The positives of globalization

Globalization may be a major contributor to the ongoing international economic crisis, but it is also promoting international collaboration and uniting people around the world in common cause. A proof of this is Rare Disease Day 2009, which takes place on Saturday, 28th February, with an international participation of thousands of patients and an impact on awareness of rare diseases that will be immeasurable.

Illustrating the opportunity to raise awareness with policy makers, the European Union Commissioner for Health, Androulla Vassiliou, will host an event on Rare Disease Day to launch the publication of a book, The Voice of 12,000 patients -- Experiences and Expectations of Rare Disease Patients on Diagnosis and Care in Europe. This is one of dozens of events taking place across the world. (For information, go to www.rarediseaseday.org.)

Rare diseases by definition affect less than 1 in 2000 people. They are a diverse group of 6000 to 8000 conditions, but sufferers face common problems. The diseases are often chronic, progressive, degenerative, and life-threatening, causing a high level of pain and suffering for patients and their families. As many as 75% of rare diseases affect children, and there are no existing effective cures.



Last year on 29th February, a rare day, the first Rare Disease Day took place in Europe, organized by EURORDIS and 14 national alliances for rare diseases. This year the event is truly global, with patients and patient groups in the United States, Canada, Argentina, Australia and China joining in the action, bringing the event to a new level.

The participation of China is symbolically charged. This means that the plight of rare disease sufferers is reaching outside the Western world into a country that has become a major economic player and where patient organizations are mobilizing and identifying themselves with this movement in an encouraging sign of civil society's participation.

Things are moving swiftly in the United States. The National Organization for Rare Diseases (NORD) has pledged all of its forces to support Rare Disease Day, catalyzing the participation of more than 130 patient organizations and generating great enthusiasm on that side of the Atlantic. This is happening against a background of promising policy developments in the United States. Only a week after the inauguration of President Obama, the FDA approved the long-awaited decision to conduct the first clinical trials for treatments based on embryonic stem cells. In addition, the new US administration has announced plans for large-scale investment in health research.

The Institute of Medicine recently announced plans to conduct a major policy analysis, leading to a Report on Rare Diseases, over the next two years. Further, the National Institutes of Health (NIH) have established a new Undiagnosed Diseases Program, jointly organized by the National Human Genome Research Institute, the NIH Office of Rare Diseases, and the NIH Clinical Center.

Meanwhile, EPPOSI, The European Platform for Patients' Organisations, Science and Industry, plans to address the impact of the global economic crisis on rare disease research and public policies during its annual Workshop in Brussels in October this year, co-chaired by EURORDIS.

The US Orphan Drug Act celebrated its 25th anniversary in 2008, and in Europe the EMEA Committee for Orphan Medicinal Products (COMP) will soon celebrate its 100th monthly session. Collaboration between the US and the EU is increasing, as demonstrated by the adoption of a common application form for orphan designation in 2007.

In December 2008, the European Commission adopted a Communication on Rare Diseases and is now pushing for a Recommendation to be approved by the Council of Ministers. This EU policy will promote the adoption of national strategies and plans on rare diseases in all member states. The European Parliament will show its support for this policy at a Dinner Debate on Rare Diseases. The idea of national plans is sprouting in Japan, Korea, Australia, New Zealand and several countries in Latin America. At the same time, thousands of patient groups are organizing activities to raise awareness of rare diseases and to draw the attention of policy-makers to the needs and expectations of the millions of people suffering from them.

All this shows the positive correlation between public awareness campaigns and public policy. The success of Rare Disease Day depends on its scale. Globalization can aggravate an economic crisis but it can also spur solidarity on a grand scale.

Yann Le Cam is Chief Executive Officer of EURORDIS, the European Organisation of Rare Diseases.