literary and artistic works including photography,
lm and music, as well as design rights to threedimensional products. Then there is the second
group of intellectual property, which does not
come with automatic legal protection. This group
consists of inventions, novel products, or innovative processes for which patent rights must rst
be obtained.
Obviously, the results of medical research fall
into this latter category of intellectual property,
which has to be protected by patents. Medical
inventions, including incremental inventions,
rst require patenting for the scientist to acquire
the intellectual property rights to his or her creation [5]. Patents are intellectual property rights
enforceable in court, which give a patent owner
the right to decide on the use of the patented
invention. Filing patents is the logical choice for
commercial drug developers, who need the exclusive rights to sell a new drug at least for some
years after it enters the market to compensate for
the costly development. Once their strictly condential research on a potential new drug shows
enough promise and for security reasons, this
decision often happens in early stages such as
lead structure development but can also be postponed until the results of pharmacological and
toxicological tests. The strategy here is to le a
patent on the drug structure and potentially also
its synthesis route. Most countries follow the
legal concept called rst-to-le (FTF), which
means that the right to the grant of a patent for a
given invention lies with the rst person to le a
patent application for protection of that invention, regardless of the date of actual invention.
Given that trends exist not only in corporate but
21 Moving Your Results fromBench toBedside: Protecting Scientic Findings
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223
also in the context of scientic research, not only
is there competition between different scientists
to come up with a suitable solution to the same
problem, this race further extends to the subsequent ling of a patent, which is necessary to
secure the rights of the inventor. This means that
the ultimate nancial benet will be with that
party who ultimately manages to le the patent
rst, not necessarily with the one who comes up
with the solution rst [6]. The downside of ling
patents early on is a reduced time for the exclusive marketing rights of the fully-developed drug,
which has to do with the fact of patents usually
having a limited lifespan of 20years. If a drug is
patented during its early development and may
require another 7–15years to reach the market,
then a pharmaceutical company would be left
with only 5–13years to make a prot on its new
product, which often explains the high costs of
new drugs [7].
21.3 Defensive Publications Oer
Freedom toOperate
Medical research typically aims at nding solutions to complex medical problems in the form of
innovative inventions or signicant improvements to the status quo. Once the scientist has
come up with a successful and unique solution,
his or her decisions in the context of establishing
proof of authorship and the publication of his or
her invention will have severe implications in the
future. Depending on the intentions and means of
the inventor, he or she must decide between different options.
There are circumstances when patents are not
practical for protecting intellectual property that
results from scientic research. Filing a patent
typically tends to be very expensive - primarily
because of the complexity of inventions and the
abundance of prior art in the respective eld of
science resulting in complex application documents, which usually must be prepared by an
experienced lawyer to ensure that a patent has a
greater chance of being successfully granted.
Consequently, attorney fees can easily cost several tens of thousands of dollars in addition to
the patent ofce ling fees. Considering that a
patent will need to be led for every single country in which the innovator wishes to posses IP
rights, big companies have a major advantage
over private innovators, who often lack the necessary funds to secure the global rights to potential high- revenue products with long-term
marketability. The high costs of pursuing
national or international patent rights may prevent private innovators from going this route
altogether, while commercial inventors usually
have the necessary experience and funds to do
so. In some cases, however, the benets afforded
from patent monopoly rights are not sufcient to
justify the cost of obtaining a patent. Either
because the products lack long-term marketability or, and this happens frequently in the corporate environment, because the patent holder
already has rights to another product that he or
she only seeks to protect against the possibility
of others patenting a technology that may make
the product redundant. In other cases, the high
costs of patenting may outweigh the benets
afforded by patent rights, irrelevant of the innovator’s nancial background.
Occasionally, e.g., in the case of academic
research, when commercialization is not a priority or its potential not even recognized, obtaining
intellectual property rights may not even be
desired. However, the innovator might still be
interested in retaining his or her freedom to operate by preventing others from ling a patent. This
regularly happens in academic settings, where
continued freedom to operate and to further rene
a technology might be of more interest than
nancial benets. Instead, the innovator may
simply wish to secure the right to continue using
and enhancing a product or technology to further
research in his respective eld. He or she might
also pursue altruistic goals, as seen in open source
projects, by wishing to put his or her invention
into the public domain to make it freely available
and prevent others from ling a patent.
Patents may be essential for incentivizing continuous scientic effort through their importance
for eventual monetization but the costs associated
with obtaining patent rights often prevent innovators from pursuing them, and some scenarios
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D. Thor
prot from a different course of action than patent application. This alternative comes in the
form of defensive publication, which is the intentional and purposeful publication of an innovation. As a cost-effective intellectual property
strategy, it consists of disclosing aspects of an
invention in a way that ensures that the invention
gets the status of prior art, thus precluding others
from obtaining a patent on the innovator’s idea.
Since the novelty of an idea is an essential precondition for obtaining a patent, the publication
of an idea can be strategically used as an IP strategy, if the previous publication date can be
proven in court and a patent ofce can readily
obtain knowledge of existing prior art [8].
Current venues for publishing defensive publications range from traditional peer-reviewed
journals to online publications. Publication in
peer-reviewed journals, as desirable as it may be
from a scientic point of view due to the additional validation by the journal, cannot be easily
or quickly obtained. Submitted articles frequently
take months to get published and the editors’ criteria for the selection of content are associated
with the possibility of eventual rejection by the
journal. While academics learned to live with
these disadvantages, this option renders too time
consuming and not suitable for most private innovators or corporate researchers, as journals also
typically avoid any content that could be interpreted as marketing. Unfortunately, many other
forms of publication such as web sites, social networks, fairs, public demonstrations, or tradeshows are however insufcient proof in the eyes
of courts, and are therefore not suitable for defensive publication. Neither would such methods of
publication offer a guarantee that the published
invention would be successfully found, identied, and considered by patent examiners and patent ofces, as they are not listed in readily
searchable databases. Therefore, a scientist
should carefully consider his or her chosen venue
for defensive publication and also consider all
possible implications of such an act.
In many countries, the inventor might lose
the right to le a patent following publication,
as it turns an invention into prior art. The
European Patent Ofce for instance regards
early disclosure an absolute bar to an EPO patent. However, other important markets such as
the United States, Russian Federation, Japan,
Canada, South Korea, Australia, Brazil,
Argentina, Malaysia, Mexico, and several others use the so-called rst- inventor- to-le system
(FITF). Following this system, the United States
Patent and Trademark Ofce (USPTO) and
other patent ofces afford early disclosers a
‘grace’ period until they need to le a patent.
This grace period starts at the time of publication and typically lasts 6–12 months during
which the inventor can still le a patent, but this
does not prevent someone else to le a patent
for the same idea during that time [9].
21.4 Trade Secrets asaCommon
Alternative
With patents being frequently too expensive for
academic researchers and defensive publications prohibiting the later exclusive rights to a
new drug or medical product, valuable information that can provide a competitive advantage is
instead often kept strictly condential, as a socalled trade secret. Unfortunately, holders of
such intellectual property are often exposed to
misappropriation (the intentional and illegal
use) of their trade secrets. While large companies possess greater resources to protect their
intellectual property, including costly patents
and funds for legally enforcing their rights,
smaller companies, in comparison, do not have
such nancial means. Consequently, smaller
companies rely on trade secrets to an even
greater extent than large companies. The problem of misappropriation has increased so much
that political institutions are now seeking new
ways to prevent misappropriation. The European
Commission has urged the European Parliament
to standardize the existing divergent national
laws against the unlawful acquisition, disclosure, and use of trade secrets. Such harmonization will give victims of trade secret
misappropriation more protection and the means
21 Moving Your Results fromBench toBedside: Protecting Scientic Findings
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225
to stop unlawful use and further disclosure of
misappropriated trade secrets, as well as the
right to compensation for any damages caused
[10]. Thus, the EU Directive on the Protection
of Trade Secrets will provide a legal framework
to discourage unfair competition, and facilitate
collaborative innovation and the sharing of valuable know-how. EU countries must bring into
force the laws and administrative provisions
necessary to comply with the Directive by June
2018. New and improved legislation to better
protect trade secrets is not, however, limited to
the EU.In fact, the Defend Trade Secrets Act of
2016 (DTSA) is a United States federal law that
allows an owner of a trade secret to sue in federal court when its trade secrets have been misappropriated [11]. International efforts by the
World Trade Organization to address this problem led to the conclusion of the Agreement on
Trade-related Aspects of Intellectual Property
Rights (the TRIPS Agreement) [12]. What all of
these regulatory efforts have in common is the
vital condition that companies must prove that a
piece of information, the trade secret, has deliberately been kept secret, for them to be able
claim protection under trade secret laws.
