Eastern European Conference on Prader Willi

SyndromeLaunching the project : Norwegian-Romanian (NoRo)

Partnership for Progress in Rare Diseases

Funded by Innovation Norway

Timisoara - April 24-25, 2009

Norwegian-Romanian (NoRo) Partnership for Progress in Rare Diseases Partners:

1. Norwegian PWS Association 2. FRAMBU – Norwegian Center for Rare Diseases 3. Ministry of Health Romania 4. Foundation ACASA Hospital 5. St. Family Church, Zalau 6. City Hall Zalau/ DASC 7. County Council Salaj/ DGASPC 8. Romanian Genetics Society 9. Medical University Timisoara 10. Romanian National Alliance for Rare Diseases

Applicant: Romanian Prader Willi Association Oslo 01.10.2008 – signed the partnership agreement

The reality of Rare Disease (RD) Rare diseases are chronic, life-threatening diseases, with a low

prevalence and high levels of complexity (5 in 10,000 people affected).

In Romania, approximately 1,500,000 patients live with RD, according to international statistics, most diagnosed incorrectly or living without a diagnosis.

Patients are particularly isolated and vulnerable. The life expectancy is significantly reduced Many have disabilities associated with problem behaviors and

obesity.

These behavioral and weight issues become a source of discrimination and reduce or destroy any educational, professional or social opportunities.

Project aim:

To improve the quality of life for people affected by rare diseases in Romania by providing equal access to early diagnosis, quality treatment and rehabilitation services through a comprehensive and accessible network of facilities and resources as set forth by the National Plan for Rare Diseases.

NoRo Project Objectives :

1. Establishing a Rare Diseases Task Force- RDTF;

2. Establishing new services for people with rare diseases in Romania;

3. Shared best practice network on RD (NoRo);

NoRo Project Objectives : 1. Establishing a Rare Diseases Task Force- RDTF:

The main criteria for selecting members of this working group will include: a. Familiarity and knowledge of the work through the

documentation: publications, training, grants; b. Made a contribution in creating protocols, recommendations; shared (partnerships) expertise with other experts ; d. Demonstrated open collaboration with patient organizations

The role of the RDTF

The role of the group: the evaluation of rare diseases in Romania (patient cases, treatments available, training needs and information) and propose actions (medical measures, educational and social, writing materials, guides to good practice, courses, curricula, etc.) and continuously update knowledge of the role which the Ministry of Health has in connection with the activity;

Functioning: The group will meet quarterly (8 X two days meetings per project) and will analyze situations presented by each member of the group, each member will propose measures and will be informing the Ministry of Health and other ministries involved in the management of rare diseases. The maximum number of group members is 20.

Budget: Each group member will have an allowance for each meeting that cover the costs of transportation, accommodation, meals. Also the costs of communication in group, conference room for meetings, supplies, printings, publications, etc are covered under the project.

Ob. 2. New services for RD

a. A training network for specialists and staff involved in the diagnosis, treatment and rehabilitation of people with rare diseases.

b. Accredited online training courses (eUniversity) about rare diseases for professionals: social workers, psychologists, nurses, teachers, doctors, etc.

c. A Pilot Reference Center for personalized intervention for those affected by rare diseases.

Ob.2. New services for rare diseases in Romania a. Developing and implementing a training network for

specialists and staff involved in the diagnosis, treatment and rehabilitation of people with rare diseases.

Organizing workshops, seminars, and training on specific areas: Selection of a training team of trainers; Creating curricula on specific jobs involved in RD, ex: genetic

councilor; Performing trainings;

b. The creation of accredited online training courses (eUniversity) about rare diseases for professionals: social workers, psychologists, nurses, teachers, doctors, etc.

Creating curricula for specific training themes; Purchasing the necessary software to perform these trainings; Authorization/ accreditation of the trainings; Organizing the first training courses;

Training

Improving training capacity in the country for the prevention, diagnosis, treatment and rehabilitation of patients with rare diseases:

Results:

- a network of training specialists and staff;

- accreditation / licensing of online courses (eUniversity) (on rare diseases, for professionals: social workers, psychologists, nurses,

teachers, doctors, etc.)

The Pilot Reference Center for personalized intervention for those affected by rare diseases 3 main components + 1 external unit:

a day center for patients with rare diseases (having behaviors on the autistic spectrum)

a comprehensive training program for patients, families, caretakers and specialists involved in the management of rare diseases (FRAMBU model)

patients newly diagnosed and their families (to learn to cope with new situations more easily)

The external unit:

A weight management unit in the Rehabilitation Hospital “ACASA” – for patients with neuromotor deficiencies.

We will plan so that patients who come to our courses can come before or after the period of the rehabilitation treatment that they take part in at ACASA Hospital

In addition, we will have a national database of patients, along with ACASA Hospital, with various diseases and be actively monitoring their progress.

Ob.3 Shared best practice network on RD (NoRo) Organizing visits of the partners Writing the final proposal of the project based on the best practice of

the partners and the shared values and experiences in the field of RD

Organizing presentation of the project’s results in the PW EE Conference (April 2009 Romania), EURORDIS membership meeting Greece 2009, IPWSO Conference Taiwan 2010, etc.

Permanent updating of the project results on the websites; Writing articles in the newsletters and RD journals to promote the

project’s results;

The dream

The space received from the City Hall

The vision

Dream vision

Achievements until now:

Relevant partnerships established during the seed money implementation;

Proper facilities for the new services; Feasibility studies completed; Grant offer letter signed; A coordination team for the project implementation

established at the Ministry of Health; Official launching of the project at the 1st Eastern

European Conference on PWS organized; The first evaluation meeting of the potential members of

the RDTF at the conference in Timisoara;

Pilot Centre for Rare Diseases: Idea of the Centre:

A residential part: To provide help for families after experience of

diagnosing a RD; A place to learn how to face the new situation and

to deal with the challenges of the disease in their everyday life;

A place to organize meetings and trainings for patients;

Support groups and counseling activities for patients and families;

Pilot Centre for Rare Diseases:

Hospitalization of the RD patients in ACASA hospital for 3 week rehabilitation visits;

Establishing a data base of patients that need to visit the rehabilitation program in ACASA;

Specializing services of the center for RD patients: weight management, individualized therapeutic approach;

Day care activities: Individualized therapeutic approach for individuals with

behaviors on the autistic spectrum; Behavior intervention therapy; Occupational Therapy and Educational activities; Recreational activities; Support groups and counseling activities; Weight management

Main intentions of the project: To develop skills to approach the RD for patients, professionals and parents;

To maintain and improve the mental and physical capabilities of patients through individualized therapeutic approaches;

To improve understanding and support of the community;

To create a data base of patients with RD in Romania;

To establish protocols of assistance and care for different RD;

To start a model network in this field through the development of a strategic partnership: NGOs from the national and international levels, Ministry of Health, Local Authorities, state departments, etc;

Document and offer transparency in the project so that similar efforts can be replicated throughout Romania

Thank you!

As parents and patients, when we lose our strengths, we are carried by hope!