With the help of health experts the WHO report identified “pharmaceutical gaps”: diseases of public health importance for which pharmaceutical treatments either do not exist or are inadequate. The report provides a public-health-based medicines development agenda, based on a systematic methodology for priority setting. National and European policies relevant to the European pharmaceutical and biotechnological industries will be influenced by the report’s recommendations. It will also provide important input to health-related policy development and research & innovation, including international cooperation.
What are the priorities?
The report is an update to the original 2004 Report Priority Medicines for Europe and the World and takes into account changes in global health and pharmaceutical innovation since 2004 in order to better address current and future patient needs.
Special emphasis was placed on identifying research needs which are relevant both within and outside of Europe. Since the last report in 2004 the burden of disease has been converging, and more commonalities have emerged between Europe and the rest of world.
While progress has been made since 2004 in a number of areas towards effective treatments, efforts are still required in areas already identified in 2004. These include for instance Alzheimer disease, HIV/AIDS or antimicrobial resistance. Several health threats have also become more pressing over the last years, such as low back pain and pneumonia.
Twenty-four diseases and disease groups have been prioritised, and reasons for their inclusion are detailed in the report. Diseases were selected on the basis of burden of disease and mortality ranking, projections, social solidarity and risk factors. The following diseases were selected, based on:
- burden of disease and mortality ranking: ischaemic heart disease, diabetes, cancer, acute stroke, HIV/AIDS, tuberculosis, malaria, Alzheimer disease and other dementias, osteoarthritis, chronic obstructive pulmonary disease, alcohol use disorders and alcoholic liver disease, hearing loss, depression, diarrhoea, pneumonia, neonatal conditions and low back pain.
- projections: antimicrobial drug resistance, pandemic influenza.
- social solidarity: rare diseases, postpartum haemorrhage, neglected tropical diseases
- risk factors: tobacco use, obesity.
In addition the report addresses several cross-cutting themes. In order to reap the benefits expected from stratified medicine targeting subpopulations for greater treatment benefit or lesser treatment harm, several research gaps must be addressed in the coming years. The particular needs of children, elderly and women are underlined. These groups have specific pharmaceutical needs due to their evolving physiology and the spectrum of diseases affecting them. Moreover their needs are often under-represented in the pharmaceutical development process.
Importantly the report also considers approaches to promote innovation, such as public-private partnerships, regulatory structures, pricing and reimbursement practices, real-life data and learning from practice, and the involvement of patients and citizens.
Providing European support
The Commission supported the update of the Priority Medicines report by WHO, and granted financial assistance through a contribution agreement. Technical assistance was also provided by setting up an advisory group of stakeholders under the Process on Corporate Responsibility in the Field of Pharmaceuticals, launched in 2010 by Commission Vice-President Antonio Tajani. Stakeholders included Member States Competent Authorities in Pricing and Reimbursement, representatives of pharmaceutical industry, patients and social security authorities, in addition to World Health Organization (WHO) experts.
In 2004 the WHO published a report recommending ways in which pharmaceutical research and innovation could best address health needs and emerging threats in Europe and the world. This report notably provided a basis for health research topics in EU Framework Programmes for Research and Development. The creation of the Innovative Medicines Initiatives, a public-private partnership between the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA), responded to the report recommendation to use public-private partnerships more frequently.
The rapid evolution of knowledge and technologies in the field of health coupled with emerging health threats called for an update of the 2004 report. Prioritising resources allocation for pharmaceutical research to foster valuable innovations that address unmet health needs is crucial in times of economic constraints.