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WHO Report
Priority Medicines for Europe and the World
EFPIA Comments
Summary
The purpose of the Priority Medicines for Europe and the World Project has been to study
pharmaceutical innovation from a public health perspective and address pharmaceutical
gaps by shaping a European research agenda in the context of EU’s Framework Research
Programmes.
The resulting report is an overall positive document, the WHO has gone through an
extensive stakeholder consultation to ensure that all relevant parties have been heard.
The report has a very diverse content, it includes a reasonable list of 16 priority diseases
demonstrating pharmaceutical gaps, statements about the need for more fixed dose
combinations, more basic research on ageing, more safety and efficacy data in children and
finally more research on female health. The basic idea of priority setting is acceptable from a
public health point of view and EFPIA has no objection to the prioritised diseases, it is
however important to emphasize that most pharmaceutical gaps are due to scientific
challenges rather than economic ones, and that industry is in fact active within the areas of
the priority diseases.
Barriers to Innovation
Section 8 of the report reviews various approaches to promoting innovation in Europe;
EFPIA’s Research Directors Group has endorsed the so-called Middleton paper on
barriers to innovation and their possible solutions.
However, it is important to single out the case made for differential pricing, the suggested
approach described in the report is based on multiplying increased life expectancy with
gross national income per capita (GNI). This approach is highly questionable because:
 Life prolongation expressed in life years is a very subjective criterion for
quantification of medical benefit. The measure and assessment would be very
difficult and vary significantly from country to country.
 Medical benefit provided by a new medicine has no relation with the GNI level. It
is a totally artificial cost effectiveness calculation.

This methodology leads to very high price differentiation between countries,
including countries with in the same economic zone. This price differentiation
will lead to increased parallel trade and to the appearance of counterfeit medicines
in the high price countries. The authors wrote “it would be perfectly possible to
segment the pharmaceutical market to have pricing set according to national
income and efficacy”; experience in Europe and in the USA has showed that this is
not the case.
EFPIA appreciates that the report acknowledges that there has been objections from many
sides to this approach and that it concludes that the topic should be further researched
and debated.
Additionally EFPIA wants to stress that promoting innovation requires rewarding
incremental innovation. Most payers and policy makers fail to take into account the
importance of incremental advances in both the near and the longer term. Defining
“innovation,” particularly in drug treatment, has created heated controversy for some time.
Medical breakthroughs and drugs that are “first-in-class” are accepted as “innovation” while
“me-too” products or line extensions usually are not. How clinical progress actually occurs,
this being in a series of small steps, is poorly understood: medical breakthroughs are the
exception rather than the rule, the nature of medical progress is step-by-step progress and
future innovation can only be funded if incremental innovation is rewarded.
The cumulative benefits of incremental innovation are underestimated. Medical advance is
only occasionally a "big bang" process, and even then depends on accumulated knowledge: a
series of minor advances add up to major progress over time. Also often missed in
discussions of innovation is the importance of new indications for marketed drugs. The new
use may be wholly unrelated to the initial indication and represent a significant innovation
in that area.
Several potential nearer-term therapeutic and economic benefits from such incremental
advances also tend to be overlooked. Any decrease in the severity, nature or frequency of
side effects and any improvement of dosing schedules or better delivery systems enhance
patient compliance and reduce the costs of treating and monitoring side effects. These
incremental therapeutic advances also contribute to the creation of a larger body of medical
and economic data about the entire class of medicines, which altogether improve treatment
and enhance economic decision-making.
European Technology Platform
The report expresses support for the concept of European Technology Platforms, EFPIA’s
Research Directors Group is currently working on a Strategic Research Agenda in the
context of a European Technology Platform with the purpose of addressing barriers to
innovation across the R&D process and improving access to medicines across Europe. The
Research Directors Group has identified main pre-competitive barriers to innovation,
around which industry and stakeholders in the drug development process can collaborate
to achieve a first class environment for R&D. The barriers are focused on improved
prediction of safety and efficacy in the drug development process. The implementation of
this Strategic Research Agenda would impact positively patients’ access to innovative
medicines.
10 November 2004
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