EuropaBio comments on WHO-Priority Medicine Project
15 September 2004
EuropaBio welcomes the opportunity which is offered to the biotechnology industry to
comment on the “WHO priority medicines project”.
EuropaBio, the European Association for Bioindustries, has 35 corporate members operating
worldwide and 24 National biotechnology associations representing some 1500 small and
medium sized enterprises (SMEs) involved in research and development, testing,
manufacturing and distribution of biotechnology products.
I. Background
Biotechnology is an important technology. Biotechnology and biological medicinal products
have produced breakthroughs in treatment for some of the most serious diseases.
Biotechnology is bringing new therapies to patients and helps to better understand the
underlying mechanisms of diseases.
In addition, biotechnology is playing an increasing role in drug discovery, even for
conventional medicines. By improving the knowledge underlying the cause of diseases,
biotechnology is also offering the potential to develop a more personalized and integrated
therapy to prevent, treat and cure previously life-threatening and serious genetic diseases,
many of them rare. And it opens new avenues to develop specifically designed drugs which
better target specific diseases with a greater efficacy and less side effects.
More than 325 million people worldwide have been helped by more than 155 biotechnology
drugs and vaccines available today. Pioneer companies provide safe, effective biologics and
use innovation to fight serious diseases and improve the quality of people's lives. Biotech
medicines now account for 20 % of all drugs on the market and for 50% of the ones in the
pipeline1.
In Europe alone, there are 1861 biotech companies2 who invest 6350 million Euro/year in
R&D and employ ca. 78.000 people.
1
Concerning the biotechnology medicines in development per therapeutic category, most of biotech medicines in the pipeline
are focusing on treating cancer (ca. 40%), followed by infections diseases (ca. 11%), autoimmune and neurologic disorders
(ca. 5%). Source: PhRMA Survey (2002)
2 Ernst and Young Global Biotechnology Report 2004. NB: Many large pharmaceutical companies – even if they have
significant proportion of their R&D activities dedicated to biotechnology -- are not considered as biotech companies and are
not taken into account here.
II. Specific comments
We understand that the WHO wants with this project to identify major diseases/healthcare
threats; assess whether robust treatments exist and, where not, if they are being developed
(i.e. pipeline development); and where nothing exists or is being developed, identify what
the public sector can do to stimulate/incentivise the necessary development of new
treatments/medicines (e.g. via public private partnerships).
However, the whole exercise is sometime very confusing. It is indeed important that policy
makers provide researchers with the tools necessary to develop health solutions which
answer public health priorities (e.g. 7th EU Research Framework Program and ad-hoc
Technology Platforms) and ensure market access for the result of their research. Therefore -if our society wants to reap the benefits of biotechnology for the research and development
of treatments, cures and diagnostic methods with the greatest public health impact -- the
following aspects must be taken into consideration by the WHO in its priority medicines
project:
From a medical perspective, the priority/classification setting must take into consideration:
 The epidemiology, i.e. look at the population (% relative and total) affected by the
disease. Need to differentiate between rare or a common disease, and between the
general population and targeted groups of patients.
 The morbidity/mortality within the general population and within the population of
patients affected by the disease. Is it a serious/disabling or life-threatening disease?
 The type of disease (e.g. acute or chronic), which also condition the clinical endpoints and symptoms to look at.
 The fact that carrying out such an exercise on global level is very difficult: indeed,
the major disease/healthcare risks are very different in the west and the developing
countries (cardiovascular disease versus malaria for example); therefore, the
incentives and approaches might have to be very different (i.e. pricing and
reimbursement and protection of innovation issues for diseases such as obesity,
cancer etc, versus direct government interventions/stimulations for diseases such as
malaria)
EuropaBio would welcome the inclusion of rare disorders in the scope of the revised WHO
report. Indeed, the therapeutic gap/need is clearly high in this field in Europe, and orphan
drugs account for a growing % of the newly approved biopharmaceuticals3. Also, it is
important to see the link between rare and common disease therapies through the research
since research into rare diseases often leads to improved knowledge and treatments for
common diseases.
In addition, there is a need for clarification about what is meant by priority:
 It is unclear from the title yet to be developed whether the WHO means to look at
priority medicines as those of higher priority between existing medicines, or wants
to look at the need for new medicines. The title should reflect this intention.
3 30 new biopharmaceuticals were approved in the US between 2000 and 2003 -- the largest number ever approved -- out of
which, orphan drugs account for 37% of the total! Source: Tufts Center for the Study of Drug Development, Regulatory Affairs
Journal, July 2004
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 Need to better define therapeutic gaps/needs. Therapeutic gaps in supply are
depending on how the therapeutic need is defined, and this is a moving target! e.g.
if we look at recent studies about prescription patterns and medical needs in
Europe, e.g. about cholesterol lowering in obesity as a growing need, but also a
lifestyle problem (same with smoking and lung cancer).
 Need to refine the classification and avoid confusion between symptoms and
causes. The disease categories identified by WHO are not science-based, as the
underlying causes of the disease may be different from the symptoms, e.g. in
cardiovascular diseases, the largest disease group indicated, and classification based
on cause may lead to very different, but scientifically more sound categories. The
current classification seems no longer acceptable in the current era of
pharmacogenetics for the purpose of public health policies as meant in the WHO
document.
