“To combat rare diseases, a national law is needed to guarantee health care and legal security”
PMS
Institute for Research and Development of Rare Diseases (city ofindependent) helps patients with rare diseases improve their quality of life. Yours, President, Jesus Ignacio Mecostands out in interviews iHealth the importance of raising awareness, research and education in this area, and the main character is the role of the patient. «It’s necessary National Rare Disease Act which guarantees health and legal security“, he emphasizes.
It also highlights the value of cultural initiatives such as Solidarity Theater in Cervantes Corralone of the actions included in the Indepf project We Go Together: 10 Months, 10 Commitmentswhose advantages intended for associations and divisions specializing in rare diseases. Thus, he notes that projects of this type help to raise public awareness of these diseases and promote social integration. «He art has a unique power to educate, inspire and unite the community around humanitarian reasons“, he emphasizes.
What work does Indepf do to improve the lives of people with rare diseases and their families?
The Institute for Rare Disease Research and Development is dedicated to advocating for and improving the quality of life of people with rare diseases and their families. Our work covers various aspects, including protecting the rights of the patient and his/her family, as well as comprehensive support for those affected, always listening to them and meeting their needs.
“We need training in uncharted areas and cross-cutting studies of new patients with specific problems.”
We carry out activities to raise public awareness and promote discussions about the specific needs of patients. For Indepf, it is essential to emphasize the leading role of the patient, which is why we advocate for the adoption of a national law on combating rare diseases that guarantees health and legal security.
In addition, we provide counseling and support to victims and their families, providing essential daily services. We focus on using all available resources to improve the work of professionals.
Likewise, we emphasize the importance of research and training. We are not limited to biomedical research or postdoctoral training; we also need training in unexplored areas and cross-sectional studies of new patients with specific problems.
What events has the Institute carried out or planned for 2024?
In 2024, Indepf has planned a number of key activities to raise awareness, education and treatment of rare diseases. First and foremost, we continue to conduct information campaigns to raise awareness of these diseases. We disseminate our work through media and social media to keep the public informed about how we work to improve the lives of patients.
“This year we will be advancing a research project focused on pain treatment and another focused on optimising chronic care for patients with complex diseases.”
We also organize educational and training events aimed at healthcare professionals, families and victims. Following the recent days in La Mancha, which saw a significant participation from professionals, we have moved our initiatives to new locations to continue disseminating knowledge and best practices in the treatment of rare diseases.
We are also advancing research projects aimed at improving the diagnosis and treatment of these diseases. This year, we will focus on two promising areas: one is a project dedicated to pain management, and the other is optimizing chronic care for patients with complex diseases.
What are the most pressing issues in the field of rare diseases?
The most pressing challenges in the field of rare diseases are diverse and complex. First, early and accurate diagnosis is a top priority. Many of these diseases are extremely difficult to detect, often delaying the initiation of appropriate treatment. This requires significant efforts to improve diagnostic processes and greater institutional accountability for implementing and maintaining advanced and effective diagnostic practices.
“Early and accurate diagnosis is a top priority for rare diseases”
Another major issue is access to treatment. There is a widespread shortage of specific treatments for rare diseases, and those that are available are often expensive and difficult to obtain. In addition, the approval process for new drugs in Spain is noticeably slow (+600 days), and the country has fewer approved orphan drugs compared to other countries in the European Union. This situation limits treatment options and exacerbates the difficulties in obtaining needed therapy.
Awareness and education are equally important. Increasing knowledge about these diseases among the community and health care workers is critical to ensure better understanding and support. Health education must be fair and equitable, which will help provide peace of mind to affected citizens and their families.
Finally, it is important to adopt a comprehensive approach that includes emotional, psychological and social support for patients and their families. There is also a need for comprehensive legislation that supports this approach, ensuring that all aspects of the support needed to improve the quality of life of people living with rare diseases are taken into account.
“Specific treatments for rare diseases are generally lacking, and those that are available are often expensive and difficult to access.”
From Indepf they are promoting the solidarity theatre initiative in the Corral Cervantes. What does it consist of?
The Solidarity Theatre initiative in Corral Cervantes is one of the activities included in our project. We Go Together: 10 Months, 10 CommitmentsIn collaboration with the Siglo de Oro Foundation, we organize a series of theatrical events in which the proceeds from ticket sales go to associations and units specializing in rare diseases. This series of events not only culturally enriches the community, but also strengthens the social fabric through active and supportive collaboration.
The aim is to share our activities with other organisations during the month, to publicise our work in Madrid and to support small projects that are usually difficult to implement. This initiative aims to support small projects that are usually difficult to implement and to raise awareness of rare diseases through art and culture.
“Cultural initiatives are needed to raise awareness of rare diseases”
What value do cultural initiatives like these bring to raising awareness of rare diseases?
Cultural initiatives are essential to raising awareness of rare diseases. Art has a unique power to educate, inspire and unite society around humanitarian causes. Through these theatrical events, we not only support associations financially, but also raise awareness of rare diseases and promote social inclusion. The public not only enjoys the cultural experience, but is also aware and motivated to participate in positive social change.
In addition, these initiatives attract the attention of the general public and the media, raising awareness and knowledge about rare diseases. They also generate resources, helping to raise funds to support supporting institutions and programs, and promote solidarity and inclusivity, creating a sense of community and mutual support among all participants.
What does the We Go Together: 10 Months, 10 Commitments plan consist of?
The plan is an initiative that has been developed over a period of ten months with the aim of improving the quality of life of people affected by rare diseases. Each month, the plan focuses on an association or unit specializing in a specific disease, providing dedicated space and time to carry out key activities. These activities include information events, awareness campaigns and fundraising actions designed to make the needs of each disease visible and support its goals. All this took place in the surroundings of Arganzuela Matadero.
“The ‘We Walk Together: 10 Months, 10 Commitments’ plan focuses each month on an association or department that specializes in a specific disease to help improve the quality of life for these patients.”
This approach allows for comprehensive coverage tailored to the specificities of each disease, while facilitating active community participation. By highlighting a different disease each month, the plan not only increases awareness and visibility, but also strengthens the support and solidarity network, promoting greater understanding and commitment to rare disease causes.