“Patient’s Experience in theDevelopment and

Implementation of NATIONAL PLANS”

Paula Brito e Costa

Amsterdam, 13th May 2011

EURORDIS Membership Meeting 2011

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Mission and Objectives

MissionRaríssimas was founded in 2002 with thegoal of helping patients, families, friendsand all the ones closely affected by RareDiseases or interested in this subject.

Strategic Objectives

Spreading information about rare diseases;

Promoting the integrated management/follow up of thepatients with rare diseases;

Contributing to a positive differentiation concerning diagnosis, information aboutmedical services/specialties, treatment and therapeutics;

Raising awareness about the importance of gaining competences concerning rarediseases subject.

• Creating a Help Line – LINHA RARA toprovide information and support tofamilies, health professionals, etc.

• Developing a New Website withupdated and quality information onRare Diseases, from A to Z ”;

• Opening two Multi-disciplinary Centersfor treatment of Rare Diseases – CentroRarÍSSIMO - Lisboa and Porto;

• Implementing investigation projects,locally, nationally and internationally;

• Building Casa dos Marcos (respitecentre for Rare Disease patients).

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Operational Goals

• In the way we present ourselves;

• In the way we communicate;

• In the way the Association is managed;

• In the way we interact with our partners/stakeholders;

• In the way we advocate for the patient;

• In the way we lobby (at the ground base, at middle levels, high levels and community levels;

• In the way we define political strategies and implement them;

• In the way we build Casa dos Marcos.

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A model based on innovation…

• Unite efforts = Unite people

• Define short-medium term strategies;

• Present defined strategies publically;

• Discuss and share strategies with politicalforces;

• Support implementation of strategies ASAP;

• Persistency

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“Because no one does anything alone”Maria Cavaco Silva – Portuguese First Lady

Consulting at the highest level:

• Juridical

• Financial

• Communication

• Education

• Image

• Strategy

• Lobbying

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But much more is needed…

Scientific Council - President - António Vaz Carneiro

Consultive Council for Strategic Reflection -Statuary Organ - President - Maria Leonor Beleza

General Assembly - President - Francisco Sá Carneiro

Fiscal Council - President - António Trindade Nunes

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Current Strengthening

Consultive Council for Strategic

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Reflection

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Consultive Council for StrategicReflection

• Because rare diseases gather innumerousunfavorable factors;

• Because Portugal has a National HealthSystem;

• Because there was already some work donein specific areas ( National Plan forHemoglobinopathies – 1987)

• Existence of National Early Diagnosis Program- 1979

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And the National Plan for DR is born!

• On an integrated level

• Building partnerships

• Assigning responsibilities to ALL social actors

• Creating a new vision of the Portuguese State

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The need to re-think intervention on Rare Diseases

Patient Associations

• Strong pressure over public institutions

• Effective desire to achieve elaboration, discussion and concretization of the measures proposed

• Strong communication component in order to achieve goals proposed in the National Plan

• Active partners

Ministry of Health

• Created by the Directorate -General of Health

• Signed by the Minister of Health in November 2008

• Intention to include the NPRD in the National Health Plan (written but not yet accomplished)

• Health Minister signed the agreement for the implementation of reference centers for RD in January 2011

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National Plan for RD – Uniting Efforts

• Portugal faces economical recession;

• On the International day for RD, the National RD Federation (FEDRA) publically denounces inequalitiesconcerning patients’ access to Orphan Drugs;

• There is a massive response all over the country;

• FEDRA sees its representativeness growing;

• FEDRA reinforces its strategy and social partners intervene to provide support;

• Government falls creating a window of opportunity for Rare Diseases;

• For the 1st time, FEDRA participates in the Parliamentary Commission for Health

• Portugal has the means to accomplish the implementation of the NPRD, but the Government is cautious;

• The Government provides replies to issues of inequalities on a daily basis;

• With a “management Government”, the Directorate-General of Health and FEDRA are rushing processes to create the 1s reference centers;

• FEDRA reinforces appeals to the Health Minister in order to see the NPRD included in the National Health Plan – no response from the Health Minister!

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“United We Are Stronger”, but…

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Rare Diseases from A to Z - Presentation

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Un unconditional support

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Un unconditional support

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Scientific Council

Building Casa dos Marcos (respite Center for RD Patients)

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Besides fighting… we createsolutions

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Besides fighting… we createsolutions

Casa dos Marcos

Assistential Model

• 1 residential unit - 24 patients

• 1 autonomous residential unit – 5 patients

• 1 unite of Occupational Activities Center – 30patients

• 1 rehabilitation unit (open to the community)

• 1 clinical unit (open to the community)

• 1 unit of continuous medical care – 30 patients,connected to the national network of medical carefacilities

• 1 private unit of medical care – 9 patients

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Besides fighting… we createWebsite aboutRare Diseasesfrom A to Z

solutions

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This is how we make history…With love and because of love

Thank you!