Access to treatment: unequal care for European rare disease patients

Authors: Sandra Courbier, Rare Barometer Leader1; Erwan Berjonneau, Rare Barometer Research Executive1; François Houyez, Treatment Information and Access Director, Health Policy Advisor1; Xavier Fournie, Corporate Medical Director & Exec VP, Global Medical Affairs2
1EURORDIS, Paris, France; 2Mapi, Lyon, France


In 1999, the European Union regulation on orphan medicinal products stated “patients suffering from rare conditions should be entitled to the same quality of treatment as other patients”1
Today, the issue of access to medical treatments is under pressure due to a number of recent developments including the economic crisis, leading to falling healthcare spending in many Member States, and an ageing population.
As for rare diseases, only a very small number of them have curative treatment. However, what is the level of access for existing rare disease treatments, either symptomatic or disease-modifying? Does it really differ from the general population?
To answer these questions, EURORDIS carried out a survey through its Rare Barometer Survey Programme ( Rare Barometer participants, i;e., patients affected by a rare disease, their parents, siblings or other family members and carers, and patient representatives who answer regularly to EURORDIS surveys, were asked about access to treatments.


Fieldwork started on 11th of February and ended on 16th of February 2017.
The question referred to medical treatment in general including medicines (disease modifiers and symptomatic treatments), surgery and medical devices. EURORDIS researchers used question 20 from the International Social Survey Programme (ISSP) focused on health and health care2 (See Figure 1). The ISSP is a continuing annual programme of cross-national collaboration on surveys covering topics important for social science research. The last wave regarding health care was carried out in 2011. Comparison were made with the results of the European continent.

Figure 1. Question 20 from the ISSP health and health care 2011 survey

For the Rare Barometer survey, a Yes/No response option was used, with “No” corresponding to: No it didn’t happened/No need of medical treatment/Non response.


A total of 1350 persons from 21 European Countries responded to the survey. The sample was composed of 54% of patients and 46% of carers (i.e., parents, siblings or other family members, carers and patient representatives). Carers answered in relation with the situation of the patient they care for. Patient representatives are frequently also patients or patients’ relatives and they represented only 5.6% of the respondents (12% of the carers category) thus had a limited impact on global responses.
24% of the rare disease sample declared that during the past 12 months they did not get the medical treatment they needed because the treatment was not available in their country (See Figure 2). The difference with the general population is striking as it is 17% higher. The gap between the general population and rare disease patients was reported as even deeper in some countries such as in the United Kingdom where 31% say that the treatment was unavailable in their country compare to only 3% of the general population.

Figure 2. Results with Response “Treatment not available”

When asked if they did not get the medical treatment they needed because they were not able to pay for it, 15% of the respondent answered yes (see Figure 3). This represents a difference of 9 percentage points compared to the general population.
Figure 3. Results with Response “Could not pay for it”

19% answered yes when asked if they were prevented from accessing the treatment they need because the waiting list was too long (see Figure 4). There is a marked difference in comparison to the general population: 10% percentage points more. This waiting time is mainly related to the time required to obtain a visit or appropriate hospitalisation.
Figure 4. Results with Response “Waiting list too long”


Compared to the general population, rare disease patients have a lower level of access to treatment. Efforts still need to be made to  fulfil the needs of people living with a rare disease while maintaining the sustainability of the European healthcare systems.


Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Official Journal L 018, 22/01/2000 P. 0001 – 0005. Available at: Last accessed April 19, 2017. Last accessed April 19, 2017.

For more information, please contact Sandra Courbier, Surveys and Social Studies Manager, Rare Barometer Leader:


Special thanks to the Rare Barometer Participants and all Rare Barometer Partners: