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					        Annex 2.3.A: Priority medicines for Europe and the World

Project Outline

Priority medicines for Europe and the world:
a public health approach to innovation
World Health Organization, Department of Essential Drugs and Medicines Policy, Geneva

Background

For a number of diseases, no effective and safe medicinal treatment is yet available (e.g.
HIV/AIDS, Alzheimer’s and several cancers). For some of these, potentially large markets
(e.g. breast cancer) exist, and associated pharmaceutical research is likely to be intensive.

For other diseases, the number of patients is low (e.g. cystic fibrosis) or the purchasing
power of those suffering from the diseases concerned is low (e.g. tuberculosis, malaria). Since
most drug research and development (R&D) is market-driven, R&D in such areas is not of
great commercial interest despite the large numbers of patients, and is therefore rarely
undertaken. For example, of about 1400 new products developed and marketed in the last 25
years, only three medicines were developed for tuberculosis.

Additionally, for some categories of patients (e.g. children, the elderly, pregnant women)
little research on safety and efficacy is carried out, and as a result, new medicines are not
specifically tested, licensed and marketed for these categories. This often leads to medicines
being used off-license, and an absence of adapted dosage forms.

Public funding for R&D, in relation to preventive and curative gaps with considerable public
health impact, should therefore be considered. In addition to existing mechanisms for
orphan drugs, and the new regulation for medicines for children, the European Union (EU)
has other opportunities for supporting innovation through targeted research funding –
namely through its 7th European Framework Programme for 20072010. Other possibilities
for supporting clinical trials include those available through the European Developing
Countries Clinical Trial Partnership, and product development through the European
Investment Bank.

The appropriate use of public funds for R&D calls for careful and transparent prioritization
of treatment needs, on the basis of sound epidemiological information, clear public health
criteria and wide consultation. The Government of the Netherlands will hold the EU
presidency in the second half of 2004 and has requested the World Health Organization
(WHO) to prepare such a research agenda on public health grounds. The current project
proposal, "A public health approach to innovation", is made in response to this request.

Objective of the project

The objective of the project is to prepare a public-health-based medicines development
agenda, for support by the EU in the immediate (20052006) and medium-term (20072010)
future, and to develop a systematic methodology in this regard.

Within the context of tackling preventive and therapeutic gaps for the citizens of Europe,
special emphasis will be put on identifying those research needs which are also relevant for
countries in economic transition (including several new EU members) and, if possible, also
for developing countries – so that a maximum benefit may be derived from a "duality of
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         Annex 2.3.A: Priority medicines for Europe and the World

interest". In addition, needs regarding better delivery mechanisms/formulations of existing
preventive and therapeutic medicines, will be identified. For example, the lack of paediatric
dosage forms for some essential medicines is recognized both as a European and a global
problem.

Time limitations within the project will make it necessary to narrow the project’s focus.
Emphasis will be put on infectious diseases, medicines for certain patient groups such as
children, and medicines for rare and/or neglected diseases. However, in the course of the
project, other needs relating to chronic or noncommunicable diseases may also be identified.
This initial focus implies that more work will be needed at a later stage. For this reason, the
project will also develop recommendations on a procedure for priority setting that could
perhaps continue to be used by the EU.

Anticipated output

   A methodology for defining research priorities on the basis of public health
    considerations.
   A first list of conditions for which public investment in drug development is
    recommended, on the basis of dual interest, with supporting public health justification
    including, if possible, a pharmacoeconomic analysis of potential benefits.

Strategy and outline of the project

Outline of proposed activities

Phase 1: Development of methodology for prioritization; inventory of potential gaps in
pharmacological treatment (August  November 2003)
Consultations with:
 research organizations (WHO/EIP, GAVI, TDR, MSF, ZonMw, EU institutions)
 professional associations with close patient contacts (general practitioners, pharmacists)
 clinical guidelines development groups and networks (Guidelines International Network
    and others)
 networks of organizations active in evidence-based medicines (Cochrane and others)
 European health insurance and health maintenance organizations; public funders
 international non-governmental organizations, consumer groups and patient
    representatives (Health Action International, International Organization of Consumers
    Unions (known as Consumers International), MSF, etc.)
 WHO (CDS, EIP, FCH, NMH, other disease programmes, regional offices)
Definition on methodology for priority setting; first draft shared with global research
institutions, European and national research councils.

Phase I:
The strategy for developing this research agenda was to first develop a draft methodology
for the prioritization process. The starting point is the experiences of other initiatives that
have tried to prioritize research needs, such as the Global Forum for Health Research, the
Global Alliance for Vaccines and Immunization (GAVI), Tropical Disease Research (TDR –
with WHO, United Nations Development Programme and the World Bank) and the Drugs
for Neglected Diseases programme of Médecins sans Frontières (MSF). Through the
Netherlands Organisation for Health Research and Development (ZonMw), a number of
European research councils will also be involved in an early stage, so that the project can
benefit from technical advice, and also to ensure shared ownership of the research agenda.


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        Annex 2.3.A: Priority medicines for Europe and the World

A first inventory of gaps in pharmacological treatment is being made through global
consultation, initially focusing on: medicines for treating acute and chronic diseases;
medicines for specific groups such as children, the elderly, and pregnant and lactating
women; and medicines to treat rare and/or neglected diseases. Within WHO, consultations
have taken place with relevant clusters such as Evidence and Information for Policy (EIP),
Family and Child Health (FCH), Communicable Diseases (CDS), and Non-communicable
Diseases and Mental Health (NMH). Outside WHO, specific contacts will be made with
professional and patient organizations, research networks and clinical guidelines networks.

A preliminary prioritisation method has been developed at WHO which uses the Cochrane
Database of Controlled Trials. For medicines needed to treat various diseases found in the
Cochrane Database, we calculate the Number Needed to Treat (NNT) as an estimate of the
present clinical benefit for that particular medicine and the Number Needed to Harm (NNH)
as an estimate of the clinical risk associated with that particular medicine. This allows us to
estimate the clinical efficacy of medicines and gives us some indication of the balance of
benefit and harm for various pharmaceutical formulations that are intended to treat certain
conditions.

Our Dutch partners have specifically requested drug industry input on the methodology. We
have presented our current view of the methodology to a senior executive at Pfizer, Ltd., Dr.
Gill Samuels and plan more extensive consultations. The pharmaceutical industry, as part of
understanding “unmet medical need” as a rationale for R&D programs, also needs to
understand the balance of benefit and harm for various pharmaceutical formulations that
treat diseases in various patient populations. It may well be that the Priority Medicines
Project methods and those of the pharmaceutical industry are complimentary and this will
be investigated.

Public health can be impacted by improving the therapeutic value of existing medicines as
well as improving the USE of existing medicines. Existing medicines are often not used
properly- in part because of “downstream” access issues but also because the medicines in
their present formulations may be inappropriately formulated and/or delivered. We would
like Phase I to be comprehensive enough so that it prompts questions as to HOW one goes
about moving a drug from a zone of “low benefit, high harm” to one that is, on balance,
more beneficial than not. Several options exist: one could create a new molecule de novo;
investigate further the disease mechanism; eliminate those medicines that are not effective,
or modify an existing molecule and make it better (new formulation, new delivery system).

Phase 2: Collecting evidence and information for prioritization (October 2003  July 2004)
Detailed review of evidence through research, collection of statistical information,
commissioned systematic reviews, further review with other international organizations:
 review burden of disease
 review potential public health impact
 review pharmacoeconomic aspects, if possible
 review current R&D agenda in these areas (study anatomical therapeutic chemical
   classification system/defined daily dose registers, applications for international
   nonproprietary names during the last 10 years, regulatory bodies)
 review potential barriers to drug development (consultation with EU Research Councils
   and pharmaceutical industry)
 interim report by March 2004




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         Annex 2.3.A: Priority medicines for Europe and the World

Phase II:
The second phase can start when Phase 1 is not yet fully completed. The process of
prioritization in Phase 2 will probably involve: a review of potential public health impact in
industrialized, transitional and developing countries; a review of the current R&D pipeline
in these areas; a review of the reasons why certain research topics are not being pursued;
further international consultation on a proposed "short-list" of priorities; and, if possible.
pharmacoeconomic analysis of selected topics. The consultations will include research
councils, countries of the EU, Eastern and Central Europe, other countries in economic
transition, and, whenever feasible, some representatives of developing countries. Some
targeted reviews may be contracted to relevant groups.

The Netherlands Organisation for Health Research and Development (ZonMw) will make an
inventory of national research programmes on priority medicines in approximately 10
European countries, including at least one of the new EU member states. Based on this
inventory, possible means of cooperation at the European level will be explored. The results
of the WHO analysis will provide important input for this project in identifying priority
areas based on societal needs. An interim report will be made available in March 2004.

Phase 3: Preparation of final list of research recommendations and final methodology for
further prioritizations (August  December 2004)
 Publication on WHO web site; invitation for comments; global electronic consultation
 Discuss, review and finalize the research agenda (WHO and Research Councils)
 Discuss, review and finalize the prioritization methodology for future use
 Presentation at EU Conference in The Hague (18 November 2004)
 Presentation at Global Health Research Summit in Mexico (19-21 November 2004)
 Preparation of final report

In the third phase of the project, the draft methodology and the final list of research priorities
will be further reviewed, discussed and prepared for publication and presentation.




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