Participants migrate to Southern hemisphere for sixth International Conference on Rare Diseases and Orphan Drugs
The International Conference on Rare Diseases and Orphan Drugs (ICORD) crossed the equator for the first time to convene in Argentina in mid-March. Hosted by the Latin American and Caribbean countries patient organisation GEISER (learn more) the conference capitalised on the efforts already underway to raise visibility and activity in the region. ICORD is an annual event conceived to promote global collaboration in the field of rare diseases and orphan drugs. The first meeting was held in 2005 in Stockholm and subsequent meetings have taken place each year in Madrid, Brussels, Washington, and Rome. This year’s theme “Global Approaches to Research and Patient Access to Diagnosis, Information and Care, And the Common Issues with Neglected Diseases in Developing Countries” has particular relevance in the region. With sixteen formal sessions and five additional activities, some 350 participants hailing from over 25 different countries (including Australia, Brazil, Chile, China, Colombia, Ecuador, Japan, Mexico, Panama, Peru and Uruguay) were in attendance. One plenary session chaired by ICORD president Stephen Groft and Kerstin Westermark explored From Pioneer Countries to the Rest of the World; and another, chaired by Ségolène Aymé and Sharon Terry focused on The Development of Information. In addition, nine round table sessions featured topics such as Turning rare diseases into an international priority; Initiatives from the public institutions; Research; Patients and family care; Best practices in the approval of orphan products; Bioethics; Linking needs with neglected diseases; Strategies for accessibility; and International initiatives. Furthermore, several Working Groups gathered to explore Regulatory needs; Research; Patient/Family; Diagnosis; Accessibility; and other topics. A satellite symposium proposed by the Pan American Health Organization (PAHO/WHO) on The impact of high cost drugs in developing countries was also held. Other notable presentations included those by GEISER founder Virginia Llera on Including the Developing Countries in the International Scenario of Rare Diseases and Orphan Drugs; Orphanet director Ségolène Aymé on A Review of the International Classification of Diseases; and Tim Coté describing The FDA Foreign Offices and its Impact in the Orphan Drugs Field. A series of related courses were also on offer from the Latin American Society for Rare Disease Medical Research (SLADIMER). Significantly, the event was declared of national importance by the Argentinean Government, and was supported by the Pan American Health Organization as well as the local Embassies of the USA and Sweden, among others. These official connections related to rare diseases are unprecedented in the region. Tokyo, Japan has tentatively been proposed as the location for ICORD 2011 conference. Dr. Domenica Taruscio from Italy has been newly elected as president of the organisation and Dr. Virginia Llera from Argentina will serve as the President-Elect for a two-year period.
A new advisory body will be created in England covering specialised services and treatments for extremely rare conditions typically affecting fewer than 500 patients. Following a consultation, the current National Commissioning Group will be dissolved and a new body, the National Commissioning Advisory Group (NCAG), will be established. The NCAG will make recommendations directly to Ministers about which services should be designated for national commissioning. The Group will also consider a small number of new technologies for small patient populations which fall outside NICE’s remit, but which may be suitable for national specialised commissioning. Any decisions that the NCAG will make about services and technologies will be guided by a decision-making framework which draws in part on work done by the 
A meeting organised by the editor of Dagens Medicin, Sweden’s most read paper for medical professionals, took place on 25 March in Stockholm. The main topic on the agenda was the recent demand from the European Union regarding a national strategy and action plan for orphan drugs and rare diseases. Over one hundred health professionals, policy-makers, patient organisation representatives, and members of companies involved in orphan drug development spent the day reviewing the difficulties faced by patients. The French strategy was presented and discussed as an example of good practice in the field. Much of the discussion focused on the assessment of marketed drugs for reimbursement as participants were not happy with some recent decisions on this issue. Participants also expressed the sentiment that Sweden is lagging behind, though it was the leading country in the field in the 1990s. Indeed, Sweden was amongst the first European countries to distinguish rare diseases – which it defined as conditions with a prevalence of less than one in 10,000, (compared to the EU definition of 5 in 10,000). It appears that the present lag is due to the absence of a mechanism that would allow stakeholders to meet and discuss - something that can be easily resolved if the political will is there. Currently a report on the present organisation of the healthcare system regarding services for rare disease patients is in preparation but without prior discussion with main third-parties - an approach which was viewed as less than optimal for the appropriate preparation of a strategy. Also, deep disappointment was voiced that the report will be descriptive only and does not offer solutions or improvements. Experts stressed the point that considerations on rare disorders need to extend beyond orphan drugs. As delineated by the European Council Recommendation on an Action in the Field of Rare Diseases, the need for specialist centres, involvement and empowerment of patients and their families, a better definition of rare diseases, and support for the development of registries and the formation of European professional groups were also evoked. Several constructive proposals were put forward during the discussions and a high-level politician participating in the meeting declared her continuing commitment to promote rare diseases in the Swedish parliament.
