The World Health Organization (WHO) today releases a groundbreaking report which recommends ways in which pharmaceutical research and innovation can best address health needs and emerging threats in Europe and the world.
Priority Medicines for Europe and the World, commissioned by the Dutch Government as current president of the European Union (EU), identifies a priority list of medicines for Europe and the rest of the world, taking into account Europe's ageing population, the increasing burden of non-communicable illnesses in developing countries and diseases which persist in spite of the availability of effective treatments. The report looks at the gaps in research and innovation for these medicines and provides specific policy recommendations on creating incentives and closing those gaps.
At present, pharmaceutical research and development are based on a market-driven incentive system relying primarily on patents and protected pricing as a prime financing mechanism. As a result, a number of health needs are left unaddressed.
The report identifies gaps for diseases for which treatments do not exist, are inadequate or are not reaching patients. Threats to public health such as antibacterial resistance or pandemic influenza, for which present treatments or preventive measures are unlikely to be effective in the future, also require immediate action.
"This report identifies health gaps and potential solutions. It is particularly timely for a continent where an ageing population faces increasing health problems, and for a world where old and new threats no longer respect national borders," said Dr. LEE Jong-wook, Director-General of WHO from the Ministerial Summit on Health Research, taking place in Mexico this week. In addition, the report addresses obstacles where effective medicines could be better delivered to the patient. It emphasizes fixed dose combination medicines (medicines which include more than one active ingredient in one pill) as worthy of further research and development. Finally, it looks at particular groups such as children, women and the elderly, who have frequently been neglected in the scientific or medicine development process.
The 17 priority conditions identified by the report are:
Future public health threats: infections due to antibacterial resistance; pandemic influenza;
Diseases for which better formulations are required: cardiovascular disease (secondary prevention); diabetes; postpartum haemorrhage, paediatric HIV/AIDS, depression in the elderly and adolescents;
Diseases for which biomarkers are absent: Alzheimer disease; osteoarthritis;
Diseases for which basic and applied research is required: cancer; acute stroke;
Neglected diseases or areas: tuberculosis; malaria and other tropical infectious diseases such as trypanosomiasis, leishmaniasis and Buruli ulcer, HIV vaccine;
Diseases for which prevention is particularly effective: chronic obstructive pulmonary disease including smoking cessation; alcohol use disorders: alcoholic liver diseases and alcohol dependency.
The report suggests that Europe can and should play a global leadership role in public health, as reflected by its history of social services provision and social safety nets for all citizens. In many developing countries, the poor are increasingly affected by the chronic diseases that are widespread in Europe, including cardiovascular disease, diabetes, tobacco-related diseases and mental illnesses such as depression. Moreover, the ten countries that joined the EU in 2004 have additional public health challenges.
For a number of diseases that affect people in all members of the EU, no effective and safe medicinal treatment is yet available (e.g. Alzheimer disease and several cancers). For some diseases, potentially large markets exist for medicines (e.g. breast cancer) and associated pharmaceutical research is likely to be intensive for certain therapeutic classes. For other categories of medicines, the number of patients is low (e.g. cystic fibrosis) or the market-driven pharmaceutical industry has failed to pursue research and development (e.g., new medicines for tuberculosis).
The report suggests that efforts to shorten the medicine development process without compromising patient safety would greatly assist in promoting pharmaceutical innovation. For instance, the EU could create and support a broad research agenda through which the European Agency for Evaluating Medicines (EMEA), national regulatory authorities, scientists, industry and the public would critically review the regulatory requirements within the medicine development process for their relevance, costing, and predictive value.
Health authorities are responsible for medicines reimbursement decisions that aim to ensure safe and effective treatment for all patients, while reconciling this with budgetary constraints. Health and reimbursement authorities and manufacturers should agree on general principles for the evaluation of future medicines. For example, the EU Commission and national authorities should support a research agenda on the various methods of rewarding clinical performance and linking prices to national income levels. The report authors believe that these measures will help encourage industry to invest in the discovery of innovative medicines that address priority health care needs.
The report maintains that where the market is strong and the problem is poor understanding of the basic biology of the disease, investment in basic research and in facilitating innovation by the pharmaceutical industry will be needed. Where the biology is well understood but the market is weak, public support for breaching the gap between basic and clinical research ? possibly through public-private partnerships and other not-for-profit product development initiatives ? will be the preferred solution. Where the biology is not well understood and there is also a weak market, then biological research can be supported while market incentives are created for the pharmaceutical industry, through reducing barriers to innovation and through improving reimbursement rewards.
The report points out that major pharmaceutical gaps have been closed in the past. For example, until 1975 the main treatment for severe peptic ulcer - a common ailment - was surgery. Following a long period of focused research in biological mechanisms underlying ulcer disease, effective medical treatments were discovered. These breakthrough discoveries, combined with the discovery that most ulceration was caused by a bacteria treatable with antibiotics, made surgery unnecessary.
The recommendations contained in the report could have a significant impact on research innovation and policy, with support from European leaders. The report will be discussed at a High Level Meeting in the Hague on November 18th 2004.