Survey: Patients’ Access to Orphan Drugs in Europe

EURORDIS CEO Yann le Cam presenting a series of rare disease patient testimonies as well as the results of the 5th EURORDIS survey on access to Orphan Drugs in Europe.
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drugsIn September 2010, EURORDIS and 10 rare disease National Alliances joined forces to obtain information about the real life situation of European patients with respect to access to orphan drugs (OD). The basic idea behind this collaborative work was to find out whether all orphan drugs, that had received EU market authorisation since the implementation of the EU Regulation on Orphan Medicinal Products in 2000, were really available for rare disease patients within their own countries.

In order to do this, national alliances were to collect information by asking their members or by surveying the national Competent Authorities, pharmacies, insurance companies and healthcare professionals in their own countries. EURORDIS provided a common template, entered the data, performed the analysis and provided feedback on the results to the participants. All 20 national alliances of the Council of National Alliances were invited to join the collaborative effort and 10 accepted (Belgium, Denmark, France, Greece, Hungary, Italy, Netherlands, Romania, Spain and Sweden)

The survey analysed access to 60 orphan drugs with a marketing authorisation in Europe. 480 out of 600 survey cards (60 drugs x 10 countries) were returned. The population of the 10 participating countries represents 256 million people in total, and the proportion of the people who could potentially be concerned by the OD surveyed, based on prevalence of condition, represents 2.1 million.

The first noticeable result of the survey was that, although patient organisations seemed to be the most obvious source of information about the availability of the drugs taken by their members, it was in fact very difficult to obtain comparable data between countries and products. Information about prices was particularly inconclusive, as many patients do not know the official price in their countries or the actual price paid by their healthcare system.

Simona Bellagambi“We needed this kind of survey to get a clear ‘proof’ of the overall situation if we want to advocate for equal access to ODs. As I started gathering information about OD prices and availability, I realised how difficult it was. Clear information and data are scarce even within patient associations,” explains Simona Bellagambi from UNIAMO. “I learnt that there are several and complicated mechanisms to reach the final price. In fact, thanks to the survey, I realised that in Italy there is a big difference between the price – fixed by the Italian Drug Agency - and the cost – covered by the National Health System.”

A key finding of the study was that, for 22% of all OD surveyed, the national alliances did not know if they were available and in 8% of cases, they confirmed that the OD were not available. “Therefore, 10 out of 100 patients, and possibly, 30 out of 100 patients across Europe, do not have access to drugs that have not only been designated and given market authorisation at the European level but for which society has given industry special incentives and support,” says EURORDIS’ CEO Yann Le Cam. “This is unacceptable and contrary both to the spirit and the text of the EU Regulation on OD.”

Another key finding was the proportion of orphan drugs available per participating country. In France, Netherlands and Denmark 90% of the 60 authorised OD are available, whereas in Spain, Greece and Romania only one-third of the authorised OD can be found on the market. A third intermediate group of countries comprising Italy, Hungary and Belgium have approximately two-thirds available. The data for Sweden could not be included in the results because it was not extensive enough. 

When looking at the availability of orphan drugs by groups of diseases, the highest availability is observed for metabolic diseases (64%) and rare cancers (59%). This is in sharp contrast with OD prescribed in other therapeutic fields such as cardiology, neurology and haematology, where the drugs are less readily available. “There is scarcely a problem of availability in all countries for OD treating metabolic conditions, which proves that it is possible to make OD available in all EU countries. This might have to do with patients being better organised, as well as medical specialists and centres of expertise being better organised and pro-active about access to new medicines,” argues Yann Le Cam.

“In some countries the percentage of patients accessing OD appears to be higher than the percentage of OD available. At first glance, this seems like good news. However, upon closer inspection, we see that it is the drugs that treat the rarer conditions that are not available. Therefore, even though less people are affected, the reality for each individual patient is dramatic,” explains Dr Faurisson, Clinical Research Advisor at EURORDIS. “Throughout the study it is obvious that the rarer the condition and the smaller the country, the less well-served patients are regarding orphan drugs.”

Another result revealed by the survey is that the orphan drugs that have been on the market the longest are not becoming more widely available. “Even though EURORDIS fourth survey on orphan drug availability in 2007 had generated the hope that OD would be increasingly available in more and more countries, the 2010 survey shows that unfortunately, the availability increase of the older drugs, after an initial period of two years, has remained desperately stable,” argues Dr Faurisson.

The survey also explored the price situation in each participating country. The first main conclusion is that official prices are different in all countries and can vary significantly. But the second and more important conclusion is that the official price can be different from the real cost. These differences in cost make it difficult to draw comparisons between countries and evaluate the financial burden of OD to the social security systems. “One thing is clear: in some countries the government is able to negotiate better prices for its population than in others,” says Dr Faurisson. “It is evident that there is the potential for a European price to avoid wholesalers in ‘cheaper’ countries re-exporting to more ‘expensive’ countries and especially to avoid patients and society paying more in countries with less negotiation power.”

For more information:
EURORDIS Survey on Patients’ Access to Orphan Drugs in Europe presentation

This article was first published in the March 2011 issue of the EURORDIS newsletter

Author: Paloma Tejada
Photo credits: © EURORDIS, Wikimedia Commons

Page created: 24/02/2011
Page last updated: 14/08/2015
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