Sandra Courbier, Rare Barometer senior Manager

15th Mai 2018

RARE BAROMETERVOICES

An evidence-based advocacy tool

2

What is Rare Barometer Voices?

3

What is the objective of the tool?

Collect patient

experiences and

opinions

Through validated methods

Generate fact and figures

Feed the rare disease community’s advocacy

work

4

Rare Barometer Voices is a group of people living with a rare disease who participate in EURORDIS surveys and studies.

Rare Barometer Voices

Patients, families, patient representatives can register

Participants provide useful information for future surveys(disease, gender, age …)

Rare Disease patients can register from any country of the world

5

eurordis.org/voices

6

Quality of the data

Carry out longitudinal studies

Inform participants on the results of the survey

More detailed anaysis

Advantages of building a panel

7

Enables data de-identification

Data storage in France

Secure and GDPR compliant data storage plateform

High level of data protection (Sphinx survey software)

Design of the questionnaire/dissemination/data analysis/results communication.

8

Registration page and all surveys and are available in 23 languages

9

More than 10 000 patients and families have registered for Rare Barometer Voices so far!

10

▪ High proportion of patients and families not engaged in

advocacy activities

Participants’ profile

11

Distribution by country

RBV databaseRepartition by country

12

➢ Raise awareness about rare diseases➢ National plans advocacy work➢ Funding applications

Data available on the following topics

▪ Impact of rare diseases on daily life▪ Participation in research▪ Data protection and data sharing▪ Experience and accessibility of treatments

Your advocacy tool

13

Availability and experienceof treatments survey

Topics covered: availability, type of treatments, access to and affordability of treatments, compassionate use programmes and the off-label use of treatments.

More than 7000 respondents

Breakdowns by :

- country/country grouping (Southern Europe, Eastern Europe, etc...)

- ERN (Epag grouping)

- adult/peadiatric population.

- oncolgy vs other type of diseases treatments

Comparison with the general population on accessibility and evolution compared to the 2017 survey.

Report will be available beginning of June

16

▪ 1st Rare Barometer surveyon the impact of rare diseases on daily life, May 2017

▪ Infographic in English for the total European sample

17

Infographic in Spanish, for the results in Spain

18

Infographic in croatian for the European results

19

Significant differences between…

Unmet care and social care needs of RD patients in Easterneuropean countries

Eastern Europe

Belarus

Bulgaria

Czech Republic

Hungary

Poland

Republic of MoldovaRomania

Russian Federation

Slovakia

Ukraine

Northern Europe

Denmark

Estonia

Finland

Iceland

Ireland

Latvia

Lithuania

Norway

Sweden

United Kingdom and Northern Ireland

Southern Europe

Albania

Andorra

Bosnia and Herzegovina

Croatia

Greece

Italy

Malta

Montenegro

North Macedonia

Portugal

Serbia

Slovenia

Spain

Western Europe

Austria

Belgium

France

Germany

Liechtenstein

Luxembourg

Monaco

Netherlands

Switzerland

Results of Rare Barometer Voices surveys: • Juggling care and daily life, the balancing act of the rare disease community, 2017• Access to treatment: unequal care for European rare disease patients, 2017

and…

Other European countries :

20

% of rare disease patients who didn’t get the medical treatment they needed in the past 12 monthsbecause ….

Eastern Europe

Other European countries

22%

14%

37%

21%

25%

17%

They could not pay for it

The waiting list was too long

It was not available where they live

+8

+16

+8

Greater difficulties in accessing treatments

21

Everyday life impact of the rare disease on families is more important

Eastern Europe

Other European countries

55%

39%

Spend more than 2 hours per day on illness-related tasks

+16

This can partly be attributed to less support they receive with (% of uncovered needs):

Day care (32%, +11)

Adapted holidays and therapeutic recreation (65%,

+21)

Adapted school (28%,

+13)

Support to adapt your house to your

needs (37%, +6)

Disability benefits (63%,

+11)

Rehabilitation services and therapies (60%, +12)

22

Greater financial pressure on families

Eastern Europe

Other European countries

81%72%

Costs are high

+9

76%

61%

Costs are hard to manage

+15

Respite care (73%, +16)

Treatment and medication(51%, +18)

Special diet(58%, +8)

Rehabilitation therapies (61%, +21)

Appointments with healthcare

professionals (42%, +11)

Hospital admissions

(29, +9)

Home care services(70%, +12)

% who can’t afford it or think it’s hard to manage

23

People living with a rare disease from eastern Europe rely more on the internet for information on treatment and care

Eastern Europe

Other European countries

36%

29%

Internet is main source of information

+7

24

Thanks for your attention!