 Need to include diagnosis/prognosis in the scope of the project. It is unclear, when
using this classification and when looking at advances in pharmacogenetics, why
WHO doesn’t include diagnostic and prognostic assays as they are more and more
linked to therapy supply. It is impossible to separate the question of which
medicines to be developed, from the diagnosis or prognosis of a disease. However,
there seems to be no reference to the need for accurate diagnosis, (or prevention for
that matter) in the document, except perhaps for TB.
From a R&D perspective, the priority setting must take into consideration the scientific
challenge and the level of knowledge about the disease. It is important to consider this when
looking at %chances of success for developing new or improved treatments, cures or
diagnosis.
 Need to recognize and reflect in the WHO project the value for innovation of
developing new pharmaceutical forms/strength/doses/routes of
administration/therapeutic indications of existing medicines.
 Need to recognize the current absence of a definition of what actually constitute
innovation and an innovative medicine, coupled with a diminishing tendency by
governments to reward and stimulate innovation
 Need to include the biotechnology tools/assays used in R&D in the scope of the
project. Those are necessary for the search of new medicines and for better
understanding the molecular basis of diseases, identifying the susceptibility of
patients to develop a disease or to respond positively to existing treatments, or
identify new biomarkers and surrogate end-points associated with a disease and
which need to be validated for evaluating safety/efficacy of drugs.
 Also, the scientific challenge is related to the lack of validated method of production
which would allow a given medicine to be produced in a controlled/GMP
environment and in sufficient therapeutic quantities. There is a need to consider this
aspect and to differentiate it from the access issues to existing medicines.
From a regulatory perspective, the priority setting must take into consideration the fact that
an enabling regulatory environment, a coherent approach and policy continuity, with
consideration for both the European and the global regulatory scene (worldwide
harmonization), are essential for healthcare biotechnology with its twenty year life-cycle of
innovation and investment. The following aspects are of importance:
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 Governments must work on a system for equitable and timely access to medicines
after EU approval. Lack of implementation or frequent changes in Member State
policy can delay access for patients to innovative medicines. Access and
reimbursement is important for patients, and represent a key factor in the ability of
SMEs to attract capital and funding. In turn, such access and related reimbursement
delays will also have an impact on the ability of companies to supply medicines for
developing countries.
 Faster access to medicines will be achieved only if the authorisation process remains
based on scientific assessment only (quality, safety and efficacy), without any
economic considerations. It is important that the primary driver for development of
health solutions should be a medical need, and not an economic need, although
there is always an economic component which has to be recognized. But especially
bodies such as the WHO should come from the societal or humanitarian angle, not
from the economic angle. It is necessary to clarify that the prioritization exercise will
not have a negative impact on the status of existing products.
 Uncertainty of the regulatory framework is key; e.g. controversy around animal
experimentation or research on embryonic stem cells. Policy makers have a
responsibility in ensuring the success for developing remedies or diagnostics for
threat to the public health which might occur tomorrow (e.g. what would be the
chances of success if such medicine would need to be tested on primates and if such
use is banned?) and entrepreneurs need to know which regulatory requirements
they have to follow.
 Novel biotechnology-based healthcare applications do not always fit existing
definitions or regulations. Is the product covered by the pharma legislation (EU, US,
worldwide)? What is the harmonisation status?
 Because of the technical complexity of biological medicinal products, scientific and
medical expertise may exist only in companies or academia, and the knowledge
about this type of product within regulatory authorities may be very low.
 The regulatory framework is often not adapted to scientific and technological
advances (e.g. validation of new biomarkers and surrogate end-points)
 The regulatory framework/authorisation procedure at EMEA is not yet fully
adapted to small and medium sized enterprises that are sponsoring those products.
 The regulatory incentives for the development of orphan drugs which currently
exist in certain regions and countries such as in Europe and in the US.
Finally, from an investor’s perspective, without sufficient protection for innovation, investors
would not be willing to take on the significant risk and financial investment required for
development of biotechnology and biotech products. This puts the development of new
medicines at stake.
 Investment is based on legal certainty. Delays in market access also can create
financial difficulties for SMEs and undermine investment which is based on a
predictable environment particularly in relation to intellectual property rights and
timely market authorisation for healthcare biotechnology products.
 Also, appropriate and consistent intellectual property and regulatory protection of
registration data are needed to ensure continued investment and innovation to meet
the needs of patients and society.
 Investment is also based on the scientific challenge. The chance of success is
inherently dependent on the acquired knowledge on a particular field and will
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condition the chance for a company to convince investors and get the funding it
needs.
Finally, there is a potential contradiction between the WHO list of priority and the way the
healthcare biotech community might look at research and market niche, and define their
priorities. It is essential for industry to be able to participate to the development of lists of
priority medicines, and to understand what would be the advantage of private investors to
follow the research agenda as proposed by the WHO (e.g. what is the WHO planning to
convince venture capitalists to invest in such EU research projects rather than others, and
why?)
EG. 15/09/2004
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