Even for the world’s most financially robust countries, finding room in their budgets for rare disease research, care and treatment is problematic. For developing countries, the challenge is Herculean. The non-profit Philippine Society for Orphan Disorders (PSOD) was created in June 2006 in order to lend support and sustainability to the small number of medical professionals working with rare disease patients in the Republic of the Philippines, a country of 7,107 islands with a total population of 92 million people. The PSOD is seeking to position itself as the country’s “central network for the advocacy and effective coordination of all viable efforts to sustain a better quality of life for the individuals with orphan or rare disorders in the Philippines”. The group’s mission is to “uphold the primary concerns and welfare of individuals with rare disorders by directly addressing, supporting and protecting their health and general well being”. The PSOD is chaired by Dr. Carmencita D. Padilla, Professor of Paediatrics at the College of Medicine, University of the Philippines Manila and Director of the Institute of Human Genetics, National Institutes of Health Philippines. Dr. Padilla also heads the Newborn Screening Reference Center, and co-chairs the National Technical Working Group on Newborn Screening at the Department of Health. Other PSOD board members consist of both professionals and parents of patients, including the organisation’s president, business woman Cynthia K. Magdaraog, the parent of a patient with Pompe disease. Amongst the goals the organisation has identified is increasing public awareness; developing and establishing a nationwide registry of relevant and material statistical information, (including medications, drug trials and other pertinent information about orphan disorders); assisting patients, their families, family support groups, doctors and researchers in the management of these conditions; promoting and supporting relevant and timely research; participating in policy formulation, advocacy and legislation of national and international relevance; developing and strengthening the relationship among institutions that are involved in the care of individuals with orphan disorders; developing and providing training for parents, families, health professionals, paramedical personnel and community health workers on the care of patients with orphan disorders; developing PSOD members’ core competencies in the management of orphan disorders; and being financially self-reliant, mobilising resources in the pursuit of organisational objectives.
In the Philippines, a rare disease is defined as a condition or illness affecting “no more than one in 20,000 individuals in the country”. The PSOD has several initiatives in place to help rare disease patients and their families, including the Endowment Fund and The Emergency Fund. The Endowment Fund is designed to help sustain long-term treatments that many diseases require. This fund also assists research for rare disorders and aims to make the PSOD self sustaining. The Emergency Fund is for the immediate needs of patients undergoing medical emergencies and can be used for life saving medicines, hospitalisation, the purchase of supportive medical devices, and other uses. The PSOD uses annual reports and other publications to communicate how donor funds are used.
The Rare Diseases Act of the Philippines
To work toward these objectives, the group has campaigned actively to put forward a bill that would make providing resources for rare disease patients a part of the country’s legal responsibilities. Currently pending, Senate Bill No. 3087: the Rare Diseases Act of the Philippines is sponsored by Senator Edgardo Angara, a fervent supporter of the scientific community. Bill 3087 seeks to ensure that every patient diagnosed with a rare disorder “has access to timely health information and adequate medical care, including drugs and other healthcare products to treat or otherwise help them cope with their condition.” Amongst the bill’s components is the call for the creation of an Office of Rare Diseases within the country’s department of health that would oversee research, create and maintain a registry, and conduct public awareness programmes. The bill also calls for “regulatory and fiscal incentives to support research and development activities on rare diseases and the import or manufacture of affordable orphan drugs or orphan products; and institutionalise a financial incentive system for agencies involved in clinical research, patient care, medical information management, and other similar activities for the benefit of persons afflicted with a rare disease”. The PSOD is sponsoring a petition to get the Rare Diseases Act passed and has set a goal of 500,000 signatures. Consult Bill 3087
Sign the Petition supporting Bill 3087
Project Rare and Care for Rare
The PSOD’s Project Rare initiative launched in February of this year. Designed to heighten public awareness, a series of activities specifically seek to identify and add patients to the rare disease registry; build patient and caregiver networks; and create an endowment fund.
Another PSOD project that got off the ground this year is the group’s first newsletter. Care for Rare reports on the activities taking place in the rare disease community, helping to foster collaboration and communication. The newly-revamped PSOD website also publicises news from the rare disease community, including Patient Welfare Activities project reports, which list donations received and how the funds were specifically applied within the rare disease community. The group further enhances its visibility by taking advantage of free networking resources such as Facebook, You Tube and Twitter.
Genzyme is a partner of the PSOD and also provides free treatments to certain Filipino patients with rare disorders Gaucher disease and Pompe disease under its International Compassionate Access Programme. The PSOD was present at the recent International Conference of National Policies and actions on Rare Diseases that took place in Taiwan and engages with several countries in the region as well as with stakeholders in the USA and Europe. OrphaNews Europe will be keeping a close watch on the PSOD and on Senate Bill 3087 in order to report any breaking developments in the struggle to secure aid for rare disease patients and their families in the Philippines.