Putting Rare Diseases Patients First!® is based in the US, but has a global outreach through its Direct to Patient strategy, this Blog, its Webinars, the website www.prdpf.org, and through its social media campaigns. To this end, our Board members and volunteers can be based anywhere in the world. Our volunteers and Board members help us to understand the situation facing rare disease patients in their own countries of birth, or where they are living currently.
In this article, Dr. Karel Petrak has provided his insights into the situation facing rare disease patients in Argentina.
As with many other countries, many rare disease patients experience barriers in access to care; fewer than 10% receive disease-specific treatment. Delayed diagnoses, limited access to resources, and absence of specific therapies often preclude patients from receiving proper, timely care.
The rare disease patient community in Argentina plays a critical role in addressing these challenges, by elevating the patient voice and partnering in the development of programs with the pharmaceutical industry and other groups, to address the needs of patients with rare diseases. For example, advocates from the Pituitary Diseases Association travel around the Argentina and provide medical updates on pituitary diseases to professionals to help identify and diagnose patients.
Countries such as the USA and the European Union have already put in place national plans or strategies to address the diagnosis, treatment, care, and support of citizens with rare diseases. These measures also include raising general awareness of rare diseases, supporting research into rare diseases, developing centers of expertise, empowering patient organizations, and strengthening healthcare infrastructure and systems.
Similar efforts are currently under-way in Argentina.
• A national policy on rare diseases has been approved and is awaiting implementation
• A streamlined authorization process is available for US or EU-approved drugs
• An Orphan Drug registration process is in place
• There is an Early Access Program in place for free access to drugs from other countries
• Legislation has been passed, but patient registries are still under development.
• Patient organizations in Argentina play an active role in patient engagement in Argentina.
In summary, as with many other countries, some progress has been made in the provision of support for rare disease patients, but there is still a long way to go. For example, although patient can request treatments available elsewhere, they would need to be aware of the medicines that are available to rare disease patients in other countries, in order to request access to these treatments. Such treatments may be approved in other countries, but not in Argentina.
Argentina’s held its very first Rare Disease Day in 2009. Since then, participants have been working with the media and patient organisations to increase awareness among the general public about rare diseases.
For Rare Disease Day 2018, there was an walk followed by food, entertainment and the presentation of a local documentary project produced with support from a community of relatives and rare disease patients. A neonatal hospital also got involved with the face painting campaign to make rare diseases more visible.
Dr. Karel Petrak
VP of Fund Raising, Grants, Donations and Major Gifts