Needless to say that academic researchers working in a university environment may nd it much
more difcult to keep such information condential than scientists working in a strictly corporate environment.
21.5 The Right toFiling IP Rights
So far this analysis of ways to protect inventions
in the academic (or corporate) environment highlighted different reasons for choosing a specic
path, often linked to its economic and organizational aspects (Table 21.1). Complexity, costs,
and revenue potential all have an important
impact on the practical suitability of each path or
instrument for protecting new innovations and
ultimately decide whether such actions should be
taken. It should be noted that in many cases the
decision however does not lie with the scientist or
inventor. Both in academic and corporate
research, the contractual agreement between the
employer and the employee doing specic
research typically stipulates that any rights to
new developments belong to the employer- may
that be a university, a research center, or a pharmaceutical company. This also extends to any
research done in the spare time of the employee if
the company can prove that any of its assets or
intellectual property have been involved and/or
used or if there is any resemblance or relation to
the scientist’s work or the company’s other activities. Even completely unrelated scientic work
has to be very carefully kept separate from any
professional endeavors. Patent applications for
inventions developed in his or her professional
capacity may be led in the scientist’s name but
will identify the employer of said researcher as
the holder of the patent rights. The failure to
make any such potentially useful information
known to the company or university could lead to
the termination of the employment agreement
and likely be linked to risks such as claims for
both compensatory damages and punitive
damages.
In the context of academic research, the right
of the institution to new intellectual property
often even transcends contractual agreements
between commercial sponsors and the individual
research unit. To avoid any disputes regarding the
ownership of IP rights deriving from research
projects sponsored by or involving third parties,
such as pharmaceutical companies, the contract
has to consider all applicable rules and laws.
Table 21.1 Comparison of Forms of IP protection
Patent Yes High Very high 20years
Defensive publication No Low Medium Unlimited
Trade-secret Yes None Non Potentially unlimited
Monetization Costs Complexity Duration
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Fig. 21.1 Timing of IP Protection in Pharmaceutical Research
D. Thor
21.6 The Right Time toDecide
21.1)
(Fig.
Protecting the results of scientic research at an
early stage has been proven to be the best paradigm, if said research has scientic or commercial potential. Even if monetization and
monopolization are not desired, retaining freedom to operate is often the decisive prerequisite
for complex scientic undertakings. To decide on
the best course of action, the scientist will however have to familiarize himself or herself with
the legal consequences of his actions, the available methods of protecting his or her interests,
and has to be capable of making qualied
assumptions as to costs of such legal steps and
the commercial potential of his invention.
Needless to say, these decisions place a lot of
responsibility on the shoulders of someone with
often very little practical experience in such matters. Having internal or external expert advisors
involved in the ongoing research efforts should
therefore be regarded as essential.
References
1. Anderson AC. The process of structure-based drug
design. Chem Biol. 2003;20(9):787–97.
2. Hirako M, McAuslane N, Salek S.A comparison of
the drug review process at ve international regulatory agencies. Therapeut Innovation Reg Sci.
2007;41(3):291–308.
3. Bently PJ, Gulbrandsen M, Kyvik S.The relationship
between basic and applied research in universities.
High Educ. 2015;70(4):689–709.
4. Schwertner HA. Patenting medical inventions. Mil
Med. 1994;159(12):729–32.
5. Anderson MH, Cislo D, Saavedra J, Cameron K.Why
international inventors might want to consider ling their rst patent application at the United States
Patent Ofce & the convergence of patent harmonization and e-commerce. Santa Clara High Technol Law
J. 2013;30(4):555–77.
6. Torjesen I.Drug development: the journey of a medi-
cine from lab to shelf. Pharm J. 2015;. online URI:
20068196
7. Barrett B.Defensive use of publications in an intellec-
tual property strategy. Nat Biotechnol. 2002;20:191–3.
