Policy, purpose, and objectives
- Section 2 establishes a State policy to protect and promote the right to health, including the right of persons with rare diseases to survival and full and healthy development through access to timely health information and adequate medical care.
- Section 2 requires the State to institutionalize a comprehensive, integrative and sustainable system that facilitates collaboration among government and nongovernment entities at national and local levels, including the private sector, professional health organizations, academic institutions, communities, and families, for early and sustainable care.
- Section 2 recognizes the crucial role of research in defining health programs and activities for rare disease needs.
- Section 2 recognizes that effective public education is vital to support early diagnosis and treatment and to prevent ridicule and stigmatization.
- Section 3 sets objectives including:
- Improving access to comprehensive medical care (including drugs and other healthcare products) and timely health information for persons diagnosed or highly suspected of rare diseases.
- Establishing and maintaining the Rare Disease Registry with data on rare diseases in the Philippines, persons afflicted, and orphan drugs and orphan products for policy formulation, program interventions, and research design.
- Integrating public educational and information campaigns in current Department of Health (DOH) programs to identify persons with rare diseases and help the public understand special needs.
- Facilitating regular collaborative activities among stakeholders to realize the Act’s objectives.
- Section 3(b) mandates regulatory and fiscal incentives to support research and development studies and to facilitate the manufacture and importation of affordable orphan drugs and orphan products.
Key definitions and concepts
- Section 4(a) defines “Commercial use” as selling orphan drugs at profit.
- Section 4(b) defines “Healthcare Practitioner” to include doctors of medicine, dentists, nurses, midwives, allied health professionals, and other healthcare professionals duly licensed by the Professional Regulatory Commission.
- Section 4(c) defines “Healthcare institutions” as hospitals, health infirmaries, health centers, lying-in centers, or puericulture centers, whether public or private.
- Section 4(d) defines “Medical care” as comprehensive and professional care encompassing correct diagnosis, treatment, and prevention of rare diseases.
- Section 4(e) defines “Medical food” for inherited metabolic diseases as special milk formula devoid of offending amino acids, organic acids or fatty acids, amino acid supplements, essential amino acid mixtures, amino acid gels or juices, and low protein food products that are part of the medical treatment regimen.
- Section 4(f) defines “Medical specialist” as:
- a pediatrician for patients zero to eighteen (0–18) years old, or
- an adult physician above eighteen (18) years old adequately trained by experts in inherited metabolic diseases to diagnose and treat rare diseases.
- Section 4(g) defines the “National Comprehensive Newborn Screening System” as the Newborn Screening (NBS) system under Republic Act No. 9288, including education of stakeholders; sample collection/transport/biochemical screening/reporting; tracking and confirmatory testing; clinical evaluation and biochemistry/medical confirmation of follow-up results; administration of drugs and/or medical/surgical management and/or dietary supplementation; and monitoring and evaluation.
- Section 4(h) defines “Newborn screening continuity clinic” as an ambulatory clinic in a secondary or tertiary hospital identified by the DOH as part of the National Comprehensive Newborn Screening System Treatment Network, equipped to facilitate continuity of care for patients confirmed with conditions included in expanded newborn screening.
- Section 4(i) defines “Orphan drug” as a drug or medicine used to treat or alleviate symptoms of persons with a rare disease and declared as such by the DOH upon recommendation of the National Institutes of Health (NIH).
- Section 4(j) defines “Orphan product” as a healthcare or nutritional product (other than a drug/medicine), including diagnostic kits, medical devices, and biological products, used to prevent, diagnose, or treat rare diseases and declared as such by the DOH upon recommendation of the NIH.
- Section 4(k) defines “Rare disease” as disorders such as inherited metabolic disorders and other diseases with similar rare occurrence recognized by the DOH upon recommendation of the NIH, excluding catastrophic forms (life threatening, seriously debilitating, or serious and chronic) of more frequently occurring diseases.
- Section 4(l) defines “Rare Disease Management Program” as a comprehensive management program encompassing diagnosis, clinical management, genetic counseling, and drug research development for persons with rare diseases.
- Section 4(m) defines “Rare Disease Registry” as a secure health information system, including an electronic database system, containing data on rare diseases, persons with rare disease, and orphan drugs/orphan products.
- Section 4(n) defines the Rare Diseases Technical Working Group (RDTWG) as a DOH-designated pool of rare disease experts that includes NIH experts, tasked with identifying rare diseases and orphan drugs/orphan products.
- Section 4(o) defines the Telegenetics Referral System as telehealth using a computer network system providing remote genetic clinical consultations to physicians in provinces for their patients.
Registry, identification, referral, and registration
- Section 5 requires the DOH, in coordination with the NIH, to create a Rare Disease Registry and to endeavor to comply with set global standards, if applicable.
- Section 5 requires that all patients diagnosed with a rare disease be included in the Rare Disease Registry.
- Section 6 provides that patients highly suspected of or diagnosed with rare disease must be referred to a newborn screening continuity clinic identified by the DOH as referral centers for rare disease treatment under the National Comprehensive Newborn Screening System.
- Section 6 provides that for patients from remote areas, the Telegenetics Referral System will be utilized.
- Section 7 requires the DOH, with NIH assistance, to develop a system to train a sufficient number of medical specialists to diagnose and manage persons with rare disease.
- Section 8 requires the DOH, with NIH assistance, to provide better access to a support system through creation of a Rare Disease Management Program under the DOH’s National Center for Disease Prevention and Control.
- Section 9 requires all healthcare practitioners and healthcare institutions to report to the Rare Disease Registry:
- diagnosed cases of rare disease, and
- reports on the status of patients.
- Section 9 requires that reporting be subject to NIH guidelines to protect the privacy of patients with rare disease.
