QuestionsQuestions (Republic Act No. 10747)
RA 10747 is known as the “Rare Diseases Act of the Philippines.” (Sec. 1)
To protect and promote the right to health of people, including persons with rare diseases, by ensuring access to timely health information and adequate medical care through a comprehensive, integrative, and sustainable system and collaboration among stakeholders. (Sec. 2)
To improve access to comprehensive medical care (including drugs/products and timely information) via an integrated healthcare system for rare diseases; establish and maintain a Rare Disease Registry; integrate public education campaigns in DOH programs; facilitate stakeholder collaboration; and provide regulatory and fiscal incentives to support R&D and make orphan drugs/products more affordable. (Sec. 3)
It refers to disorders such as inherited metabolic disorders and other diseases with similar rare occurrence as recognized by DOH upon NIH recommendation, excluding catastrophic (life threatening, seriously debilitating, or serious and chronic) forms of more frequently occurring diseases. (Sec. 4(l))
Any duly licensed: doctor of medicine, dentist, nurse, midwife, allied health professional, and other healthcare professional licensed by the Professional Regulatory Commission. (Sec. 4(b))
It is a secure health information system/electronic database for data on rare diseases, persons with rare diseases, and orphan drugs and orphan products, used for policy formulation, program interventions, and research design. (Sec. 4(m))
DOH convenes it. It determines rare diseases and orphan drugs/products and updates the list; formulates policies regulating orphan drug/product approval/certification; and establishes a system to regularly update information, diagnosis, and treatment. (Sec. 12)
DOH, with NIH coordination, creates the Registry and endeavors to comply with global standards if applicable; all patients diagnosed with rare disease shall be included in the registry. (Sec. 5)
To a newborn screening continuity clinic identified by DOH as referral centers for treatment under the National Comprehensive Newborn Screening System; for remote areas, DOH uses the Telegenetics Referral System. (Sec. 6)
DOH, with NIH assistance, shall develop a system to train a sufficient number of medical specialists to diagnose and manage persons with rare diseases. (Sec. 7)
A comprehensive management program encompassing diagnosis, clinical management, genetic counseling, and drug research development for people with rare diseases, created by DOH with NIH assistance under the National Center for Disease Prevention and Control. (Sec. 8)
They are required to report diagnosed cases of rare disease to the Rare Disease Registry and provide reports on patient status, subject to NIH-issued guidelines to protect patient privacy. (Sec. 9)
Yes. Persons with rare disease shall be considered persons with disabilities (PWDs) in accordance with RA 7277 as amended (Magna Carta for Disabled Persons). This entitles them to the corresponding rights and privileges under disability laws. (Sec. 10)
DSWD ensures social welfare and benefits under RA 7277 as amended are granted; DOLE adopts programs promoting work and employment opportunities for able persons with rare disease. (Sec. 11)
Orphan drug: a drug/medicine to treat or alleviate symptoms of persons with a rare disease, declared as such by DOH upon NIH recommendation. Orphan product: healthcare or nutritional product (not a drug/medicine), including diagnostic kits, medical devices, biological products used to prevent/diagnose/treat rare diseases, declared as such by DOH upon NIH recommendation. (Sec. 4(i)–(j))
Within 120 days from effectivity of the Act, DOH shall publish a list of orphan drugs; similarly, DOH shall publish a list of orphan products within 120 days. (Secs. 14–15)
Yes, any person may import it for compassionate use by securing a compassionate special permit from the FDA in accordance with DOH AO No. 4, s. 1992 and future guidelines. (Sec. 16)
DOH is the lead agency. It must establish the RDTWG; coordinate with NIH for technical assistance; coordinate with involved agencies; support newborn screening continuity clinics and designate referral centers; organize specialists pool; conduct public education campaigns; develop IRR within 180 days; and allot budget for implementation. (Sec. 17)
IRR must be issued within 180 days from effectivity. The Act takes effect 15 days after publication in at least two newspapers of general circulation. (Secs. 23 and 26)