Question & AnswerQ&A (Republic Act No. 10747)
Republic Act No. 10747 is known as the "Rare Diseases Act of the Philippines."
The State declares it as policy to protect and promote the right to health of persons suffering from rare diseases, ensuring their survival and full and healthy development through access to timely health information and adequate medical care.
The objectives include improving access to comprehensive medical care for persons with rare diseases, establishing a Rare Disease Registry, integrating public education and information campaigns to identify rare disease cases, facilitating collaboration among stakeholders, and providing regulatory and fiscal incentives for research and manufacture of affordable orphan drugs and products.
A rare disease is defined as disorders such as inherited metabolic disorders and other diseases with similar rare occurrence as recognized by the DOH upon recommendation of the NIH, excluding catastrophic forms of more frequently occurring diseases.
Healthcare practitioners include any doctor of medicine, dentist, nurse, midwife, allied health professional, and other healthcare professionals duly licensed by the Professional Regulatory Commission.
The Rare Disease Registry is a secure health information system, including an electronic database system, which contains data on rare diseases, persons with rare diseases, and orphan drugs and products, utilized for policy making and research.
The Department of Health is the lead agency responsible for implementation, establishing the Rare Diseases Technical Working Group (RDTWG), coordinating with other agencies, supporting newborn screening clinics, organizing medical specialists, conducting public education, issuing regulations, and budgeting for the Act.
Persons with rare diseases are considered Persons with Disabilities (PWDs) and are entitled to the same rights and privileges under Republic Act No. 7277, including assistance from the Department of Social Welfare and Development and employment programs from the Department of Labor and Employment.
An orphan drug is any drug or medicine used to treat or alleviate symptoms of persons afflicted with rare diseases, designated as such by the DOH upon recommendation of the National Institutes of Health.
Persons may import orphan drugs or products for compassionate use by securing a compassionate special permit from the Food and Drug Administration (FDA), which must issue the permit within 30 days, expedited in emergencies, valid for three years and renewable thereafter.