Rare disease as disability; rights and agencies
- Section 10 provides that persons with rare disease are considered persons with disabilities (PWDs) under Republic Act No. 7277, as amended, the Magna Carta for Disabled Persons.
- Section 11 requires the appropriate national government agency to ensure persons with rare disease are accorded the same rights and privileges as PWDs.
- Section 11(a) mandates that the Department of Social Welfare and Development (DSWD) provide assistance so social welfare and benefits under Republic Act No. 7277, as amended, are granted.
- Section 11(b) mandates that the Department of Labor and Employment (DOLE) adopt programs promoting opportunities for work and employment of persons with rare disease who are able-persons.
Rare disease and orphan status designation
- Section 12(a) requires the DOH to convene the RDTWG to determine which disorders are rare diseases and what orphan drugs and orphan products are, and to update the list periodically.
- Section 12(b) requires the RDTWG to formulate policies regulating the approval and certification of orphan drugs and orphan products.
- Section 12(c) requires the RDTWG to establish a system for regular updating of information, diagnosis, and treatment of rare diseases for comprehensive healthcare.
- Section 13 empowers the DOH, upon RDTWG recommendation, to designate any disease recognized to rarely afflict the population of the country as a rare disease.
- Section 14 provides that the DOH may designate an orphan drug:
- motu proprio or upon application by any interested person, and
- upon RDTWG recommendation.
- Section 14 mandates that within one hundred twenty (120) days from the effectivity of this Act, the DOH shall publish a list of orphan drugs for these rare diseases.
- Section 15 provides that the DOH may designate an orphan product:
- motu proprio or upon application by any interested person, and
- upon RDTWG recommendation.
- Section 15 mandates that within one hundred twenty (120) days from the effectivity of this Act, the DOH shall publish a list of orphan products for these rare diseases.
Restricted-use permits for orphan products
- Section 16(a) allows any person to import an orphan drug/orphan product for compassionate use.
- Section 16(a) requires the importer to secure a compassionate special permit from the Food and Drug Administration (FDA) in accordance with DOH Administrative Order No. 4, series of 1992, and any future guidelines for the same purpose.
- Section 16(a) requires the FDA, within thirty (30) days from receipt of the requirements, to issue a permit for restricted use of the orphan drug/orphan product.
- Section 16(a) provides that the restricted-use permit is effective for three (3) years and is renewable for three (3) years thereafter.
- Section 16(a) requires the FDA to expedite the permit in cases of emergency.
Implementation duties and lead agency
- Section 17(a) designates the DOH as the lead agency in implementing the Act.
- Section 17 requires the DOH to:
- establish the RDTWG;
- coordinate with the NIH for technical assistance;
- coordinate with government and nongovernment agencies involved in implementation;
- support newborn screening continuity clinics and designate referral centers in strategic locations for timely and sustainable management of persons with rare disease;
- organize a pool of medical specialists responsible for diagnosis and management of persons with rare disease and their families;
- conduct culturally sensitive public education and information campaigns on rare diseases, identification, special needs, and the right against ridicule and discrimination, with NIH assistance and through professional societies and nongovernment organizations;
- develop the implementing rules and regulations within one hundred eighty (180) days from enactment; and
- allot budget for implementation.
- Section 18(a) requires the FDA, NIH, DILG, DepED, DSWD, DOLE, DOST, and other relevant agencies to implement designated tasks.
- Section 18(a)(a) assigns to the FDA ensuring medical foods, orphan drugs, and orphan products are permitted in the country for treating rare diseases and developing a system addressing emergency cases.
- Section 18(a)(b) assigns to the NIH technical assistance to the DOH.
- Section 18(a)(c) assigns to DILG, DepED, DSWD, and DOLE ensuring persons with rare disease are given opportunities to be productive members of society and provided the same rights and benefits as PWDs.
- Section 18(a)(d) assigns to DOST mechanisms to further research on rare diseases and develop low-cost medical foods and orphan products for patients.
- Section 18(a)(e) requires other relevant agencies to assist in full implementation.
- Section 19 requires healthcare practitioners attending to a person with rare disease to inform the patient and family of available resources and refer them to the nearest available specialist.
- Section 20 requires the DOH and NIH, with health professional societies and academic healthcare institutions, to:
- conduct continuing education, information, and training for practitioners on identification and referral for medical management; and
- educate practitioners on the importance of reporting cases to the Rare Disease Registry.
Financial assistance and fiscal incentives
- Section 21(a) provides that persons with rare disease may avail of a basic benefit package from the Philippine Health Insurance Corporation, provided in accordance with its guidelines.
- Section 21(b) provides that persons with rare disease may avail of medical assistance as provided in Section 8 of Republic Act No. 10351 or the Sin Tax Reform Act of 2012.
- Section 22(a) exempts from all taxes and customs duties, as applicable, whether national or local:
- donations intended for researches on rare diseases, maintenance of the Rare Disease Registry, or purchase of orphan drugs or orphan products for use solely by patients with rare diseases.
- Section 22(b) exempts from all taxes and customs duties, as applicable, whether national or local:
- orphan drugs and orphan products for use solely by patients with rare diseases, as certified by the FDA.
Rules, repeals, separability, and effectivity
- Section 23 requires the DOH, in consultation with the NIH, to issue the implementing rules and regulations within one hundred eighty (180) days from the effectivity of this Act.
- Section 24 repeals or modifies all general and special laws, decrees, executive orders, proclamations, and administrative regulations—or parts of them—that are inconsistent with the Act.
- Section 25 provides separability: unconstitutional or invalid parts do not affect other parts that remain in force and effect.
- Section 26 establishes effectivity as fifteen (15) days after publication in at least two (2) newspapers of general circulation.