The future availability of medicines orphans and ultra huérfanos will be compromised if it applies the cost saving measure announced by the Minister of health, social policy and equal.

AELMHU is positioned before the savings measures announced by the Government at the meeting of the plenary of the Interterritorial Council of the SNS

-the discount of 15% of the price of the innovative medicines that will take more than 10 years in the market and which do not have generic, applied to the orphan drugsit seriously jeopardizes the future availability of medicines indicated for the treatment of patients with rare and ultra-raras diseases.

Spain, 2011-July the Association Spanish of laboratories of medicines orphans and Ultra-Huérfanos (AELMHU), formed by companies that investigate, develop and market medicines for the treatment of rare and ultra-raras diseases, expresses great concern the extent of reduction of 15% of the price of the innovative medicineshave been more than 10 years in the market and that they are not generic. Apply this discount to medicines orphans and ultra-huérfanos, the future availability of these drugs (indicated for the treatment of patients with rare and ultra-raras diseases) will be seriously compromised.

AELMHU believes that, if the Government decides to implement this measure of reduction in the area of rare diseases, will be serious danger the future ability of companies to continue investing in the research needed for the development of new medicines to meet the health needs of these patients. With this measure, the Government would not recognizing the disproportionate negative impact that a new cut will have on the development of orphan medicinal products. In contrast, it should be noted and be aware of the savings for the health system will be minimal since due to the low prevalence of rare diseases, spending on orphan drugs and ultra-huérfanos in the health budget is negligible.

It should also be noted that many of the companies that market orphan drugs are newly formed, very small in terms of its infrastructure and even some of them have a marketed product. These companies invest and continue to invest in r & d in Spain. For this reason, a reduction in this area would be a serious threat to the viability of these enterprises, engines of research and development of new medicines for diseases without therapeutic alternatives.

Orphan drugs already suffered a discount in the price of 4% (RD 8/2010 of urgent measures for the control of the public deficit). An additional measure of lowering of prices of medicines orphans and ultra-huérfanos would be a direct conflict with the efforts made over the past decade, both European and national, to not recognize rare diseases, and drugs orphans who deal with, as a social priority that deserves a unique and specific treatment.

The creation of a selection of medicines Committee composed of experts from the ACS. should not result in an increase in the time in which a drug orphan becomes available in the market, which would run counter to the processes of rapid authorisation which is granted to these drugsdue to the fact that patients do not have any alternative therapy available.

The national health system must ensure to maintain support in the area of rare diseases with the aim that produce an efficient investment that will allow to have an increasing flow of drugs patients innovative orphans. Therefore, we firmly believe that the orphan drugs and ultra-huérfanos should be excluded from this price cut in the innovative medicines that will take more than 10 years in the market.

Medicines orphans and ultra-huérfanos

According to the definition of the European Union (EU), rare or uncommon diseases are those debilitating, chronic or life-threatening diseases affecting less than 5 per 10,000 inhabitants. Within this group are the disease ultra-huérfanas, which are those that affect less than 1 per 50,000 inhabitants. For many years the Group of patients with rare diseases not has been available since effective treatments that, under normal market conditions, the process of r & d of orphan medicinal products is not profitable for the pharmaceutical industry, due to the low number of patients affected. Therefore Regulation EC 141/2000 of the European Parliament was adopted in the year 2000 and of the Council with the aim of stimulating the development and marketing of orphan medicinal products, intended to diagnose, prevent or treat rare diseases. In addition, the European Commission has designated rare diseases as one of the priorities of health policy in the field of public health within the seventh framework programme. Also, rare diseases are still a priority for action within the programme of public health 2008-2013.

This whole set of European regulations applied by each of the Member States, has achieved the goal of promoting research in areas unattractive from the point of view of profitability, fact which has led to the commercialization of 62 orphan medicinal products. These efforts must, without doubt, continue since there are thousands of rare and ultra-raras diseases without any authorized treatment.