8. Horton R. Helping or hindering the genius of
America?: e conict in congress surrounding the rstinventor-to-le and revised grace period and the resulting impact. Santa Clara Law Rev. 2015;55(3):730–67.
21 Moving Your Results fromBench toBedside: Protecting Scientic Findings
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227
9. Directive (EU) 2016/943 of the European Parliament
and of the Council on the protection of undisclosed
know-how and business information (trade secrets)
against their unlawful acquisition, use and disclosure
Trade Secrets.
intellectual-property/trade-secrets_de
Feb 2018.
10. Cohen BA, Renaud MT, Armington MW.Explaining
the defend trade secrets act business law today.
http://ec.europa.eu/growth/industry/
. Accessed 28
2016.
blt/2016/09/03_cohen.html
11. World Intellectual Property Organization. WIPO
Intellectual Property Handbook: Policy, law and use.
Geneva: WIPO; 2004.
12. World Trade Organization. Trade-Related Aspects
of Intellectual Property Rights. 1994
wto.org/english/docs_e/legal_e/27-trips_01_e.htm
Accessed 15 Feb 2018.
https://www.americanbar.org/publications/
. Accessed 25 Feb 2018.
https://www.
.
The Regulatory Landscape ofCell-
https://t.me/medicina_free
andTissue-Based Regenerative
Medicine: Current Challenges
andEmerging Issues
SarahKhalidAlageel, MartinHildebrandt,
andAnaValériaGouveiade Andrade
22
22.1 Introduction
Rather than managing sustained disease or damage, the eld of Regenerative Medicine (RM) is
aimed at restoring or establishing normal function by replacing or regenerating human cells,
tissues, or organs [1]. As a subdivision of translational research in molecular and cell biology, biomaterial science, and organ and tissue engineering
[2], RM holds great promise in addressing the
S. K. Alageel
Department for Plastic and Hand Surgery, Klinikum
rechts der Isar, Technical University of Munich,
Munich, Germany
TUM Cells Interdisciplinary Center for Cellular
Therapies, TUM School of Medicine, Klinikum
rechts der Isar, Technical University of Munich,
Munich, Germany
e-mail: Sarah.Alageel@mri.tum.de
M. Hildebrandt
TUM Cells Interdisciplinary Center for Cellular
Therapies, TUM School of Medicine, Klinikum
rechts der Isar, Technical University of Munich,
Munich, Germany
Institute of Clinical Chemistry and
Pathobiochemistry, Klinikum rechts der Isar,
Technical University of Munich,
Munich, Germany
e-mail: Martin.Hildebrandt@mri.tum.de
A. V. G. de Andrade (*)
TUM Cells Interdisciplinary Center for Cellular
Therapies, TUM School of Medicine, Klinikum
rechts der Isar, Technical University of Munich,
Munich, Germany
e-mail: ana.andrade@mri.tum.de
global lack of organ supply, aging-related diseases, and congenital or acquired defects by
either actively reconstructing de novo organs [1]
and/or tissues or functionally healing previously
irreparable tissues or organs by stimulating the
body’s own repair mechanisms [1].
Under this umbrella, researchers have been
working vigorously on the development and
bench to bedside translation of a variety of innovative therapeutic products such as: human cell
and tissue products, tissue engineered therapeutic
products, gene therapy products, and combined
products. However, despite the continuous
advances in science and technology paving the
way in the development of Regenerative Medicine
Therapeutics (RMT), to date, only few products
have been authorized for marketing in the United
States (US) and the European Union (EU).
To better understand how the EU and the US
manage the development and manufacture of RM
products, details regarding the regulatory process
from the rst step of classication until market
approval will be addressed here.
22.2 EU andUS: Dierent
Approaches When it Comes
toMedicinal Products
In the EU, the evaluation and regulation of the
translation and marketing of RMTs is overseen
by the European Medicines Agency (EMA).
© Springer Nature Switzerland AG 2019
D. Duscher, M. A. Shiffman (eds.), Regenerative Medicine and Plastic Surgery,
https://doi.org/10.1007/978-3-030-19958-6_22
229
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